title: An Open-Label, Multicenter, Dose-Escalation Phase I Study to Evaluate the Safety, Pharmacokinetics, and Therapeutic Activity of RO6958688, A Novel T-cell Bispecific Antibody That Targets the Human Carcinoembryonic Antigen (CEA) on Tumor Cells and CD3 on T Cells, Administered Intravenously in Patients With Locally Advanced and/or Metastatic CEA(+) Solid Tumors

summary: Study BP29541 is a first-in-human, open-label, multi-center, dose-escalation Phase I clinical study of single-agent RO6958688 in participants with locally advanced and/or metastatic carcinoembryonic antigen (CEA) positive solid tumors who have progressed on standard treatment, are intolerant to standard of care (SOC), and/or are non-amenable to SOC. The study will be conducted in two parts. Part I of the study will investigate the safety and pharmacokinetics of a single dose of RO6958688 in single participant cohorts with dosing starting from a minimal anticipated biological effect level dose of 0.05 milligrams (mg) and up to a maximum dose of 2.5 mg. Part II will establish the appropriate therapeutic dose based on safety, pharmacokinetics, and the maximum tolerated dose (MTD) of RO6958688 for the once per week (QW) regimen, every three weeks (Q3W) regimen, and for the step up dosing regimen.

ReferenceCitation: Martinez-Sabadell A, Morancho B, Rius Ruiz I, Roman Alonso M, Ovejero Romero P, Escorihuela M, Chicote I, Palmer HG, Nonell L, Alemany-Chavarria M, Klein C, Bacac M, Arribas J, Arenas EJ. The target antigen determines the mechanism of acquired resistance to T cell-based therapies. Cell Rep. 2022 Oct 18;41(3):111430. doi: 10.1016/j.celrep.2022.111430.

location_country:
Denmark
Spain
Italy
Canada
United States
France
Netherlands

location_facility: Cedars Sinai Medical Center; Samuel-Oschin Comprehensive Cancer Institute

location_facility: Hospital Univ Vall d'Hebron; Servicio de Oncologia

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: IRCCS IST. Tumori Fondaz. Pascale; S.C. Oncologia Medica,Melanoma,Immunoterapia E Terapie Innovative

location_facility: Centre Leon Berard; Departement Oncologie Medicale

location_facility: Azienda Ospedaliera Universitaria Senese, U.O.C. Immunoterapia Oncologica

location_facility: Hospital Universitario 12 de Octubre; Servicio de Oncologia

location_facility: START Madrid. Centro Integral Oncologico Clara Campal; CIOCC

location_facility: START Madrid-FJD, Hospital Fundacion Jimenez Diaz


title: Clinical Study of the BreathID® System to Train the Algorithm for the ¹³C-Octanoate Breath Test With or Without the ¹³C-Methacetin Breath Test (OBT and MBT Respectively) for Correlation With Histological Findings Associated With Non-Alcoholic Steatohepatitis (NASH) and Other Liver Diseases

summary: This study's aim is to develop an algorithm for the ¹³C-Octanoate Breath Test with or without the ¹³C-Methacetin Breath Test (OBT and MBT respectively) for correlation with histological findings associated with of Non-Alcoholic Steatohepatitis (NASH) and other liver diseases using the BreathID® System

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Diagnostic

enrollment_type: Actual

lead_sponsor: Exalenz Bioscience LTD.

location_country:
United States
France
United Kingdom
Belgium

location_facility: Northwestern University, Feinberg School of Medicine

intervention_type: Device

lead_sponsor_type: industriel

status_simplified: Completed


type: Other Identifier

title: RIFTOS MKI - Radioactive Iodine reFractory Asymptomatic Patients in Differentiated Thyroid Cancer - an Observational Study to Assess the Use of Multikinase Inhibitors

acronym: RIFTOS MKI

summary: The purpose of the study was to assess the use of Multikinase Inhibitors (MKIs) in the treatment of patients with a progressive differentiated thyroid carcinoma (DTC) refractory to radioactive iodine (RAI) who do not have any symptoms.

study_type: Observational

time_perspective: Prospective

ReferenceType: derived

ReferenceCitation: Brose MS, Smit JWA, Lin CC, Tori M, Bowles DW, Worden F, Shen DH, Huang SM, Tsai HJ, Alevizaki M, Peeters RP, Takahashi S, Rumyantsev P, Guan R, Babajanyan S, Ozgurdal K, Sugitani I, Pitoia F, Lamartina L. Multikinase Inhibitors for the Treatment of Asymptomatic Radioactive Iodine-Refractory Differentiated Thyroid Cancer: Global Noninterventional Study (RIFTOS MKI). Thyroid. 2022 Sep;32(9):1059-1068. doi: 10.1089/thy.2022.0061.

ReferenceCitation: Porcelli T, Luongo C, Sessa F, Klain M, Masone S, Troncone G, Bellevicine C, Schlumberger M, Salvatore D. Long-term management of lenvatinib-treated thyroid cancer patients: a real-life experience at a single institution. Endocrine. 2021 Aug;73(2):358-366. doi: 10.1007/s12020-021-02634-z. Epub 2021 Feb 3.

ReferenceCitation: Brose MS, Smit J, Lin CC, Pitoia F, Fellous M, DeSanctis Y, Schlumberger M, Tori M, Sugitani I. Timing of multikinase inhibitor initiation in differentiated thyroid cancer. Endocr Relat Cancer. 2017 May;24(5):237-242. doi: 10.1530/ERC-17-0016. Epub 2017 Mar 7.

lead_sponsor: Bayer

location_country:
Taiwan
Brazil
Saudi Arabia
Greece
Russian Federation
Lebanon
Netherlands
Argentina
Spain
Philippines
Algeria
United States
France
India
Turkey
Mexico
Japan
Germany
Egypt

intervention_type: Drug


title: LUMINIST: LUng Cancer Molecular Insights Non Interventional Study

acronym: LUMINIST

summary: The recent development of therapies targeting specific biomarkers mutations is changing the standards of care and prognosis of patients with advanced NSCLC, but very few data are currently available on those emerging biomarkers. In addition, the correlation of biomarkers with patients' clinical outcomes in a standard of care setting is poorly understood. This study aims to address that need.

lead_sponsor: AstraZeneca

location_country:
Spain
Italy
United Kingdom
Ukraine
Peru
Romania
Belgium
Poland
Portugal
Austria
Australia
Russian Federation
Israel
Turkey
Mexico
Sweden
Canada
Hungary
Germany
Bulgaria
France
Chile
Netherlands

intervention_type: Other


primary_purpose: Treatment

acronym: CHERISH

intervention_type: Procedure

title: A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular Atrophy

lead_sponsor: Biogen

type: EudraCT Number

source: euctr

design_allocation: Randomized

ReferenceCitation: Darras BT, Farrar MA, Mercuri E, Finkel RS, Foster R, Hughes SG, Bhan I, Farwell W, Gheuens S. An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials. CNS Drugs. 2019 Sep;33(9):919-932. doi: 10.1007/s40263-019-00656-w.

ReferenceCitation: Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS; CHERISH Study Group. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504.

location_country:
Hong Kong
Spain
Italy
Canada
Sweden
Korea, Republic of
United States
France
Japan
Germany

location_facility: UCLA Clinical and Translational Research Center

location_facility: University Hospital Freiberg, Center for Paediatrics and Adolescent Medicine, Department of Neuropaediatrics and Muscular Disease

location_facility: Washington University School of Medicine

location_facility: University of Gothenburg, The Queen Silvia Children's Hospital

location_facility: Ann and Robert H. Lurie Children's Hospital of Chicago

location_facility: AOU Policlinico G. Martino Dipartimento di Neuroscienze e Centro Clinico Nemo Sud

location_facility: McGill University Health Centre-Glen Site-CIM

location_facility: Children's Hospital - London Health Sciences Centre

location_facility: Children's Hospital of Philadelphia - Neurology

location_facility: Armand Trousseau Hospital, I-Motion, Clinical Trials Platform

location_facility: The University of Hong Kong, Queen Mary Hospital, Department of Paediatrics and Adolescent Medicine

location_facility: Seoul National University Children's Hospital

location_facility: Fondazione Policlinico Universitario Agostino Gemelli-Universita Cattolica de Sacro Cuore-UOC Neuropsichiatre Infantile

location_facility: Lucile Packard Children's Hospital at Stanford

summary: The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secondary objective is to examine the safety and tolerability of nusinersen administered intrathecally to participants with later-onset SMA.


title: Study Title: A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of GS-6615 on Exercise Capacity in Subjects With Symptomatic Hypertrophic Cardiomyopathy

acronym: LIBERTY-HCM

summary: The primary objective of this study was to evaluate the effect of eleclazine (GS-6615) on exercise capacity as measured by Peak oxygen uptake (VO2) achieved during cardiopulmonary exercise testing (CPET), in participants with symptomatic hypertrophic cardiomyopathy (HCM).

status: Terminated

ReferenceType: background

ReferenceCitation: Olivotto I, Hellawell JL, Farzaneh-Far R, Blair C, Coppini R, Myers J, Belardinelli L, Maron MS. Novel Approach Targeting the Complex Pathophysiology of Hypertrophic Cardiomyopathy: The Impact of Late Sodium Current Inhibition on Exercise Capacity in Subjects with Symptomatic Hypertrophic Cardiomyopathy (LIBERTY-HCM) Trial. Circ Heart Fail. 2016 Mar;9(3):e002764. doi: 10.1161/CIRCHEARTFAILURE.115.002764.

lead_sponsor: Gilead Sciences

location_country:
United Kingdom
Italy
Australia
Netherlands
United States
Israel
France
Germany

location_facility: University of Iowa Hospitals and Clinics

location_facility: Azienda Ospedaliero Universitaria Di Bologna

location_facility: Azienda Ospedaliero Universitaria di Parma

location_facility: Azienda Ospedaliera San Camillo Forlanini

location_facility: Azienda Ospedaliera Universitaria Careggi

location_facility: Columbia University Medical Center/ New York Presbyterian

location_facility: Brigham & Women's Hospital and Harvard Medical School

status_simplified: Unknown


title: A Phase IIIb, Multinational, Multicenter, Open-Label Extension Study Assessing the Long-Term Safety of PRN Intravitreal Injections of DE-109 in Subjects With Non-Infectious Uveitis of the Posterior Segment of the Eye Who Have Participated in the SAKURA Development Program

acronym: SPRING

summary: The objective of this extension study is to evaluate the long-term safety of treatment with DE-109 (440 μg) in subjects with non-infectious uveitis of the posterior segment of the eye who have participated in the SAKURA development program.

lead_sponsor: Santen Inc.

location_country:
Italy
Austria
India
United States
France
Turkey


title: A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks

summary: The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.

ipd_sharing: No

lead_sponsor: Alnylam Pharmaceuticals

location_country:
Poland
Taiwan
Spain
Italy
United Kingdom
Australia
Mexico
Finland
Netherlands
Switzerland
Korea, Republic of
United States
France
Israel
South Africa
Belgium
Norway
Germany
Bulgaria
Czechia


title: A Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults.

summary: The primary objective of this study was to evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo in participants with Lennox-Gastaut syndrome (LGS).

ReferenceType: result

ReferenceCitation: Devinsky O, Patel AD, Cross JH, Villanueva V, Wirrell EC, Privitera M, Greenwood SM, Roberts C, Checketts D, VanLandingham KE, Zuberi SM; GWPCARE3 Study Group. Effect of Cannabidiol on Drop Seizures in the Lennox-Gastaut Syndrome. N Engl J Med. 2018 May 17;378(20):1888-1897. doi: 10.1056/NEJMoa1714631.

ReferenceCitation: Brigo F, Jones K, Eltze C, Matricardi S. Anti-seizure medications for Lennox-Gastaut syndrome. Cochrane Database Syst Rev. 2021 Apr 7;4:CD003277. doi: 10.1002/14651858.CD003277.pub4.

ReferenceCitation: Privitera M, Bhathal H, Wong M, Cross JH, Wirrell E, Marsh ED, Mazurkiewicz-Beldzinska M, Villanueva V, Checketts D, Knappertz V, VanLandingham K. Time to onset of cannabidiol (CBD) treatment effect in Lennox-Gastaut syndrome: Analysis from two randomized controlled trials. Epilepsia. 2021 May;62(5):1130-1140. doi: 10.1111/epi.16878. Epub 2021 Apr 2.

ReferenceCitation: Perry MS. Don't Fear the Reefer-Evidence Mounts for Plant-Based Cannabidiol as Treatment for Epilepsy. Epilepsy Curr. 2019 Mar-Apr;19(2):93-95. doi: 10.1177/1535759719835671.

lead_sponsor: Jazz Pharmaceuticals

location_country:
United Kingdom
United States
Spain
France


title:
A Double-Blind, Placebo-Controlled, Randomized-Withdrawal,
 Multicenter Study of the Efficacy and Safety of Xyrem with an Open- Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects with Narcolepsy with Cataplexy

ReferenceCitation: Lecendreux M, Plazzi G, Dauvilliers Y, Rosen CL, Ruoff C, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Long-term safety and maintenance of efficacy of sodium oxybate in the treatment of narcolepsy with cataplexy in pediatric patients. J Clin Sleep Med. 2022 Sep 1;18(9):2217-2227. doi: 10.5664/jcsm.10090.

ReferenceCitation: Wang YG, Menno D, Chen A, Steininger TL, Morris S, Black J, Profant J, Johns MW. Validation of the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) questionnaire in pediatric patients with narcolepsy with cataplexy aged 7-16 years. Sleep Med. 2022 Jan;89:78-84. doi: 10.1016/j.sleep.2021.11.003. Epub 2021 Nov 11.

ReferenceCitation: Plazzi G, Ruoff C, Lecendreux M, Dauvilliers Y, Rosen CL, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Treatment of paediatric narcolepsy with sodium oxybate: a double-blind, placebo-controlled, randomised-withdrawal multicentre study and open-label investigation. Lancet Child Adolesc Health. 2018 Jul;2(7):483-494. doi: 10.1016/S2352-4642(18)30133-0. Epub 2018 May 21.

location_country:
Netherlands
United States
Italy
France

location_facility: University Hospitals Cleveland Medical Center

location_facility: Dipartimento di Scienze Biomediche e Biomotorie

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Children's Hospital of The King's Daughters

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago

summary: The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy.


title: A Post-market Clinical Follow-up Study of the TITAN™ Reverse Shoulder System Used in Primary or Revision Total Shoulder Arthroplasty

summary: The Integra® TITAN™ Reverse Shoulder System (TRS) is a semi-constrained total shoulder construct. When used under the conditions and purposes intended the TRS will relieve pain and restore some functional joint motion to the affected shoulder. The purpose of this study is to assess the short, mid and long term outcomes of the TITAN™ Reverse Shoulder System.

status: Active, not recruiting

study_completion_date_type: Anticipated

lead_sponsor: Smith & Nephew, Inc.

location_facility: Hospital of the University of Pennsylvania

status_simplified: Ongoing


primary_purpose: Prevention

title: Open Label, Single Arm Safety Prospective Cohort Study of Dabigatran Etexilate for Secondary Prevention of Venous Thromboembolism in Children From 0 to Less Than 18 Years

lead_sponsor: Boehringer Ingelheim

ReferenceCitation: Brandão LR, Tartakovsky I, Albisetti M, Halton J, Bomgaars L, Chalmers E, Luciani M, Saracco P, Felgenhauer J, Lvova O, Simetzberger M, Sun Z, Mitchell LG. Dabigatran in the treatment and secondary prophylaxis of venous thromboembolism in children with thrombophilia. Blood Adv. 2022 Nov 22;6(22):5908-5923. doi: 10.1182/bloodadvances.2021005681.

ReferenceCitation: Brandão LR, Albisetti M, Halton J, Bomgaars L, Chalmers E, Mitchell LG, Nurmeev I, Svirin P, Kuhn T, Zapletal O, Tartakovsky I, Simetzberger M, Huang F, Sun Z, Kreuzer J, Gropper S, Brueckmann M, Luciani M; DIVERSITY Study Investigators. Safety of dabigatran etexilate for the secondary prevention of venous thromboembolism in children. Blood. 2020 Feb 13;135(7):491-504. doi: 10.1182/blood.2019000998. Erratum in: Blood. 2020 May 7;135(19):1720.

location_country:
Italy
Lithuania
Ukraine
United States
Belgium
Czechia
Austria
Russian Federation
Israel
Turkey
Mexico
Norway
Sweden
Brazil
Canada
Switzerland
Hungary
Germany
Denmark
Taiwan
Thailand
France

location_facility: HMCP - Hospital e Maternidade Celso Pierro - PUC-Campinas

location_facility: Morozovskaya Children Clin.Hosp.,Haematological Dept, Moscow

location_facility: Rigshospitalet, København, Børneonkologisk Afsnit 5002

location_facility: Reg Clin.Hosp.#1,Healthcare Tyumen Region,Cardiovas.Surgery

location_facility: Oslo Universitetssykehus HF, Rikshospitalet

location_facility: Children Intensive Care Hosp,Anaesthesiology Dept,Vilnius

location_facility: University Hospital Plzen, Plzen-Lochotin

location_facility: Shaare Zedek Medical Center, Jerusalem 91031

location_facility: Child.CityClin.Hos.na.ZA Bashlyaeva MoscowHealth Dep,Cardiol

location_facility: St.Petersburg State Pediatric Univ.Ministry of Healthcare RF

location_facility: Ege Universitesi Tip Fakultesi Cocuk Hematolojisi Bilim Dali

location_facility: Western Ukrainian Spec.Children Med.Center,Lviv

location_facility: Necmettin Erbakan Universitesi Meram Tip Fakultesi

location_facility: Istanbul Universitesi Cerrahpasa Tip Fakultesi

location_facility: Children Rep.Clin.Hosp of MoH,Cardio Vas.surgery Dept, Kazan

location_facility: Science Res.Instit.CV Diseases,Scientific Res.Dept,Kemerovo

location_facility: Istanbul Saglik Bilimleri Uni. Kanuni Sultan Suleyman EAH

location_facility: State Budget Healthcare Institution "Republican children's clinical hospital"

location_facility: Cukurova Universitesi Tip Fakultesi Cocuk Sagligi

location_facility: Providence Sacred Heart Medical Center and Children's Hospital

location_facility: Instituto de Oncologia Pediatrica - IOP / GRAAC - UNIFESP

location_facility: Faculdade de Ciencias Medicas da UNICAMP

summary: This open-label, single arm prospective cohort study will assess the safety of dabigatran etexilate in secondary prevention of venous thromboembolism in paediatric patients. Children from 0 to less than 18 years of age will be eligible to participate.


title: A Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy

acronym: ENDEAR

summary: The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal muscular atrophy (SMA). The secondary objective of the study is to examine the safety and tolerability of nusinersen administered intrathecally to participants with infantile-onset SMA.

ReferenceCitation: Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guérin A, Pivneva I, Wu EQ, Arjunji R, Feltner D, Sproule DM. Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1. Adv Ther. 2019 May;36(5):1164-1176. doi: 10.1007/s12325-019-00923-8. Epub 2019 Mar 16.

ReferenceCitation: Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752.

ReferenceCitation: Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, Yamashita M, Rigo F, Hung G, Schneider E, Norris DA, Xia S, Bennett CF, Bishop KM. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016 Dec 17;388(10063):3017-3026. doi: 10.1016/S0140-6736(16)31408-8. Epub 2016 Dec 7.

location_country:
Spain
Italy
United Kingdom
Australia
Canada
Sweden
Korea, Republic of
United States
France
Turkey
Belgium
Japan
Germany

location_facility: Washington University School of Medicine

location_facility: University of Gothenburg, The Queen Silvia Children's Hospital

location_facility: Ann and Robert H. Lurie Children's Hospital of Chicago

location_facility: Hospital Universitario La Paz, Pediatric Neurology Department

location_facility: Istituto Giannina Gaslini, Centro Traslazionale di Miologia e Patologie Neurodegenerative

location_facility: UT Southwestern Medical Center/Children's Medical Center Dallas

location_facility: Pediatric Neurology Unit, Catholic University

location_facility: Primary Children's Medical Center (University of Utah)

location_facility: Children's Hospital of Philadelphia - Neurology

location_facility: MRC Centre for Neuromuscular Diseases at Newcastle, Institute of Genetic Medicine Newcastle University

location_facility: UCL Institute of Child Health/Great Ormond Street

location_facility: Hôpital Universitaire des Enfants Reine FABIOLA (HUDERF)

location_facility: Royal Children's Hospital, Children's Neuroscience Centre

location_facility: British Columbia Children's Hospital/UBC

location_facility: Universtatsklinikum Freiburg, Zentrum fur Kinder-und Jugendmedizin , Abteilung Neuropadiatrie und Muskelerkrankungen


title: A Prospective Short-term Study to Evaluate Methodologies for the Assessment of Disease Extent, Impact, and Wound Evolution in Patients With Dystrophic Epidermolysis Bullosa (DEB)

summary: The purpose of this study is to better understand disease extent and to identify appropriate methodologies to evaluate (dystrophic epidermolysis bullosa) DEB in a quantitative and qualitative manner.

ReferenceCitation: Paller AS, Pope E, Rudin D, Malyala A, Ramsdell D, Johnson R, Landy H, Murrell DF; DEB Investigators. A prospective short-term study to evaluate methodologies for the assessment of disease extent, impact, and wound evolution in patients with dystrophic epidermolysis bullosa. Orphanet J Rare Dis. 2022 Aug 13;17(1):314. doi: 10.1186/s13023-022-02461-z.

lead_sponsor: Shire

location_country:
Italy
Austria
Australia
United States
France
Germany
Czechia

location_facility: Cincinnati Childrens Hospital Medical Center

location_facility: Istituto Dermopatico Dell'immacolata IRCCS

location_facility: Ann and Robert H Lurie Childrens Hospital of Chicago

location_facility: Service de Génétique et INSERM UMR 1163, Laboratoire de Génétique des maladies cutanées - Institut Imagine


title: Phase III, Randomized, Double-Blind, Double-Dummy, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy (Induction of Remission) and Safety of Etrolizumab Compared With Adalimumab and Placebo in Patients With Moderate to Severe Ulcerative Colitis Who Are Naive to TNF Inhibitors

acronym: HIBISCUS II

summary: This Phase III, double-blind, placebo and active-comparator controlled, multicenter study will investigate the efficacy and safety of etrolizumab in induction of remission in participants with moderately to severely active ulcerative colitis (UC) who are naIve to tumor necrosis factor (TNF) inhibitors and refractory to or intolerant of prior immunosuppressant and/or corticosteroid treatment. In addition to this study, a second Phase III trial with identical study design (GA28948; NCT02163759) was independently conducted.

ReferenceCitation: Rubin DT, Dotan I, DuVall A, Bouhnik Y, Radford-Smith G, Higgins PDR, Mishkin DS, Arrisi P, Scalori A, Oh YS, Tole S, Chai A, Chamberlain-James K, Lacey S, McBride J, Panés J; HIBISCUS Study Group. Etrolizumab versus adalimumab or placebo as induction therapy for moderately to severely active ulcerative colitis (HIBISCUS): two phase 3 randomised, controlled trials. Lancet Gastroenterol Hepatol. 2022 Jan;7(1):17-27. doi: 10.1016/S2468-1253(21)00338-1. Epub 2021 Nov 17. Erratum in: Lancet Gastroenterol Hepatol. 2022 Apr;7(4):e8.

ReferenceCitation: Sandborn WJ, Vermeire S, Tyrrell H, Hassanali A, Lacey S, Tole S, Tatro AR; Etrolizumab Global Steering Committee. Etrolizumab for the Treatment of Ulcerative Colitis and Crohn's Disease: An Overview of the Phase 3 Clinical Program. Adv Ther. 2020 Jul;37(7):3417-3431. doi: 10.1007/s12325-020-01366-2. Epub 2020 May 22.

lead_sponsor: Hoffmann-La Roche

location_country:
Croatia
Poland
Brazil
Greece
Latvia
Australia
New Zealand
Argentina
Lithuania
Russian Federation
Ukraine
Hungary
United States
France
Turkey
Colombia
Malaysia
Bulgaria
Czechia

location_facility: Klaipeda Seamen's Hospital, Public Institution

location_facility: Hospital of Lithuanian University of Health. Sciences Kaunas Clinics

location_facility: Private Small Enterprise Medical Center Pulse

location_facility: Irkutsk State Medical Academy of Continuing Education

location_facility: GI L.T.Malaya Therapy National Institute of the NAMS of Ukraine

location_facility: Pavlov First Saint Petersburg State Medical University

location_facility: A.Novak Transcarpathian Regional Clinical Hospital

location_facility: Pusat Perubatan Universiti Kebangsaan Malaysia

location_facility: Medical Center "Nov Rehabilitatsionen Tsentar", EOOD

location_facility: Cotton-O'Neil Clinical Research Center, Digestive Health

location_facility: FSBEI HE "Stavropol State Medical University" of Ministry of Healthcare of Russian Federation

location_facility: M.V. Sklifosovskyi Poltava RCH Dept of Gastroenterology HSEIU UMSA

location_facility: Center of Emergency and Radiation Medicine; Pulmonology

location_facility: BHI of Omsk region Clinical Oncology Dispensary

location_facility: Great Lakes Gastroenterology Research, LLC

location_facility: Niepubliczny Zaklad Opieki Zdrowotnej SONOMED

location_facility: Hospital Universitario Prof Edgar Santos-Ufba; Ambulatorio Magalhaes Neto 3Andar- Dermatologia

location_facility: Gaziantep University Medical Faculty Sahinbey Educational Research Hospital; Medical Oncology

location_facility: SBEI HPE Altai State Medical University of MoH and SD; Out-patient Department

location_facility: FSBI "Scientific Research Institute of Physyology and Basic Medicine" under the SB of RAMS

location_facility: Haydarpasa Numune Training and Research Hospital; Gastroenterology

location_facility: SI inst. of Gastroenterology of NAMSU Dept of Stomach & Duodenum Diseases, D&ThN SI DMA of MoHU

location_facility: Pauls Stradins Clinical University Hospital

location_facility: Vilnius University Hospital Santariskiu Clinic, Public Institution; Cardiology

location_facility: CI Kherson Afanasii and Olha Tropiny City Clinical Hospital

location_facility: Hospital Universitario Walter Cantidio - UFC

location_facility: Allmedica Badania Kliniczne Sp z o.o. Sp K.

location_facility: Centro de Investigaciones Medicas Mar Del Plata

location_facility: Uniwersyteckie Centrum Kliniczne im. prof. K. Gibinskiego SUM

location_facility: CI of SRC Sumy RCH Dept of Gasroenterology Sumy SU MI

location_facility: University General Hospital of Heraklion

location_facility: Hospital Raja Perempuan Zainab II; Department of Medicine

location_facility: Med Center of International Institute of Clinical Trials LLC; Medical Center "OK!Clinic+"

location_facility: Ankara Diskapi Yildirim Beyazit Training and Research Hospital; Gastroenterology

location_facility: CI of Kyiv RC Kyiv Regional Clinical Hospital


title: Long-Term Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Tocilizumab in Patients With Polyarticular-Course and Systemic Juvenile Idiopathic Arthritis

location_country:
Brazil
Spain
Italy
Russian Federation
Australia
United Kingdom
Argentina
Canada
United States
France
Mexico
Germany

location_facility: Hospital Universitario la Fe: Servicio de Reumatologia Pediatrica

location_facility: Bristol Royal Hospital for Children; Rheumatology Department

location_facility: Hospital Sant Joan De Deu; Servicio de Reumatologia Pediatrica

location_facility: Royal Children'S Hospital; Paediatric Rheumatology

location_facility: SI Sceintific children health center RAMS

location_facility: Hospital de La Paz; Unidad de Reumatologia Pediatrica

location_facility: University of Utah; Immunology/Rheumatology/Allergy

location_facility: Arkansas Children's Hospital Research Institute

location_facility: Hackensack University Medical Center; Pediatric Rheumatology

location_facility: Asklepios Klinik; Zentrum für Allgemeine Pädiatrie und Neonatologie

location_facility: Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria

location_facility: Alder Hey Children's NHS Foundation Trust

location_facility: Hospital Ramon y Cajal ; Servicio de Reumatologia

location_facility: Universidade Federal de Sao Paulo - UNIFES

location_facility: Cincinnati Children'S Hospital Medical Center; Division of Rheumatology

location_facility: Westmead Hospital; Paediatric Rheumatology

location_facility: Uniklinikum Freiburg Zentrum für Kinder- und Jugendmedizin; Pädiatrische Infektio- u. Rheumatologie

location_facility: Hospital Infantil de México "Federico Gomez"; Rheumatology

location_facility: Irccs Ospedale Pediatrico Bambin Gesu - Dip. Di Medicina

location_facility: Hospital de Ninos de la Santisima Trinidad; Hematología

summary: This open-label extension of the JIGSAW studies (WA28117 [NCT01904279] and WA28118 [NCT01904292]) is designed to evaluate the long-term safety and efficacy of subcutaneous (SC) tocilizumab treatment in participants with polyarticular-course and systemic juvenile idiopathic arthritis (pJIA and sJIA). Participants from the 2 JIGSAW studies will continue to receive 162 milligrams (mg) of SC tocilizumab with treatment schedule according to arthritis subtype and body weight. Participants will receive the treatment until commercial availability of the drug or for a maximum of 5 years, whichever is earlier.


title: Phase 3 Efficacy and Safety Study of CT-P6 and Herceptin as Neoadjuvant and Adjuvant Treatment in Patients With Her2-positive Early Breast Cancer

summary: This study will determine whether CT-P6 and Herceptin are equivalent in patients with early-stage breast cancer undergoing neoadjuvant chemotherapy. Our hypothesis is that the pathologic complete response rate will be equivalent in patients treated with neoadjuvant CT-P6 or Herceptin. Patients will receive 8 cycles of neoadjuvant systemic therapy and up to 10 cycles of therapy in the adjuvant setting.

ReferenceCitation: Stebbing J, Baranau YV, Baryash V, Manikhas A, Moiseyenko V, Dzagnidze G, Zhavrid E, Boliukh D, Pikiel J, Eniu AE, Li RK, Tiangco B, Lee SJ, Kim S. Long-term efficacy and safety of CT-P6 versus trastuzumab in patients with HER2-positive early breast cancer: final results from a randomized phase III trial. Breast Cancer Res Treat. 2021 Aug;188(3):631-640. doi: 10.1007/s10549-021-06240-5. Epub 2021 Jun 20.

ReferenceCitation: Esteva FJ, Baranau YV, Baryash V, Manikhas A, Moiseyenko V, Dzagnidze G, Zhavrid E, Boliukh D, Stroyakovskiy D, Pikiel J, Eniu AE, Li RK, Rusyn AV, Tiangco B, Lee SJ, Lee SY, Yu SY, Stebbing J. Efficacy and safety of CT-P6 versus reference trastuzumab in HER2-positive early breast cancer: updated results of a randomised phase 3 trial. Cancer Chemother Pharmacol. 2019 Oct;84(4):839-847. doi: 10.1007/s00280-019-03920-4. Epub 2019 Aug 19.

ReferenceCitation: Stebbing J, Baranau Y, Baryash V, Manikhas A, Moiseyenko V, Dzagnidze G, Zhavrid E, Boliukh D, Stroyakovskii D, Pikiel J, Eniu A, Komov D, Morar-Bolba G, Li RK, Rusyn A, Lee SJ, Lee SY, Esteva FJ. CT-P6 compared with reference trastuzumab for HER2-positive breast cancer: a randomised, double-blind, active-controlled, phase 3 equivalence trial. Lancet Oncol. 2017 Jul;18(7):917-928. doi: 10.1016/S1470-2045(17)30434-5. Epub 2017 Jun 4. Erratum in: Lancet Oncol. 2017 Aug;18(8):e433. Lancet Oncol. 2017 Sep;18(9):e510.

lead_sponsor: Celltrion

location_country:
Spain
Italy
Ukraine
Peru
Romania
Philippines
Japan
South Africa
Poland
Portugal
Russian Federation
Argentina
India
Mexico
Bosnia and Herzegovina
Hungary
Taiwan
Belarus
Latvia
Georgia
France
Chile


title: Assessment of Patterns of Patient-reported Outcomes in Adults With Congenital Heart Disease - International Study

acronym: APPROACH-IS

summary: The purpose of this study is to examine the differences in perceived health, psychosocial functioning, behavioral outcomes and quality of life of adults with congenital heart disease who are living in different areas of the world, and how these differences can be understood (e.g., differences in sense of coherence or illness perceptions).

time_perspective: Cross-Sectional

ReferenceCitation: Moons P, Luyckx K, Thomet C, Budts W, Enomoto J, Sluman MA, Yang HL, Jackson JL, Khairy P, Cook SC, Chidambarathanu S, Alday L, Oechslin E, Eriksen K, Dellborg M, Berghammer M, Johansson B, Mackie AS, Menahem S, Caruana M, Veldtman G, Soufi A, Fernandes SM, White K, Callus E, Kutty S, Kovacs AH; APPROACH-IS consortium. Patient-reported outcomes in the aging population of adults with congenital heart disease: results from APPROACH-IS. Eur J Cardiovasc Nurs. 2022 Jul 28. pii: zvac057. doi: 10.1093/eurjcn/zvac057. [Epub ahead of print]

ReferenceCitation: Lu CW, Wang JK, Yang HL, Kovacs AH, Luyckx K, Ruperti-Repilado FJ, Van De Bruaene A, Enomoto J, Sluman MA, Jackson JL, Khairy P, Cook SC, Chidambarathanu S, Alday L, Oechslin E, Eriksen K, Dellborg M, Berghammer M, Johansson B, Mackie AS, Menahem S, Caruana M, Veldtman G, Soufi A, Fernandes SM, White K, Callus E, Kutty S, Apers S, Moons P; APPROACH-IS consortium, the International Society for Adult Congenital Heart Disease (ISACHD) *. Heart Failure and Patient-Reported Outcomes in Adults With Congenital Heart Disease from 15 Countries. J Am Heart Assoc. 2022 May 3;11(9):e024993. doi: 10.1161/JAHA.121.024993. Epub 2022 Apr 26.

ReferenceCitation: Fox KR, Hardy RY, Moons P, Kovacs AH, Luyckx K, Apers S, Cook SC, Veldtman G, Fernandes SM, White K, Kutty S, Jackson JL; APPROACH-IS Consortium and the International Society for Adult Congenital Heart Disease (ISACHD). Smoking among adult congenital heart disease survivors in the United States: Prevalence and relationship with illness perceptions. J Behav Med. 2021 Dec;44(6):772-783. doi: 10.1007/s10865-021-00239-5. Epub 2021 Jun 29.

ReferenceCitation: Moons P, Luyckx K, Thomet C, Budts W, Enomoto J, Sluman MA, Lu CW, Jackson JL, Khairy P, Cook SC, Chidambarathanu S, Alday L, Eriksen K, Dellborg M, Berghammer M, Johansson B, Mackie AS, Menahem S, Caruana M, Veldtman G, Soufi A, Fernandes SM, White K, Callus E, Kutty S, Ombelet F, Apers S, Kovacs AH; APPROACH-IS Consortium and the International Society for Adult Congenital Heart Disease (ISACHD). Physical Functioning, Mental Health, and Quality of Life in Different Congenital Heart Defects: Comparative Analysis in 3538 Patients From 15 Countries. Can J Cardiol. 2021 Feb;37(2):215-223. doi: 10.1016/j.cjca.2020.03.044. Epub 2020 Apr 6.

ReferenceCitation: Moons P, Apers S, Kovacs AH, Thomet C, Budts W, Enomoto J, Sluman MA, Wang JK, Jackson JL, Khairy P, Cook SC, Chidambarathanu S, Alday L, Oechslin E, Eriksen K, Dellborg M, Berghammer M, Johansson B, Mackie AS, Menahem S, Caruana M, Veldtman G, Soufi A, Fernandes SM, White K, Callus E, Kutty S, Luyckx K; APPROACH-IS consortium and the International Society for Adult Congenital Heart Disease (ISACHD). Sense of coherence in adults with congenital heart disease in 15 countries: Patient characteristics, cultural dimensions and quality of life. Eur J Cardiovasc Nurs. 2021 Feb 11;20(1):48-55. doi: 10.1177/1474515120930496.

ReferenceCitation: Van Bulck L, Goossens E, Luyckx K, Apers S, Oechslin E, Thomet C, Budts W, Enomoto J, Sluman MA, Lu CW, Jackson JL, Khairy P, Cook SC, Chidambarathanu S, Alday L, Eriksen K, Dellborg M, Berghammer M, Johansson B, Mackie AS, Menahem S, Caruana M, Veldtman G, Soufi A, Fernandes SM, White K, Callus E, Kutty S, Moons P; APPROACH-IS consortium and the International Society for Adult Congenital Heart Disease (ISACHD). Healthcare system inputs and patient-reported outcomes: a study in adults with congenital heart defect from 15 countries. BMC Health Serv Res. 2020 Jun 3;20(1):496. doi: 10.1186/s12913-020-05361-9.

ReferenceCitation: Holbein CE, Veldtman GR, Moons P, Kovacs AH, Luyckx K, Apers S, Chidambarathanu S, Soufi A, Eriksen K, Jackson JL, Enomoto J, Fernandes SM, Johansson B, Alday L, Dellborg M, Berghammer M, Menahem S, Caruana M, Kutty S, Mackie AS, Thomet C, Budts W, White K, Sluman MA, Callus E, Cook SC, Khairy P, Cedars A; APPROACH-IS consortium and the International Society for Adult Congenital Heart Disease (ISACHD). Perceived Health Mediates Effects of Physical Activity on Quality of Life in Patients With a Fontan Circulation. Am J Cardiol. 2019 Jul 1;124(1):144-150. doi: 10.1016/j.amjcard.2019.03.039. Epub 2019 Apr 10.

ReferenceCitation: Apers S, Kovacs AH, Luyckx K, Alday L, Berghammer M, Budts W, Callus E, Caruana M, Chidambarathanu S, Cook SC, Dellborg M, Enomoto J, Eriksen K, Fernandes SM, Jackson JL, Johansson B, Khairy P, Kutty S, Menahem S, Rempel G, Sluman MA, Soufi A, Thomet C, Veldtman G, Wang JK, White K, Moons P; APPROACH-IS consortium; International Society for Adult Congenital Heart Disease (ISACHD). Assessment of Patterns of Patient-Reported Outcomes in Adults with Congenital Heart disease - International Study (APPROACH-IS): rationale, design, and methods. Int J Cardiol. 2015 Jan 20;179:334-42. doi: 10.1016/j.ijcard.2014.11.084. Epub 2014 Nov 8.

lead_sponsor: Philip Moons

location_country:
Taiwan
Italy
Australia
Argentina
Canada
Switzerland
Sweden
United States
France
India
Malta
Belgium
Japan
Netherlands
Norway

location_facility: Northern Alberta Adult Congenital Heart Clinic

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Washington University and Barnes Jewish Heart & Vascular Center


acronym: PROBESE

summary:
Postoperative respiratory failure, particularly after surgery under general anesthesia, adds to the morbidity and mortality of surgical patients. Anesthesiologists inconsistently use positive end-expiratory pressure (PEEP) and recruitment maneuvers in the hope that this may improve oxygenation and protect against postoperative pulmonary complications (PPCs), especially in obese patients. While anesthesiologists tend to use PEEP higher than in non-obese patients. While it is uncertain whether a strategy that uses higher levels of PEEP with recruitment maneuvers truly prevents PPCs in these patients, use of higher levels of PEEP with recruitment maneuvers could compromise intra-operative hemodynamics.

The investigators aim to compare a ventilation strategy using higher levels of PEEP with recruitment maneuvers with one using lower levels of PEEP without recruitment maneuvers in obese patients at an intermediate-to-high risk for PPCs.

We hypothesize that an intra-operative ventilation strategy using higher levels of PEEP and recruitment maneuvers, as compared to ventilation with lower levels of PEEP without recruitment maneuvers, prevents PPCs in obese patients at an intermediate-to-high risk for PPC.

ReferenceCitation: Ellenberger C, Pelosi P, de Abreu MG, Wrigge H, Diaper J, Hagerman A, Adam Y, Schultz MJ, Licker M; PROBESE investigators, of the PROtective VEntilation Network (PROVEnet); Clinical Trial Network of the European Society of Anaesthesiology and Intensive Care (ESAIC). Distribution of ventilation and oxygenation in surgical obese patients ventilated with high versus low positive end-expiratory pressure: A substudy of a randomised controlled trial. Eur J Anaesthesiol. 2022 Nov 1;39(11):875-884. doi: 10.1097/EJA.0000000000001741. Epub 2022 Sep 12.

ReferenceCitation: Writing Committee for the PROBESE Collaborative Group of the PROtective VEntilation Network (PROVEnet) for the Clinical Trial Network of the European Society of Anaesthesiology, Bluth T, Serpa Neto A, Schultz MJ, Pelosi P, Gama de Abreu M; PROBESE Collaborative Group, Bluth T, Bobek I, Canet JC, Cinnella G, de Baerdemaeker L, Gama de Abreu M, Gregoretti C, Hedenstierna G, Hemmes SNT, Hiesmayr M, Hollmann MW, Jaber S, Laffey J, Licker MJ, Markstaller K, Matot I, Mills GH, Mulier JP, Pelosi P, Putensen C, Rossaint R, Schmitt J, Schultz MJ, Senturk M, Serpa Neto A, Severgnini P, Sprung J, Vidal Melo MF, Wrigge H. Effect of Intraoperative High Positive End-Expiratory Pressure (PEEP) With Recruitment Maneuvers vs Low PEEP on Postoperative Pulmonary Complications in Obese Patients: A Randomized Clinical Trial. JAMA. 2019 Jun 18;321(23):2292-2305. doi: 10.1001/jama.2019.7505. Erratum in: JAMA. 2019 Nov 12;322(18):1829-1830.

ReferenceCitation: Bluth T, Teichmann R, Kiss T, Bobek I, Canet J, Cinnella G, De Baerdemaeker L, Gregoretti C, Hedenstierna G, Hemmes SN, Hiesmayr M, Hollmann MW, Jaber S, Laffey JG, Licker MJ, Markstaller K, Matot I, Müller G, Mills GH, Mulier JP, Putensen C, Rossaint R, Schmitt J, Senturk M, Serpa Neto A, Severgnini P, Sprung J, Vidal Melo MF, Wrigge H, Schultz MJ, Pelosi P, Gama de Abreu M; PROBESE investigators; PROtective VEntilation Network (PROVEnet); Clinical Trial Network of the European Society of Anaesthesiology (ESA). Protective intraoperative ventilation with higher versus lower levels of positive end-expiratory pressure in obese patients (PROBESE): study protocol for a randomized controlled trial. Trials. 2017 Apr 28;18(1):202. doi: 10.1186/s13063-017-1929-0. Erratum in: Trials. 2017 Jun 1;18(1):247.

lead_sponsor: Technische Universität Dresden

location_country:
Brazil
Spain
Italy
Austria
Netherlands
United Kingdom
Canada
Switzerland
Sweden
Hungary
United States
France
Israel
Turkey
Belgium
Germany

location_facility: Saint Michael's Hospital, University of Toronto

location_facility: Department of Anesthesiology and Intensive Care, University Hospital Carl Gustav Carus

location_facility: Massachusetts General Hospital, Harvard University

location_facility: Academic Medical Center, University of Amsterdam

location_facility: Hospital Universitari Germans Trias I Pujol

lead_sponsor_type: academique


title: Radium-223 Alpha Emitter Agent in Non-intervention Safety Study in mCRPC popUlation for Long-teRm Evaluation

acronym: REASSURE

summary: Observational study in the routine clinical practice setting to evaluate the short and long term safety profile of Radium-223 in metastatic castration resistant prostate cancer patients and to evaluate the risk of developing second primary cancers.

ReferenceCitation: Sartor O, Fougère C, Essler M, Ezziddin S, Kramer G, Ellinger J, Nordquist L, Sylvester J, Paganelli G, Peer A, Bögemann M, Meltzer J, Sandström P, Verholen F, Song DY. (177)Lu-Prostate-Specific Membrane Antigen Ligand After (223)Ra Treatment in Men with Bone-Metastatic Castration-Resistant Prostate Cancer: Real-World Clinical Experience. J Nucl Med. 2022 Mar;63(3):410-414. doi: 10.2967/jnumed.121.262240. Epub 2021 Jun 24. Erratum in: J Nucl Med. 2022 Jul;63(7):100-1000.

location_country:
Denmark
Portugal
Luxembourg
Spain
Colombia
Austria
Greece
Italy
Argentina
Canada
Mexico
Netherlands
Sweden
United States
France
Israel
United Kingdom
Belgium
Germany
Czechia


title: A Multicentre, Long-term Safety, Efficacy, and Pharmacokinetics Study of Lubiprostone in Paediatric Subjects Aged ≥ 6 Years to < 18 Years With Functional Constipation

lead_sponsor: Sucampo Pharma Americas, LLC

ReferenceCitation: Benninga MA, Hussain SZ, Sood MR, Nurko S, Hyman P, Clifford RA, O'Gorman M, Losch-Beridon T, Mareya S, Lichtlen P, Di Lorenzo C. Lubiprostone for Pediatric Functional Constipation: Randomized, Controlled, Double-Blind Study With Long-term Extension. Clin Gastroenterol Hepatol. 2022 Mar;20(3):602-610.e5. doi: 10.1016/j.cgh.2021.04.005. Epub 2021 Apr 7.

location_country:
Poland
United Kingdom
Canada
United States
France
Belgium
Netherlands

location_facility: UnityPoint Health Des Moines/ Blank Children's Clinic

location_facility: Pediatric Gastroenterology Carilion Medical Center

location_facility: Pediatric Research of Charlottesville, LLC

location_facility: University of South Alabama Women's and Children's Hospital

location_facility: Nemours/Alfred I. duPont Hospital for Children

location_facility: Arkansas Childrens Hospital Research Institute

location_facility: St. Christopher's Hospital for Children Drexel University

location_facility: University of Oklahoma Health Sciences Center

location_facility: Coastal Pediatric Associates - James Island

location_facility: Children's Center for Digestive Health Care/GI Care for Kids

location_facility: INOVA Pediatric Digestive Diseases Center

location_facility: Chelsea and Westminster Hospital NHS Foundation Trust

location_facility: University of Mississippi Medical Center

location_facility: Coastal Pediatrics Associates - Mount Pleasant

location_facility: MultiCare Health System Institute for Research and Innovation

location_facility: University of Louisville Research Foundation Inc. Kosair Charities Pediatric Clinical Research Unit

location_facility: Morgan Stanley Children's Hospital of New York Presbyterian

location_facility: Arizona Children's Center at Maricopa Medical Center

summary:
This study is for children with constipation.

Children who completed 3 months of treatment in the earlier study (NCT02042183):

were invited to participate
will receive lubiprostone for 9 more months
will see if lubiprostone safely relieves their constipation if taken for a whole year


title: A Natural History Study to Observe Disease Progression, Standard of Care and Investigate Biomarkers in Alport Syndrome Patients

summary:
There is limited published clinical data about the natural history of renal disease in Alport syndrome. The RG012-01 study will collect data to characterize the progression of renal dysfunction in Alport syndrome patients.

Patients with a confirmed diagnosis of Alport syndrome who have qualifying GFR will be considered for enrollment. The sequential sampling of subjects' urine and/or blood will allow an assessment of the rate of change of established clinical endpoints, such as GFR and/or the rate of change of other renal biomarkers (proteinuria and β-2 microglobulin) in subjects whose renal function is steadily declining. The identification of surrogate markers that track the decline of renal function and could correlate with time to end-stage renal disease (ESRD) is a key goal of the natural history study.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
United Kingdom
Australia
Canada
United States
France
Germany


title: A Phase 1 Study to Evaluate the Safety and Tolerability of MEDI4736 as Monotherapy or in Combination With Tremelimumab With or Without Azacitidine in Subjects With Myelodysplastic Syndrome After Treatment With Hypomethylating Agents

summary: This is a multicenter, open-label, Phase 1 study to assess the safety and antitumor activity of MEDI4736 as Monotherapy or in Combination with Tremelimumab with or without Azacitidine in Subjects with myelodysplastic syndrome after treatment with hypomethylating agents

design_allocation: Non-Randomized

lead_sponsor: MedImmune LLC

location_country:
Germany
United States
United Kingdom
France

intervention_type: Biological


title: Fractional Flow Reserve Versus Angiography for Multivessel Evaluation (FAME) 3 Trial A Comparison of Fractional Flow Reserve-Guided Percutaneous Coronary Intervention and Coronary Artery Bypass Graft Surgery in Patients With Multivessel Coronary Artery Disease

acronym: FAME 3

summary: The purpose of this study is to determine whether Fractional flow reserve (FFR, (coronary pressure wire-based index for assessing the ischemic potential of a coronary lesion)-guided percutaneous coronary intervention (PCI) in patients with multivessel coronary artery disease (CAD) will result in similar outcomes to coronary artery bypass graft surgery (CABG).

ReferenceCitation: Zimmermann FM, De Bruyne B, Pijls NH, Desai M, Oldroyd KG, Park SJ, Reardon MJ, Wendler O, Woo J, Yeung AC, Fearon WF. Rationale and design of the Fractional Flow Reserve versus Angiography for Multivessel Evaluation (FAME) 3 Trial: a comparison of fractional flow reserve-guided percutaneous coronary intervention and coronary artery bypass graft surgery in patients with multivessel coronary artery disease. Am Heart J. 2015 Oct;170(4):619-626.e2. doi: 10.1016/j.ahj.2015.06.024. Epub 2015 Jul 9.

ReferenceCitation: Zimmermann FM, De Bruyne B, Pijls NHJ, Desai M, Oldroyd KG, Reardon MJ, Wendler O, Woo J, Yeung AC, Fearon WF. A protocol update of the Fractional Flow Reserve versus Angiography for Multivessel Evaluation (FAME) 3 trial: A comparison of fractional flow reserve-guided percutaneous coronary intervention and coronary artery bypass graft surgery in patients with multivessel coronary artery disease. Am Heart J. 2019 Aug;214:156-157. doi: 10.1016/j.ahj.2019.04.012. Epub 2019 Apr 29.

ReferenceCitation: Fearon WF, Zimmermann FM, De Bruyne B, Piroth Z, van Straten AHM, Szekely L, Davidavičius G, Kalinauskas G, Mansour S, Kharbanda R, Östlund-Papadogeorgos N, Aminian A, Oldroyd KG, Al-Attar N, Jagic N, Dambrink JE, Kala P, Angerås O, MacCarthy P, Wendler O, Casselman F, Witt N, Mavromatis K, Miner SES, Sarma J, Engstrøm T, Christiansen EH, Tonino PAL, Reardon MJ, Lu D, Ding VY, Kobayashi Y, Hlatky MA, Mahaffey KW, Desai M, Woo YJ, Yeung AC, Pijls NHJ; FAME 3 Investigators. Fractional Flow Reserve-Guided PCI as Compared with Coronary Bypass Surgery. N Engl J Med. 2022 Jan 13;386(2):128-137. doi: 10.1056/NEJMoa2112299. Epub 2021 Nov 4.

ReferenceCitation: Piroth Z, Otsuki H, Zimmermann FM, Ferenci T, Keulards DCJ, Yeung AC, Pijls NHJ, De Bruyne B, Fearon WF. Prognostic Value of Measuring Fractional Flow Reserve After Percutaneous Coronary Intervention in Patients With Complex Coronary Artery Disease: Insights From the FAME 3 Trial. Circ Cardiovasc Interv. 2022 Nov;15(11):884-891. doi: 10.1161/CIRCINTERVENTIONS.122.012542. Epub 2022 Sep 19.

ReferenceCitation: Fearon WF, Zimmermann FM, Ding VY, Zelis JM, Piroth Z, Davidavicius G, Mansour S, Kharbanda R, Ostlund-Papadogeorgos N, Oldroyd KG, Wendler O, Reardon MJ, Woo YJ, Yeung AC, Pijls NHJ, De Bruyne B, Desai M, Hlatky MA; FAME 3 Investigators. Quality of Life After Fractional Flow Reserve-Guided PCI Compared With Coronary Bypass Surgery. Circulation. 2022 May 31;145(22):1655-1662. doi: 10.1161/CIRCULATIONAHA.122.060049. Epub 2022 Apr 2.

lead_sponsor: Stanford University

location_country:
Denmark
Serbia
United Kingdom
Australia
New Zealand
Canada
Lithuania
Sweden
Hungary
Korea, Republic of
United States
France
Belgium
Norway
Netherlands
Czechia

location_facility: Karolinska Institutet, Dep of clinical science and education, Södersjukhuset

location_facility: Masaryk University and University Hospital Brno

location_facility: Vilnius University Hospital Santariskiu Klinikos

location_facility: Le'Centre Hospitalier de l'Universite de Montreal

location_facility: University Hospitals Coventry and Warwickshire

location_facility: Southampton University Hospitals NHS Trust


title: An Open-Label, Non-randomised, Parallel Group, Multicentre, Phase I Study to Assess the Safety and Effect of Olaparib at Steady State on the Pharmacokinetics of the Anti-hormonal Agents Anastrozole, Letrozole and Tamoxifen at Steady State, and the Effect of the Anti-hormonal Agents on Olaparib, Following Administration in Patients With Advanced Solid Cancer

summary: This is an open-label 2-part Phase I study in patients with advanced solid tumours. Part A of the study (mandatory) will assess the effect of olaparib on the pharmacokinetics (PK) of anastrozole, letrozole and tamoxifen and vice versa; Part B will allow patients (if eligible) continued access to olaparib after the PK phase and will provide additional safety data.

primary_purpose: Basic Science

ReferenceCitation: Plummer R, Verheul HM, De Vos FYFL, Leunen K, Molife LR, Rolfo C, Grundtvig-Sørensen P, De Grève J, Rottey S, Jerusalem G, Italiano A, Spicer J, Dirix L, Goessl C, Birkett J, Spencer S, Learoyd M, Bailey C, Dean E. Pharmacokinetic Effects and Safety of Olaparib Administered with Endocrine Therapy: A Phase I Study in Patients with Advanced Solid Tumours. Adv Ther. 2018 Nov;35(11):1945-1964. doi: 10.1007/s12325-018-0804-z. Epub 2018 Oct 15.

lead_sponsor: AstraZeneca

location_country:
Denmark
United Kingdom
France
Belgium
Netherlands


title: EXPERT, EXPosurE Registry RiociguaT in Patients With Pulmonary Hypertension

acronym: EXPERT

summary: In accordance with the regulatory guidance this registry has been designed to collect information about the long-term safety of Adempas in real clinical practice outside the regulated environment of a controlled clinical study.

lead_sponsor: Bayer

location_country:
Luxembourg
Saudi Arabia
Italy
Spain
United Kingdom
Belgium
Czechia
Portugal
Russian Federation
Austria
Australia
Argentina
Turkey
Norway
Sweden
Slovakia
Canada
Switzerland
Germany
Denmark
Taiwan
Greece
Ireland
Estonia
Finland
France
Colombia
Netherlands


title: An Open-Label, Single-Group Clinical Study to Evaluate the Pharmacokinetics, Safety,and Efficacy of Omacetaxine Mepesuccinate Given Subcutaneously as a Fixed Dose inPatients With Chronic Phase or Accelerated Phase Chronic Myeloid Leukemia Who Have Failed 2 or More Tyrosine Kinase Inhibitor Therapies

summary: To satisfy a postmarketing requirement, the sponsor has been requested to conduct a Phase 1/Phase 2 single-group clinical study to investigate the pharmacokinetics and preliminary safety and efficacy of omacetaxine following a fixed-dose administration to patients with CP or AP CML who have failed 2 or more tyrosine kinase inhibitor (TKI) therapies.

primary_purpose: Other

lead_sponsor: Teva Branded Pharmaceutical Products R&D, Inc.

location_country:
Korea, Republic of
United States
France
Belgium

location_facility:
Teva Investigational Site 12547
Teva Investigational Site 37047
Teva Investigational Site 12546
Teva Investigational Site 12545
Teva Investigational Site 12544
Teva Investigational Site 12543
Teva Investigational Site 37048
Teva Investigational Site 35157
Teva Investigational Site 12550
Teva Investigational Site 87026


title: A Phase 1, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects With Advanced Solid Tumors, Including Glioma, With an IDH1 Mutation

summary: The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced solid tumors, including glioma, that harbor an IDH1 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose. The second portion of the study is a dose expansion phase where four arms of patients will receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose. Anticipated time on study treatment is until disease progression, unacceptable toxicity occurs or at Investigator discretion.

enrollment_type: Anticipated

ReferenceCitation: Ellingson BM, Kim GHJ, Brown M, Lee J, Salamon N, Steelman L, Hassan I, Pandya SS, Chun S, Linetsky M, Yoo B, Wen PY, Mellinghoff IK, Goldin J, Cloughesy TF. Volumetric measurements are preferred in the evaluation of mutant IDH inhibition in non-enhancing diffuse gliomas: Evidence from a phase I trial of ivosidenib. Neuro Oncol. 2022 May 4;24(5):770-778. doi: 10.1093/neuonc/noab256.

ReferenceCitation: Aguado-Fraile E, Tassinari A, Ishii Y, Sigel C, Lowery MA, Goyal L, Gliser C, Jiang L, Pandya SS, Wu B, Bardeesy N, Choe S, Deshpande V. Molecular and morphological changes induced by ivosidenib correlate with efficacy in mutant-IDH1 cholangiocarcinoma. Future Oncol. 2021 Jun;17(16):2057-2074. doi: 10.2217/fon-2020-1274. Epub 2021 Mar 12.

ReferenceCitation: Mellinghoff IK, Ellingson BM, Touat M, Maher E, De La Fuente MI, Holdhoff M, Cote GM, Burris H, Janku F, Young RJ, Huang R, Jiang L, Choe S, Fan B, Yen K, Lu M, Bowden C, Steelman L, Pandya SS, Cloughesy TF, Wen PY. Ivosidenib in Isocitrate Dehydrogenase 1-Mutated Advanced Glioma. J Clin Oncol. 2020 Oct 10;38(29):3398-3406. doi: 10.1200/JCO.19.03327. Epub 2020 Jun 12.

ReferenceCitation: Tap WD, Villalobos VM, Cote GM, Burris H, Janku F, Mir O, Beeram M, Wagner AJ, Jiang L, Wu B, Choe S, Yen K, Gliser C, Fan B, Agresta S, Pandya SS, Trent JC. Phase I Study of the Mutant IDH1 Inhibitor Ivosidenib: Safety and Clinical Activity in Patients With Advanced Chondrosarcoma. J Clin Oncol. 2020 May 20;38(15):1693-1701. doi: 10.1200/JCO.19.02492. Epub 2020 Mar 24.

ReferenceCitation: Lowery MA, Burris HA 3rd, Janku F, Shroff RT, Cleary JM, Azad NS, Goyal L, Maher EA, Gore L, Hollebecque A, Beeram M, Trent JC, Jiang L, Fan B, Aguado-Fraile E, Choe S, Wu B, Gliser C, Agresta SV, Pandya SS, Zhu AX, Abou-Alfa GK. Safety and activity of ivosidenib in patients with IDH1-mutant advanced cholangiocarcinoma: a phase 1 study. Lancet Gastroenterol Hepatol. 2019 Sep;4(9):711-720. doi: 10.1016/S2468-1253(19)30189-X. Epub 2019 Jul 9.

ReferenceCitation: Fan B, Mellinghoff IK, Wen PY, Lowery MA, Goyal L, Tap WD, Pandya SS, Manyak E, Jiang L, Liu G, Nimkar T, Gliser C, Prahl Judge M, Agresta S, Yang H, Dai D. Clinical pharmacokinetics and pharmacodynamics of ivosidenib, an oral, targeted inhibitor of mutant IDH1, in patients with advanced solid tumors. Invest New Drugs. 2020 Apr;38(2):433-444. doi: 10.1007/s10637-019-00771-x. Epub 2019 Apr 26.

ipd_sharing: Yes

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.

lead_sponsor: Institut de Recherches Internationales Servier

location_country:
United States
France


title: A Double-blind, Placebo-controlled, Parallel-group, Multicenter Study Of The Efficacy And Safety Of Pregabalin As Adjunctive Therapy In Children 1 Month Through <4 Years Of Age With Partial Onset Seizures

summary: This study is designed to evaluate the effectiveness of 2 doses of pregabalin to reduce seizure frequency as an add on therapy in pediatric subjects 1 month to <4 years of age with refractory partial onset seizures. It is hypothesized that both doses of pregabalin will demonstrate superior efficacy when compared to placebo by reducing the partial onset seizure frequency and that pregabalin will be safe and well tolerated.

ReferenceCitation: Mann D, Antinew J, Knapp L, Almas M, Liu J, Scavone J, Yang R, Modequillo M, Makedonska I, Ortiz M, Kyrychenko A, Nordli D, Farkas V, Farkas MK; A0081042 study group. Pregabalin adjunctive therapy for focal onset seizures in children 1 month to <4 years of age: A double-blind, placebo-controlled, video-electroencephalographic trial. Epilepsia. 2020 Apr;61(4):617-626. doi: 10.1111/epi.16466. Epub 2020 Mar 18.

lead_sponsor: Pfizer's Upjohn has merged with Mylan to form Viatris Inc.

location_country:
Serbia
Spain
Ukraine
United States
Romania
Philippines
Belgium
Russian Federation
Israel
Turkey
Malaysia
Bosnia and Herzegovina
Lebanon
Hungary
China
Germany
Bulgaria
Taiwan
Belarus
Greece
Thailand
Korea, Republic of
France

location_facility: Semmelweis Egyetem, I. Sz. Gyermekgyogyaszati Klinika

location_facility: Pediatric Epilepsy & Neurology Specialists, PA

location_facility: FSFEI HE N.I. Pirogov RNRMU of Minzdrav of Russia

location_facility: Pharmacy of Tel Aviv Sourasky Medical Center

location_facility: Komunalnyi zaklad "Dnipropetrovska dytiacha miska klinichna likarnia #5"

location_facility: St. Petersburg State Pediatric Medical University

location_facility: Chang Gung Memorial Hospital- Kaohsiung branch

location_facility: Siriraj Hospital, Faculty of Medicine, Mahidol University

location_facility: Karadeniz Teknik Universitesi Tip Fakultesi Farabi Hastanesi

location_facility: The First Bethune Hospital of Jilin University

location_facility: DZ "Ukrainskyi medychnyi tsentr reabilitatsii ditei z orhanichnym urazhenniam

location_facility: Perm State Medical University n. a. acad. E.A. Vagner

location_facility: RSBHI Smolensk Regional Clinical Hospital

location_facility: Mother and Child Healthcare Institute Dr Vukan Cupic

location_facility: Oblasnyi klinichnyi tsentr neirokhirurhii ta nevrolohii,viddilennia neirokhirurhii #2

location_facility: Szent János Kórház es Észak Budai Egyesitett Kórházak

location_facility: University Clinical Centre of the Republic of Srpska

location_facility: Dr. Kenessey Albert Korhaz es Rendelointezet

location_facility: Ivano-Frankivska oblasna dytiacha klinichna likarnia

location_facility: Saint George Hospital - University Medical Center

location_facility: Spitalul Clinic de Urgente pentru Copii "Sf. Maria"

location_facility: Eskisehir Osmangazi Universitesi Tip Fakultesi

location_facility: Pediatric Epilepsy Center of Central Florida

location_facility: Izmir Tepecik Training and Research Hospital

location_facility: SHI Ulyanovsk Regional Children's Clinical Hospital n. a. Y.F.Goryachev

location_facility: American University of Beirut Medical Center

location_facility: GU Republican Scientific and Practical Center Mother and Child

location_facility: Magyarorszagi Reformatus Egyhaz Bethesda Gyermekkorhaz, Gyermekneurologia

location_facility: Pharmacy of The E. Wolfson Medical Center

location_facility: The University of Texas Health Science Center at Houston

location_facility: Institute for Child and Youth Healthcare of Vojvodina

location_facility: Komunalnyi zaklad "Dnipropetrovska oblasna dytiacha klinichna likarnia"

location_facility: General Hospital of Thessaloniki Ippokratio


title: A Prospective, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel-group, 12-week Study to Evaluate the Safety and Tolerability of Macitentan in Subjects With Combined Pre- and Post-capillary Pulmonary Hypertension (CpcPH) Due to Left Ventricular Dysfunction

summary: Study to evaluate if macitentan is safe and tolerable enough to be used for treatment of subjects with combined pre- and post-capillary pulmonary hypertension (CpcPH) due to left ventricular dysfunction.

lead_sponsor: Actelion

location_country:
Spain
Italy
Austria
Canada
Switzerland
United States
France
Israel
Belgium
Germany
Czechia

location_facility: IKEM (Institut klinické a experimentální medicíny, Institute for Clinical and Experimental Medicine)

location_facility: Krankenhaus der Elisabethinen Linz, 2. Interne Abteilung

location_facility: Washington University School of Medicine - Center for Advanced Med

location_facility: A.O. Universitaria Policlinico S. Orsola-Malpighi - Dipartimento di Medicina Specialistica, Diagnostica e Sperimentale - Unità Operativa di Cardiologia

location_facility: Institute of Pulmonology Hadassah Medical Centre : Ein Karem

location_facility: Kaplan Medical Centre / Pulmonary Institute and Department of Medicine

location_facility: University of Michigan Internal Medicine Cardiology, Pulmonary Hypertension Program

location_facility: Hospital Vall d´Hebron Servicio de Cardiologia

location_facility: Hospital Universitario 12 Octubre Servicio de Cardiología

location_facility: Universitätsklinikum Köln Herzzentrum / Klinik III für Innere Medizin (Kardiologie, Pneumologie, Angiologie und Intensivmedizin)

location_facility: Centre Hospitalier Universitaire Vaudois Service de Cardiologie

location_facility: The Chaim Sheba Medical Center / The Institute of Pulmonology, Physiology and Exercise

location_facility: Universitätsklinik Schleswig-Holstein Campus Kiel Klinik für Innere Medizin III Kardiologie und Angiologie

location_facility: Houston Methodist Hospital - Heart Failure/Pulm Hypertension

location_facility: Universitätsklinik für Kardiologie Schweizer Herz- und Gefässzentrum Bern

location_facility: Hôpital Erasme, Cliniques Universitaires de Bruxelles, Cardiologie

location_facility: Hôpital Charles Nicolle Service de Cardiologie

location_facility: Klinikum der Universität München Medizinische Klinik und Poliklinik 1 - Großhadern Schwerpunkt Pneumologie

location_facility: DRK Klinken Berlin Kopenick Klinik für Innere Medizin Kardiologie

location_facility: University Hospital Gasthuisberg / Interne Geneeskunde - I.G. Pneumologie

location_facility: FN Olomouc, 1. Interní klinika - kardiologická

location_facility: Medical University of Vienna and AKH Cardiology

location_facility: Hospital Reina Sofia Servicio de Cardiologia

location_facility: FN Brno-Bohunice, I. interní kardiologická klinika

location_facility: Lékařská fakulta a Všeobecná fakultní nemocnice v Praze, II. Interní klinika kardiologie a angiologie


title: A Multicenter, Open-label, Long-term Follow-up Safety and Efficacy Study of GLPG0634 Treatment in Subjects With Moderately to Severely Active Rheumatoid Arthritis

lead_sponsor: Galapagos NV

ReferenceCitation: Combe B, Besuyen R, Gómez-Centeno A, Matsubara T, Sancho Jimenez JJ, Yin Z, Buch MH. Geographic Analysis of the Safety and Efficacy of Filgotinib in Rheumatoid Arthritis. Rheumatol Ther. 2022 Oct 7. doi: 10.1007/s40744-022-00494-1. [Epub ahead of print]

ReferenceCitation: Kavanaugh A, Westhovens RR, Winthrop KL, Lee SJ, Tan Y, An D, Ye L, Sundy JS, Besuyen R, Meuleners L, Stanislavchuk M, Spindler AJ, Greenwald M, Alten R, Genovese MC. Safety and Efficacy of Filgotinib: Up to 4-year Results From an Open-label Extension Study of Phase II Rheumatoid Arthritis Programs. J Rheumatol. 2021 Aug;48(8):1230-1238. doi: 10.3899/jrheum.201183. Epub 2021 Feb 1.

location_country:
Spain
Ukraine
United States
Romania
Belgium
Czechia
Poland
Russian Federation
Australia
Argentina
Israel
Mexico
Guatemala
Hungary
Germany
Bulgaria
Moldova, Republic of
Latvia
New Zealand
France
Chile
Colombia

location_facility: Centro de Estudios de Investigacion Basica y Clinica, SC

location_facility: Centro de Investigacion en Reumatologia y Especialidades Medicas (CIREEM)

location_facility: Municipal Non-profit Enterprise Consultative and Diagnostic Center of Pechersk District of Kiev city

location_facility: Specjalisty Czne Centrum Medyczne Nowomed

location_facility: Kherson City Clinical Hospital N. A. Afanasii and Olga Tropin

location_facility: Powiatowy Zakrad Opieki Zdrowotnej w Starachowicach

location_facility: CIMeL Centro dee Investigacion Medico Lanus

location_facility: Instituto de Asistencia Reumatologia Integral - IARI

location_facility: Diagnostic Consultative Center "Sveta Anna" LTD

location_facility: NZOZ "DOBRY LEKARZ" Specjalistyczne Poradnie Lekarskie

location_facility: Sanatorio Nuestra Señora de la Esperanza

location_facility: Municipal Non-Profit Institution Consultative and Diagnostic Centre of Desnyasky District of Kyiv

location_facility: Hospital Regional "Guillermo Grant Benavente"

location_facility: Spitalul Clinic Judetean de Urgenta Sf. Apostol Andrei Galati

location_facility: Multiprofile Hospital for Active Treatment - Ruse

location_facility: NZOZ "Nasz Lekarz" Pratyka Grupowa Lekarzy Rodzinnychz

location_facility: Complejo Hospitalario Universitario A Coruña (CHUAC)

location_facility: Kharkiv Medical Academy of Postgraduate Education, Department of Cardiology

location_facility: MVZ Rheumatologie and Autoimmun Medizin HH GmbH

location_facility: GBOU VPO Orenburg State Medical University

location_facility: National Multiprofile Transport Hospital "Tsar Boris III," Sofia, Clinical of Internal Diseases

location_facility: L.T. Malaya Therapy Institute of National Academy of Medical Sciences of Ukraine

location_facility: Vladimir Regional State Instituion of Healthcare

location_facility: Vinnitsya Regional Clinical Hospital Named after M.I.Pirogov, Rheumatology Department

location_facility: First Moscow State Medical University n.a. I.M. Sechenova of the Ministry of Health

location_facility: University Multiprofile Hospital for Active Treatment "SV. Ivan Rilski" EAD, Sofia, Rheumatology Clinic

location_facility: University "Multiprofile Hospital for Active Treatment - Kaspela" LTD

location_facility: Scientific Research Institute of Rheumatology

location_facility: Organizacion Medica de Investigaciones (OMI)

location_facility: Communal Institution of Healthcare - Kharkiv City Clinical Hospital #13

location_facility: Arizona Arthritis & Rheumatology Research PLLC

location_facility: Schlossparkklinik - Akad. Lehrkrankenhaus Charite

location_facility: Clinica Medica Especializada en Reumatologia

summary:
The primary objective of the study is to evaluate the long-term safety and tolerability of filgotinib (formerly GLPG0634) for the treatment of rheumatoid arthritis.

Participants will be enrolled in this open-label long-term follow-up study after they have completed one of the two core studies, GLPG0634-CL-203 (DARWIN1) or GLPG0634-CL-204 (DARWIN2), and will be evaluated for any side effects that may occur (long-term safety and tolerability) when taking filgotinib. During the course of the study, participants will also be examined for long-term effects of filgotinib administration on disease activity (efficacy), subjects' disability, fatigue, and quality of life.


title: A Phase 2 Clinical Trial of Nivolumab, or Nivolumab Combinations in Recurrent and Metastatic Microsatellite Instability High (MSI-H) and Non-MSI-H Colon Cancer

summary: The purpose of this study is to examine if Nivolumab by itself, or Nivolumab in combination with other anti-cancer drugs, will result in meaningful tumor size reduction, in participants with colon cancer that has come back or has spread, and who have a specific biomarker in their tumors.

ReferenceCitation: Lenz HJ, Van Cutsem E, Luisa Limon M, Wong KYM, Hendlisz A, Aglietta M, García-Alfonso P, Neyns B, Luppi G, Cardin DB, Dragovich T, Shah U, Abdullaev S, Gricar J, Ledeine JM, Overman MJ, Lonardi S. First-Line Nivolumab Plus Low-Dose Ipilimumab for Microsatellite Instability-High/Mismatch Repair-Deficient Metastatic Colorectal Cancer: The Phase II CheckMate 142 Study. J Clin Oncol. 2022 Jan 10;40(2):161-170. doi: 10.1200/JCO.21.01015. Epub 2021 Oct 12.

ReferenceCitation: Overman MJ, Lonardi S, Wong KYM, Lenz HJ, Gelsomino F, Aglietta M, Morse MA, Van Cutsem E, McDermott R, Hill A, Sawyer MB, Hendlisz A, Neyns B, Svrcek M, Moss RA, Ledeine JM, Cao ZA, Kamble S, Kopetz S, André T. Durable Clinical Benefit With Nivolumab Plus Ipilimumab in DNA Mismatch Repair-Deficient/Microsatellite Instability-High Metastatic Colorectal Cancer. J Clin Oncol. 2018 Mar 10;36(8):773-779. doi: 10.1200/JCO.2017.76.9901. Epub 2018 Jan 20.

ReferenceCitation: Overman MJ, McDermott R, Leach JL, Lonardi S, Lenz HJ, Morse MA, Desai J, Hill A, Axelson M, Moss RA, Goldberg MV, Cao ZA, Ledeine JM, Maglinte GA, Kopetz S, André T. Nivolumab in patients with metastatic DNA mismatch repair-deficient or microsatellite instability-high colorectal cancer (CheckMate 142): an open-label, multicentre, phase 2 study. Lancet Oncol. 2017 Sep;18(9):1182-1191. doi: 10.1016/S1470-2045(17)30422-9. Epub 2017 Jul 19. Erratum in: Lancet Oncol. 2017 Sep;18(9):e510.

lead_sponsor: Bristol-Myers Squibb

location_country:
Spain
Ireland
Italy
Australia
Canada
United States
France
Belgium

location_facility:
Local Institution - 0037
Local Institution - 0022
Local Institution - 0036
Local Institution - 0012
Local Institution - 0027
Local Institution - 0011
Local Institution - 0040
Local Institution - 0005
Local Institution - 0029
Local Institution - 0033
Local Institution - 0010
Local Institution - 0025
Local Institution - 0003
Local Institution - 0018
Local Institution - 0030
Local Institution - 0024
Local Institution - 0016
Local Institution - 0034
Local Institution - 0023
Local Institution - 0028
Local Institution - 0001
Local Institution - 0020
Local Institution - 0041
Local Institution - 0019
Local Institution - 0039
Local Institution - 0035
Local Institution - 0013
Local Institution - 0002
Local Institution - 0004
Local Institution - 0008
Allina Health Cancer Institute
Local Institution - 0006
Local Institution - 0032


acronym: ACH

title: A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children With Achondroplasia

lead_sponsor: BioMarin Pharmaceutical

ReferenceCitation: Chan ML, Qi Y, Larimore K, Cherukuri A, Seid L, Jayaram K, Jeha G, Fisheleva E, Day J, Huntsman-Labed A, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Horton WA, Henshaw J. Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia. Clin Pharmacokinet. 2022 Feb;61(2):263-280. doi: 10.1007/s40262-021-01059-1. Epub 2021 Aug 25.

ReferenceCitation: Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019 Jul 4;381(1):25-35. doi: 10.1056/NEJMoa1813446. Epub 2019 Jun 18.

location_country:
Australia
United States
United Kingdom
France

location_facility: Johns Hopkins McKusick - Institute of Genetic Medicine

location_facility: Children's Hospital & Research Center Oakland

location_facility: Guys & St. Thomas NHS Foundation Trust Evelina Hospital

location_facility: Ann and Robert H. Lurie Childrens Hospital of Chicago

summary: This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia.


title: A Phase 1 Open-label Study of Safety and Tolerability of MEDI4736 in Subjects With Metastatic or Unresectable Melanoma in Combination With Dabrafenib and Trametinib or With Trametinib Alone

summary: The purpose of this study is to determine the maximum tolerated dose and characterize the safety profile of durvalumab (MEDI4736) in combination with dabrafenib and trametinib or with trametinib alone in participants with metastatic or unresectable melanoma with BRAF-mutation positive or wild-type (WT) BRAF, respectively.

ReferenceCitation: Gordon MS, Lutzky J, Lawrence D, Butler M, Ascierto PA, Hug B, et al. Phase 1 study evaluating safety and tolerability of MEDI4736, an anti-programmed cell death ligand-1 (PD-L1) antibody, in combination with dabrafenib and trametinib or trametinib alone in patients with unresectable or metastatic melanoma. Ann Oncol 2014; 25(suppl_4): iv374-iv393 (abstract 8004).

ReferenceCitation: Ribas A, Butler M, Lutzky J, Lawrence DP, Robert C, Miller W, et al. Phase 1 study combining anti-PD-L1 (MEDI4736) and BRAF (dabrafenib) and/or MEK (trametinib) inhibitors in advanced melanoma. J Clin Oncol 2015; 33 (15_suppl): (abstract 3003).

ReferenceCitation: Ribas A, Algazi A, Ascierto PA, Butler MO, Chandra S, Gordon M, Hernandez-Aya L, Lawrence D, Lutzky J, Miller WH Jr, Campbell KM, Delafont B, Marshall S, Mueller N, Robert C. PD-L1 blockade in combination with inhibition of MAPK oncogenic signaling in patients with advanced melanoma. Nat Commun. 2020 Dec 7;11(1):6262. doi: 10.1038/s41467-020-19810-w. Erratum in: Nat Commun. 2021 Aug 12;12(1):5022.

location_country:
United States
Italy
Canada
France


title: Multicenter, Open-label Extension Study to Investigate the Long-term Safety and Efficacy of IgPro20 in Maintenance Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in Subjects Completing Study IgPro20_3003

summary:
This study is an extension study to the pivotal study IgPro20_3003 (NCT01545076). The purpose of this extension study is to investigate the long-term treatment of CIDP with IgPro20, with regard to safety and efficacy.

Subjects who have completed subcutaneous (SC) Week 25 or were successfully rescued from a CIDP relapse during the SC Treatment Period of pivotal study IgPro20_3003 (NCT01545076) will have the option to receive open-label low-dose IgPro20 (0.2 g/kg bodyweight [bw]) weekly for up to 48 weeks. Subjects relapsing on low-dose IgPro20 will either return to high-dose IgPro20 (0.4 g/kg) immediately or be discontinued, depending on investigator's judgment. Subjects returning to high-dose IgPro20 will continue on high-dose until they have completed a total of 48 weeks of IgPro20 treatment. If subjects do not successfully recover from CIDP relapse within 4 weeks, they will be withdrawn.

The treatment duration will be up to 48 weeks, followed by a completion visit (week 49).

lead_sponsor: CSL Behring

location_country:
Spain
Italy
United Kingdom
Australia
Netherlands
Canada
United States
France
Japan
Germany
Czechia

location_facility:
Site Reference 2760069
Site Reference 2760049
Site Reference 2760047
Site Reference 2760055
Site Reference 0360011
Site Reference 2760039
Site Reference 3920035
Site Reference 2030002
Site Reference 3800031
Site Reference 1240009
Site Reference 8400169
Site Reference 2760052
Site Reference 5280001
Site Reference 2760094
Site Reference 8260019
Site Reference 3920038
Site Reference 7240011
Site Reference 1240007
Site Reference 0360017
Site Reference 8400181
Site Reference 2760054
Site Reference 3920037
Site Reference 8400182
Site Reference 2030009
Site Reference 8400166
Site Reference 3920061
Site Reference 3920040
Site Reference 7240010
Site Reference 8400167
Site Reference 2500022
Site Reference 3920065
Site Reference 8260032
Site Reference 2760072


title: A Randomized, Placebo-controlled, Double-blind, Parallel Group, Multicenter Study to Investigate the Efficacy and Safety of 5 Fixed Doses of BAY85-3934 Administered Orally in the Correction of Anemia in Pre-dialysis Subjects With Chronic Kidney Disease Not Currently Treated With Erythropoiesis-stimulating Agent in Europe and Asia Pacific

acronym: DIALOGUE 1

summary:
Anaemia is a condition in which blood has a lower than normal number of red blood cells. It can also occur if red blood cells do not contain enough haemoglobin, an oxygen carrying part of blood. Anaemia is common in patients with chronic kidney disease. Healthy kidneys produce a hormone called erythropoietin, which stimulates the bone marrow to produce the proper number of red blood cells needed to carry oxygen to vital organs. Chronic kidney disease is a general term that means that the kidneys are not functioning to their full potential. The study drug, BAY85-3934, is being evaluated as a drug to increase the body's ability to produce erythropoietin.

The purpose of this study is to find out if the study drug, a tablet taken orally, is safe and effective for the treatment of anaemia associated with chronic kidney disease.

The study will enroll 120 patients at multiple locations in Europe, Asia and Australia. Participation will involve a screening visit and between 12 and 14 study visits scheduled over a period of approximately 5 to 7 months. The estimated total duration of study treatment will be 16 weeks. During these scheduled visits patients will undergo a number of procedures to confirm efficacy and safety of the study drug, including measurement of heart rate and blood pressure, physical examination, Electrocardiogram and blood/urine sample collection for laboratory tests.

The study will be conducted at 5 hospitals in the UK. Bayer HealthCare AG is funding this research.

ReferenceCitation: Natale P, Palmer SC, Jaure A, Hodson EM, Ruospo M, Cooper TE, Hahn D, Saglimbene VM, Craig JC, Strippoli GF. Hypoxia-inducible factor stabilisers for the anaemia of chronic kidney disease. Cochrane Database Syst Rev. 2022 Aug 25;8:CD013751. doi: 10.1002/14651858.CD013751.pub2. Review.

location_country:
Poland
Spain
Italy
United Kingdom
Australia
Hungary
Korea, Republic of
France
Israel
Turkey
Romania
Japan
Germany
Bulgaria

location_facility:
Ankara Univ. Medical Faculty
Sifa University Medical Faculty
Baskent University Medical Faculty


title: A Phase 1b Study of the Safety and Pharmacology of Atezolizumab (Anti-PD-L1 Antibody) Administered With Erlotinib or Alectinib in Patients With Advanced Non-Small Cell Lung Cancer

summary:
This open-label, multicenter study will assess the safety, tolerability, and pharmacokinetics of intravenous (IV) dosing of atezolizumab in combination with oral erlotinib or alectinib in participants with NSCLC.

This study has two stages. In the erlotinib group, the combination treatment will be given to participants with epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI)-treatment-naive, advanced (nonresectable) NSCLC in a safety-evaluation stage and to participants with previously untreated EGFR mutation-positive, advanced NSCLC in an expansion stage (Stage 2). In the alectinib group, for both the safety-evaluation and expansion stages (Stages 1 and 2), the combination will be given to participants who are treatment-naive with anaplastic lymphoma kinase (ALK)-positive advanced NSCLC.

In Stage 1, erlotinib will be given at a starting dose of 150 milligrams (mg) by mouth (PO) once daily (QD) and the starting dose of alectinib will be 600 mg twice daily (BID), for 28 consecutive days during Cycle 1 and on Days 1 through 21 of each cycle thereafter. The starting dose of atezolizumab will be 1200 mg, administered every 3 weeks (q3W) starting on Day 8 of Cycle 1. If the starting regimen for a combination treatment is not tolerated, alternative doses and/or schedules of erlotinib and atezolizumab or alectinib and atezolizumab may be tested to determine potential recommended Phase 2 dose (RP2D) for that combination treatment. In Stage 2, a potential RP2D and schedule for each combination treatment will be investigated in an expansion cohort.

For both stages, continuation of treatment beyond Cycle 1 will be at the discretion of the treating investigator. Study treatment will be discontinued in participants who experience disease progression or unacceptable toxicity, are not compliant with the study protocol, or, in their opinion or in the opinion of the investigator, are not benefiting from study treatment. However, in the absence of unacceptable toxicity, participants with second-line or greater NSCLC who are still receiving atezolizumab at the time of radiographic disease progression may be permitted to continue study treatment.

lead_sponsor: Hoffmann-La Roche

location_country:
Hong Kong
Spain
United Kingdom
Korea, Republic of
United States
France

location_facility: Massachusetts General Hospital;Hematology/ Oncology

location_facility: Case Western Reserve University; Medicine-Hematology and Oncology

location_facility: Memorial Sloan Kettering - Basking Ridge

location_facility: Karmanos Cancer Center; Department of Oncology

location_facility: Hospital Clinico Universitario de Valencia

location_facility: START Madrid. Centro Integral Oncologico Clara Campal; CIOCC

location_facility: Beth Israel Deaconess Med Ctr; Neurology/MS Center


title: A Phase 1B Dose Escalation Study to Investigate the Safety, Tolerability and Preliminary Efficacy for the Combination Dasatinib (BMS-354825) Plus Nivolumab (BMS-936558) in Patients Chronic Myeloid Leukemia (CML)

summary: The purpose of this study is to find a dose of Nivolumab that can be safely added to Dasatinib in patients with Chronic Myeloid Leukemia.

location_country:
Spain
Italy
Australia
Canada
United States
France
Germany

location_facility: The University Of Texas MD Anderson Cancer Center

location_facility: Ut Southwestern Medical Center At Dallas

location_facility: Froedtert Hospital & Medical College of Wisconsin


title: Micra Transcatheter Pacing Study

summary: The purpose of this clinical study is to evaluate the safety and efficacy of the Micra Transcatheter Pacing System and to assess long term performance.

ReferenceCitation: Piccini JP, Cunnane R, Steffel J, El-Chami MF, Reynolds D, Roberts PR, Soejima K, Steinwender C, Garweg C, Chinitz L, Ellis CR, Stromberg K, Fagan DH, Mont L. Development and validation of a risk score for predicting pericardial effusion in patients undergoing leadless pacemaker implantation: experience with the Micra transcatheter pacemaker. Europace. 2022 Jul 21;24(7):1119-1126. doi: 10.1093/europace/euab315.

ReferenceCitation: El-Chami MF, Shinn T, Bansal S, Martinez-Sande JL, Clementy N, Augostini R, Ravindran B, Sagi V, Ramanna H, Garweg C, Roberts PR, Soejima K, Stromberg K, Fagan DH, Zuniga N, Piccini JP. Leadless pacemaker implant with concomitant atrioventricular node ablation: Experience with the Micra transcatheter pacemaker. J Cardiovasc Electrophysiol. 2021 Mar;32(3):832-841. doi: 10.1111/jce.14881. Epub 2021 Jan 23.

ReferenceCitation: Garg A, Koneru JN, Fagan DH, Stromberg K, Padala SK, El-Chami MF, Roberts PR, Piccini JP, Cheng A, Ellenbogen KA. Morbidity and mortality in patients precluded for transvenous pacemaker implantation: Experience with a leadless pacemaker. Heart Rhythm. 2020 Dec;17(12):2056-2063. doi: 10.1016/j.hrthm.2020.07.035. Epub 2020 Aug 4.

ReferenceCitation: Tjong FVY, Beurskens NEG, de Groot JR, Waweru C, Liu S, Ritter P, Reynolds D, Wilde AAM, Knops RE; Micra Investigators. Health-related quality of life impact of a transcatheter pacing system. J Cardiovasc Electrophysiol. 2018 Dec;29(12):1697-1704. doi: 10.1111/jce.13726. Epub 2018 Oct 15.

ReferenceCitation: El-Chami MF, Al-Samadi F, Clementy N, Garweg C, Martinez-Sande JL, Piccini JP, Iacopino S, Lloyd M, Viñolas Prat X, Jacobsen MD, Ritter P, Johansen JB, Tondo C, Liu F, Fagan DH, Eakley AK, Roberts PR. Updated performance of the Micra transcatheter pacemaker in the real-world setting: A comparison to the investigational study and a transvenous historical control. Heart Rhythm. 2018 Dec;15(12):1800-1807. doi: 10.1016/j.hrthm.2018.08.005. Epub 2018 Aug 10.

ReferenceCitation: Okabe T, El-Chami MF, Lloyd MS, Buck B, Gornick CC, Moore JC, Augostini RS, Hummel JD. Leadless pacemaker implantation and concurrent atrioventricular junction ablation in patients with atrial fibrillation. Pacing Clin Electrophysiol. 2018 May;41(5):504-510. doi: 10.1111/pace.13312. Epub 2018 Apr 16.

ReferenceCitation: Soejima K, Asano T, Ishikawa T, Kusano K, Sato T, Okamura H, Matsumoto K, Taguchi W, Stromberg K, Lande J, Kobayashi Y; Micra Transcatheter Pacing Study Group. Performance of Leadless Pacemaker in Japanese Patients vs. Rest of the World - Results From a Global Clinical Trial. Circ J. 2017 Oct 25;81(11):1589-1595. doi: 10.1253/circj.CJ-17-0259. Epub 2017 May 30.

ReferenceCitation: Lloyd M, Reynolds D, Sheldon T, Stromberg K, Hudnall JH, Demmer WM, Omar R, Ritter P, Hummel J, Mont L, Steinwender C, Duray GZ. Rate adaptive pacing in an intracardiac pacemaker. Heart Rhythm. 2017 Feb;14(2):200-205. doi: 10.1016/j.hrthm.2016.11.016. Epub 2016 Nov 15.

ReferenceCitation: Reynolds D, Duray GZ, Omar R, Soejima K, Neuzil P, Zhang S, Narasimhan C, Steinwender C, Brugada J, Lloyd M, Roberts PR, Sagi V, Hummel J, Bongiorni MG, Knops RE, Ellis CR, Gornick CC, Bernabei MA, Laager V, Stromberg K, Williams ER, Hudnall JH, Ritter P; Micra Transcatheter Pacing Study Group. A Leadless Intracardiac Transcatheter Pacing System. N Engl J Med. 2016 Feb 11;374(6):533-41. doi: 10.1056/NEJMoa1511643. Epub 2015 Nov 9.

ReferenceCitation: Ritter P, Duray GZ, Steinwender C, Soejima K, Omar R, Mont L, Boersma LV, Knops RE, Chinitz L, Zhang S, Narasimhan C, Hummel J, Lloyd M, Simmers TA, Voigt A, Laager V, Stromberg K, Bonner MD, Sheldon TJ, Reynolds D; Micra Transcatheter Pacing Study Group. Early performance of a miniaturized leadless cardiac pacemaker: the Micra Transcatheter Pacing Study. Eur Heart J. 2015 Oct 1;36(37):2510-9. doi: 10.1093/eurheartj/ehv214. Epub 2015 Jun 4.

ReferenceCitation: Ritter P, Duray GZ, Zhang S, Narasimhan C, Soejima K, Omar R, Laager V, Stromberg K, Williams E, Reynolds D; Micra Transcatheter Pacing Study Group. The rationale and design of the Micra Transcatheter Pacing Study: safety and efficacy of a novel miniaturized pacemaker. Europace. 2015 May;17(5):807-13. doi: 10.1093/europace/euv026. Epub 2015 Apr 7.

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
Denmark
Serbia
Spain
Greece
Austria
Australia
Italy
Canada
Netherlands
United Kingdom
Hungary
United States
France
China
India
South Africa
Japan
Malaysia
Czechia

location_facility: University of Oklahoma Health Science Center (OUHSC)

location_facility: Institut Jantung Negara - National Heart Institute

location_facility: St. Antonius Ziekenhuis - Locatie Nieuwegein

location_facility: Hospital Universitari Clínic de Barcelona

location_facility: Fuwai Hospital Chinese Academy of Medical Sciences and Peking Union Medical College

location_facility: Azienda Ospedaliero Universitaria Pisana - Stabilimento Santa Chiara

location_facility: National Cerebral and Cardiovascular Center

location_facility: University General Hospital of Heraklion

location_facility: University of Pittsburgh Medical Center UPMC Presbyterian

location_facility: Lancaster Heart & Vascular Research Institute

location_facility: Southampton General Hospital - University Hospital Southampton NHS Foundation Trust

location_facility: Institut Universitaire de Cardiologie et de Pneumologie de Québec (IUCPQ)


title: A Phase 2/3, Multicenter, Randomized, Double-blinded, Placebo-controlled, Repeat-dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics, and Pharmacokinetics of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

acronym: ASCEND

summary:
Primary Objective:

The primary objective of this phase 2/3 study is to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult participants with acid sphingomyelinase deficiency (ASMD) by assessing changes in: 1) spleen volume as measured by abdominal magnetic resonance imaging (MRI) (and, for the United States [US] only, in association with participant perception related to spleen volume as measured by splenomegaly-related score [SRS]); and 2) infiltrative lung disease as measured by the pulmonary function test, diffusing capacity of the lung for carbon monoxide (DLCO).

Secondary Objectives:

To confirm the safety of olipudase alfa administered intravenously once every 2 weeks for 52 weeks.
To characterize the effect of olipudase alfa on the participant perception related to spleen volume as measured by the SRS after 52 weeks of study drug administration. (For the US, the effect of olipudase alfa on the SRS is part of the primary objective).
To characterize the effect of olipudase alfa after 52 weeks of study drug administration on the following outcome measures assessed sequentially:
The effect of olipudase alfa on liver volume;
The effect of olipudase alfa on platelet count;
The effect of olipudase alfa on fatigue;
The effect of olipudase alfa on pain;
The effect of olipudase alfa on dyspnea.

ipd_sharing_description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org.

location_country:
Brazil
Portugal
Spain
Italy
United Kingdom
Australia
Netherlands
Argentina
Tunisia
United States
Chile
France
Turkey
Belgium
Japan
Germany
Bulgaria

location_facility:
Investigational Site Number :620002
Investigational Site Number :392001
Investigational Site Number :724001
Investigational Site Number :840006
Investigational Site Number :826001
Investigational Site Number :792002
Investigational Site Number :380001
Investigational Site Number :276001
Investigational Site Number :528001
Investigational Site Number :100001
Investigational Site Number :840005
Investigational Site Number :380002
Investigational Site Number :032001
Investigational Site Number :056001
Investigational Site Number :788001
Investigational Site Number :840003
Investigational Site Number :250001
Investigational Site Number :826002
Investigational Site Number :076001
Investigational Site Number :152001
Investigational Site Number :792003
Investigational Site Number :792001
Investigational Site Number :036001


title: Cochlear-Implanted Recipient Observational Study

acronym: IROS

summary:
The purpose of this study is to collect patient related benefit data following treatment for permanent hearing loss with a hearing implant from the company Cochlear over a period of 2 years post treatment.

Assessment of benefits is based on standard questionnaires of hearing ability and quality of life in general.

ReferenceCitation: Maes IH, Joore MA, Cima RF, Vlaeyen JW, Anteunis LJ. Assessment of health state in patients with tinnitus: a comparison of the EQ-5D and HUI mark III. Ear Hear. 2011 Jul-Aug;32(4):428-35. doi: 10.1097/AUD.0b013e3181fdf09f.

ReferenceCitation: Noble W, Tyler R, Dunn C, Bhullar N. Unilateral and bilateral cochlear implants and the implant-plus-hearing-aid profile: comparing self-assessed and measured abilities. Int J Audiol. 2008 Aug;47(8):505-14. doi: 10.1080/14992020802070770.

ReferenceCitation: Mauch H, Kaur J, Irwin C, Wyss J. Design, implementation, and management of an international medical device registry. Trials. 2021 Nov 25;22(1):845. doi: 10.1186/s13063-021-05821-5.

lead_sponsor: Cochlear

location_country:
Poland
Brazil
Belgium
Portugal
Slovenia
Austria
Netherlands
Spain
Argentina
Sweden
Hungary
France
South Africa
Turkey
Colombia
Germany

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 2 w Poznaniu

location_facility: Hospital Clinico Universitario Lozano Blesa

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 4 w Lublinie

location_facility: Universitätsklinik für Hals-, Nasen- und Ohrenheilkunde

location_facility: Durban Cochlear Implant Pogramme, Parklands Hospital

location_facility: Hospital Universitario Clinica San Rafael

location_facility: Instituto Nacional de Otologia Garcia Gomez

location_facility: Wojewodzki Szpital Dzieciecy w Bydgoszczy

location_facility: Uniwersytecki Szpital Kliniczny w Bialymstoku

location_facility: Hospital Clinico Universitario de Salamanca

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Centro de Investigaciones Otoaudiologicas (CIOA)

location_facility: Instytut Centrum Zdrowia Matki Polki w Lodzi

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 1 w Szczecinie

location_facility: Implantes Cocleares Equipo Multicentrico (ICEM)

location_facility: Johannesburg Cochlear Implant Center, Wits Donald Gordon Medical Centre

location_facility: Uniwersytecki Szpital Kliniczny nr 1 w Lodzi