acronym: CALM

title: Phase I, Open Label, Dose-escalation Study Followed by a Safety Expansion Part to Evaluate the Safety, Expansion and Persistence of a Single Dose of UCART19 (Allogeneic Engineered T-cells Expressing Anti-CD19 Chimeric Antigen Receptor), Administered Intravenously in Patients With Relapsed or Refractory CD19 Positive B-cell Acute Lymphoblastic Leukaemia (B-ALL)

ReferenceCitation: Benjamin R, Graham C, Yallop D, Jozwik A, Mirci-Danicar OC, Lucchini G, Pinner D, Jain N, Kantarjian H, Boissel N, Maus MV, Frigault MJ, Baruchel A, Mohty M, Gianella-Borradori A, Binlich F, Balandraud S, Vitry F, Thomas E, Philippe A, Fouliard S, Dupouy S, Marchiq I, Almena-Carrasco M, Ferry N, Arnould S, Konto C, Veys P, Qasim W; UCART19 Group. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies. Lancet. 2020 Dec 12;396(10266):1885-1894. doi: 10.1016/S0140-6736(20)32334-5.

ReferenceCitation: Benjamin R, Jain N, Maus MV, Boissel N, Graham C, Jozwik A, Yallop D, Konopleva M, Frigault MJ, Teshima T, Kato K, Boucaud F, Balandraud S, Gianella-Borradori A, Binlich F, Marchiq I, Dupouy S, Almena-Carrasco M, Pannaux M, Fouliard S, Brissot E, Mohty M; CALM Study Group. UCART19, a first-in-class allogeneic anti-CD19 chimeric antigen receptor T-cell therapy for adults with relapsed or refractory B-cell acute lymphoblastic leukaemia (CALM): a phase 1, dose-escalation trial. Lancet Haematol. 2022 Nov;9(11):e833-e843. doi: 10.1016/S2352-3026(22)00245-9. Epub 2022 Oct 10.

ipd_sharing_description:
Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data.

Access can be requested for all interventional clinical studies:

used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

sponsored by Servier
with a first patient enrolled as of 1 January 2004 onwards
for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

location_country:
Japan
United States
United Kingdom
France

location_facility: University of Texas MD Anderson Cancer Center

location_facility: King's College Hospital NHS Foundation Trust

location_facility: Hospital of the University of Pennsylvania

summary: The study is in two parts: a dose escalation then a safety dose expansion. The purpose of the dose escalation part is to evaluate the safety and tolerability of ascending doses of UCART19 (dose-escalation part) given as a single infusion in patients with relapsed / refractory (R/R) B-cell acute lymphoblastic leukaemia (B-ALL), to determine the maximum tolerated dose (MTD), the recommended dose and the lymphodepletion regimen. The purpose of the safety dose expansion is to assess the safety and tolerability of the RD for UCART19.


title: Arimoclomol Prospective Doubleblind, Randomised, Placebo-controlled Study in Patients Diagnosed With NiemannPick Disease Type C

summary:
A prospective, randomised, double-blind, placebo controlled therapeutic study in patients with confirmed diagnosis of NiemannPick disease type C (NPC).

The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the patient's current prescribed best standard of care; patient's standard of care may, or may not, include miglustat.

The CT-ORZY-NPC-002 study has been expanded to include an open label paediatric sub-study including patients aged 6 to <24 months at study enrolment.

study_start_date_type: Actual

status: Active, not recruiting

study_completion_date_type: Anticipated

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: Mengel E, Patterson MC, Da Riol RM, Del Toro M, Deodato F, Gautschi M, Grunewald S, Grønborg S, Harmatz P, Héron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Andreasen AK, Geist MA, Havnsøe Torp Petersen N, Ingemann L, Hansen T, Blaettler T, Kirkegaard T, Í Dali C. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. J Inherit Metab Dis. 2021 Nov;44(6):1463-1480. doi: 10.1002/jimd.12428. Epub 2021 Sep 7.

ReferenceCitation: Patterson MC, Lloyd-Price L, Guldberg C, Doll H, Burbridge C, Chladek M, íDali C, Mengel E, Symonds T. Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale. Orphanet J Rare Dis. 2021 Feb 12;16(1):79. doi: 10.1186/s13023-021-01719-2.

lead_sponsor: KemPharm Denmark A/S

location_country:
Denmark
Poland
Spain
Italy
United Kingdom
Switzerland
United States
France
Germany

location_facility: UCSF Benioff Children's Hospital Oakland

location_facility: University Hospital Copenhagen (Rigshospitalet)

location_facility: Dr. von Haunersches Kinderspital der Universität München

location_facility: Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" di Udin

location_facility: Villa Metabolica, Universitätsmedizin Mainz

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Ongoing


title: A Phase Ib Dose Escalation Study of BCL201 in Combination With Idelalisib in Patients With Follicular Lymphoma (FL) and Mantle Cell Lymphoma (MCL)

summary:
This is a phase Ib multi-center, open-label study: escalation part followed by expansion part. The primary purpose of the Phase Ib CBCL201X2102C study is to characterize the safety and tolerability of BCL201 combined with idelalisib in patients with FL and MCL.

Approximately 65 patients are to be enrolled.

The primary endpoint for the Phase Ib is frequency, severity and seriousness of AEs, lab abnormalities and other safety parameters such as ECG changes. An adaptive Bayesian logistic regression model (BLRM) will guide the dose escalation to determine the MTD/RDE in phase Ib. In addition Bayesian regression models will be used to estimate the dose-exposure relationships for both BCL201 and idelalisib in order to guide the escalation steps. A Bayesian method for the expansion part will be used for the primary activity objective.

The study data will be analyzed and reported based on all patients' data of the escalation and expansion part.

status: Completed

study_completion_date_type: Actual

design_allocation: Non-Randomized

ipd_sharing: No

lead_sponsor: Novartis Pharmaceuticals

location_country:
Austria
Germany
United States
France

status_simplified: Completed


title: A Prospective, Non-comparative, Multi-center Clinical Trial to Evaluate the Safety and Efficacy of the STARflo Glaucoma Implant in Patients With Open Angle Glaucoma

summary: Prospective, Non-comparative, Multi-center clinical trial to evaluate the safety and efficacy of the STARflo Glaucoma Implant in patients with refractory open angle glaucoma.

lead_sponsor: iSTAR Medical

location_country: Switzerland France Belgium Germany Bulgaria

location_facility: Universitatsklinik fur Augenheilkunde Inselspital

intervention_type: Device


title: A Randomised Controlled Trial to Compare Seizure Remission Outcome Following Resective Surgery With or Without Prior Treatment With Ketogenic Diet in Children With Epilepsy the Result of Focal Cortical Dysplasia Type II

acronym: EDIBLE

summary: The investigators are undertaking the first European Randomised Controlled Trial (RCT) for epilepsy surgery in children with FCD type II, to prospectively evaluate the role of the KD prior to surgery in improving seizure outcome. The investigators will evaluate the role of KD as a disease-modifying treatment to achieve seizure control and improve neurodevelopment and quality of life. Children age 3 - 15 years with pharmacoresistant epilepsy believed to be the result of focal cortical dysplasia type II, considered to be surgically treatable, will be randomised to either receive 6m treatment presurgery with a ketogenic diet, or to proceed direct to surgery (no pretreatment). Primary outcome will be the time to achieve a period of 6 months of seizure freedom from the date of randomisation. Tissue resected at surgery will also be evaluated with regard to the degree of any methylation of DNA.

status: Terminated

ReferenceType: background

ReferenceCitation: Wiebe S, Blume WT, Girvin JP, Eliasziw M; Effectiveness and Efficiency of Surgery for Temporal Lobe Epilepsy Study Group. A randomized, controlled trial of surgery for temporal-lobe epilepsy. N Engl J Med. 2001 Aug 2;345(5):311-8.

ReferenceCitation: Engel J Jr, McDermott MP, Wiebe S, Langfitt JT, Stern JM, Dewar S, Sperling MR, Gardiner I, Erba G, Fried I, Jacobs M, Vinters HV, Mintzer S, Kieburtz K; Early Randomized Surgical Epilepsy Trial (ERSET) Study Group. Early surgical therapy for drug-resistant temporal lobe epilepsy: a randomized trial. JAMA. 2012 Mar 7;307(9):922-30. doi: 10.1001/jama.2012.220.

ReferenceCitation: Harvey AS, Cross JH, Shinnar S, Mathern GW; ILAE Pediatric Epilepsy Surgery Survey Taskforce. Defining the spectrum of international practice in pediatric epilepsy surgery patients. Epilepsia. 2008 Jan;49(1):146-55. Epub 2007 Nov 27. Erratum in: Epilepsia. 2013 Jun;54(6):1140. Mathern, Bary W [corrected to Mathern, Gary W].

ReferenceCitation: Lerner JT, Salamon N, Hauptman JS, Velasco TR, Hemb M, Wu JY, Sankar R, Donald Shields W, Engel J Jr, Fried I, Cepeda C, Andre VM, Levine MS, Miyata H, Yong WH, Vinters HV, Mathern GW. Assessment and surgical outcomes for mild type I and severe type II cortical dysplasia: a critical review and the UCLA experience. Epilepsia. 2009 Jun;50(6):1310-35. doi: 10.1111/j.1528-1167.2008.01998.x. Epub 2009 Jan 21. Review.

ReferenceCitation: Blümcke I, Thom M, Aronica E, Armstrong DD, Vinters HV, Palmini A, Jacques TS, Avanzini G, Barkovich AJ, Battaglia G, Becker A, Cepeda C, Cendes F, Colombo N, Crino P, Cross JH, Delalande O, Dubeau F, Duncan J, Guerrini R, Kahane P, Mathern G, Najm I, Ozkara C, Raybaud C, Represa A, Roper SN, Salamon N, Schulze-Bonhage A, Tassi L, Vezzani A, Spreafico R. The clinicopathologic spectrum of focal cortical dysplasias: a consensus classification proposed by an ad hoc Task Force of the ILAE Diagnostic Methods Commission. Epilepsia. 2011 Jan;52(1):158-74. doi: 10.1111/j.1528-1167.2010.02777.x. Epub 2010 Nov 10.

ReferenceCitation: Levy RG, Cooper PN, Giri P. Ketogenic diet and other dietary treatments for epilepsy. Cochrane Database Syst Rev. 2012 Mar 14;(3):CD001903. doi: 10.1002/14651858.CD001903.pub2. Review. Update in: Cochrane Database Syst Rev. 2016;2:CD001903.

ReferenceCitation: Neal EG, Chaffe H, Schwartz RH, Lawson MS, Edwards N, Fitzsimmons G, Whitney A, Cross JH. The ketogenic diet for the treatment of childhood epilepsy: a randomised controlled trial. Lancet Neurol. 2008 Jun;7(6):500-6. doi: 10.1016/S1474-4422(08)70092-9. Epub 2008 May 2.

ReferenceCitation: Neal EG, Chaffe H, Schwartz RH, Lawson MS, Edwards N, Fitzsimmons G, Whitney A, Cross JH. A randomized trial of classical and medium-chain triglyceride ketogenic diets in the treatment of childhood epilepsy. Epilepsia. 2009 May;50(5):1109-17. doi: 10.1111/j.1528-1167.2008.01870.x. Epub 2008 Nov 19.

ReferenceCitation: Sharma S, Sankhyan N, Gulati S, Agarwala A. Use of the modified Atkins diet for treatment of refractory childhood epilepsy: a randomized controlled trial. Epilepsia. 2013 Mar;54(3):481-6. doi: 10.1111/epi.12069. Epub 2013 Jan 7.

ReferenceCitation: Jung DE, Kang HC, Kim HD. Long-term outcome of the ketogenic diet for intractable childhood epilepsy with focal malformation of cortical development. Pediatrics. 2008 Aug;122(2):e330-3. doi: 10.1542/peds.2008-0012.

ReferenceCitation: Hahnen E, Hauke J, Tränkle C, Eyüpoglu IY, Wirth B, Blümcke I. Histone deacetylase inhibitors: possible implications for neurodegenerative disorders. Expert Opin Investig Drugs. 2008 Feb;17(2):169-84. doi: 10.1517/13543784.17.2.169. Review.

ReferenceCitation: Göttlicher M, Minucci S, Zhu P, Krämer OH, Schimpf A, Giavara S, Sleeman JP, Lo Coco F, Nervi C, Pelicci PG, Heinzel T. Valproic acid defines a novel class of HDAC inhibitors inducing differentiation of transformed cells. EMBO J. 2001 Dec 17;20(24):6969-78.

ReferenceCitation: Feil R, Fraga MF. Epigenetics and the environment: emerging patterns and implications. Nat Rev Genet. 2012 Jan 4;13(2):97-109. doi: 10.1038/nrg3142. Review.

ReferenceCitation: Feng J, Zhou Y, Campbell SL, Le T, Li E, Sweatt JD, Silva AJ, Fan G. Dnmt1 and Dnmt3a maintain DNA methylation and regulate synaptic function in adult forebrain neurons. Nat Neurosci. 2010 Apr;13(4):423-30. doi: 10.1038/nn.2514. Epub 2010 Mar 14.

ReferenceCitation: Kobow K, Kaspi A, Harikrishnan KN, Kiese K, Ziemann M, Khurana I, Fritzsche I, Hauke J, Hahnen E, Coras R, Mühlebner A, El-Osta A, Blümcke I. Deep sequencing reveals increased DNA methylation in chronic rat epilepsy. Acta Neuropathol. 2013 Nov;126(5):741-56. doi: 10.1007/s00401-013-1168-8. Epub 2013 Sep 5.

lead_sponsor: University College, London

location_country:
United Kingdom
Italy
Austria
Switzerland
United States
France
Germany
Czechia

location_facility: Great Ormond Street Hospital for Children NHS Trust

intervention_type: Dietary Supplement

lead_sponsor_type: academique

status_simplified: Unknown


type: EudraCT Number

title: Clinical Study of the BreathID® LF System to Train the Algorithm for the ¹³C-Methacetin Breath Test (MBT) in Assessment of Portal Hypertension in Patients With Compensated Liver Cirrhosis

summary: This study will be used to train an algorithm using Methacetin breath test (MBT) measures and to select a cut-off to determine presence or absence of Clinically Significant Portal Hypertension (CSPH) as defined by Hepatic Venous Gradient Pressure (HVPG) ≥ 10mmHg,

primary_purpose: Diagnostic

lead_sponsor: Exalenz Bioscience LTD.

location_country:
United States
Switzerland
Spain
France

location_facility: (University of Barcelona) Hospital Clinic


title: A Phase 2 Multi-center, Randomized, Double-blind, Comparator-Controlled Dose Finding Study to Evaluate MP4CO for the Acute Treatment of Vaso-occlusive Crises in Subjects With Sickle Cell Disease

summary:
Sickle Cell disease is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body. In Sickle Cell disease, as red blood cells circulate and oxygen is released, the deoxygenated abnormal Hemoglobin S can begin to polymerize and cause red cells to become sticky and elongated. These "sickled" red cells are less flexible and will obstruct small blood vessels and prevent normal red cells from circulating freely, which limits oxygen delivery to tissues and organs. This is known as a "sickling crisis" or "vaso-occlusive crisis" and is the leading cause of hospitalization in patients with Sickle Cell disease.

Patients suffering from a sickle crisis experience severe pain and are at risk of stroke, heart attack or even death. Current therapy is limited to hydration and symptomatic pain relief. The administration of MP4CO as an adjunct treatment to standard therapy may alleviate pain associated with a sickling crisis and potentially reduce the severity and duration of a crisis. This may shorten the time in hospital and potentially improve the quality of life for patients with sickle cell anemia.

status: Withdrawn

ReferenceCitation: Vandegriff KD, Young MA, Lohman J, Bellelli A, Samaja M, Malavalli A, Winslow RM. CO-MP4, a polyethylene glycol-conjugated haemoglobin derivative and carbon monoxide carrier that reduces myocardial infarct size in rats. Br J Pharmacol. 2008 Aug;154(8):1649-61. doi: 10.1038/bjp.2008.219. Epub 2008 Jun 9.

ReferenceCitation: Vandegriff KD, Bellelli A, Samaja M, Malavalli A, Brunori M, Winslow RM. Kinetics of NO and O2 binding to a maleimide poly(ethylene glycol)-conjugated human haemoglobin. Biochem J. 2004 Aug 15;382(Pt 1):183-9.

ReferenceCitation: Tsai AG, Vandegriff KD, Intaglietta M, Winslow RM. Targeted O2 delivery by low-P50 hemoglobin: a new basis for O2 therapeutics. Am J Physiol Heart Circ Physiol. 2003 Oct;285(4):H1411-9. Epub 2003 Jun 12.

ReferenceCitation: Cole RH, Vandegriff KD. MP4, a vasodilatory PEGylated hemoglobin. Adv Exp Med Biol. 2011;701:85-90. doi: 10.1007/978-1-4419-7756-4_12.

ReferenceCitation: Vandegriff KD, Malavalli A, Mkrtchyan GM, Spann SN, Baker DA, Winslow RM. Sites of modification of hemospan, a poly(ethylene glycol)-modified human hemoglobin for use as an oxygen therapeutic. Bioconjug Chem. 2008 Nov 19;19(11):2163-70. doi: 10.1021/bc8002666.

ReferenceCitation: Svergun DI, Ekström F, Vandegriff KD, Malavalli A, Baker DA, Nilsson C, Winslow RM. Solution structure of poly(ethylene) glycol-conjugated hemoglobin revealed by small-angle X-ray scattering: implications for a new oxygen therapeutic. Biophys J. 2008 Jan 1;94(1):173-81. Epub 2007 Sep 7.

ReferenceCitation: Tsai AG, Cabrales P, Manjula BN, Acharya SA, Winslow RM, Intaglietta M. Dissociation of local nitric oxide concentration and vasoconstriction in the presence of cell-free hemoglobin oxygen carriers. Blood. 2006 Nov 15;108(10):3603-10. Epub 2006 Jul 20.

ReferenceCitation: Vandegriff KD, McCarthy M, Rohlfs RJ, Winslow RM. Colloid osmotic properties of modified hemoglobins: chemically cross-linked versus polyethylene glycol surface-conjugated. Biophys Chem. 1997 Nov;69(1):23-30.

ReferenceCitation: Ballas SK, Gupta K, Adams-Graves P. Sickle cell pain: a critical reappraisal. Blood. 2012 Nov 1;120(18):3647-56. doi: 10.1182/blood-2012-04-383430. Epub 2012 Aug 24. Review.

ReferenceCitation: Belcher JD, Young M, Chen C, Nguyen J, Burhop K, Tran P, Vercellotti GM. MP4CO, a pegylated hemoglobin saturated with carbon monoxide, is a modulator of HO-1, inflammation, and vaso-occlusion in transgenic sickle mice. Blood. 2013 Oct 10;122(15):2757-64. doi: 10.1182/blood-2013-02-486282. Epub 2013 Aug 1.

ReferenceCitation: Belcher JD, Mahaseth H, Welch TE, Otterbein LE, Hebbel RP, Vercellotti GM. Heme oxygenase-1 is a modulator of inflammation and vaso-occlusion in transgenic sickle mice. J Clin Invest. 2006 Mar;116(3):808-16. Epub 2006 Feb 16.

ReferenceCitation: Bilban M, Haschemi A, Wegiel B, Chin BY, Wagner O, Otterbein LE. Heme oxygenase and carbon monoxide initiate homeostatic signaling. J Mol Med (Berl). 2008 Mar;86(3):267-79. Epub 2007 Nov 22. Review.

ReferenceCitation: Piantadosi CA, Withers CM, Bartz RR, MacGarvey NC, Fu P, Sweeney TE, Welty-Wolf KE, Suliman HB. Heme oxygenase-1 couples activation of mitochondrial biogenesis to anti-inflammatory cytokine expression. J Biol Chem. 2011 May 6;286(18):16374-85. doi: 10.1074/jbc.M110.207738. Epub 2011 Mar 18.

ReferenceCitation: Powars DR, Chan LS, Hiti A, Ramicone E, Johnson C. Outcome of sickle cell anemia: a 4-decade observational study of 1056 patients. Medicine (Baltimore). 2005 Nov;84(6):363-376. doi: 10.1097/01.md.0000189089.45003.52.

lead_sponsor: Sangart

location_country:
Brazil
Bahrain
United Kingdom
Lebanon
Qatar
France
Turkey
Belgium
Netherlands

location_facility: Rio de Janerio Instituto Estadual de Hematologie

location_facility: Georges Pompidou European University Hospital


title: PHASE ANGLE PROJECT

acronym: PAP

summary:
Critically ill patients feature a loss of fat-free mass (FFM) up to 440 g/day, which is associated with increased morbidity and prolonged recovery. In several clinical conditions, FFM or phase angle (PhA)derived from BIA have been associated with clinical outcome. However, solid data to support this association in ICU patients are lacking. Only one retrospective study of 51 ICU patients with acute respiratory failure correlated loss of active cell mass with mortality. In a pilot study performed in 55 ICU patients, the investigators observed that five kHz BIA PhA was significantly related to SOFA (r=0.38, P=0.03). The relation between PhA and mortality remains to be determined in ICU patients.

Classic ICU validated severity scores (e.g. Acute Physiology and Chronic Health Evaluation (APACHE), Sequential Organ Failure Assessment (SOFA), Simplified Acute Physiology Score (SAPS)) and recent nutritional scores have been developed to foresee the clinical outcome of ICU patients. Most of these scores are time consuming and suffer some degree of discriminative power (i.e. APACHE II and SAPS II are not validated in cardiovascular surgery patients).

PhA is reflecting intracellular status: altered intracellular water (ICW) to extracellular water (ECW) distribution is suggested by low PhA. PhA measurement does not require anamnestic parameters, body weight, and lab tests. It could easily, accurately and repeatedly measured at bedside. PhA has been correlated with the disease prognosis in HIV infection, hemodialysis, peritoneal dialysis, chronic renal failure and liver cirrhosis patients. These studies suggest that PhA may be useful in determining increased risk of morbidity in the ICU.

Computerized tomography (CT) images targeted on the 3rd lumbar vertebrae (L3) could accurately measure FFM13 and predict survival in cancer patients. Body composition evaluation by CT presents great practical significance due to its routine ICU use in the initial diagnosis or follow-up. The usefulness of measuring FFM with L3-targeted CT has never been evaluated in ICU patients.

Therefore, the investigators aim to conduct an international multicentre prospective observational study in ICU patients to assess the prognosis value of BIA PhA at admission, and to compare the performances of BIA and L3-targeted CT for FFM measurement.

study_type: Observational

time_perspective: Prospective

ReferenceCitation: Thibault R, Makhlouf AM, Mulliez A, Cristina Gonzalez M, Kekstas G, Kozjek NR, Preiser JC, Rozalen IC, Dadet S, Krznaric Z, Kupczyk K, Tamion F, Cano N, Pichard C; Phase Angle Project Investigators. Fat-free mass at admission predicts 28-day mortality in intensive care unit patients: the international prospective observational study Phase Angle Project. Intensive Care Med. 2016 Sep;42(9):1445-53. doi: 10.1007/s00134-016-4468-3. Epub 2016 Aug 11.

lead_sponsor: University Hospital, Geneva

location_country: Croatia Poland Brazil Spain Lithuania Switzerland France Israel Belgium

location_facility:
Erasme University Hospital
Universidade Federal de Pelotas
Geneva University Hospital
Rabin Medical Center
Vilnius University Hospital
Univ. Hospital Center Zagreb
Stanley Dudrick's Memorial Hospital
Clinica USP Palmaplanas
Clermont-Ferrand University Hospital
Rouen University Hospital


title: The "DeSScipher" Project - to Decipher the Best Treatment for Systemic Sclerosis - Observational Trial 2: Improvement of Hand Dysfunction by Arthritis in Systemic Sclerosis

summary:
Systemic sclerosis (SSc) is an orphan, multiorgan disease affecting the connective tissue of the skin and several internal organs. Beside skin involvement, digital ulcers, tendinitis, calcinosis and flexion contractures, the presence of hand arthritis is a major contributor to impairment of hand function in systemic sclerosis. Several immunomodulatory drugs used in other rheumatic diseases (including methotrexate, leflunomide, azathioprine, mycophenolate mofetil and low-dose corticosteroids) can potentially improve arthritis and consequently hand function in systemic sclerosis. For the assessment of arthritis, the CDAI (clinical disease activity index) is validated in rheumatoid arthritis, and may be useful for SSc-related arthritis, too.

This observational trial is part of the collaborative project "DeSScipher", one out of five observational trials to decipher the optimal management of systemic sclerosis. Aim of this observational trial is to:

investigate the efficacy and safety of different treatments on hand dysfunction in systemic sclerosis patients with hand arthritis and
to validate the CDAI for arthritis in systemic sclerosis.

status: Unknown status

enrollment_type: Anticipated

lead_sponsor: Prof. Laszlo Czirjak

location_country:
Croatia
Romania
Serbia
Spain
Italy
Russian Federation
United Kingdom
Switzerland
Hungary
France
Turkey
Belgium
Germany
Egypt

location_facility: Clinic of Nephrology, Internal and Occupational Diseases

location_facility: Pecsi Tudomanyegyetem - University of Pecs

location_facility: Institute of Rheumatology, Russian Academy of Medical Science

location_facility: University of Marmara, Department of Rheumatology

location_facility: University of Florence, Denothe Centre, Division of Rheumatology AOUC, Department of Biomedicine

location_facility: Hospital Universitario Madrid Norte Sanchinarro

location_facility: Centre for Pediatric Rheumatology, Klinikum Eilbek

location_facility: Department of Internal Medicine Hôpital Claude Huriez

location_facility: GR.T.Popa Center for Biomedical Research, European Center for Translational Research, "GR.T.Popa" University of Medicine and Pharmacy, Rehabilitation Hospital

location_facility: University of Zurich, Department of Rheumatology

location_facility: University of Ghent, Department of Rheumatology

location_facility: Justus-Liebig-University Gießen, Kerckhoff Clinic, Departement of Rheumatology and Clinical Immunology

location_facility: Divisione di Reumatologia, Università di Roma La Sapienza, Dipartimento di Clinica e Terapia medica applicata, Policlinico Umberto I

location_facility: Royal Free Hospital, University College London

location_facility: Charité Universitätsmedizin Berlin, Charité Centrum 12 für Innere Medizin und Dermatologie, Medizinische Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie

location_facility: University of Manchester, Rheumatic Diseases Centre, Clinical Sciences

location_facility: Reumatologie, University of Medicine & Pharmacy,"Iuliu Hatieganu" Cluj

location_facility: Istituto di Clinica Medica Generale, Ematologia ed Immunologia Clinica, Università Politecnica delle Marche, Polo Didattico, University of Ancona

location_facility: Université Paris Descartes, Hôpital Cochin, Service de Rhumatologie A & INSERM 1016

location_facility: Department of Rheumatology, University of Cagliari-Policlinico Universitario

location_facility: The Universitiy of Leeds, Division of Rheumatic and Musculoskeletal Disease, St James's University Hospital

location_facility: Department of Internal Medicine and Rheumatology Clinic, Ion Cantacuzino Clinical Hospital

location_facility: Assiut and Sohage University Hospital Rheumatology Department Assiut University Hospital


title: Phase IIb Study to Assess the Efficacy, Safety and Tolerability of Two Dosing Regimens of AllerT in Comparison to Placebo in Adult Subjects Allergic to Birch Pollen

summary: The main objective of the trial is to demonstrate the efficacy of a two months pre-seasonal treatment with AllerT 100 µg maintenance dose in reducing symptoms of allergic rhinoconjunctivitis during the following birch pollen season

lead_sponsor: Anergis

location_country: Denmark Poland Latvia Lithuania Switzerland France Sweden

location_facility: Viktorija Vevere private practice of Allergology

location_facility: Paul Stradins Clinical University Hospital - Pulmonology Allergology

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Antakalnio affiliation of the Vilnius City Allergy Center

location_facility: Silesian Piasts University of Medicine in Wrocław

location_facility: SPZOZ Uniwersytecki Szpital Kliniczny - Allergology

location_facility: Center of examination and treatment of allergic diseases


title: An Open-label Extension of Study AC-066A301 Investigating the Safety and Tolerability of ACT-385781A in Patients With Pulmonary Arterial Hypertension (PAH)

summary: This is an open-label, nonrandomized extension to study AC-066A301. The study will assess safety and tolerability of ACT-385781A while providing a means for continuing treatment after ending participation in study AC-066A301.

lead_sponsor: Actelion

location_country: Spain Italy Canada France Belgium Netherlands

location_facility: Sir Mortimer B Davis Jewish General Hospital


title: A Multicenter, Single-arm, Open-label, Phase 3b Study to Assess the Effects of Switching From Flolan® to EFI/ACT-385781A in Patients With Pulmonary Arterial Hypertension

source: euctr

location_facility: University Health Network, Toronto TGH - 10 EN - 220

summary: This study is investigating the effect of switching from Flolan® to Epoprostenol for Injection (EFI/ACT-385781A) in pulmonary arterial hypertension patients currently treated with Flolan®. For this purpose patients being treated for at least 12 months with Flolan® will be switched from Flolan® to EFI/ACT-385781A and followed-up for 90 days. During these 90 days safety and tolerability of EFI/ACT-385781A will closely be monitored in all treated patients. This 90 follow-up will provide clinical evidence on the safety, tolerability, efficacy and treatment satisfaction of switching from Flolan® to EFI/ACT-385781A in patients with pulmonary arterial hypertension.


title: An Open-label, Phase I/IIa, Dose Escalating Study of 2B3-101 in Patients With Solid Tumors and Brain Metastases or Recurrent Malignant Glioma.

summary: The purpose of this study is to determine the safety, tolerability, and pharmacokinetics (PK) of 2B3-101 both as single agent and in combination with trastuzumab. Furthermore, the study will explore the preliminary antitumor activity of 2B3-101 as single agent in patients with with solid tumors and brain metastases or recurrent malignant glioma as well as in patients with various forms of breast cancer with and in combination with trastuzumab in HER2+ breast cancer patients with brain metastases.

lead_sponsor: BBB-Therapeutics B.V.

location_country:
Netherlands
United States
France
Belgium

location_facility: Leids Universitair Medisch Centrum (LUMC)

location_facility: UNC Lineberger Comprehensive Cancer Center

location_facility: Vrije Universiteit medisch centrum (Vumc)


title: REVOLUTION (WFCC-133): Clinical Workflow Study for the Evaluation of the Multi-Electrode Pulmonary Vein Isolation System for the Treatment of Paroxysmal Atrial Fibrillation (PAF)

acronym: REVOLUTION

summary: The purpose of this study is to assess the safety and effectiveness of the Circular and Crescent Mapping and Ablation catheters and the workflow of the Multi-Electrode Irrigated Pulmonary Vein Isolation System when used for the treatment of drug refractory symptomatic paroxysmal atrial fibrillation (PAF).

lead_sponsor: Biosense Webster, Inc.

location_country: Denmark Italy France Belgium Germany Czechia

location_facility: Institute for Clinical and Experimental Medicine (IKEM)


acronym: EARLY

title: A Randomized, Double-Blind, Placebo-Controlled, Crossover Multi-Center Study to Assess the Efficacy and Safety of Inhaled Tobramycin Nebuliser Solution (TOBI®) for the Treatment of Early Infections of P. Aeruginosa in Cystic Fibrosis Subjects Aged From 3 Months to Less Than 7 Years

ReferenceCitation: Ratjen F, Moeller A, McKinney ML, Asherova I, Alon N, Maykut R, Angyalosi G; EARLY study group. Eradication of early P. aeruginosa infection in children <7 years of age with cystic fibrosis: The early study. J Cyst Fibros. 2019 Jan;18(1):78-85. doi: 10.1016/j.jcf.2018.04.002. Epub 2018 Apr 21.

location_country:
Greece
Italy
Russian Federation
Canada
Switzerland
Hungary
France
Romania
Germany
Egypt

summary: This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.


title: International, Multicenter, Prospective, Randomized, Parallel Group, Open Label Protocol to Evaluate Safety and Efficacy of Isolated Pancreas Transplantation Compared to Intensive Insulin Therapy in Type 1 Diabetic Patients With Overt Diabetic Nephropathy and Mildly Reduced Renal Function

acronym: PANCREAS

summary: Current medical therapies are not able to prevent progression of established macroproteinuira (i.e. diabetic nephropathy) to end-stage renal failure in type 1 (insulin dependent) diabetic patients. In this setting, proteinuria is a major risk factor for mortality. Pancreas transplantation, on the contrary, can revert diabetic nephropathy and thereby prevent end-stage chronic renal failure, with theoretically lower risk of death as compared to current medical therapies.The main objective of this study is to assess superiority of isolated pancreas transplantation versus intensive exogenous insulin therapy in type 1 diabetic patients with overt diabetic nephropathy and mildly reduced renal function. The primary endpoint is a composite efficacy/failure end-point including: patient mortality and renal function impairment during 5 years in patients with badly controlled diabetes and nephropathy resisting to up-to-date nephroprotective therapies.Main secondary objectives are safety and efficacy of both regimens, including proteinuria and renal histology evaluation, metabolic control and quality of life, acute and chronic extrarenal complications of diabetes, pancreas survival and all risks related to the transplant procedure (anaesthesia, surgery and immunosuppression side-effects) and to the intensive insulin therapy management.

lead_sponsor: Nantes University Hospital

location_country:
Brazil
Italy
Czech Republic
United States
France

location_facility: Hôpital Edouard-Herriot - Hospices Civils de Lyon

location_facility: Diabetes Center - Institute for Clinical and Experimental Medicine

location_facility: Centre Hospitalier et Universitaire de Nantes

location_facility: Istituto Scientifico Ospedale San Raffaele

intervention_type: Procedure


title: The Cardiovascular Morbidity in Children With Chronic Renal Failure Study

summary:
Children and adolescents with chronic kidney disease (CKD) are at high risk for cardiovascular (CV) morbidity and mortality. Recent studies suggest that pediatric patients with even moderately impaired kidney function may be afflicted with significant early cardiac and vascular abnormalities.

The pathogenesis and the natural course of CV comorbidity in pediatric CKD patients is still elusive. In this multicenter, prospective, observational study the prevalence, degree and progression of CV comorbidity in children will be characterized and related to CKD progression. The morphology and function of the heart and vessels will be monitored by sensitive, non-invasive methods and will be compared with aged matched healthy controls. Multiple potential clinical, anthropometric, biochemical, and pharmacological risk factors will be monitored prospectively and will be related to CV status. Genotyping might identify predisposing genetic factors for progression of CV comorbidity and underlying nephropathies.

lead_sponsor: Heidelberg University

location_country:
Poland
Portugal
Serbia
United Kingdom
Italy
Austria
Lithuania
Czech Republic
Switzerland
Sweden
Hungary
France
Turkey
Belgium
Germany

location_facility: Haseki Educational and Research Hospital

location_facility: Center for Pediatrics and Adolescent Medicine

location_facility: Goztepe Educational and Research Hospital


title: A 2-Part Trial: a Randomized 6-day Repeat-dose, Parallel-group Study in Subjects With T2DM to Assess the Safety and Tolerability of GSK1614235 Compared to Placebo and Sitagliptin; and a Randomized Single-dose, Food Effect Study in Healthy Volunteers to Assess Safety and Tolerability of GSK1614235

summary:
The purpose of Part A of this study is to test whether repeated doses of the study drug (GSK1614235) are safe and well tolerated (i.e. do not produce unacceptable side effects) and whether we can obtain some preliminary information as to whether it works in lowering blood glucose levels. We will do this by comparing the effect of the study drug with placebo (no drug present) and against a drug (sitagliptin) known to control blood glucose in the treatment of diabetes.

The purpose of Part B of this study is to determine the how the timing of dosing, relative to meals, affects the response to study drug.

ReferenceCitation: Dobbins RL, Greenway FL, Chen L, Liu Y, Breed SL, Andrews SM, Wald JA, Walker A, Smith CD. Selective sodium-dependent glucose transporter 1 inhibitors block glucose absorption and impair glucose-dependent insulinotropic peptide release. Am J Physiol Gastrointest Liver Physiol. 2015 Jun 1;308(11):G946-54. doi: 10.1152/ajpgi.00286.2014. Epub 2015 Mar 12.

lead_sponsor: GlaxoSmithKline

location_country:
Germany
United States
United Kingdom
France

intervention_type: Other


title: A Phase I/II Multi-center, Open Label Study of TKI258 Administered Orally on an Intermittent Schedule in Adult Patients With Advanced or Metastatic Renal Cell Cancer (RCC)

location_country:
Taiwan
Spain
Netherlands
United States
France
Germany

summary: This is a phase I/II open-label study to delineate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of TKI258. The eligible subject population consists of subjects who have been diagnosed with advanced or metastatic renal cell cancer that is refractory to standard therapy or for which no curative standard therapy exists.


title: A Randomised, Controlled and Double-blind Clinical Investigation on the Effectiveness and Safety of a Foam Dressing Biatain Ibu Non-adhesive vs. Biatain Non-adhesive, in Painful Chronic Venous Leg Ulcers

summary: The objective of this investigation is to demonstrate the effectiveness and safety of Biatain Ibu non-adhesive foam dressing compared to Biatain non-adhesive foam dressing.

lead_sponsor: Coloplast A/S

location_country: Denmark France Germany Spain

location_facility: Department for Dermatology, University School of Medicine

location_facility: C.S. SERGAS Rosalía de Castro - Enfermero

location_facility: Fachartzpraxis für Dermatologie, Allergologie, Lasermedizin


title: Therapeutic Impact of Albumin Dialysis With the Molecular Adsorbents Recirculating System (MARS®) in Severely Decompensated Chronic Liver Disease

acronym: RELIEF

summary: The objective of this trial is to evaluate the impact of elimination of albumin bound substances during albumin dialysis (MARS®) on mortality and the clinical time course in patients with a recent severe clinical deterioration of chronic liver disease caused by a precipitating (trigger) event within 4 weeks manifested by jaundice, encephalopathy and/or renal failure.

ReferenceCitation: Stange J, Mitzner S, Ramlow W, Gliesche T, Hickstein H, Schmidt R. A new procedure for the removal of protein bound drugs and toxins. ASAIO J. 1993 Jul-Sep;39(3):M621-5.

ReferenceCitation: Stange J, Ramlow W, Mitzner S, Schmidt R, Klinkmann H. Dialysis against a recycled albumin solution enables the removal of albumin-bound toxins. Artif Organs. 1993 Sep;17(9):809-13.

lead_sponsor: Baxter Healthcare Corporation

location_country:
Denmark
Spain
Italy
Austria
United Kingdom
Switzerland
France
Belgium
Germany

location_facility: Martin Luther Universität Halle-Wittenberg


title: A Phase I/II Open Label Dose Escalation Study to Investigate the Safety and Tolerability of Acadesine in Patients With B-cell Chronic Lymphocytic Leukemia

summary: The main aim of this study is to test the safety of acadesine in patients with B-CLL and see what effects it has on patients and their leukaemia. The study also aims to examine the way acadesine is processed by the body. The study will look at the effects of acadesine in the body and the concentration of the drug and its main by-product (ZMP) in the blood to determine the dose and the frequency of dosing that is likely to be the most effective.

lead_sponsor: Advancell - Advanced In Vitro Cell Technologies, S.A.

location_country: France Spain Belgium

location_facility: Cliniques universitaires Saint-Luc Haematology Dept

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Service des maladies du sang Hôpital HURIEZ, CHRU

location_facility: Hematologia Clinica, Institut Catala d'Oncologia


title: A Multicenter, Phase III, Randomised Study of Photodynamic Therapy With Metvix Cream 160 mg/g in Comparison With Cryotherapy in Patients With Primary Superficial Basal Cell Carcinoma

summary:
The purpose of this study is to compare the efficacy of Photodynamic Therapy (PDT) with methyl aminolevulinate (MAL) cream to cryotherapy, in treatment of patients with primary superficial basal cell carcinoma.

Secondary objectives are to compare cosmetic outcome and tolerability (adverse events) in these patients, 3 months after treatment. In addition the recurrence rates in the two treatment groups will be compared up to five years after treatment.

lead_sponsor: Galderma R&D

location_country:
United Kingdom
Italy
Austria
Finland
France
Belgium
Sweden

location_facility: Service de Dermatologie, C.H.U Saint Louis


title: An Open-labeled Trial With a Dose-escalation Part and a Parallel Group Design(1) Investigating Zalutumumab, an Anti-EGF Receptor Antibody, in Combination With Chemo-Radiation as First Line Treatment of Patients With Cancer of the Head and Neck (1) The Parallel Group Part Was Cancelled

summary: The purpose of this study is to investigate the safety of zalutumumab in combination with chemotherapy and radiotherapy as treatment of patients with head and neck cancer

lead_sponsor: Genmab

location_country:
Sweden
United States
France
Belgium
Netherlands

location_facility: Hopital Bretonneau Clinique d'Oncologie et Radiothérapie

location_facility: Centre Georges-Francois Leclerc Hospital


title: Profile of Depressive Symptoms in Parkinson's Disease (PRODEST-PD)

summary: To explore or establish the relationship between cognitive, mood and motor symptoms in PD to scores on depression rating scales in a naturalistic setting.

lead_sponsor: Boehringer Ingelheim

location_country:
Spain
Italy
Austria
Netherlands
United Kingdom
Switzerland
France
Germany

location_facility: Universitatsklinikum Carl Gustav Carus Dresden

location_facility: Boehringer Ingelheim Investigational Site


title: Enzymatic Debridement in Burns Patients (Children & Adults): A Comparison to Standard of Care (Protocol MW 2004-11-02)

summary:
Burns represent one of the most severe and dreaded traumas. Burned and traumatized tissue is known as eschar. The dead eschar, if not removed, often becomes heavily contaminated and is the source of local and/or systemic infection or sepsis. The local inflammation and infection destroy healthy surrounding tissues and extends the original damage. In order to prevent these complications, and in order to minimize the risk of infection, it is imperative to evaluate the burn and remove all of the offending eschar at the earliest possible opportunity. This removal of dead tissue is termed "debridement".

The most direct debridement method for eschar removal is surgery. Traditional, conservative non-surgical debridement is a lengthy process which often involves many complications.

The objective of this study is to evaluate the safety and enzymatic debriding efficacy of Debrase Gel Dressing (DGD) in hospitalized patients with deep partial thickness and/or full thickness thermal burns and to compare DGD to standard of care (SOC).

ReferenceCitation: Rosenberg L, Lapid O, Bogdanov-Berezovsky A, Glesinger R, Krieger Y, Silberstein E, Sagi A, Judkins K, Singer AJ. Safety and efficacy of a proteolytic enzyme for enzymatic burn debridement: a preliminary report. Burns. 2004 Dec;30(8):843-50.

ReferenceCitation: Rosenberg L, Krieger Y, Bogdanov-Berezovski A, Silberstein E, Shoham Y, Singer AJ. A novel rapid and selective enzymatic debridement agent for burn wound management: a multi-center RCT. Burns. 2014 May;40(3):466-74. doi: 10.1016/j.burns.2013.08.013. Epub 2013 Sep 26.

lead_sponsor: MediWound Ltd

location_country:
Poland
Brazil
United Kingdom
Italy
Australia
France
Israel
Romania
Germany
Slovakia

location_facility: Direttore U.O. Chirurgia Plastica e Centro Ustioni Ospedale Civico

location_facility: Clinic of Burns and Reconstructive Surgery Hospital Kosice

location_facility: Hospital do Servidor Publico do Estado de Sao Paulo

location_facility: Center for Burns & Reconstructive Surgery, University Hopsital Bratislava

location_facility: Emergency Clinic Hospital "Bagdazar-Arsenie"

location_facility: Centre Hospitalier Regional et Universitaire de Marseille, Service de Chirurgie Plastique Reparatrice et Esthetique

location_facility: Pronto Socorro para Queimaduras de Goiania


type: Registry Identifier

title: A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Efficacy and Safety of TAK-475 or Placebo When Co-administered With Current Lipid-lowering Therapy in Subjects With Homozygous Familial Hypercholesterolemia.

summary: The purpose of the study is to determine the efficacy of lapaquistat acetate, once daily (QD), to lower cholesterol in subjects with homozygous familial hypercholesterolemia undergoing lipid-lowering treatment.

ReferenceType: result

ReferenceCitation: Stein EA, Bays H, O'Brien D, Pedicano J, Piper E, Spezzi A. Lapaquistat acetate: development of a squalene synthase inhibitor for the treatment of hypercholesterolemia. Circulation. 2011 May 10;123(18):1974-85. doi: 10.1161/CIRCULATIONAHA.110.975284. Epub 2011 Apr 25.

lead_sponsor: Takeda

location_country:
Poland
United Kingdom
Canada
United States
Israel
France


title: A Comparison of the Inflammatory Control of Asthma Provided by One Inhalation of Symbicort® Turbuhaler® 160/4.5 µg/Inhalation b.i.d. Plus As-needed Versus One Inhalation of Symbicort® Turbuhaler® 320/9 µg/Inhalation b.i.d. + One Inhalation of Pulmicort® Turbuhaler® 400 µg/Dose b.i.d. Plus Terbutaline Turbuhaler® 0.4 mg/Inhalation As-needed. A 12-month, Randomised, Double-blind, Parallel-group, Active Controlled, Multinational, Phase IIIB Study in Adult Patients With Asthma, EOS

summary: The purpose of this study is to determine if a flexible dose of Symbicort has a similar effect on the airways of patients with asthma as a higher fixed-dose of Symbicort with additional glucocorticosteroids added.

lead_sponsor: AstraZeneca

location_country:
Denmark
Spain
United Kingdom
Sweden
France
Germany


title: A Phase IIIb, Randomized, Open Label Study to Compare the Safety, Efficacy and Tolerability of Anagrelide Hydrochloride Versus Hydroxyurea in High-Risk Essential Thrombocythaemia Patients.

summary: Essential thrombocythaemia is a disorder of bone marrow, which causes too many platelets to be produced. Platelets are small cells carried around in the blood, which help form blood clots. When patients have too many platelets, there is a risk of blood clots forming unnecessarily and excessive bleeding. The aim of this study is to gain additional information on the safety profile of Anagrelide (Xagrid(r)) and Hydroxyurea (also known as hydroxycarbamide).

ReferenceCitation: Birgegård G, Folkvaljon F, Garmo H, Holmberg L, Besses C, Griesshammer M, Gugliotta L, Wu J, Achenbach H, Kiladjian JJ, Harrison CN. Leukemic transformation and second cancers in 3649 patients with high-risk essential thrombocythemia in the EXELS study. Leuk Res. 2018 Nov;74:105-109. doi: 10.1016/j.leukres.2018.10.006. Epub 2018 Oct 11.

ReferenceCitation: Gotic M, Egyed M, Gercheva L, Warzocha K, Kvasnicka HM, Achenbach H, Wu J. Cardiovascular Safety of Anagrelide Hydrochloride versus Hydroxyurea in Essential Thrombocythaemia. Cardiovasc Toxicol. 2021 Mar;21(3):236-247. doi: 10.1007/s12012-020-09615-0. Epub 2020 Oct 29.

lead_sponsor: Shire

location_country: Poland Portugal Serbia Hungary France Bulgaria

location_facility: Hopital Saint Louis - Centre d'Investigation Clinique

location_facility: CHU Angers Services des Maladies du Sang

location_facility: Klinika Hematologii Instytut Hematologii i Transfuzjologi

location_facility: University of Debrecen Medical and Health Science Centre

location_facility: University Multiprofile Hospital for Active Treatment ''Dr Georgi Stranski'' - Pleven

location_facility: University Multiprofile Hospital for Active Treament ''Sv. Marina'' - Varna Haematology Clinic

location_facility: Katedra i Klinika Onkologii i Chorob Wewnetrznych Akademii Medycznej

location_facility: University Multiprofile Hospital for active Treatment ''Alexandrovska'' Clinic of Haematology

location_facility: Institute for Haematology of Clinical Centre of Serbia

location_facility: Uniwersyteckie Centrum Kliniczne Katedra i Klinika Hematologii i Transplantologii

location_facility: Samodzielny Publiczny Szpital Kliniczny Nr 1


title: Non-Randomized, Prospective, Multi-center Evaluation of the ABSOLUTE™ .035 Peripheral Self-Expanding Stent System for Occluded or Stenotic Superficial Femoral or Proximal Popliteal Arteries

summary: The purpose of this study is to investigate the performance of the ABSOLUTE™ .035 peripheral self-expanding stent system in preventing restenosis of occluded or stenotic superficial femoral or proximal popliteal arteries.

lead_sponsor: Abbott Medical Devices

location_country: Spain Greece Austria Italy France Belgium Germany

location_facility: Universitäres Herz & Gefässzentrum Hamburg

location_facility: Allgemeines Krankenhaus der Stadt Wien (AKH Wien)


title: An EORTC Randomized, Double Blind, Placebo-Controlled, Phase II Multi-Center Trial Of Anastrozole (Arimidex) In Combination With ZD 1839 (Iressa) Or Placebo In Patients With Advanced Breast Cancer

summary:
RATIONALE: Estrogen can stimulate the growth of breast cancer cells. Hormone therapy using anastrozole may fight breast cancer by reducing the production of estrogen. Gefitinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Combining anastrozole with gefitinib may kill more tumor cells.

PURPOSE: Randomized phase II trial to compare the effectiveness of anastrozole with or without gefitinib in treating postmenopausal women who have metastatic or locally recurrent breast cancer.

ReferenceCitation: Mauriac L, Cameron D, Dirix L: Results of randomized phase II trial combining Iressa (gefitinib) and arimidex in women with advanced breast cancer (ABC): EORTC protocol 10021. [Abstract] 31st Annual San Antonio Breast Cancer Symposium, December 10-14, 2008, San Antonio, Texas. A-6133, 2008.

ReferenceCitation: Tryfonidis K, Basaran G, Bogaerts J, Debled M, Dirix L, Thery JC, Tjan-Heijnen VC, Van den Weyngaert D, Cufer T, Piccart M, Cameron D; EORTC-Breast Cancer Group. A European Organisation for Research and Treatment of Cancer randomized, double-blind, placebo-controlled, multicentre phase II trial of anastrozole in combination with gefitinib or placebo in hormone receptor-positive advanced breast cancer (NCT00066378). Eur J Cancer. 2016 Jan;53:144-54. doi: 10.1016/j.ejca.2015.10.012. Epub 2015 Dec 24.

lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

location_country:
United Kingdom
Slovenia
France
Belgium
Netherlands

location_facility: Universitair Medisch Centrum St. Radboud - Nijmegen

location_facility: Netherlands Cancer Institute - Antoni van Leeuwenhoek Hospital

location_facility: Edinburgh Cancer Centre at Western General Hospital


title: Oral Empirical Therapy of Fever in Low-Risk Neutropenic Cancer Patients: A Prospective, Double-Blind, Randomized, Multicenter Trial Comparing Monotherapy (Single Daily Dose Moxifloxacin) With Combination Therapy (Ciprofloxacin Plus Amoxicillin/Clavulanic Acid)

summary:
RATIONALE: Antibiotics such as amoxicillin, ciprofloxacin, and moxifloxacin may be effective in preventing or controlling fever and neutropenia in patients with cancer. It is not yet known whether moxifloxacin alone is more effective than amoxicillin combined with ciprofloxacin in treating neutropenia and fever.

PURPOSE: This randomized clinical trial is studying how well moxifloxacin works and compares it to ciprofloxacin together with amoxicillin in treating neutropenia and fever in patients with cancer.

primary_purpose: Supportive Care

ReferenceCitation: Kern WV, Marchetti O, Drgona L, Akan H, Aoun M, Akova M, de Bock R, Paesmans M, Viscoli C, Calandra T. Oral antibiotics for fever in low-risk neutropenic patients with cancer: a double-blind, randomized, multicenter trial comparing single daily moxifloxacin with twice daily ciprofloxacin plus amoxicillin/clavulanic acid combination therapy--EORTC infectious diseases group trial XV. J Clin Oncol. 2013 Mar 20;31(9):1149-56. doi: 10.1200/JCO.2012.45.8109. Epub 2013 Jan 28.

location_country: Italy Switzerland France Israel Turkey Belgium Germany Slovakia

location_facility: Hacettepe University - Faculty of Medicine

location_facility: Frauenklinik - Universitaetsklinikum Rostock am Klinikum Sudstadt

location_facility: Charite - Universitaetsmedizin Berlin - Campus Benjamin Franklin

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Medizinische Universitaetsklinik I at the University of Cologne

location_facility: Klinikum der Albert - Ludwigs - Universitaet Freiburg

location_facility: Istituto Nazionale per la Ricerca sul Cancro

location_facility: Ruprecht - Karls - Universitaet Heidelberg


title: A Study of Two Different Doses of ABT-378/Ritonavir in HIV-Infected Patients Who Have Taken Protease Inhibitors and Non-Nucleoside Reverse Transcriptase Inhibitors

summary: The purpose of this study is to determine the safety and effectiveness of a high dose of ABT-378/ritonavir

lead_sponsor: Abbott

location_country:
United States
Spain
France

location_facility: The University of North Carolina at Chapel Hill


title: Multicenter Phase III Open Label Randomized Trial Comparing CPT-11 In Combination With A 5-FU/FA Infusional Regimen To The Same 5-FU/FA Infusional Regimen Alone As Adjuvant Treatment Of Stage III Colon Cancer

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug and giving them after surgery may kill more tumor cells.

PURPOSE: Randomized phase III trial to compare the effectiveness of fluorouracil and leucovorin with or without irinotecan in treating patients who have undergone surgery for stage III colorectal cancer.

ReferenceCitation: Punt CJ, Buyse M, Köhne CH, Hohenberger P, Labianca R, Schmoll HJ, Påhlman L, Sobrero A, Douillard JY. Endpoints in adjuvant treatment trials: a systematic review of the literature in colon cancer and proposed definitions for future trials. J Natl Cancer Inst. 2007 Jul 4;99(13):998-1003. Epub 2007 Jun 27. Review.

ReferenceCitation: Roth AD, Tejpar S, Delorenzi M, Yan P, Fiocca R, Klingbiel D, Dietrich D, Biesmans B, Bodoky G, Barone C, Aranda E, Nordlinger B, Cisar L, Labianca R, Cunningham D, Van Cutsem E, Bosman F. Prognostic role of KRAS and BRAF in stage II and III resected colon cancer: results of the translational study on the PETACC-3, EORTC 40993, SAKK 60-00 trial. J Clin Oncol. 2010 Jan 20;28(3):466-74. doi: 10.1200/JCO.2009.23.3452. Epub 2009 Dec 14.

ReferenceCitation: Bosman FT, Yan P, Tejpar S, Fiocca R, Van Cutsem E, Kennedy RD, Dietrich D, Roth A. Tissue biomarker development in a multicentre trial context: a feasibility study on the PETACC3 stage II and III colon cancer adjuvant treatment trial. Clin Cancer Res. 2009 Sep 1;15(17):5528-33. doi: 10.1158/1078-0432.CCR-09-0741. Epub 2009 Aug 18.

ReferenceCitation: Roth AD, Tejpar S, Yan P, et al.: Correlation of molecular markers in colon cancer with stage-specific prognosis: results of the translational study on the PETACC 3 - EORTC 40993-SAKK 60-00 trial. [Abstract] American Society of Clinical Oncology 2009 Gastrointestinal Cancers Symposium, 15-17 January 2009, San Francisco, CA. A-288, 2009.

ReferenceCitation: Tejpar S, Bosman F, Delorenzi M, et al.: Microsatellite instability (MSI) in stage II and III colon cancer treated with 5FU-LV or 5FU-LV and irinotecan (PETACC 3-EORTC 40993-SAKK 60/00 trial). [Abstract] J Clin Oncol 27 (Suppl 15): A-4001, 2009.

ReferenceCitation: Van Cutsem E, Labianca R, Bodoky G, Barone C, Aranda E, Nordlinger B, Topham C, Tabernero J, André T, Sobrero AF, Mini E, Greil R, Di Costanzo F, Collette L, Cisar L, Zhang X, Khayat D, Bokemeyer C, Roth AD, Cunningham D. Randomized phase III trial comparing biweekly infusional fluorouracil/leucovorin alone or with irinotecan in the adjuvant treatment of stage III colon cancer: PETACC-3. J Clin Oncol. 2009 Jul 1;27(19):3117-25. doi: 10.1200/JCO.2008.21.6663. Epub 2009 May 18.

ReferenceCitation: Roth AD, Yan P, Dietrich D, et al.: Does UGT1A1*28 homozygosity predict for severe toxicity in patients treated with 5-fluorouracil (5-FU)-irinotecan (IRI)? Results of the PETACC 3-EORTC 40993-SAKK 60/00 trial comparing IRI/5-FU/folinic acid (FA) to 5-FU/FA in stage II-III colon cancer. [Abstract] American Society of Clinical Oncology 2008 Gastrointestinal Cancers Symposium, 25-27 January 2008, Orlando, FL. A-277, 2008.

ReferenceCitation: Roth AD, Yan P, Dietrich D, et al.: Is UGT1A1*28 homozygosity the strongest predictor for severe hematotoxicity in patients treated with 5-fluorouracil (5-FU)-irinotecan (IRI)? Results of the PETACC 3 - EORTC 40993 -SAKK 60/00 trial comparing IRI/5-FU/folinic acid (FA) to 5-FU/FA in stage II- III colon cancer (COC) patients. [Abstract] J Clin Oncol 26 (Suppl 15): A-4036, 2008.

ReferenceCitation: van Cutsem E, Labianca R, Hossfeld D, et al.: Randomized phase III trial comparing infused irinotecan / 5-fluorouracil (5-FU)/folinic acid (IF) versus 5-FU/FA (F) in stage III colon cancer patients (pts). (PETACC 3). [Abstract] J Clin Oncol 23 (Suppl 16): A-LBA8, 3s, 2005.

ReferenceCitation: Taieb J, Shi Q, Pederson L, Alberts S, Wolmark N, Van Cutsem E, de Gramont A, Kerr R, Grothey A, Lonardi S, Yoshino T, Yothers G, Sinicrope FA, Zaanan A, André T. Prognosis of microsatellite instability and/or mismatch repair deficiency stage III colon cancer patients after disease recurrence following adjuvant treatment: results of an ACCENT pooled analysis of seven studies. Ann Oncol. 2019 Sep 1;30(9):1466-1471. doi: 10.1093/annonc/mdz208.

ReferenceCitation: Klingbiel D, Saridaki Z, Roth AD, Bosman FT, Delorenzi M, Tejpar S. Prognosis of stage II and III colon cancer treated with adjuvant 5-fluorouracil or FOLFIRI in relation to microsatellite status: results of the PETACC-3 trial. Ann Oncol. 2015 Jan;26(1):126-132. doi: 10.1093/annonc/mdu499. Epub 2014 Oct 30.

lead_sponsor: Pfizer

location_country:
Portugal
Spain
Italy
Austria
United Kingdom
Switzerland
France
Belgium
Germany
Egypt

location_facility: Hopital Cantonal Universitaire de Geneva

location_facility: Universita Degli Studi di Firenze - Policlin. di Careggi

location_facility: Instituto Portugues de Oncologia do Porto


title: A Feasibility Study Of Primary Chemotherapy Followed By Concomitant Chemoradiation With And Without Amifostine In Patients With Locally Advanced Undifferentiated Nasopharyngeal Cancer (UNPC)

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining radiation therapy with chemotherapy may kill more tumor cells. Giving chemoprotective drugs such as amifostine may protect normal cells from the side effects of chemotherapy and radiation therapy.

PURPOSE: Randomized phase II trial to compare the effectiveness of paclitaxel and carboplatin followed by cisplatin plus radiation therapy with or without amifostine in treating patients who have locally advanced cancer of the nasopharynx.

location_country:
Spain
Italy
United Kingdom
Turkey
France
Belgium

location_facility: Istituto Nazionale per lo Studio e la Cura dei Tumori

location_facility: Istanbul University-Institute of Oncology

intervention_type: Radiation


title: A Randomized, Open-label, Multicenter, Multi-arm, Phase 1b/2 Platform Study to Evaluate Safety and Efficacy of Investigational Immunotherapies in Participants With Previously Treated Unresectable or Metastatic Melanoma

summary: This is a platform study evaluating the safety and efficacy of multiple novel investigational products (IPs) that target unresectable or metastatic cutaneous melanoma in participants who have failed standard treatment.

study_start_date_type: Anticipated

lead_sponsor: Innovent Biologics (Suzhou) Co. Ltd.

location_country:
Spain
United Kingdom
Australia
Switzerland
United States
France
Germany

location_facility: Hospital Universitario Reina Sofia, Cordoba

location_facility: University of California San Francisco Medical Center

location_facility: Cambridge University Hospitals NHS Foundation Trust (Oxford)

location_facility: Institut Catala d'Oncologia Hospital Universitari Germans Trials I Pujol, Barcelona

location_facility: Hospices Civil De Lyon Nord - Centre Hospitalier Lyon Sud - Dermatologie

intervention_type: Combination Product


title: A Phase IB Open-Label, Multicenter Study Evaluating the Safety, Efficacy, and Pharmacokinetics of Mosunetuzumab in Patients With Relapsed or Refractory Chronic Lymphocytic Leukemia

summary: This study will assess the safety, tolerability, pharmaokinetics, and preliminary efficacy of mosunetuzumab in participants with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL).

status: Recruiting

ipd_sharing: Yes

ipd_sharing_description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

lead_sponsor: Hoffmann-La Roche

location_country:
Spain
Italy
United Kingdom
Australia
United States
France
Germany

location_facility: Osp. San Raffaele; Dip. Di Oncoematologia

location_facility: Azienda Ospedaliera Di Perugia Ospedale s. Maria Della Misericordia; Oncologia Medica

location_facility: Uniklinik Koln; Klinik I fur Innere Medizin

location_facility: Churchill Hospital; Department of Oncology

location_facility: Hospital de la Santa Creu i Sant Pau; Servicio de Hematologia

location_facility: Hospital Universitari Vall d'Hebron; Servicio de Hematologia

location_facility: Princess Alexandra Hospital Woolloongabba; Clinical Hematology and Medical Oncology

location_facility: A.O. Spedali Civili Di Brescia-P.O. Spedali Civili;U.O. Ematologia

location_facility: CHU DE CLERMONT FERRAND; Service de Thérapie Cellulaire et d'Hématologie clinique adultes

location_facility: Universitätsklinikum Ulm; Medizinische Uni-Klinik III Abt. Innere Medizin III Hämatologie u. Onkolo.

location_facility: Memorial Sloan-Kettering Cancer Center; Hematology/Oncology

location_facility: ASST GRANDE OSPEDALE METROPOLITANO NIGUARDA; Struttura Complessa di Ematologia


title: A Phase I/IIa Open-label Dose Escalation and Expansion Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of AZD7789, an Anti-PD-1 and Anti-TIM-3 Bispecific Antibody, in Participants With Advanced or Metastatic Solid Tumors.

summary: This is a Phase I/IIa study designed to evaluate if experimental anti-PD-1 and anti-TIM-3 bispecific antibody, AZD7789 is safe, tolerable and efficacious in participants with advanced solid tumors.

ipd_sharing_description:
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal.

All request will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

location_country:
Spain
Canada
United States
France
Netherlands


title: Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis

summary:
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG.

Trial details include:

The maximum trial duration for each individual participant will be approximately 28 weeks
The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)

ipd_sharing: Undecided

lead_sponsor: argenx

location_country:
Poland
Spain
Italy
Austria
Georgia
Netherlands
United States
France
Belgium
Germany

location_facility:
Investigator Site 4 - FR0330032
Investigator Site 12 - FR0330041
Investigator Site 1 - GEO9950017
Investigator Site 7 - ES0340040
Investigator Site 13 - IT0390047
Investigator Site 9 - PL0480035
Investigator Site 16 - US0010123
Investigator Site 8 - PL0480038
Investigator Site 15 - US0010003
Investigator Site 6 - PL0480034
Investigator Site 2 - GEO9950018
Investigator Site 5 - BE0320021
Investigator Site 10 - NL0310001
Investigator Site 3 - ES0340041
Investigator Site 11 - AT0430010
Investigator Site 14 - DE0490032

intervention_type: Biological


title: A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Apremilast in Children From 5 to Less Than 18 Years of Age With Active Juvenile Psoriatic Arthritis (PEAPOD)

acronym: PEAPOD

summary: The study will aim to estimate the efficacy of apremilast compared with placebo in the treatment of juvenile psoriatic arthritis (JPsA) in pediatric participants 5 to less than 18 years of age.

ipd_sharing_description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

lead_sponsor: Amgen

location_country:
Poland
Portugal
Spain
Greece
Austria
Italy
Netherlands
United Kingdom
France
Turkey
Belgium
Germany

location_facility: Hospices Civils de Lyon Hopital Femme Mere Enfant

location_facility: SPZOZ Centralny Szpital Kliniczny Uniwersytetu Medycznego w Lodzi

location_facility: Centro Hospitalar Universtario de Sao Joao, EPE

location_facility: Ziekenhuis Netwerk Antwerpen Jan Palfijn

location_facility: Narodowy Instytut Geriatrii Reumatologii i Rehabilitacji im prof dr hab med Eleonory Reicher

location_facility: Charite Universitaetsmedizin Berlin Campus Virchow-Klinikum

location_facility: Wojewodzki Specjalistyczny Szpital Dzieciecy im sw Ludwika w Krakowie

location_facility: Hospital Universitari i Politecnic La Fe

location_facility: Istanbul Universitesi Istanbul Tip Fakultesi

location_facility: Azienda Socio Sanitaria Territoriale Centro Specialistico Ortopedico Traumatologico Gaetano Pini

location_facility: Istanbul Universitesi Cerrahpasa Tip Fakultesi

location_facility: Centre Hospitalier Regional de la Citadelle

location_facility: Asklepios Kinderklinik Sankt Augustin GmbH


acronym: ILLUMINATE-C

title: ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1)

lead_sponsor: Alnylam Pharmaceuticals

ReferenceCitation: Michael M, Groothoff JW, Shasha-Lavsky H, Lieske JC, Frishberg Y, Simkova E, Sellier-Leclerc AL, Devresse A, Guebre-Egziabher F, Bakkaloglu SA, Mourani C, Saqan R, Singer R, Willey R, Habtemariam B, Gansner JM, Bhan I, McGregor T, Magen D. Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial. Am J Kidney Dis. 2022 Jul 14. pii: S0272-6386(22)00771-5. doi: 10.1053/j.ajkd.2022.05.012. [Epub ahead of print]

location_country:
Jordan
Italy
Australia
Lebanon
United States
France
Israel
Turkey
Belgium
Netherlands
United Arab Emirates

summary: The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1 (PH1).


title: An Open-Label, Multi-Centre Phase I/IIa Study Evaluating the Safety and Clinical Activity of Neoantigen Reactive T Cells in Patients With Advanced Non-Small Cell Lung Cancer

summary: This is a first-in-human, open-label, multi-centre, phase I/IIa study to characterise the safety and clinical activity autologous clonal neoantigen reactive T cells (cNeT) administered intravenously in adults with advanced non-small cell lung cancer (NSCLC).

lead_sponsor: Achilles Therapeutics UK Limited

location_country:
United Kingdom
Spain
United States
France
Germany

location_facility: University College London Hospital (UCLH)

location_facility: H Lee Moffitt Cancer Center and Research Unit

location_facility: Universitätsklinikum Carl Gustav Carus an der TU Dresden Anstalt des öffentlichen Rechts des Freistaates Sachsen

location_facility: University Hospital Southampton NHS Foundation Trust

location_facility: Guys and St Thomas' NHS Foundation Trust

location_facility: Hospital Universitario Fundación Jiménez Díaz

location_facility: Cambridge University Hospitals NHS Foundation Trust

location_facility: University Hospitals Birmingham NHS Foundation Trust


title: Stereotactic Ablative Body Radiotherapy (SABR) Combined With Immunotherapy (L19-IL2) in Stage IV NSCLC Patients, ImmunoSABR: a Multicentre, Randomised Controlled Open-label Phase II Trial

summary:
This will be a phase II trial testing if the combination of stereotactic ablative body radiotherapy (SABR) and L19-IL2 improve the progression-free survival in patients with limited metastatic non-small cell lung cancer (NSCLC). The trial consists of one cohort with two arms; C-arm and an E-arm.

Patients with oligometastatic disease will receive SABR to minimal 1 and max all metastatic sites (max 5 sites irradiated) and patients with diffuse metastatic lesions (6 to max 10) will receive radiotherapy to max 5 sites. In the experimental arm, immunotherapy will be given after irradiation.

ReferenceCitation: Olivo Pimentel V, Marcus D, van der Wiel AM, Lieuwes NG, Biemans R, Lieverse RI, Neri D, Theys J, Yaromina A, Dubois LJ, Lambin P. Releasing the brakes of tumor immunity with anti-PD-L1 and pushing its accelerator with L19-IL2 cures poorly immunogenic tumors when combined with radiotherapy. J Immunother Cancer. 2021 Mar;9(3). pii: e001764. doi: 10.1136/jitc-2020-001764.

ReferenceCitation: Lieverse RIY, Van Limbergen EJ, Oberije CJG, Troost EGC, Hadrup SR, Dingemans AC, Hendriks LEL, Eckert F, Hiley C, Dooms C, Lievens Y, de Jong MC, Bussink J, Geets X, Valentini V, Elia G, Neri D, Billiet C, Abdollahi A, Pasquier D, Boisselier P, Yaromina A, De Ruysscher D, Dubois LJ, Lambin P. Stereotactic ablative body radiotherapy (SABR) combined with immunotherapy (L19-IL2) versus standard of care in stage IV NSCLC patients, ImmunoSABR: a multicentre, randomised controlled open-label phase II trial. BMC Cancer. 2020 Jun 15;20(1):557. doi: 10.1186/s12885-020-07055-1.

lead_sponsor: Maastricht University

location_country:
United Kingdom
Italy
Netherlands
France
Belgium
Germany

location_facility: INSTITUT régional du CANCER MONTPELLIER - ICM - VAL d'AURELLE

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS Università Cattolica del Sacro Cuore

location_facility: Academisch Ziekenhuis Maastricht (Leading Centre)


acronym: LEADER 75

title: LowEr Administered Dose With highEr Relaxivity: Gadovist vs Dotarem (LEADER 75)

lead_sponsor: Bayer

ReferenceCitation: Liu BP, Rosenberg M, Saverio P, Weon YC, Peters S, Ardellier FD, Boeckenhoff A, Endrikat J. Clinical Efficacy of Reduced-Dose Gadobutrol Versus Standard-Dose Gadoterate for Contrast-Enhanced MRI of the CNS: An International Multicenter Prospective Crossover Trial (LEADER-75). AJR Am J Roentgenol. 2021 Nov;217(5):1195-1205. doi: 10.2214/AJR.21.25924. Epub 2021 Jun 16.

location_country:
United Kingdom
Italy
Switzerland
Korea, Republic of
United States
France
Germany

location_facility: Universitätsklinikum Schleswig-Holstein / AÖR

location_facility: University of Texas Southwestern Medical Center

location_facility: Universität Rostock - Medizinische Fakultät

location_facility: Penn State Milton S. Hershey Medical Center

summary:
The study was conducted to gain knowledge about a new dose of a diagnostic drug that is used for contrast-enhanced Magnetic Resonance Imaging (MRI) of the human central nervous system (CNS). MRI can visualize the anatomy of the body and is used to detect medical conditions. Diagnostic drugs like gadobutrol and gadoterate contain an element called gadolinium that is applied to improve the analysability of MRI-images.

The purpose of this study was to examine if contrast-enhanced MRI using a reduced dose of the gadolinium-based contrast agent gadobutrol delivers images of similar quality to those obtained when a full dose of the gadolinium-based contrast agent gadoterate was used.


title: A Phase 1/2 Open-label Safety and Dose-finding Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A

ipd_sharing_description:
Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

location_country:
United Kingdom
Netherlands
United States
France
Germany
Bulgaria

location_facility: Hopital Necker les enfants malades - Paris

location_facility: SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD

summary: In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the disease. By replacing the defective gene with a healthy copy the human body may produce clotting factor on its own. Hemophilia A is a bleeding disorder in which the human body does not have enough clotting factor VIII, a protein that controls bleeding. Researcher want to find the optimal dose of BAY 2599023 (DTX201) so that the body may produce enough clotting factor on its own.


title: Trial to Evaluate Treatment With Abbott Transcatheter Clip Repair System in Patients With Moderate or Greater Tricuspid Regurgitation (TRILUMINATE)

acronym: TRILUMINATE

summary: The primary purpose of this study is to evaluate safety and effectiveness of the Tricuspid Valve Repair System (TVRS) for treating symptomatic moderate or greater tricuspid regurgitation (TR) in patients currently on medical management and who are deemed appropriate for percutaneous transcatheter intervention.

ReferenceCitation: Lurz P, Stephan von Bardeleben R, Weber M, Sitges M, Sorajja P, Hausleiter J, Denti P, Trochu JN, Nabauer M, Tang GHL, Biaggi P, Ying SW, Trusty PM, Dahou A, Hahn RT, Nickenig G; TRILUMINATE Investigators. Transcatheter Edge-to-Edge Repair for Treatment of Tricuspid Regurgitation. J Am Coll Cardiol. 2021 Jan 26;77(3):229-239. doi: 10.1016/j.jacc.2020.11.038.

ReferenceCitation: Nickenig G, Weber M, Lurz P, von Bardeleben RS, Sitges M, Sorajja P, Hausleiter J, Denti P, Trochu JN, Näbauer M, Dahou A, Hahn RT. Transcatheter edge-to-edge repair for reduction of tricuspid regurgitation: 6-month outcomes of the TRILUMINATE single-arm study. Lancet. 2019 Nov 30;394(10213):2002-2011. doi: 10.1016/S0140-6736(19)32600-5. Epub 2019 Nov 7. Erratum in: Lancet. 2020 Mar 14;395(10227):870.

location_country:
Spain
Italy
Switzerland
United States
France
Germany

location_facility: Clinical and Provincial Hospital of Barcelona

location_facility: HerzKlinik Hirslanden - Klinik Hirslanden

location_facility: Universitatsmedizin der Johannes Gutenberg-Universitat Mainz

location_facility: Ludwig-Maximilian University of Munich (LMU)

location_facility: Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele - Presidio Ferrarotto


title: A Randomized, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effects on Heterotopic Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva

summary:
This is a three period study design consisting of a 6-month, randomized, double-blind placebo-controlled treatment (period 1) followed by a 6-month, open-label treatment (period 2) and a follow-up treatment period (period 3).

Primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP).

Primary efficacy objective of the study is to assess the effect of REGN2477 versus placebo on the change from baseline in heterotopic ossification (HO) in patients with FOP, as determined by 18-NaF uptake in HO lesions by positron emission tomography (PET) and in total volume of HO lesions by computed tomography (CT).

Key Secondary objectives are:

To compare the effect of REGN2477 versus placebo on pain due to FOP, as measured by the area under the curve (AUC) for pain based on daily pain numeric rating scale (NRS) scores
To assess the effect of REGN2477 versus placebo on the change from baseline in HO, as determined by the number of new HO lesions identified by 18F-NaF PET or by CT
To assess the effect of REGN2477 versus placebo on the change from baseline in 18F-NaF standardized uptake value maximum (SUVmax) of individual active HO site(s) by PET
To assess the effect of REGN2477, between week 28 and week 56, on the number, activity, and volume of HO lesions identified by 18F-NaF PET or by CT in patients who switch from placebo to REGN2477 at week 28 versus the same patients between baseline and week 28
To assess the effect of REGN2477 versus placebo on the change from baseline in biochemical markers of bone formation
To characterize the concentrations of total activin A at baseline and over time following the first dose of study drug
To characterize the concentration-time profile (pharmacokinetics [PK]) of REGN2477 in patients with FOP
To assess the immunogenicity of REGN2477

ReferenceCitation: Vanhoutte F, Liang S, Ruddy M, Zhao A, Drewery T, Wang Y, DelGizzi R, Forleo-Neto E, Rajadhyaksha M, Herman G, Davis JD. Pharmacokinetics and Pharmacodynamics of Garetosmab (Anti-Activin A): Results From a First-in-Human Phase 1 Study. J Clin Pharmacol. 2020 Nov;60(11):1424-1431. doi: 10.1002/jcph.1638. Epub 2020 Jun 18.

ReferenceCitation: Aykul S, Corpina RA, Goebel EJ, Cunanan CJ, Dimitriou A, Kim HJ, Zhang Q, Rafique A, Leidich R, Wang X, McClain J, Jimenez J, Nannuru KC, Rothman NJ, Lees-Shepard JB, Martinez-Hackert E, Murphy AJ, Thompson TB, Economides AN, Idone V. Activin A forms a non-signaling complex with ACVR1 and type II Activin/BMP receptors via its finger 2 tip loop. Elife. 2020 Jun 9;9. pii: e54582. doi: 10.7554/eLife.54582.

lead_sponsor: Regeneron Pharmaceuticals

location_country:
Poland
Spain
Italy
United Kingdom
Canada
United States
France
Netherlands

location_facility: Hopital Lariboisiere,Hospitalier Universitaire Nord

location_facility: Royal National Orthopaedic Hospital, Brockley Hill


acronym: Expanded treatment protocol for CTL019.

title: Phase IIIb Study for Relapsed/Refractory Pediatric/Young Adult Acute Lymphoblastic Leukemia Patients to be Treated With CTL019

lead_sponsor: Novartis Pharmaceuticals

ReferenceCitation: Laetsch TW, Maude SL, Balduzzi A, Rives S, Bittencourt H, Boyer MW, Buechner J, De Moerloose B, Qayed M, Phillips CL, Pulsipher MA, Hiramatsu H, Tiwari R, Grupp SA. Tisagenlecleucel in pediatric and young adult patients with Down syndrome-associated relapsed/refractory acute lymphoblastic leukemia. Leukemia. 2022 Jun;36(6):1508-1515. doi: 10.1038/s41375-022-01550-z. Epub 2022 Apr 14.

ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

location_country: Spain Italy Austria Canada France Norway Belgium Japan Germany

summary: This is a single arm, open-label, multi-center, phase III B study to determine the safety and efficacy of CTL019 in pediatric/young adult patients with r/r B-cell Acute Lymphoblastic Leukemia (ALL).


title: A Real World Evaluation of the ELUVIA Drug Eluting Stent in All-Comers With Superficial Femoral Artery and Proximal Popliteal Artery Disease

acronym: REGAL

summary: The REGAL study is a European, prospective, multi-center Post-Market Clinical Follow-up (PMCF) trial providing additional data including health economics data to support the use of the ELUVIA stent in the treatment of lesions located in the femoropopliteal arteries.

lead_sponsor: Boston Scientific Corporation

location_country: Spain Italy Austria France Belgium

location_facility: Centre Hospitalier Universitaire Grenoble-Alpes

location_facility: Hospital Azienda Ospedaliera Universitaria Senese

location_facility: Foundation for the Aragonese Healthcare Research Institute

location_facility: Hosp. Complejo Hosp Universitario (CHUO)


title: A Phase 2 Randomized Open-Label Study of Oral Darolutamide (ODM-201) vs. Androgen Deprivation Therapy (ADT) With LHRH Agonists or Antagonist in Men With Hormone Naive Prostate Cancer

summary:
This is an open label non-comparative controlled randomized phase II study. The experimental arm is the group receiving ODM-201. The group receiving androgen-deprivation therapy (ADT) is included as an internal control.

The primary trial objective is to demonstrate that ODM-201 produces prostate-specific antigen (PSA) response rates at 24 weeks (defined as ≥80% reduction compared to baseline) that are in the range of those achieved with 24 weeks of ADT.

In total, this 1:1 randomized study will therefore require randomization of at least 250 patients, 125 to each arm.

location_country: France Italy Spain Belgium

location_facility: CHU de Dijon - Centre Georges-Francois-Leclerc

location_facility: Fundacion Instituto Valenciano De Oncologia

location_facility: Institut Catala d'Oncologia - ICO Badalona - Hospital Germans Trias i Pujol

location_facility: Hospital Universitario Virgen De La Victoria

location_facility: Azienda Ospedaliera Citta della Salute e della Scienza di Torino - Ospedale Molinette

location_facility: Hopitaux Universitaires Bordet-Erasme - Hopital Universitaire Erasme


title: MERIT - A Phase II Marker Identification Trial for Tarceva in Second Line NSCLC Patients

acronym: MERIT

summary: This study will assess potentially predictive markers of efficacy in participants with NSCLC receiving oral erlotinib (Tarceva) therapy. The anticipated time on study treatment is until disease progression, unacceptable toxicity or death.

location_country:
Poland
Taiwan
Hong Kong
Spain
Ireland
Italy
Estonia
Russian Federation
United Kingdom
France
Singapore
Belgium
Germany
Bulgaria


title: A Phase I Dose Finding Study of Oral LXH254 in Adult Patients With Advanced Solid Tumors Harboring MAPK Pathway Alterations

summary: A Phase I Study of LXH254 in Patients With Advanced Solid Tumors That Harbor MAPK Pathway Alterations.

location_country:
Spain
Italy
Netherlands
Canada
Switzerland
Korea, Republic of
United States
France
Japan
Germany

location_facility: UT M.D Anderson Cancer Center SC - LXH254X2101

location_facility: Massachusetts General Hospital MGH Cancer Center

location_facility: Memorial Sloan Kettering Cancer Center SC - LXH254X2101


title: An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy

acronym: FSHD

summary: The purpose of this study is to assess the safety and biological activity of ATYR1940 in patients with early onset facioscapulohumeral muscular dystrophy (FSHD).

lead_sponsor: aTyr Pharma, Inc.

location_country:
United States
Italy
France

location_facility: Centre d'nvestigation Clinique - Centre de Pharmacologie Clinique et d'Evaluations Thérapeutiques (CICCPCET)

location_facility: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta


title: A Phase I, Multicenter, Open-label, Single-sequence Drug-drug Interaction Study to Assess the Effect of INC280 on the Pharmacokinetics of Midazolam and Caffeine in Patients With cMET-dysregulated Advanced Solid Tumors

summary: Drg-drug Interaction (DDI) study to assess the effect of INC280 on the pharmacokinetics of midazolam and caffeine in patients with cMET-dysregulated advanced solid tumors

location_country:
Denmark
Spain
Italy
United Kingdom
United States
France
Bulgaria

location_facility: Washington University School of Medicine

location_facility: Emory University School of Medicine/Winship Cancer Institute SC-2


primary_purpose: Screening

acronym: MORA

title: A Multicenter Randomized Parallel Group Phase III Study Comparing the Bowel Cleansing Efficacy, Safety and Tolerability of NER1006 (a Low Volume Bowel Cleansing Solution) Versus MOVIPREP Using 2-Day Split-Dosing and 1-Day Morning Split-Dosing Regimen in Adults.

lead_sponsor: Norgine

ReferenceCitation: Cash BD, Allen C, Poppers DM. Transient alterations in plasma sodium concentrations with NER1006 bowel preparation: an analysis of three phase III, randomized clinical trials. BMC Gastroenterol. 2022 Sep 5;22(1):412. doi: 10.1186/s12876-022-02484-7.

ReferenceCitation: Bisschops R, Manning J, Clayton LB, Ng Kwet Shing R, Álvarez-González M; MORA Study Group. Colon cleansing efficacy and safety with 1 L NER1006 versus 2 L polyethylene glycol + ascorbate: a randomized phase 3 trial. Endoscopy. 2019 Jan;51(1):60-72. doi: 10.1055/a-0638-8125. Epub 2018 Jul 19.

location_country:
Poland
Spain
Italy
United Kingdom
France
Belgium
Germany

location_facility: Centro di Riferimento Oncologico (C.R.O.) S.O.C Gastroenterologia

location_facility: Hôpital Avicenne- Service de Gastro-Entérologie

location_facility: Hospital General Universitario de Alicante

location_facility: Praxis für Innere Medizin, Gastroenterologie und Allg. Medizin

location_facility: Klinikum der Friedrich Schiller Universität Jena

location_facility: Royal Albert Edward Infirmary Department

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: Ospedale Valduce U.O. Gastroenterologia e Endoscopia

location_facility: A.O.U. di Bologna - Policlinico S. Orsola-Malpighi

location_facility: Uniwersytecki Szpital Kliniczny w Bialymstoku

location_facility: NZOZ Centrum Medyczne-Szpital Swietej Rodziny

location_facility: Centralny Szpital Kliniczny Ministerstwa Spraw Wewnetrznych

location_facility: Gabinet Internistyczny dr n. med. Krzysztof Janik

location_facility: Pol. Univ. A. Gemelli U.O. di Endoscopia Digestiva Chirurgica

location_facility: Hospital Ramon y Cajal - Ctra. De Colmenar km. 9, 100

location_facility: Kliniken Essen-Mitte; Abteilung für Gastroenterologie

summary: This study evaluates the efficacy, safety and tolerability of NER1006 versus MOVIPREP in adult patients requiring bowel cleansing prior to any procedure that requires a clean bowel, using a 2-Day evening/morning Split-Dosing and 1-Day morning only Split-Dosing regimens. Approximately 810 patients will be randomised with the aim of achieving a minimum of 245 patients in each of the 3 groups.


title: A Prospective Colorectal Liver Metastasis Database With an Integrated Quality Assurance Program

acronym: CLIMB

summary:
This prospective database has two main objectives;

to evaluate the complication rates, 30-day and 90-day mortality from different surgical strategies for unresectable, borderline resectable or initially unresectable liver metastasis from colorectal cancer.
to establish baseline quality parameters for different surgical strategies for unresectable, borderline and initially unresectable colorectal liver metastasis (CRLM) patients.

location_country:
Denmark
Serbia
Spain
Italy
Austria
Netherlands
United Kingdom
Switzerland
Sweden
France
Belgium
Germany

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

location_facility: Der Wiener Krankenanstaltenverbund - Krankenanstalt Rudolfstiftung


title: An Open Label Study to Evaluate the Effects of Ezogabine/Retigabine Added to Existing Anti-epileptic Drug(s) on Urinary Voiding Function in Subjects With Partial Onset Seizures

summary:
This is a multicentre, open label study to examine the effect of ezogabine/retigabine on the voiding function of adult subjects with drug-resistant partial onset seizures (POS). Subjects fulfilling the study entry criteria at Screening and at Baseline including a comprehensive eye examination by an ophthalmologist or retina specialist and a skin assessment by the investigator will receive ezogabine/retigabine.

The starting dose of ezogabine/retigabine will be 300 mg/day. Subjects will be up titrated by 150 mg/day weekly up to the maximum ezogabine/retigabine daily dose of 1200 mg (or the highest tolerated dose). During the 49 days of the treatment phase, subjects will undergo three repeat non-invasive assessments of voiding function. In addition, subjects who meet pre-determined criteria for voiding dysfunction will undergo multichannel cystometry in order to characterise bladder hypocontractility, bladder outlet obstruction or a combination of events which clinically is manifest with difficulty emptying the bladder or acute urinary retention.

At the end of the Treatment Phase, all subjects will enter the Taper Phase, a 3-week down titration period. Subjects who have new findings of abnormal pigmentation of the retina, unexplained vision loss, pigmentation of non-retinal ocular tissue or discoloration of skin, lip, nail, or mucosa since baseline will be asked to enter the Safety Follow-Up / Continuation Phase. All subjects will undergo 6-monthly comprehensive eye examinations during the Safety Follow-Up / Continuation Phase. Subjects who have not developed abnormal discoloration of the skin, lips, nails or mucosa will continue to undergo skin assessments by the investigator. Any subject who has developed abnormal discoloration of the skin, lips, nails or mucosa since baseline will be referred to a dermatologist for evaluation and 6-monthly follow up assessments. All subjects will continue to be followed until the pigmentation and/or discoloration has resolved or stabilised, as defined by no change over 2 consecutive 6-monthly assessments conducted over at least 12 months after discontinuation of ezogabine/retigabine.

lead_sponsor: GlaxoSmithKline

location_country:
Poland
United States
France


title: An Open Label, 52-week, Safety and Tolerability Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.

summary: This study was to determine long-term safety and tolerability, and continued efficacy in lowering triglycerides of LCQ908 in subjects with Familial Chylomicronemia Syndrome (FCS) (HLP type I).

location_country:
United Kingdom
Netherlands
Canada
United States
France
South Africa
Germany


title: An Open-label, Multi-center Phase I Dose-finding Study of RAD001 (Everolimus, Afinitor®) in Combination With BEZ235 in Patients With Advanced Solid Tumors

summary:
Study has two parts:

Dose-finding: to determine the maximum tolerated dose (MTD) and to evaluate the safety and tolerability of RAD001 (everolimus , Afinitor®) in combination with BEZ235 in patients with advanced solid tumors.
Dose-expansion: to assess safety and tolerability of RAD001 and BEZ235 at the MTD in patients with ER+/HER2- metastatic breast cancer and metastatic renal cell cancer

location_country:
Spain
Italy
United Kingdom
New Zealand
United States
France
Belgium

location_facility: Highlands Oncology Group Dept of Highlands Oncology Grp

location_facility: Washington University School of Medicine Washington University (16)


title: A Multi-arm Dose-finding Phase Ib Multicenter Study of Imatinib in Combination With the Oral Phosphatidyl-inositol 3-kinase (PI3-K) Inhibitor BKM120 in Patients With Gastrointestinal Stromal Tumor (GIST) Who Failed Prior Therapy With Imatinib and Sunitinib

summary: The purpose of this study is to determine a maximum tolerated dose and/or recommended phase 2 dose of a combination of imatinib and BKM120 in the treatment of 3rd line GIST patients.

ReferenceCitation: Gelderblom H, Jones RL, George S, Valverde Morales C, Benson C, Jean-Yves Blay, Renouf DJ, Doi T, Le Cesne A, Leahy M, Hertle S, Aimone P, Brandt U, Schӧffski P. Imatinib in combination with phosphoinositol kinase inhibitor buparlisib in patients with gastrointestinal stromal tumour who failed prior therapy with imatinib and sunitinib: a Phase 1b, multicentre study. Br J Cancer. 2020 Apr;122(8):1158-1165. doi: 10.1038/s41416-020-0769-y. Epub 2020 Mar 9.

location_country:
Spain
United Kingdom
Canada
United States
France
Belgium
Japan
Netherlands

location_facility:
Novartis Investigative Site
Dana Farber Cancer Institute SC (2)
Seattle Cancer Care Alliance Onc


title: A Multi-center, Open Label, First in Human Phase I Dose Escalation Study of Single Agent RO5503781, a Small Molecule MDM2 Antagonist, Administered Orally in Patients With Advanced Malignancies, Except Leukemia

summary: This multicenter, open label, dose-escalating study will evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of RO5503781, administered once daily (QD) or once weekly (QW) in participants with advanced malignancies except leukemia. Participants will receive multiple escalating oral doses in two different dosing schedules (Sch) until disease progression or unacceptable toxicity occurs.

ReferenceCitation: Italiano A, Miller WH Jr, Blay JY, Gietema JA, Bang YJ, Mileshkin LR, Hirte HW, Higgins B, Blotner S, Nichols GL, Chen LC, Petry C, Yang QJ, Schmitt C, Jamois C, Siu LL. Phase I study of daily and weekly regimens of the orally administered MDM2 antagonist idasanutlin in patients with advanced tumors. Invest New Drugs. 2021 Dec;39(6):1587-1597. doi: 10.1007/s10637-021-01141-2. Epub 2021 Jun 28.

location_country:
Australia
Canada
Korea, Republic of
France
Netherlands


title: A Multicentre, Open-Label, Long-Term, Safety and Tolerability Study of Retigabine Immediate Release (IR) in Adults With Partial-Onset Seizures (Extension of Study RGB113905)

acronym: IR

summary: The purpose of this Phase III study is to assess the long-term safety, tolerability and efficacy of flexibly dosed retigabine Immediate Release (IR) as adjunctive therapy in adult subjects with partial-onset seizures. In addition, those subjects who successfully completed 20 weeks of adjunctive treatment with retigabine IR in the parent study, RGB113905, and who were thought to have benefitted from treatment will be provided continued access to retigabine IR.

ipd_sharing_description: IPD for this study will be made available via the Clinical Study Data Request site.

location_country:
Poland
Italy
Russian Federation
Netherlands
Thailand
Ukraine
France
Belgium
Germany
Bulgaria


title: A Proof of Concept, Open-label, Forced Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of 10-weeks Treatment of LCI699 in Patients With Cushing's Disease

summary:
This exploratory study is a proof of concept study to determine whether LCI699 can safely reduce the level of urinary free cortisol in patients with Cushing's disease.

In addition, this study evaluated the long term efficacy and safety of LCI699 including an additional 12 week of treatment followed by a 12 month long term optional extension.

A second extension provided patients who were clinically benefitting from LCI699 an opportunity to continue to have access to the drug until LCI699 was commercially available and reimbursed or through the availability of a local access program.

ReferenceCitation: Bertagna X, Pivonello R, Fleseriu M, Zhang Y, Robinson P, Taylor A, Watson CE, Maldonado M, Hamrahian AH, Boscaro M, Biller BM. LCI699, a potent 11β-hydroxylase inhibitor, normalizes urinary cortisol in patients with Cushing's disease: results from a multicenter, proof-of-concept study. J Clin Endocrinol Metab. 2014 Apr;99(4):1375-83. doi: 10.1210/jc.2013-2117. Epub 2013 Dec 11.

location_country:
Japan
United States
Italy
France

location_facility: Massachusetts General Hospital Neuroendocrine Unit

location_facility: Northwestern University Endo, Metabolism and Molecular


title: A 2-year, Multicenter, Double-masked, Randomized, Parallel Study of the Safety of LUMIGAN® 0.1 mg/mL Compared With LUMIGAN® 0.3 mg/mL in Patients With Glaucoma or Ocular Hypertension

summary: This study will evaluate the long-term safety of bimatoprost 0.01% ophthalmic solution compared with bimatoprost 0.03% ophthalmic solution in patients with glaucoma or ocular hypertension.

lead_sponsor: Allergan

location_country:
Poland
Spain
Italy
United Kingdom
Hungary
France
Israel
Belgium
Germany
Czechia

location_facility: Dipartimento di Scienze Otorino-Odonto-Oftalmologiche e Cerv

location_facility: Department of Ophthalmology, University of Tuebingen

location_facility: Azlenda Ospedaliero Universitaria Careggi Viale

location_facility: Fundacion Oftalmologica Del Mediterraneo

location_facility: Centre Hospitalier Universitaire de Bordeaux

location_facility: Fondazione G.B. Bietti per l'Oftalmologia I.R.C.C.S.

location_facility: Akad. Med. w Warszawie - Ketedra I Klin. Okulistki Wyzialu L

location_facility: Szpital Kliniczny Przemienienia Panskiego Uniwersytetu

location_facility: Huntingdon Glaucoma Diagnostic & Research Centre

location_facility: University of Szeged Szent-Gyorgyi Albert Clinical Center

location_facility: Osrodek Badan Klinicznych Euromedis Sp. z o.o.

location_facility: Glaucoma Service The Rabin Medical Center