summary: The study will evaluate the safety and efficacy of the intravitreal implant of dexamethasone with Anti-VEGF treatment vs. Anti-VEGF alone (with sham dexamethasone injection) in patients with subfoveal choroidal neovascularization secondary to age-related macular degeneration.

ReferenceCitation: Kuppermann BD, Goldstein M, Maturi RK, Pollack A, Singer M, Tufail A, Weinberger D, Li XY, Liu CC, Lou J, Whitcup SM; Ozurdex® ERIE Study Group. Dexamethasone Intravitreal Implant as Adjunctive Therapy to Ranibizumab in Neovascular Age-Related Macular Degeneration: A Multicenter Randomized Controlled Trial. Ophthalmologica. 2015;234(1):40-54. doi: 10.1159/000381865. Epub 2015 Jun 18.

lead_sponsor: Allergan

location_country:
Portugal
United Kingdom
Italy
Australia
New Zealand
Korea, Republic of
United States
Israel
France


title: A Randomized, Open-label, Multi-center Study of Larotaxel at 90mg/m2 or Docetaxel Every 3 Weeks, Alone or in Combination With Trastuzumab According to Her2neu Status, Administered After a Combination of Anthracycline and Cyclophosphamide as Pre-operative Therapy in Patients With High Risk Localized Breast Cancer.

acronym: SATIN

summary:
The primary objective of this study is to assess the pathological Complete Response (pCR) rate by treatment arm (according to Chevallier criteria).

The secondary objectives are:

to assess in each treatment arm the clinical Response Rate (RR), the rate of breast conservation, the Progression-Free Survival (PFS), the Overall Survival (OS), the safety and tolerability profile, the pathological Complete Response rate (pCR) according to NSABP and Sataloff criteria,
to rank docetaxel and larotaxel alone in Her2 -ve patients, or combined with trastuzumab in Her2 +ve patients, according to the pCR rate.

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

lead_sponsor: Sanofi

location_country:
Poland
Brazil
United Kingdom
Tunisia
Uruguay
France
Belgium
Germany

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


title: A Randomized, Double-Bind, Placebo-Controlled, Parallel-Group, Fixed-Dose, Multicenter Study to Assess Efficacy and Safety of Rimonabant 20 mg Versus Placebo on Weight Loss and Frequency of Binge Episodes in Obese Patients With Food Craving

summary:
The primary objective is to assess the effect of rimonabant compared to placebo on weight loss over a period of 6 months when prescribed with a mild hypocaloric diet in obese patients with binge eating disorder.

The secondary objectives are:

to assess the effect of rimonabant on the number of binge episodes per week, to assess the effect of rimonabant on eating behavior using the Binge Eating Scale (BES) and Three Factor Eating Questionnaire (TFEQ),
to evaluate the safety and tolerability of rimonabant over a period of 6 months.

ReferenceType: derived

ReferenceCitation: Pataky Z, Gasteyger C, Ziegler O, Rissanen A, Hanotin C, Golay A. Efficacy of rimonabant in obese patients with binge eating disorder. Exp Clin Endocrinol Diabetes. 2013 Jan;121(1):20-6. doi: 10.1055/s-0032-1329957. Epub 2012 Nov 12.

location_country:
Portugal
Finland
Switzerland
Sweden
United States
France
Netherlands


title: Anamorelin HCl in the Treatment of Non-Small Cell Lung Cancer-Cachexia (NSCLC-C): An Extension Study

summary: The administration of Anamorelin HCl in patients with Non-Small Cell Lung Cancer-Cachexia (NSCLC-C) is expected to increase appetite, lean body mass, weight gain, and muscle strength.

ReferenceCitation: Currow D, Temel JS, Abernethy A, Milanowski J, Friend J, Fearon KC. ROMANA 3: a phase 3 safety extension study of anamorelin in advanced non-small-cell lung cancer (NSCLC) patients with cachexia. Ann Oncol. 2017 Aug 1;28(8):1949-1956. doi: 10.1093/annonc/mdx192.

lead_sponsor: Helsinn Therapeutics (U.S.), Inc

location_country:
Poland
Belarus
Serbia
Italy
Russian Federation
Australia
Slovenia
Canada
Spain
Ukraine
Hungary
United States
France
Israel
Belgium
Germany
Czechia


acronym: paradigm™ 4

title: Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B

lead_sponsor: Novo Nordisk A/S

type: EudraCT Number

type: Other Identifier

type: Registry Identifier

source: euctr

design_allocation: Non-Randomized

study_start_date_type: Actual

ReferenceType: background

ReferenceCitation: Young G, Collins P, Tehranchi R, Chuansumrit A, Hanabusa H, Lentz SR, Mahlangu J, Mauser-Bunschoten E, Negrier C, Oldenburg J, Patiroglu T, Santagostino E, Zak M, Abdul Karim F. Safety and efficacy of nonacog beta pegol (N9-GP) for prophylaxis and treatment of bleeding episodes in previously-treated patients with hemophilia B: results from an extension trial. American Society of Hematology - 56th Annual Meeting (ASH) in San Francisco, CA, US

ReferenceType: result

ReferenceCitation: Young G, Collins PW, Colberg T, Chuansumrit A, Hanabusa H, Lentz SR, Mahlangu J, Mauser-Bunschoten EP, Négrier C, Oldenburg J, Patiroglu T, Santagostino E, Tehranchi R, Zak M, Karim FA. Nonacog beta pegol (N9-GP) in haemophilia B: A multinational phase III safety and efficacy extension trial (paradigm™4). Thromb Res. 2016 May;141:69-76. doi: 10.1016/j.thromres.2016.02.030. Epub 2016 Mar 2.

ReferenceCitation: Chowdary P, Kearney S, Regnault A, Hoxer CS, Yee DL. Improvement in health-related quality of life in patients with haemophilia B treated with nonacog beta pegol, a new extended half-life recombinant FIX product. Haemophilia. 2016 Jul;22(4):e267-74. doi: 10.1111/hae.12995. Epub 2016 Jun 28.

location_country: Macedonia, The Former Yugoslav Republic of

summary:
This trial is conducted in Asia, Europe, Japan, North America and South Africa. The aim is to evaluate the safety and efficacy of nonacog beta pegol (NNC-0156-0000-0009) after long-term exposure in patients with haemophilia B.

This trial is an extension to trials NN7999-3747 (NCT01333111/paradigm™ 2) and NN7999-3773 (NCT01386528/paradigm™ 3).


title: A Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multi-Centre Study To Investigate The Safety And Efficacy Of CP-690,550 For Maintenance Therapy In Subjects With Moderate To Severe Crohn's Disease

summary: This study investigates safety and efficacy of CP-690,550 in adult patients with moderate to severe Crohn's disease who completed the double-blind induction treatment in Study A3921083 and achieved clinical response-100 and/or clinical remission (CDAI<150) at Week 8.

ReferenceCitation: Panés J, Sandborn WJ, Schreiber S, Sands BE, Vermeire S, D'Haens G, Panaccione R, Higgins PDR, Colombel JF, Feagan BG, Chan G, Moscariello M, Wang W, Niezychowski W, Marren A, Healey P, Maller E. Tofacitinib for induction and maintenance therapy of Crohn's disease: results of two phase IIb randomised placebo-controlled trials. Gut. 2017 Jun;66(6):1049-1059. doi: 10.1136/gutjnl-2016-312735. Epub 2017 Feb 16.

lead_sponsor: Pfizer

location_country:
Spain
Greece
Austria
Australia
Netherlands
Canada
Czech Republic
Ukraine
Hungary
Korea, Republic of
United States
France
Israel
South Africa
Japan
Germany
Bulgaria

location_facility: Municipal Institution "Odesa Regional Clinical Hospital", Odesa Regional Centre of Gastroenterology.

location_facility: North Florida Gastroenterology Research, LLC

location_facility: Charite, Universitaetsmedizin Berlin, Campus Virchow-Klinikum

location_facility: General Hospital of Athens "Evangelismos",1st Gastroenterology Department

location_facility: National Hospital Organization Sendai Medical Center

location_facility: Hokkaido P.W.F.A.C Sapporo-Kosei general Hospital

location_facility: Investigational Drug Services - University Hospitals Case Medical Center

location_facility: Gasteroenterology Consultants of Clearwater

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

location_facility: ASAN Medical Center,Division of Gastroenterology,Department of Internal Medicine

location_facility: Szent Janos Korhaz es Eszak-budai Egyesitett Korhazak/I. Belgyogyaszat es Gastroenterologia

location_facility: East Ann Arbor Health and Geriatrics Center

location_facility: The Hospital of Hyogo College of Medicine

location_facility: Bugat Pal Korhaz Egeszsegugyi Szolgaltato Kozhasznu Nonprofit Kft.,

location_facility: Donetsk National Medical University n.a M. Gorky, Faculty of Internal Medicine #2

location_facility: Metropolitan Gastroenterology Group, P C

location_facility: Hôpital Haut-Lévêque/Service d hépato-gastroentérologie

location_facility: Shands Hospital at the University of Florida

location_facility: Wisconsin Center for Advanced Research - GI Associates, LLC

location_facility: Severance Hospital, Yonsei University Health System

location_facility: Gastroenterology Associates of Central Georgia, LLC

location_facility: Peterfy Sandor Utcai Korhaz, Rendelointezet es Baleseti Kozpont, I. sz. Belgyogyaszat

location_facility: Universitaetsklinikum Ulm, Klinik Fuer Innere Medizin I

location_facility: Advanced Clinical Research Institute-Phase 1, LLC

location_facility: Laboratórium Kft. Somogy Megyei Mikrobiológiai Laboratórium

location_facility: Montreal General Hospital - McGill University Health Centre

location_facility: Medical Clinical Research Center of Medical Center "Health Clinic" LLC

location_facility: Laboratorium Kft. Fovarosi és Pest Megyei Mikrobiologiai Laboratorium

location_facility: University of Florida - College of Medicine

location_facility: Premier Medical Group of the Hudson Valley, PC

location_facility: NYU Langone Nassau Gastroenterology Associates

location_facility: MBAL Sofiamed OOD, Otdelenie po gastroenterologia

location_facility: Medial Pharma spol.s.r.o. (Pharmacy only)

location_facility: Office of Drs. Ranjith Andrew Singh and Jamie D. Papp,

location_facility: Samsung Medical Center, Division of Gastroenterology, Department of Internal Medicine

location_facility: Gulfshore Endoscopy Center (Endoscopies Only)

location_facility: London Health Sciences Centre - University Hospital

location_facility: AKH Wien Universitaetsklinik fuer Innere Medizin III


title: Phase 3 Prospective, Randomized, Blinded, and Controlled Investigation of HepaSphere/QuadraSphere Microspheres for Delivery of Doxorubicin for the Treatment of Hepatocellular Carcinoma

acronym: HiQuality

summary: The purpose of this study is to evaluate overall survival in patients diagnosed with hepatocellular cancer (HCC) treated with HepaSphere/QuadraSphere Microspheres loaded with chemotherapeutic agent doxorubicin compared to conventional transarterial chemoembolization with particle PVA, lipiodol, and doxorubicin.

status: Terminated

ipd_sharing: No

lead_sponsor: Merit Medical Systems, Inc.

location_country:
Italy
Greece
United States
France
Belgium

location_facility: MUSC Medical Center (Medical University of South Carolina)

location_facility: Hospital of the University of Pennsylvania

location_facility: Palo Alto Veterans Institute for Research

location_facility: VA Greater Los Angeles Healthcare System

location_facility: University Hospitals K.U. Leuven (Dept of Hematology)

intervention_type: Procedure

status_simplified: Unknown


title: Anamorelin HCl in the Treatment of Non-Small Cell Lung Cancer-Cachexia (NSCLC-C): A Randomized Double-Blind Placebo-Controlled Multicenter Phase III Study to Evaluate the Safety and Efficacy of Anamorelin HCl in Patients With NSCLC-C

summary: The administration of Anamorelin in patients with Stage III-IV non-small cell lung cancer-cachexia (NSCLC-C) is expected to increase appetite, lean body mass, weight gain, and muscle strength.

ReferenceCitation: Temel JS, Abernethy AP, Currow DC, Friend J, Duus EM, Yan Y, Fearon KC. Anamorelin in patients with non-small-cell lung cancer and cachexia (ROMANA 1 and ROMANA 2): results from two randomised, double-blind, phase 3 trials. Lancet Oncol. 2016 Apr;17(4):519-531. doi: 10.1016/S1470-2045(15)00558-6. Epub 2016 Feb 20.

location_country:
Poland
Belarus
Serbia
Italy
Russian Federation
Netherlands
Slovenia
Canada
Spain
Ukraine
Hungary
United States
France
Belgium
Germany
Czechia


title: Prospective Randomized Study of the Incidence and Outcome of Veno-Occlusive Disease (VOD) With the Prophylactic Use of Defibrotide (DF) in Pediatric Stem Cell Transplantation

acronym: VOD-DF

summary: The aim of this trial is to evaluate whether the prophylactic use of Defibrotide (DF) in pediatric patients (age less than 18 years) undergoing stem cell transplantation and who are at high risk of developing hepatic Veno-occlusive Disease (VOD) will have an impact on the incidence and severity of the disease. Patients will be randomly assigned to one of two treatment arms: Those allocated to the Prophylactic Arm will receive the study drug (Defibrotide) from the day of conditioning onwards. Patients allocated to the Control Arm will receive the study drug (Defibrotide) from the day that VOD is diagnosed.

primary_purpose: Prevention

enrollment_type: Anticipated

ReferenceCitation: Corbacioglu S, Cesaro S, Faraci M, Valteau-Couanet D, Gruhn B, Rovelli A, Boelens JJ, Hewitt A, Schrum J, Schulz AS, Müller I, Stein J, Wynn R, Greil J, Sykora KW, Matthes-Martin S, Führer M, O'Meara A, Toporski J, Sedlacek P, Schlegel PG, Ehlert K, Fasth A, Winiarski J, Arvidson J, Mauz-Körholz C, Ozsahin H, Schrauder A, Bader P, Massaro J, D'Agostino R, Hoyle M, Iacobelli M, Debatin KM, Peters C, Dini G. Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial. Lancet. 2012 Apr 7;379(9823):1301-9. doi: 10.1016/S0140-6736(11)61938-7. Epub 2012 Feb 23.

lead_sponsor: European Society for Blood and Marrow Transplantation

location_country:
United Kingdom
Ireland
Austria
Italy
Netherlands
Switzerland
Sweden
France
Israel
Germany

location_facility:
Schneider Children's MC
Poliklinik Kinderheilkunde
Hopital Cantonal Universitaire
Institute G. Gaslini
Universitätsspital
Christian-Albrecht -University
Kinderklinik
Children's Hospital
St Anna Kinderspital
Johann-Wolfgang Goethe Universität
Clinica di Oncoematologia Pediatrica
Inst. Gustave Roussy
University Hospital
Great Ormond Street Hospital
Medical School
Universitätsspital Eppendorf
Ospedale S. Gerardo
Our Lady's Hospital for Sick Children
Sahlgrenska University Hospital
Kinderpoliklinik Uni
Klinik Kinder-Onkologie
University Children Hospital
Inselspital


title: A Randomised, Double-blind, Dose-finding, Multicenter Study of the Safety, Tolerability, and Efficacy of GSK2251052 Therapy Compared to Imipenem-cilastatin in the Treatment of Adult Subjects With Febrile Complicated Lower Urinary Tract Infections and Acute Pyelonephritis

summary: This study is being conducted to evaluate the safety, efficacy (clinical and microbiological), pharmacokinetics/pharmacodynamics of GSK2251052 and to assess whether it would be a suitable antibiotic for the treatment for febrile lower cUTI and pyelonephritis(complicated and uncomplicated). GSK2251052 will be compared to imipenem-cilastatin, which is an antibiotic commonly used to treat serious cUTI infections. GSK2251052 has a spectrum of microbiological activity that includes pathogens responsible for cUTI.

ReferenceCitation: O'Dwyer K, Spivak AT, Ingraham K, Min S, Holmes DJ, Jakielaszek C, Rittenhouse S, Kwan AL, Livi GP, Sathe G, Thomas E, Van Horn S, Miller LA, Twynholm M, Tomayko J, Dalessandro M, Caltabiano M, Scangarella-Oman NE, Brown JR. Bacterial resistance to leucyl-tRNA synthetase inhibitor GSK2251052 develops during treatment of complicated urinary tract infections. Antimicrob Agents Chemother. 2015 Jan;59(1):289-98. doi: 10.1128/AAC.03774-14. Epub 2014 Oct 27.

lead_sponsor: GlaxoSmithKline

location_country:
Spain
Greece
Russian Federation
Canada
United States
France

intervention_type: Other


title: Different Treatment Regimens of Calcipotriol Cream and Combination (Calcipotriol/Betamethasone Dipropionate) Ointment in Psoriasis Vulgaris

summary:
Patients with psoriasis vulgaris of the trunk and/or limbs are randomised to treatment with:

4 weeks of ointment containing calcipotriol and betamethasone dipropionate followed by 8 weeks of calcipotriol cream
4 weeks of (calcipotriol plus betamethasone dipropionate) ointment followed by 8 weeks of calcipotriol cream on weekdays/ (calcipotriol plus betamethasone dipropionate) ointment on weekends
4 weeks of (calcipotriol plus betamethasone dipropionate) ointment followed by 8 weeks of vehicle of calcipotriol cream

The objective is to compare the efficacy and safety of the different treatment regimens

lead_sponsor: LEO Pharma

location_country:
Spain
United Kingdom
Netherlands
Canada
France
Belgium
Germany

location_facility: Klinikum der Goethe-Universität, Zentrum für Dermatologie und Venerologie

location_facility: Clatterbridge Hospital, Department of Dermatology

location_facility: Hospital Mutua de Terrassa Dermatology Service


title: A Post-Market Study to Evaluate the Safety and Performance of the Ovation™ or Ovation Prime™ Abdominal Stent Graft System

summary: This is a prospective observational post-market study of subjects receiving the Ovation™ or Ovation Prime™ Abdominal Stent Graft System ("Ovation™ or Ovation Prime™ Abdominal Stent Graft System Post-Market Study") in the treatment of abdominal aortic aneurysms (AAA). The Ovation™ or Ovation Prime™ Abdominal Stent Graft System Post-Market study is intended to expand the clinical knowledge base by collecting data on subjects treated with the Ovation™ or Ovation Prime™ Abdominal Stent Graft System in actual clinical practice following commercial approval.

study_type: Observational

time_perspective: Prospective

lead_sponsor: TriVascular, Inc.

location_country:
United Kingdom
Greece
Austria
Italy
Sweden
France
Belgium
Germany

location_facility: Frimley Park Hospital NHS Foundation Trust

location_facility: Medical University of Vienna Dept. for Radiology, Section for Cardiovascular and Interventional Radiology

location_facility: Universitäres Herz und Gefaßzentrum Hamburg

location_facility: Städtisches klinikum Karlsruhe Klinik für Gefäß- und Thoraxchirurgie

location_facility: Università La Sapienza/Policlinico Umberto I

location_facility: A.O. Ospedale di Circolo e Fondazione Macchi

location_facility: Univeristà La Sapienza/Policlinico Umberto I

location_facility: "CA Gefäßchirurgie Theresienkrankenhaus/St. Hedwig"

intervention_type: Device


title: Efficacy of Maintenance Therapy With Rituximab After Induction Chemotherapy (R-CHOP vs. R-FC) for Elderly Patients With Mantle Cell Lymphoma Not Suitable for Autologous Stem Cell Transplantation

acronym: MCLelderly

summary:
The aim of this study is to answer the following independent questions in the treatment of mantle cell lymphomas:

Can rituximab-fludarabine, cyclophosphamide (R-FC) improve the reduction of lymphoma mass compared to rituximab-cyclophosphamide, doxorubicin, vincristine, prednisone (R-CHOP) and so become a new standard for initial cytoreductive therapy?
Can maintenance with rituximab substitute the interferon maintenance and even improve the progression free survival in patients after successful initial cytoreductive therapy?

status: Unknown status

study_completion_date_type: Anticipated

ReferenceCitation: Kluin-Nelemans HC, Hoster E, Hermine O, Walewski J, Trneny M, Geisler CH, Stilgenbauer S, Thieblemont C, Vehling-Kaiser U, Doorduijn JK, Coiffier B, Forstpointner R, Tilly H, Kanz L, Feugier P, Szymczyk M, Hallek M, Kremers S, Lepeu G, Sanhes L, Zijlstra JM, Bouabdallah R, Lugtenburg PJ, Macro M, Pfreundschuh M, Procházka V, Di Raimondo F, Ribrag V, Uppenkamp M, André M, Klapper W, Hiddemann W, Unterhalt M, Dreyling MH. Treatment of older patients with mantle-cell lymphoma. N Engl J Med. 2012 Aug 9;367(6):520-31. doi: 10.1056/NEJMoa1200920.

ReferenceCitation: Kluin-Nelemans HC, Hoster E, Hermine O, Walewski J, Geisler CH, Trneny M, Stilgenbauer S, Kaiser F, Doorduijn JK, Salles G, Szymczyk M, Tilly H, Kanz L, Schmidt C, Feugier P, Thieblemont C, Zijlstra JM, Ribrag V, Klapper W, Pott C, Unterhalt M, Dreyling MH. Treatment of Older Patients With Mantle Cell Lymphoma (MCL): Long-Term Follow-Up of the Randomized European MCL Elderly Trial. J Clin Oncol. 2020 Jan 20;38(3):248-256. doi: 10.1200/JCO.19.01294. Epub 2019 Dec 5.

ReferenceCitation: Hoster E, Klapper W, Hermine O, Kluin-Nelemans HC, Walewski J, van Hoof A, Trneny M, Geisler CH, Di Raimondo F, Szymczyk M, Stilgenbauer S, Thieblemont C, Hallek M, Forstpointner R, Pott C, Ribrag V, Doorduijn J, Hiddemann W, Dreyling MH, Unterhalt M. Confirmation of the mantle-cell lymphoma International Prognostic Index in randomized trials of the European Mantle-Cell Lymphoma Network. J Clin Oncol. 2014 May 1;32(13):1338-46. doi: 10.1200/JCO.2013.52.2466. Epub 2014 Mar 31.

ReferenceCitation: Pott C, Hoster E, Delfau-Larue MH, Beldjord K, Böttcher S, Asnafi V, Plonquet A, Siebert R, Callet-Bauchu E, Andersen N, van Dongen JJ, Klapper W, Berger F, Ribrag V, van Hoof AL, Trneny M, Walewski J, Dreger P, Unterhalt M, Hiddemann W, Kneba M, Kluin-Nelemans HC, Hermine O, Macintyre E, Dreyling M. Molecular remission is an independent predictor of clinical outcome in patients with mantle cell lymphoma after combined immunochemotherapy: a European MCL intergroup study. Blood. 2010 Apr 22;115(16):3215-23. doi: 10.1182/blood-2009-06-230250. Epub 2009 Dec 23.

lead_sponsor: European Mantle Cell Lymphoma Network

location_country:
Denmark
Poland
Italy
Netherlands
Czech Republic
France
Germany

location_facility: Ospedale Ferratotto, Divisione Di Ematologia

location_facility: General University Hospital, 1St Department of Medicine

location_facility: The Maria Sklodowska Memorial, Cancer Center - Inst. of Oncology

location_facility: HOVON - Dutch Haemato-Oncology Association (HOVON-Datacenter)

location_facility: Groupe D´Etudes des Lymphomes De l´Adulte (GELA)


title: The Effectiveness of Duloxetine Compared With Placebo in the Treatment of Predominant Stress Urinary Incontinence

summary: The study evaluates the effectiveness of duloxetine in reducing urinary incontinence occurrences in women due to physical stress(e.g. stomach pressure on bladder). Incontinence is the inability to control bladder function with leakage of urine.

lead_sponsor: Eli Lilly and Company

location_country:
Brazil
Spain
Italy
Canada
Puerto Rico
France
Mexico
Germany

location_facility: For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY(1-877-285-4559 or 1-317-615-4559) Mon-Fri from 9am to 5pm Eastern time(UTC/GMT - 5 hours, EST) or speak with your personal physician

location_facility: For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY(1-877-285-4559 OR 1-317-615-4559) Mon-Fri from 9am to 5pm Eastern time(UTC/GMT - 5 hours, EST) or speak with your personal physician


title: A Two Arm Phase II Study Comparing Docetaxel/Cisplatin Induction Therapy Followed By Concurrent Chemoradiotherapy Versus Concurrent Chemoradiotherapy Followed By Consolidation Docetaxel/Cisplatin in Patients With Locally Advanced Unresectable NSCLC (Stage IIIA-Multiple cN2 or IIIB)

summary:
Primary Objective:

To evaluate the toxicity/safety profile of docetaxel/cisplatin induction therapy followed by concurrent chemoradiotherapy versus concurrent chemoradiotherapy followed by consolidation docetaxel/cisplatin in patients with locally advanced unresectable NSCLC (stage IIIA- multiple cN2 or IIIB).

Secondary Objective:

To estimate efficacy parameters in overall response rate, progression free survival and 1 year survival for each of the two above mentioned arms.

ReferenceCitation: van Sörnsen de Koste JR, Senan S, Underberg RW, Oei SS, Elshove D, Slotman BJ, Lagerwaard FJ. Use of CD-ROM-based tool for analyzing contouring variations in involved-field radiotherapy for Stage III NSCLC. Int J Radiat Oncol Biol Phys. 2005 Oct 1;63(2):334-9.

location_country:
Spain
Italy
United Kingdom
Finland
France
Turkey
Belgium
Netherlands


title: An Open-label, Phase 2a Study to Evaluate Pazopanib Eye Drops Administered for 12 Weeks to Patients With Neovascular Age-related Macular Degeneration

summary: The purpose of this 12 week, open-label study is to investigate the safety and efficacy of a single dose regimen of pazopanib eye drop for neovascular AMD.

ReferenceCitation: Singh R, Wurzelmann JI, Ye L, Henderson L, Hossain M, Trivedi T, Kelly DS. Clinical evaluation of pazopanib eye drops in healthy subjects and in subjects with neovascular age-related macular degeneration. Retina. 2014 Sep;34(9):1787-95. doi: 10.1097/IAE.0000000000000179.

location_country:
Germany
United States
France


title: A Phase 2 Efficacy And Safety Study Of SU011248 Administered In A Continuous Daily Regimen In Patients With Cytokine-Refractory Metastatic Renal Cell Carcinoma

summary: To evaluate the anti-tumor activity of SU011248 (sunitinib) in cytokine-refractory metastatic renal cell carcinoma (RCC) when administered in a continuous treatment regimen

ReferenceCitation: de Velasco G, McKay RR, Lin X, Moreira RB, Simantov R, Choueiri TK. Comprehensive Analysis of Survival Outcomes in Non-Clear Cell Renal Cell Carcinoma Patients Treated in Clinical Trials. Clin Genitourin Cancer. 2017 Dec;15(6):652-660.e1. doi: 10.1016/j.clgc.2017.03.004. Epub 2017 Mar 21.

ReferenceCitation: Grünwald V, Lin X, Kalanovic D, Simantov R. Early Tumour Shrinkage: A Tool for the Detection of Early Clinical Activity in Metastatic Renal Cell Carcinoma. Eur Urol. 2016 Dec;70(6):1006-1015. doi: 10.1016/j.eururo.2016.05.010. Epub 2016 May 26.

ReferenceCitation: Grünwald V, McKay RR, Krajewski KM, Kalanovic D, Lin X, Perkins JJ, Simantov R, Choueiri TK. Depth of remission is a prognostic factor for survival in patients with metastatic renal cell carcinoma. Eur Urol. 2015 May;67(5):952-8. doi: 10.1016/j.eururo.2014.12.036. Epub 2015 Jan 7.

ReferenceCitation: Escudier B, Roigas J, Gillessen S, Harmenberg U, Srinivas S, Mulder SF, Fountzilas G, Peschel C, Flodgren P, Maneval EC, Chen I, Vogelzang NJ. Phase II study of sunitinib administered in a continuous once-daily dosing regimen in patients with cytokine-refractory metastatic renal cell carcinoma. J Clin Oncol. 2009 Sep 1;27(25):4068-75. doi: 10.1200/JCO.2008.20.5476. Epub 2009 Aug 3.

location_country:
Greece
Netherlands
Switzerland
Sweden
United States
France
Germany


title: A Phase II Study of Metronomic and Targeted Anti-angiogenesis Therapy for Children With Recurrent/Progressive Medulloblastoma, Ependymoma and ATRT

acronym: MEMMAT

summary: Patients with relapsed medulloblastoma, ependymoma and ATRT have a very poor prognosis whether treated with conventional chemotherapy, high-dose chemotherapy with stem cell rescue, irradiation or combinations of these modalities. Antiangiogenetic therapy has emerged as new treatment option in solid malignancies. The frequent, metronomic schedule targets both proliferating tumor cells and endothelial cells, and minimizes toxicity. In this study the investigators will evaluate the use of biweekly intravenous bevacizumab in combination with five oral drugs (thalidomide, celecoxib, fenofibrate, and alternating cycles of daily low-dose oral etoposide and cyclophosphamide), augmented with alternating courses of intrathecal etoposide and cytarabine. The aim of the study is to extend therapy options for children with recurrent or progressive medulloblastoma, ependymoma and ATRT, for whom no known curative therapy exists, by prolonging survival while maintaining good quality of life. The primary objective of the MEMMAT trial is to evaluate the activity of this multidrug antiangiogenic approach in these heavily pretreated children and young adults. Additionally, progression-free survival (PFS), overall survival (OS), as well as feasibility and toxicity will be examined.

status: Recruiting

ReferenceCitation: Slavc I, Mayr L, Stepien N, Gojo J, Aliotti Lippolis M, Azizi AA, Chocholous M, Baumgartner A, Hedrich CS, Holm S, Sehested A, Leblond P, Dieckmann K, Haberler C, Czech T, Kool M, Peyrl A. Improved Long-Term Survival of Patients with Recurrent Medulloblastoma Treated with a "MEMMAT-like" Metronomic Antiangiogenic Approach. Cancers (Basel). 2022 Oct 19;14(20):5128. doi: 10.3390/cancers14205128.

lead_sponsor: Medical University of Vienna

location_country:
Denmark
Spain
Austria
United States
France
Norway
Sweden
Czechia

location_facility: Kepler Universitätsklinikum Med Campus IV

location_facility: Dana-Farber Cancer Institute and Boston Children's Hospital

location_facility: Onkologisk-hematologisk seksjon Barneklinikken Haukeland universitetssjukehus

location_facility: Dell Children's Medical Group SFC-HEM/ONC

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

location_facility: Hospital Infantil Universitario Nino Jesus

lead_sponsor_type: academique

status_simplified: Ongoing


title: International Protocol for the Treatment of Childhood Anaplastic Large Cell Lymphoma

acronym: ALCL 99

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. It is not yet known which combination chemotherapy regimen is more effective for treating anaplastic large cell lymphoma.

PURPOSE: This randomized phase III trial is studying several different regimens of combination chemotherapy to compare how well they work in treating children with anaplastic large cell lymphoma.

ReferenceCitation: Attarbaschi A, Mann G, Rosolen A, Williams D, Uyttebroeck A, Marky I, Lamant L, Horibe K, Wrobel G, Beishuizen A, Wössmann W, Reiter A, Mauguen A, Le Deley MC, Brugières L; European Intergroup for Childhood Non-Hodgkin Lymphoma (EICNHL) ALCL99 Trial. Limited stage I disease is not necessarily indicative of an excellent prognosis in childhood anaplastic large cell lymphoma. Blood. 2011 May 26;117(21):5616-9. doi: 10.1182/blood-2010-12-324012. Epub 2011 Mar 28.

ReferenceCitation: Wrobel G, Mauguen A, Rosolen A, Reiter A, Williams D, Horibe K, Brugières L, Le Deley MC; European Inter-Group for Childhood, Non-Hodgkin Lymphoma (EICNHL). Safety assessment of intensive induction therapy in childhood anaplastic large cell lymphoma: report of the ALCL99 randomised trial. Pediatr Blood Cancer. 2011 Jul 1;56(7):1071-7. doi: 10.1002/pbc.22940. Epub 2011 Jan 28.

ReferenceCitation: Le Deley MC, Rosolen A, Williams DM, Horibe K, Wrobel G, Attarbaschi A, Zsiros J, Uyttebroeck A, Marky IM, Lamant L, Woessmann W, Pillon M, Hobson R, Mauguen A, Reiter A, Brugières L. Vinblastine in children and adolescents with high-risk anaplastic large-cell lymphoma: results of the randomized ALCL99-vinblastine trial. J Clin Oncol. 2010 Sep 1;28(25):3987-93. doi: 10.1200/JCO.2010.28.5999. Epub 2010 Aug 2.

lead_sponsor: Gustave Roussy, Cancer Campus, Grand Paris

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

location_facility: Karolinska University Hospital - Huddinge

location_facility: Hospital Clinico Universitario de Valencia


title: A Multicenter Open-label Extension Study For Subjects Who Participated In Study B0151003 (Andante Ii)

acronym: ANDANTE II

summary: This is a multi-center Phase 2, open label, safety extension study in subjects with moderate to severe CD who are anti-TNF inadequate responders. Subjects eligible for this study will have completed the 12-week induction period of study B0151003 and will be enrolled as either responders or non responders.

ReferenceCitation: Danese S, Vermeire S, Hellstern P, Panaccione R, Rogler G, Fraser G, Kohn A, Desreumaux P, Leong RW, Comer GM, Cataldi F, Banerjee A, Maguire MK, Li C, Rath N, Beebe J, Schreiber S. Randomised trial and open-label extension study of an anti-interleukin-6 antibody in Crohn's disease (ANDANTE I and II). Gut. 2019 Jan;68(1):40-48. doi: 10.1136/gutjnl-2017-314562. Epub 2017 Dec 15.

location_country:
Denmark
Brazil
United Kingdom
Ireland
Italy
Australia
New Zealand
Canada
Czech Republic
Switzerland
Hungary
United States
France
Israel
Belgium
Germany

location_facility: Pathology, Haematology and Biochemistry Laboratories, St. Vincent's Healthcare Group

location_facility: Institut klinicke a experimentalni mediciny

location_facility: Mater Misericordiae Hospital, Department of Clinical Chemistry and Clinical Haematology

location_facility: The University of Chicago Medical Center

location_facility: Universita Campus Biomedico UOC di Gastroenterologia

location_facility: Barts Health NHS Trust-Royal London Hospital

location_facility: Vanderbilt University Medical Center: IBD Clinic

location_facility: Rabin Medical Center, Beilinson Hospital

location_facility: Laboratório de Análises Clínicas do HUCFF/UFRJ

location_facility: Hadassah university Hospital (HUH) - Ein Karem, Hadassah Medical Organization (HMO)

location_facility: The University of Texas Health Science Center at Houston

location_facility: Oklahoma Foundation for Digestive Research

location_facility: Gastroenterology Associates of Western Michigan

location_facility: Gastointestinal Specialists of Georgia, PC

location_facility: Research Protocol Management Specialists

location_facility: London Health Science Centre - University Hospital

location_facility: Szegedi Tudomanyegyetem altalanos Orvostudomanyi Kar / I. sz. Belgyogyaszati Klinika

location_facility: IBD Center-Vanderbilt University Medical Center

location_facility: Weill Cornell Imaging at New York Presbyterian Hospital

location_facility: Rocky Mountain Gastroenterology Associates

location_facility: Vanderbilt University Medical Center-IDS

location_facility: Debreceni Egyetem Orvos- es Egeszsegtudomanyi Centrum

location_facility: New York Presbyterian Hospital - Weill Cornell Medical College Investigational Pharmacy

location_facility: Atlanta Center for Gastroenterology, P.C.

location_facility: "Kirurgisk Afdeling 0143 Hilleroed Hospital

location_facility: Hopital Saint-Antoine - Service De Gastroenterologie

location_facility: St Bartholomew's Hospital (Barts Health NHS Trust)

location_facility: Aarhus Universitetshospital, Aarhus Sygehus

location_facility: University of Kentucky Chandler Medical Center

location_facility: Montreal General Hospital - McGill University Health Centre

location_facility: Hospital Universitário Fraga Filho da UFRJ

location_facility: The University of TX Health Sci. Ctr at Houston

location_facility: Heritage Medical Research Clinic - University of Calgary

location_facility: Medicinsk Afdeling, Gastroenterologisk Sektion

location_facility: Setor de Cardiologia do Hospital Nossa Senhora das Gracas

location_facility: Clinical Pathologiy Laboratories, Inc, DBA DRL Labs

location_facility: International Clinical Research - US, LLC

location_facility: Setor de Endoscopia Digestiva do Hospital Nossa Senhora das Gracas

location_facility: Azienda Ospedaliera - Universita di Padova

location_facility: Medical Research Center of Connecticut, LLC

location_facility: Colonoscopy and X-rays: OU Physicians Building

location_facility: University Hospital Leuven, Campus Gasthuisberg

location_facility: Hepato-Gastroenterologie HK, s.r.o. Poliklinika III

location_facility: VC Medical Center Investigative Drug Service (IDS) [Ship Drug To]

location_facility: Weill Cornell Medical College of Cornell University-Greenberg

location_facility: Gastroenterology Consultants of Clearwater

location_facility: Vanderbilt University Medical Center-GI Research

intervention_type: Biological


title: PMCF Study - Verification of Activa IM-Nail™ Safety and Clinical Performance

acronym: BRINFF

summary: The Activa IM-Nail™ is used in the fixation of forearm fractures with cast to achieve a level of reduction and stabilisation that is appropriate to the age of the child. The post-market clinical follow-up study will be performed to identify the residual risk related to re-fracture and to determine its impact to the risk/benefit ratio of Activa IM-Nail™.

ReferenceCitation: Korhonen L, Perhomaa M, Kyrö A, Pokka T, Serlo W, Merikanto J, Sinikumpu JJ. Intramedullary nailing of forearm shaft fractures by biodegradable compared with titanium nails: Results of a prospective randomized trial in children with at least two years of follow-up. Biomaterials. 2018 Dec;185:383-392. doi: 10.1016/j.biomaterials.2018.09.011. Epub 2018 Sep 11.

lead_sponsor: Thomas Klestil

location_country: Denmark Portugal Austria Switzerland Hungary France Germany

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Pécs University Hospital, Department of Pediatrics

location_facility: Péterfy Hospital, National Trauma Center, Department of Pediatric Trauma Surgery

location_facility: Lille University Center, Jeanne de Flandre Hospital

location_facility: University Medical Center Schleswig-Holstein, Campus Lübeck Department of Pediatric Surgery

location_facility: Herlev and Gentofte University Hospital Department of Orthopedic Surgery


ipd_sharing: Yes

status: Active, not recruiting

acronym: APPOINT-PNH

title: A Multicenter, Single-arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

lead_sponsor: Novartis Pharmaceuticals

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

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

location_country:
United Kingdom
Italy
Korea, Republic of
France
Malaysia
China
Singapore
Germany

summary: The purpose of this Phase 3 study is to determine whether iptacopan is efficacious and safe for the treatment of PNH patients who are naive to complement inhibitor therapy.


title: A Multicenter Study to Estimate the Prevalence of HER2 Low and Describe the SoC, Treatment Patterns, and Outcome in Real-world Practice Among Unresectable and/or Metastatic Breast Cancer Patients With HER2-low Status - the RetroBC-HER2L Study

summary: This is a worldwide, multicenter, non-interventional, retrospective study of patient medical records from metastatic breast cancer (mBC) patients previously identified as human epidermal growth factor receptor 2 negative (HER2-neg), regardless of hormone status.

time_perspective: Retrospective

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

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

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

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

lead_sponsor: AstraZeneca

location_country:
Portugal
United Kingdom
Italy
Australia
Canada
Korea, Republic of
United States
France
Japan
Germany


acronym: ICC APL Study 02

title: Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia

lead_sponsor: Associazione Italiana Ematologia Oncologia Pediatrica

location_country: Denmark Portugal Spain Ireland Italy Netherlands Sweden France Israel Belgium Germany Czechia

location_facility: Azienda Ospedaliera di Padova - Oncoematologia Pediatrica

location_facility: Childrens hematology and oncology Uppsala University

location_facility: Fondazione IRCCS Policlinico San Matteo - Oncoematologia Pediatrica

location_facility: Fondazione Monza e Brianza per il Bambino e la sua Mamma (MBBM) - Ospedale San Gerardo

location_facility: Instituto Português de Oncologia de Lisboa Francisco Gentil, EPE

location_facility: Pediatrics and Adolescent Medicine Aarhus University Hospital

location_facility: Ospedale Pediatrico Microcitemico "A.Cau", Az.Ospedaliera Brotzu - SC Oncoematologia Ped. e Patologia della coagulazione

location_facility: AOU Città della Salute e della Scienza di Torino - Presidio Infantile Regina Margherita

location_facility: Universitätsklinikum Essen (AöR) Zentrum für Kinder-und Jugendmedizin Klinik für Kinderheilkunde III

location_facility: Rappaport Children'S Hospital, Rambam Health Care Campus

location_facility: AOU Policlinico Sant'Orsola-Malpighi - Oncologia ed Ematologia Pediatrica

location_facility: IRCCS Istituto Gannina Gaslini - Dipartimento di Oncoematologia

location_facility: Policlinico Umberto I Università "LA Sapienza" - Dip. Biotecnologie cellulari ed ematologia UOS Ematologia Pediatrica

location_facility: Ospedale Papa Giovanni XXIII - USS Oncoematologia Pediatrica

location_facility: Univerità degli Studi della Campania- Luigi Vanvitelli - Sevizio di Oncologia Pediatrica

location_facility: Hôpital Universitaire des Enfants Reine Fabiola (Huderf)

location_facility: Valencia University Medical School University Hospital La Fe

location_facility: Ospedale "Casa Sollievo della Sofferenza" - UO Oncoematologia Pediatrica

location_facility: AOU Policlinico Dipartimento di Pediatria

location_facility: Instituto Português de Oncologia do Porto Francisco Gentil, E. P. E.

location_facility: ARNAS Civico di Cristina e Benfratelli - UOC Oncoematologia Pediatrica

location_facility: A.O. Universitaria Meyer - DAI Oncoematologia Pediatrica

location_facility: Centro Hospitalar Universitário de Coimbra - Hospital Pediátrico de Coimbra

location_facility: Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica - Ospedale Pediatrico "Bambino Gesù"

location_facility: Ospedale santa Chiara - AOU Pisana, UO Oncoematologia Pediatrica

location_facility: AOU Policlinico Vittorio Emanuele - UOC Ematologia ed Oncologia Pediatrica con TNO

summary: The trial is open to all patients with a diagnosis of acute promyelocytic leukemia (APL) who are PCR-positive for the PML-RARα transcript and less than 18 years of age.


title: A Phase 2a, Randomized, Double-blind, Placebo Controlled, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency

acronym: ESTRELLA

summary:
This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ A1ATD-associated liver disease.

The study will be conducted in 2 parallel cohorts. A total of up to 27 participants may be enrolled in a single cohort. The 2 cohorts are differentiated only by the duration of the treatment period, the number of doses administered, and the timing of the end-of-treatment (EOT) liver biopsy. Participants in Cohort 1 will undergo EOT biopsy at 24 weeks, and participants in Cohort 2 will undergo EOT biopsy at 48 weeks.

primary_purpose: Other

lead_sponsor: Dicerna Pharmaceuticals, Inc.

location_country:
Portugal
Spain
United Kingdom
Australia
Netherlands
Canada
New Zealand
Sweden
France
Belgium
Germany

location_facility: Centro Hospitalar Universitario de Sao Joao

location_facility: Centro Hospitalar de Trás-os-Montes e Alto Douro, EPE

location_facility: Hospital Universitario Marques de Valdecilla Santander

location_facility: Centre Hospitalier de l'Universite de Montreal (CHUM)

location_facility: Universitaetsklinikum Schleswig-Holstein Campus Kiel

location_facility: Hospital da Senhora da Oliveira - Guimaraes

location_facility: CTC Clinical Trial Consultants AB Uppsala

location_facility: CHU Bordeaux - Hopital Haut-Leveque - Centre François Magendie

location_facility: Addenbrooke's Hospital, Cambridge University


title: SUrgical Versus PERcutaneous AXillary Artery International Registry A Physician-initiated, International, Multicentric, Retrospective, Observational, Voluntary Registry - SUPER AXA International Registry

summary: The purpose of the Study is to compare the outcomes of the surgical and the percutaneous approach to the upper extremity access (axillary or brachial artery) during endovascular procedures on the aortic valve, the aorta, and its side branches.

ReferenceCitation: Mirza AK, Oderich GS, Sandri GA, Tenorio ER, Davila VJ, Kärkkäinen JM, Hofer J, Cha S. Outcomes of upper extremity access during fenestrated-branched endovascular aortic repair. J Vasc Surg. 2019 Mar;69(3):635-643. doi: 10.1016/j.jvs.2018.05.214. Epub 2018 Oct 24.

ReferenceCitation: Branzan D, Steiner S, Haensig M, Scheinert D, Schmidt A. Percutaneous Axillary Artery Access for Endovascular Treatment of Complex Thoraco-abdominal Aortic Aneurysms. Eur J Vasc Endovasc Surg. 2019 Sep;58(3):344-349. doi: 10.1016/j.ejvs.2019.01.011. Epub 2019 Jul 30.

ReferenceCitation: Malgor RD, Marques de Marino P, Verhoeven E, Katsargyris A. A systematic review of outcomes of upper extremity access for fenestrated and branched endovascular aortic repair. J Vasc Surg. 2020 May;71(5):1763-1770.e2. doi: 10.1016/j.jvs.2019.09.028. Epub 2019 Nov 15.

ReferenceCitation: Deuschl F, Schofer N, Seiffert M, Hakmi S, Mizote I, Schaefer A, Schirmer J, Reichenspurner H, Blankenberg S, Conradi L, Schäfer U. Direct percutaneous transaxillary implantation of a novel self-expandable transcatheter heart valve for aortic stenosis. Catheter Cardiovasc Interv. 2017 Dec 1;90(7):1167-1174. doi: 10.1002/ccd.26986. Epub 2017 Mar 15.

ReferenceCitation: Schäfer U, Deuschl F, Schofer N, Frerker C, Schmidt T, Kuck KH, Kreidel F, Schirmer J, Mizote I, Reichenspurner H, Blankenberg S, Treede H, Conradi L. Safety and efficacy of the percutaneous transaxillary access for transcatheter aortic valve implantation using various transcatheter heart valves in 100 consecutive patients. Int J Cardiol. 2017 Apr 1;232:247-254. doi: 10.1016/j.ijcard.2017.01.010. Epub 2017 Jan 7.

ReferenceCitation: Fröhlich GM, Baxter PD, Malkin CJ, Scott DJ, Moat NE, Hildick-Smith D, Cunningham D, MacCarthy PA, Trivedi U, de Belder MA, Ludman PF, Blackman DJ; National Institute for Cardiovascular Outcomes Research. Comparative survival after transapical, direct aortic, and subclavian transcatheter aortic valve implantation (data from the UK TAVI registry). Am J Cardiol. 2015 Nov 15;116(10):1555-9. doi: 10.1016/j.amjcard.2015.08.035. Epub 2015 Sep 3.

ReferenceCitation: Arnett DM, Lee JC, Harms MA, Kearney KE, Ramos M, Smith BM, Anderson EC, Tayal R, McCabe JM. Caliber and fitness of the axillary artery as a conduit for large-bore cardiovascular procedures. Catheter Cardiovasc Interv. 2018 Jan 1;91(1):150-156. doi: 10.1002/ccd.27416. Epub 2017 Nov 11.

ReferenceCitation: Kappetein AP, Head SJ, Généreux P, Piazza N, van Mieghem NM, Blackstone EH, Brott TG, Cohen DJ, Cutlip DE, van Es GA, Hahn RT, Kirtane AJ, Krucoff MW, Kodali S, Mack MJ, Mehran R, Rodés-Cabau J, Vranckx P, Webb JG, Windecker S, Serruys PW, Leon MB; Valve Academic Research Consortium (VARC)-2. Updated standardized endpoint definitions for transcatheter aortic valve implantation: the Valve Academic Research Consortium-2 consensus document (VARC-2). Eur J Cardiothorac Surg. 2012 Nov;42(5):S45-60. doi: 10.1093/ejcts/ezs533. Epub 2012 Oct 1.

ReferenceCitation: Bertoglio L, Grandi A, Melloni A, Kahlberg A, Melissano G, Chiesa R. Percutaneous AXillary Artery (PAXA) Access at the First Segment During Fenestrated and Branched Endovascular Aortic Procedures. Eur J Vasc Endovasc Surg. 2020 Jun;59(6):929-938. doi: 10.1016/j.ejvs.2020.01.027. Epub 2020 Feb 20.

ReferenceCitation: Schaefer A, Schirmer J, Schofer N, Schneeberger Y, Deuschl F, Blankenberg S, Reichenspurner H, Conradi L, Schäfer U. Transaxillary transcatheter aortic valve implantation utilizing a novel vascular closure device with resorbable collagen material: a feasibility study. Clin Res Cardiol. 2019 Jul;108(7):779-786. doi: 10.1007/s00392-018-1407-z. Epub 2018 Dec 17.

ReferenceCitation: De Palma R, Rück A, Settergren M, Saleh N. Percutaneous axillary arteriotomy closure during transcatheter aortic valve replacement using the MANTA device. Catheter Cardiovasc Interv. 2018 Nov 1;92(5):998-1001. doi: 10.1002/ccd.27383. Epub 2017 Oct 25.

ReferenceCitation: Puippe GD, Kobe A, Rancic Z, Pfiffner R, Lachat M, Pfammatter T. Safety of percutaneous axillary artery access with a suture-mediated closing device for parallel endograft aortic procedures - a retrospective pilot study. Vasa. 2018 Jun;47(4):311-317. doi: 10.1024/0301-1526/a000702. Epub 2018 Mar 27.

ipd_sharing: Undecided

lead_sponsor: IRCCS San Raffaele

location_country:
Spain
Italy
Austria
United Kingdom
Switzerland
Sweden
United States
France
Belgium
Germany

location_facility: Ascension St John Hospital and Medical Center

location_facility: Nuffield Dept. of Clinical Neurosciences, University of Oxford

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: University Heart and Vascular Center Hamburg

location_facility: Service de Chirurgie Thoracique et Cardiovasculaire


acronym: INSIGHT

title: A Phase 2 Randomized, Open-label, Parallel-group, Multicenter Study to Assess Intrahepatic and Peripheral Changes of Immunologic and Virologic Markers in Response to Combination Regimens Containing JNJ-73763989 and Nucleos(t)Ide Analog With or Without JNJ-56136379 in Patients With Chronic Hepatitis B Virus Infection

lead_sponsor: Janssen Research & Development, LLC

ipd_sharing_description:
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency.

As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

location_country:
Poland
United Kingdom
Italy
New Zealand
Canada
United States
France
Belgium
Germany

location_facility:
UZ Antwerpen
Kings College Hospital
New Zealand Clinical Research
ID Clinic
Irccs Ospedale Maggiore Di Milano
Toronto General Hospital
Hôpital Beaujon
Grahame Hayton Unit
University Medical Center
Johns Hopkins University

summary: The purpose of this study is to assess changes in intrahepatic hepatitis B surface antigen (HBsAg) between baseline and on-treatment liver biopsy in response to JNJ-3989-based combination treatment.


acronym: paradigm™ 2

title: A Multi-centre, Single-blind Trial Evaluating Safety and Efficacy, Including Pharmacokinetics, of NNC-0156-0000-0009 When Used for Treatment and Prophylaxis of Bleeding Episodes in Patients With Haemophilia B

lead_sponsor: Novo Nordisk A/S

ReferenceCitation: Collins PW, Young G, Knobe K, Karim FA, Angchaisuksiri P, Banner C, Gürsel T, Mahlangu J, Matsushita T, Mauser-Bunschoten EP, Oldenburg J, Walsh CE, Negrier C; paradigm 2 Investigators. Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial. Blood. 2014 Dec 18;124(26):3880-6. doi: 10.1182/blood-2014-05-573055. Epub 2014 Sep 26.

location_country: Macedonia, The Former Yugoslav Republic of

summary: This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of NNC-0156-0000-0009 (nonacog beta pegol) when used for treatment and prophylaxis of bleeding episodes in patients with haemophilia B.


title: Safety and Pharmacokinetics of ODM-201 in Patients With Castrate Resistant Prostate Cancer: Open, Non-randomised, Uncontrolled, Multicentre, Multiple Dose Escalation Study With a Randomised Phase II Expansion Component

summary: The purpose of this study is to evaluate safety, tolerability and pharmacokinetics of ODM-201 in patients with castrate resistant prostate cancer.

ReferenceCitation: Fizazi K, Massard C, Bono P, Jones R, Kataja V, James N, Garcia JA, Protheroe A, Tammela TL, Elliott T, Mattila L, Aspegren J, Vuorela A, Langmuir P, Mustonen M; ARADES study group. Activity and safety of ODM-201 in patients with progressive metastatic castration-resistant prostate cancer (ARADES): an open-label phase 1 dose-escalation and randomised phase 2 dose expansion trial. Lancet Oncol. 2014 Aug;15(9):975-85. doi: 10.1016/S1470-2045(14)70240-2. Epub 2014 Jun 25.

lead_sponsor: Orion Corporation, Orion Pharma

location_country:
United Kingdom
Estonia
Finland
Czech Republic
United States
France

location_facility: Klinika onkologie a radioterapie LFUK a FN

location_facility: Eastern CT Hematology and Oncology Associates

location_facility: Oddeleni Radiacni a Klinicke Onkologie Nemocnice Znojmo


title: Myeloproliferative Neoplasms (MPN) and COVID-19

acronym: MPN-COVID

summary: An increased risk of both venous and arterial thromboembolism was noted in reports from SARS-CoV-2-infected patients in China and has been confirmed in autopsy findings from patients who experienced sudden death. Myeloproliferative Neoplasms (MPNs), which encompass polycythemia vera, essential thrombocythemia and primary myelofibrosis, are thrombophilic disorders with a natural propensity to thrombosis that is fuelled by the intrinsic activation of inflammatory cytokines. It therefore follows that an underlying diagnosis of MPN may increase the risk of worse clinical outcomes and death during periods of active Covid-19 disease. This ambispective, observational study aims to elucidate the key factors which affect the clinical course of patients with MPN who develop Covid-19 disease.

time_perspective: Other

ipd_sharing_description: Each participant can visualize its own data on eCRF after the completion patient's electronic data form.

lead_sponsor: Fondazione per la Ricerca Ospedale Maggiore

location_country:
Croatia
Poland
Spain
Italy
United Kingdom
United States
France
Germany
Bulgaria

location_facility: University Hospital Halle, Department of Hematology/Oncology

location_facility: University Clinic for Hematology, Oncology, Hemostaseology and Palliative Care, Johannes Wesling Medical Center

location_facility: Hospital Universitario Príncipe de Asturias

location_facility: Policlinico Universitario Fondazione Agostino Gemelli

location_facility: A.O.U. Città della Salute e della Scienza di Torino

location_facility: Azienda Ospedaliero Universitaria Maggiore della Carità

location_facility: University hospital Dubrava-School of Medicine University of Zagreb

location_facility: University Medicine Greifswald - Hematology, Oncology, Stem Cell Transplantation and Palliative Care

location_facility: ICO L'Hospitalet - Hospital Moisès Broggi

location_facility: ICO Girona Hospital Josep Trueta Servei d'hematologia

location_facility: National Specialised Hospital for Active Treatment of Hematological Diseases

location_facility: Institut Català d' Oncologia - Hospital Germans Trias i Pujol

location_facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

location_facility: Guy's and St. Thomas' NHS Foundation Trust.

location_facility: New York-Presbyterian/Weill Cornell Medical Center

location_facility: A.S.O. SS. Antonio e Biagio e C.Arrigo di Alessandria

location_facility: Ospedale Policlinico "G.B. Rossi" Borgo Roma

location_facility: Servicio de Hematología Hospital General Universitario de Albacete

location_facility: FEA Hematología Hospital Universitario de Móstoles


title: Multi-centric Observational Study to Analyse the Diagnostic Molecular Features in the Clinical Setting of Kidney Allograft Biopsies

summary: The current standard for biopsy-based diagnoses of dysfunction of kidney transplants is the Banff Classification which represents arbitrary international consensus. Recent data-driven approaches using molecular and conventional technologies indicate that mere consensus produces frequently incorrect diagnoses with potential harm to patients due to inappropriate treatment. To address this unmet need and improve diagnostics in the area of organ transplantation, the Alberta Transplant Applied Genomics Centre (ATAGC) has developed a new diagnostic system that combines the molecular and histopathological features of transplant biopsies, plus clinical and laboratory parameters, to create the first Integrated Diagnostic System. The present study will validate and refine this system in 500 prospectively unselected biopsies for clinical indications from American, Canadian and European centres in addition to 300 biopsies already collected. Due to a considerable interest and support from participating Centers, the study is further extended to 1500 prospective biopsies. Thus this is the extension of the INTERCOM study (INTERCOMEX). In addition to demonstrating the feasibility and value of this System in routine patient care and clinical trials, the study will develop and optimize a transparent and user-friendly reporting format to communicate this information to clinicians and obtain detailed feedback on how this system can best improve patient care.

ReferenceCitation: Halloran PF, Famulski KS, Reeve J. Molecular assessment of disease states in kidney transplant biopsy samples. Nat Rev Nephrol. 2016 Sep;12(9):534-48. doi: 10.1038/nrneph.2016.85. Epub 2016 Jun 27. Review.

ReferenceCitation: Einecke G, Reeve J, Gupta G, Böhmig GA, Eskandary F, Bromberg JS, Budde K, Halloran PF; INTERCOMEX investigators. Factors associated with kidney graft survival in pure antibody-mediated rejection at the time of indication biopsy: Importance of parenchymal injury but not disease activity. Am J Transplant. 2021 Apr;21(4):1391-1401. doi: 10.1111/ajt.16161. Epub 2020 Jul 31.

ReferenceCitation: Halloran PF, Pereira AB, Chang J, Matas A, Picton M, De Freitas D, Bromberg J, Serón D, Sellarés J, Einecke G, Reeve J. Potential impact of microarray diagnosis of T cell-mediated rejection in kidney transplants: The INTERCOM study. Am J Transplant. 2013 Sep;13(9):2352-63. doi: 10.1111/ajt.12387. Epub 2013 Aug 5.

ReferenceCitation: Halloran PF, Pereira AB, Chang J, Matas A, Picton M, De Freitas D, Bromberg J, Serón D, Sellarés J, Einecke G, Reeve J. Microarray diagnosis of antibody-mediated rejection in kidney transplant biopsies: an international prospective study (INTERCOM). Am J Transplant. 2013 Nov;13(11):2865-74. doi: 10.1111/ajt.12465. Epub 2013 Oct 3.

ReferenceCitation: Loupy A, Lefaucheur C, Vernerey D, Chang J, Hidalgo LG, Beuscart T, Verine J, Aubert O, Dubleumortier S, Duong van Huyen JP, Jouven X, Glotz D, Legendre C, Halloran PF. Molecular microscope strategy to improve risk stratification in early antibody-mediated kidney allograft rejection. J Am Soc Nephrol. 2014 Oct;25(10):2267-77. doi: 10.1681/ASN.2013111149. Epub 2014 Apr 3.

ReferenceCitation: Halloran PF, Chang J, Famulski K, Hidalgo LG, Salazar ID, Merino Lopez M, Matas A, Picton M, de Freitas D, Bromberg J, Serón D, Sellarés J, Einecke G, Reeve J. Disappearance of T Cell-Mediated Rejection Despite Continued Antibody-Mediated Rejection in Late Kidney Transplant Recipients. J Am Soc Nephrol. 2015 Jul;26(7):1711-20. doi: 10.1681/ASN.2014060588. Epub 2014 Nov 6.

ReferenceCitation: Halloran PF, Merino Lopez M, Barreto Pereira A. Identifying Subphenotypes of Antibody-Mediated Rejection in Kidney Transplants. Am J Transplant. 2016 Mar;16(3):908-20. doi: 10.1111/ajt.13551. Epub 2016 Jan 6.

ReferenceCitation: Reeve J, Chang J, Salazar ID, Lopez MM, Halloran PF. Using Molecular Phenotyping to Guide Improvements in the Histologic Diagnosis of T Cell-Mediated Rejection. Am J Transplant. 2016 Apr;16(4):1183-92. doi: 10.1111/ajt.13572. Epub 2016 Jan 5.

ReferenceCitation: Eskandary F, Bond G, Kozakowski N, Regele H, Marinova L, Wahrmann M, Kikić Ž, Haslacher H, Rasoul-Rockenschaub S, Kaltenecker CC, König F, Hidalgo LG, Oberbauer R, Halloran PF, Böhmig GA. Diagnostic Contribution of Donor-Specific Antibody Characteristics to Uncover Late Silent Antibody-Mediated Rejection-Results of a Cross-Sectional Screening Study. Transplantation. 2017 Mar;101(3):631-641. doi: 10.1097/TP.0000000000001195.

ReferenceCitation: Aubert O, Loupy A, Hidalgo L, Duong van Huyen JP, Higgins S, Viglietti D, Jouven X, Glotz D, Legendre C, Lefaucheur C, Halloran PF. Antibody-Mediated Rejection Due to Preexisting versus De Novo Donor-Specific Antibodies in Kidney Allograft Recipients. J Am Soc Nephrol. 2017 Jun;28(6):1912-1923. doi: 10.1681/ASN.2016070797. Epub 2017 Mar 2.

ReferenceCitation: Halloran PF, Reeve J, Akalin E, Aubert O, Bohmig GA, Brennan D, Bromberg J, Einecke G, Eskandary F, Gosset C, Duong Van Huyen JP, Gupta G, Lefaucheur C, Malone A, Mannon RB, Seron D, Sellares J, Weir M, Loupy A. Real Time Central Assessment of Kidney Transplant Indication Biopsies by Microarrays: The INTERCOMEX Study. Am J Transplant. 2017 Nov;17(11):2851-2862. doi: 10.1111/ajt.14329. Epub 2017 May 30.

ReferenceCitation: Reeve J, Böhmig GA, Eskandary F, Einecke G, Lefaucheur C, Loupy A, Halloran PF; MMDx-Kidney study group. Assessing rejection-related disease in kidney transplant biopsies based on archetypal analysis of molecular phenotypes. JCI Insight. 2017 Jun 15;2(12). pii: 94197. doi: 10.1172/jci.insight.94197. eCollection 2017 Jun 15.

ReferenceCitation: Madill-Thomsen KS, Wiggins RC, Eskandary F, Böhmig GA, Halloran PF. The Effect of Cortex/Medulla Proportions on Molecular Diagnoses in Kidney Transplant Biopsies: Rejection and Injury Can Be Assessed in Medulla. Am J Transplant. 2017 Aug;17(8):2117-2128. doi: 10.1111/ajt.14233. Epub 2017 Mar 23.

ReferenceCitation: Halloran PF, Venner JM, Famulski KS. Comprehensive Analysis of Transcript Changes Associated With Allograft Rejection: Combining Universal and Selective Features. Am J Transplant. 2017 Jul;17(7):1754-1769. doi: 10.1111/ajt.14200. Epub 2017 Feb 25.

ReferenceCitation: Halloran PF, Matas A, Kasiske BL, Madill-Thomsen KS, Mackova M, Famulski KS. Molecular phenotype of kidney transplant indication biopsies with inflammation in scarred areas. Am J Transplant. 2019 May;19(5):1356-1370. doi: 10.1111/ajt.15178. Epub 2018 Dec 13.

ReferenceCitation: Reeve J, Böhmig GA, Eskandary F, Einecke G, Gupta G, Madill-Thomsen K, Mackova M, Halloran PF; INTERCOMEX MMDx-Kidney Study Group. Generating automated kidney transplant biopsy reports combining molecular measurements with ensembles of machine learning classifiers. Am J Transplant. 2019 Oct;19(10):2719-2731. doi: 10.1111/ajt.15351. Epub 2019 Apr 10. Erratum in: Am J Transplant. 2020 Feb;20(2):620-621.

ReferenceCitation: Madill-Thomsen K, Perkowska-Ptasińska A, Böhmig GA, Eskandary F, Einecke G, Gupta G, Halloran PF; MMDx-Kidney Study Group. Discrepancy analysis comparing molecular and histology diagnoses in kidney transplant biopsies. Am J Transplant. 2020 May;20(5):1341-1350. doi: 10.1111/ajt.15752. Epub 2020 Jan 23.

ReferenceCitation: Halloran PF, Böhmig GA, Bromberg J, Einecke G, Eskandary FA, Gupta G, Myslak M, Viklicky O, Perkowska-Ptasinska A, Madill-Thomsen KS; INTERCOMEX Investigators. Archetypal Analysis of Injury in Kidney Transplant Biopsies Identifies Two Classes of Early AKI. Front Med (Lausanne). 2022 Apr 7;9:817324. doi: 10.3389/fmed.2022.817324. eCollection 2022.

ReferenceCitation: Halloran PF, Madill-Thomsen KS, Böhmig GA, Myslak M, Gupta G, Kumar D, Viklicky O, Perkowska-Ptasinska A, Famulski KS; INTERCOMEX Investigators. A 2-fold Approach to Polyoma Virus (BK) Nephropathy in Kidney Transplants: Distinguishing Direct Virus Effects From Cognate T Cell-mediated Inflammation. Transplantation. 2021 Nov 1;105(11):2374-2384. doi: 10.1097/TP.0000000000003884.

ReferenceCitation: Madill-Thomsen KS, Böhmig GA, Bromberg J, Einecke G, Eskandary F, Gupta G, Hidalgo LG, Myslak M, Viklicky O, Perkowska-Ptasinska A, Halloran PF; INTERCOMEX Investigators. Donor-Specific Antibody Is Associated with Increased Expression of Rejection Transcripts in Renal Transplant Biopsies Classified as No Rejection. J Am Soc Nephrol. 2021 Nov;32(11):2743-2758. doi: 10.1681/ASN.2021040433. Epub 2021 Jul 12.

ReferenceCitation: Halloran PF. Integrating molecular and histologic interpretation of transplant biopsies. Clin Transplant. 2021 Apr;35(4):e14244. doi: 10.1111/ctr.14244. Epub 2021 Feb 17.

ReferenceCitation: Halloran PF, Böhmig GA, Bromberg JS, Budde K, Gupta G, Einecke G, Eskandary F, Madill-Thomsen K, Reeve J; INTERCOMEX investigators. Discovering novel injury features in kidney transplant biopsies associated with TCMR and donor aging. Am J Transplant. 2021 May;21(5):1725-1739. doi: 10.1111/ajt.16374. Epub 2020 Nov 30.

ReferenceCitation: Venner JM, Famulski KS, Reeve J, Chang J, Halloran PF. Relationships among injury, fibrosis, and time in human kidney transplants. JCI Insight. 2016 Jan 21;1(1):e85323. doi: 10.1172/jci.insight.85323.

lead_sponsor: University of Alberta

location_country:
Croatia
Poland
Slovenia
Ireland
Austria
Spain
United Kingdom
Canada
Switzerland
Korea, Republic of
United States
France
Germany
Czechia

location_facility: Department of Surgery, University of Usan, College of Medicine

location_facility: University of Maryland School of Medicine

location_facility: University of British Columbia, St. Paul's Hospital

location_facility: Institute for Experimental and Clinical Medicine (IKEM)

location_facility: Pomeranian Medical University in Szczecin

location_facility: University of Wisconsin School of Medicine and Public Health

location_facility: Department of Medicine, University of Alberta

location_facility: Virginia Commonwealth University School of Medicine

location_facility: Texas Transplant Institute - Methodist Healthcare System


title: Evaluation of the Xpert® Bladder Cancer Monitor Assay Compared to Cystoscopy for the Follow-up of Patients With History of Low or Intermediate Risk Non-muscle-invasive Bladder Cancer (NMIBC): an Observational Prospective International Multicenter Study

acronym: ANTICIPATE X

summary: Bladder cancer is the 5th most common cancer in Europe, with more than 151,000 new cases diagnosed in 2012 (4% of the total). Bladder cancer has the highest recurrence rate of any malignancy, often as high as 70% within 5 years of successful treatment. This high recurrence rate requires diligent and accurate monitoring as a means for early diagnosis and treatment. Considering the burden associated to repeated invasive cystoscopies, there is a need for robust but accurate tests for surveillance. In that prospect, urinary molecular tests have been developed although none were deemed adequate in the European clinical guidelines to replace cystoscopies. The Xpert Bladder Cancer Monitor Assay is a qualitative in vitro diagnostic test designed to monitor for the recurrence of bladder cancer in patients previously diagnosed with this cancer. The test provides a fast and accurate result, is non-invasive and easy to perform. The aim of this study is to assess the non-inferiority of the Xpert Bladder Cancer Monitor assay in detecting recurrences in comparison to cystoscopy in the follow-up of patients with low or intermediate risk non-muscle-invasive bladder cancer (NMIBC).

lead_sponsor: Cepheid

location_country:
Spain
Italy
Austria
Netherlands
United Kingdom
Sweden
France
Germany
Czechia

location_facility: University Clinic for Urology and Andrology

location_facility: Humanitas University - Gradenigo Hospital of Turin

location_facility: CHU Toulouse / Institut Universitaire du Cancer Toulouse Oncopole

intervention_type: Diagnostic Test


title: Persistence Of Major Molecular Remission In Chronic Myeloid Leukemia After A Second Stop Of Tki Treatment In Patients Who Failed An Initial Stop Attempt: A Multicenter Prospective Trial

summary: This study will enroll CML patients who have failed a first TKI stopping attempt. After failure and at least a year of TKI treatment, patients will proceed to dasatinib treatment for another 2 years. If MR4 or better is re-achieved and maintained for at least one year, patients will be eligible for a second stop. After verification of MR4, TKI treatment will be stopped and patients followed in the same manner as after first stop. If MMR is lost (BCR-ABL >0.1% (IS)), TKI treatment will once again be restarted.

ipd_sharing_description: Study results will be published as soon as possible. Steering committee will decide on sharing IPD. This is still pending.

lead_sponsor: Amsterdam UMC, location VUmc

location_country: Denmark Netherlands Finland Sweden France Norway Germany

location_facility: Uppsala University Hospital (Akademiska)

location_facility: St Olavs Hospital-Trondheim University Hospital


title: The Effect of Low Protein, Extensively Hydrolyzed Infant Formula on Allergy Prevention in At-risk Infants up to 1 Year of Age: a Randomized, Double-blind, Controlled Intervention Study and the Long-term Effect on Allergy Prevention of Early Nutrition Given in the First 120 Days of Life in At-risk Infants Until the Child is 6 Years of Age

summary: A multi-center, prospective, randomized, controlled, doubleblind intervention clinical Trial is performed to proof the efficiency of hydrolyzed infant formula showing a risk reduction of developing an allergy.

lead_sponsor: HiPP GmbH & Co. Vertrieb KG

location_country: Poland Portugal Serbia Spain Italy Finland France Germany Bulgaria Czechia

location_facility: Complejo Hospitalario Universitario de Santiago

location_facility: Second specialized obstetric and gynecological hospital for active treatment Sheinovo EAD

location_facility: Studienambulanz der pädiatrische Pneumologie und AllergologieKlinik für Kinder- und Jugendmedizin (KKJM)

location_facility: Klinik für Kinder- und Jugendmedizin der Ruhruniversität Bochum im St. Josef Hospital

location_facility: Clinical Hospital Center "Dr Dragisa Misovic-Dedinje"

location_facility: Klinik für Kinder- und Jugendmedizin Marienhospital gGmbH Wesel


title: Incidence of Dyssynchronous Spontaneous Breathing Effort, Breath-stacking and Reverse Triggering in Early ARDS

acronym: BEARDS

summary:
Patients sedated under mechanical ventilation with acute hypoxemic respiratory failure with a PaO2/FiO2 equal or less than 200mmHg (Acute Respiratory Distress Syndrome, ARDS and non-ARDS) will be included in the study early in the course of the disease (first week of mechanical ventilation). At enrollment, data on the clinical condition of the patient will be recorded together with ventilation settings: ventilation mode, the fraction of inspired oxygen (FiO2), PEEP, tidal volume, set pressure, respiratory rate, time of the respiratory cycle, recent blood gas parameters.

Airway pressure, flow, and esophageal pressure (or alternatively electrical activity of the diaphragm, Eadi) will be recorded 3 times a day for 7 days:

Period 1 (morning): duration 20-30 minutes
Period 2 (afternoon): duration 20-30 minutes
Period 3 (evening / night): duration 20-30 minutes

Registration will be ended at extubation, death or at eight days from the first recording.

Monitoring of vital parameters (hemodynamic and respiratory) will be continuous throughout the duration of the study, as per normal clinical practice. All drugs used during the day of the measurements will be recorded. The patient will then be followed until discharge from the ICU and after 60 days of discharge to evaluate mortality.

As an ancillary study, in a subgroup of patients continuous simplified measurement of respiratory recordings together with hourly clinical data on sedation and extended simplified polysomnography recordings will be performed within the first 7 days from inclusion.

The analysis of the recorded waveforms will be performed in a single center by a centralized system that will quantify dyssynchrony and its intensity, calculate pressure time product, collect clinical and physiological data and outcome, and investigate possible correlations.

ReferenceCitation: Bianchi I, Grassi A, Pham T, Telias I, Teggia Droghi M, Vieira F, Jonkman A, Brochard L, Bellani G. Reliability of plateau pressure during patient-triggered assisted ventilation. Analysis of a multicentre database. J Crit Care. 2022 Apr;68:96-103. doi: 10.1016/j.jcrc.2021.12.002. Epub 2021 Dec 21. Erratum in: J Crit Care. 2022 Apr 1;:154030.

ReferenceCitation: Pham T, Montanya J, Telias I, Piraino T, Magrans R, Coudroy R, Damiani LF, Mellado Artigas R, Madorno M, Blanch L, Brochard L; BEARDS study investigators. Automated detection and quantification of reverse triggering effort under mechanical ventilation. Crit Care. 2021 Feb 15;25(1):60. doi: 10.1186/s13054-020-03387-3.

ipd_sharing_description: We plan to share physiologic tracings with no participant identification.

lead_sponsor: Unity Health Toronto

location_country: Taiwan Spain Greece Italy Netherlands Canada Thailand France China Germany

location_facility: Centre Hospitalier Universitaire - CHU Angers

location_facility: National Cheng-Kung University and Hospital

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

location_facility: Beijing Tiantan Hospital, Capital Medical University

location_facility: Azienda Ospedaliero - Universitaria OORR Ospedali Riuniti di Foggia


title: Use of the VisuMax Femtosecond Laser Lenticule Removal Procedure for the Correction of Hyperopia With or Without Astigmatism

summary: The correction of farsightedness using ReLEx SMILE for hyperopia is focus of this investigation. The objective is to evaluate the safety and effectiveness.

lead_sponsor: Carl Zeiss Meditec AG

location_country:
United Kingdom
India
France
China
Germany
Czechia

location_facility: Medical Center Nethradhama Superspeciality Eye Hospital

location_facility: University Medical Center Universitätsklinikum Gießen Marburg


acronym: RESTORE

title: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials (RESTORE)

lead_sponsor: GenSight Biologics

ReferenceCitation: Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison. Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021.

location_country:
United Kingdom
Italy
United States
France
Germany

location_facility: LMU Klinikum der Universität München / Friedrich-Baur-Institut

summary: The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in the Rescue or Reverse studies.


title: The Multi-National Gilenya Pregnancy Exposure Registry in Multiple Sclerosis

summary: The purpose of the Multi-National Gilenya Pregnancy Exposure Registry in Multiple Sclerosis (MS) is to continuously monitor, evaluate, and assess for major and minor teratogenic effects in the offspring of women exposed to fingolimod before (up to 8 weeks before last menstrual period (LMP)) and during pregnancy in routine clinical practice. The overall aim is to collect and evaluate data on maternal, fetal, and infant outcomes and compare it with reference populations.

location_country:
Saudi Arabia
Italy
Spain
United Kingdom
United States
Belgium
Romania
Czechia
Poland
Portugal
Slovenia
Austria
Australia
Russian Federation
Argentina
Israel
Mexico
Norway
Sweden
Slovakia
Cyprus
Brazil
Lebanon
Canada
Switzerland
Hungary
Germany
Denmark
Greece
Ireland
Finland
France
Colombia
Netherlands
United Arab Emirates


title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

lead_sponsor: BioMarin Pharmaceutical

ReferenceCitation: Schweighardt B, Tompkins T, Lau K, Jesaitis L, Qi Y, Musson DG, Farmer P, Haller C, Shaywitz AJ, Yang K, O'Neill CA. Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial. Clin Ther. 2015 May 1;37(5):1012-1021.e6. doi: 10.1016/j.clinthera.2014.11.005. Epub 2014 Dec 6.

location_country:
Denmark
Taiwan
Brazil
Portugal
Saudi Arabia
Italy
United Kingdom
Netherlands
Qatar
Argentina
Canada
Korea, Republic of
United States
France
Colombia
Japan
Germany

summary:
This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).

There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for MPS IVA patients. BMN 110 is administered to MPS IVA patients by IV infusion, allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes.

This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate (KS) in tissue macrophages, hyaline cartilage, other connective tissues, and heart valve, and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders.


title: A Multicenter Longitudinal Study for Disease Profiling of Asthma and Chronic Obstructive Pulmonary Disease

summary: The purpose of this study is to characterize the clinical, physiologic, and molecular profiles of healthy participants, participants with mild, moderate, and severe asthma; and participants with moderate to severe Chronic Obstructive Pulmonary Disease (COPD).

primary_purpose: Basic Science

ReferenceCitation: Cass SP, Yang Y, Xiao J, McGrath JJC, Fantauzzi MF, Thayaparan D, Wang F, Liang Z, Long F, Stevenson CS, Chen R, Stampfli MR. Current smoking status is associated with reduced sputum immunoglobulin M and G expression in COPD. Eur Respir J. 2021 Feb 4;57(2). pii: 1902338. doi: 10.1183/13993003.02338-2019. Print 2021 Feb.

ReferenceCitation: Loza MJ, Djukanovic R, Chung KF, Horowitz D, Ma K, Branigan P, Barnathan ES, Susulic VS, Silkoff PE, Sterk PJ, Baribaud F; ADEPT (Airways Disease Endotyping for Personalized Therapeutics) and U-BIOPRED (Unbiased Biomarkers for the Prediction of Respiratory Disease Outcome Consortium) investigators. Validated and longitudinally stable asthma phenotypes based on cluster analysis of the ADEPT study. Respir Res. 2016 Dec 15;17(1):165.

location_country:
Denmark
United Kingdom
Netherlands
Canada
United States
France
Romania
Germany


title: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome

summary: This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.

ReferenceCitation: Berry-Kravis E, Des Portes V, Hagerman R, Jacquemont S, Charles P, Visootsak J, Brinkman M, Rerat K, Koumaras B, Zhu L, Barth GM, Jaecklin T, Apostol G, von Raison F. Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Sci Transl Med. 2016 Jan 13;8(321):321ra5. doi: 10.1126/scitranslmed.aab4109.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Denmark
Spain
Italy
United Kingdom
Australia
Canada
Switzerland
United States
France
Germany


title: A Phase 3, Open-label Study of the Safety and Effectiveness of AA4500 Administered Twice Per Treatment Cycle for up to Four Treatment Cycles (2 x 4) in Men With Peyronie's Disease

summary:
This study is a Phase 3, open-label study of the safety and efficacy of AA4500 0.58 mg in subjects with Peyronie's disease. Subjects will be screened for study eligibility within 21 days before the initial injection of study drug in the first treatment cycle.

After up to four treatment cycles, each subject will be followed for additional safety and efficacy assessments on Days 168 (± 7 days) and 252 (± 7 days) (nominal weeks 24 and 36).

After the final injection of each treatment cycle, the investigator will model the plaque in an attempt to stretch or elongate the plaque. If the subject's penile curvature is reduced to <15° after the first, second, or third cycle of injections or if the investigator determines further treatment is not clinically indicated (eg, adverse events; allergic reaction), subsequent treatment cycles will not be administered.

Approximately 300 subjects will be enrolled.

ReferenceCitation: Levine LA, Cuzin B, Mark S, Gelbard MK, Jones NA, Liu G, Kaufman GJ, Tursi JP, Ralph DJ. Clinical safety and effectiveness of collagenase clostridium histolyticum injection in patients with Peyronie's disease: a phase 3 open-label study. J Sex Med. 2015 Jan;12(1):248-58. doi: 10.1111/jsm.12731. Epub 2014 Nov 12.

lead_sponsor: Endo Pharmaceuticals

location_country:
Denmark
Spain
Italy
United Kingdom
New Zealand
Sweden
United States
France
Germany

location_facility: Connecticut Clinical Research Center, LLC

location_facility: Waitemata Urology Research North Shore Hospital

location_facility: Tristate Urologic Services PSC, Inc./ dba TUG Research

location_facility: Maimonides Medical Center, Division of Urology

location_facility: Aarhus Universitiy Hospital, Aalborg Sygehus

location_facility: Universitatsklinikum Freiburg, Abteilung Urologie

location_facility: Urology of Virginia-Sentara Medical Group


title: An Open-label, Multicenter, Randomized Phase 2 Study Evaluating the Safety and Efficacy of Cisplatin and Pemetrexed With or Without Cixutumumab as First-Line Therapy in Patients With Advanced Nonsquamous Non-Small Cell Lung Carcinoma

summary: The primary purpose of this study is to evaluate the hypothesis that cixutumumab given in combination with cisplatin and pemetrexed is superior to cisplatin and pemetrexed as first-line therapy for patients with advanced nonsquamous non-small cell lung carcinoma (NSCLC).

ipd_sharing_description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

lead_sponsor: Eli Lilly and Company

location_country:
Brazil
Spain
Italy
Netherlands
Argentina
Canada
United States
France
Israel
Turkey
Belgium
Germany

location_facility: For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.


intervention_type: Genetic

title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

lead_sponsor: bluebird bio

ReferenceCitation: Locatelli F, Thompson AA, Kwiatkowski JL, Porter JB, Thrasher AJ, Hongeng S, Sauer MG, Thuret I, Lal A, Algeri M, Schneiderman J, Olson TS, Carpenter B, Amrolia PJ, Anurathapan U, Schambach A, Chabannon C, Schmidt M, Labik I, Elliot H, Guo R, Asmal M, Colvin RA, Walters MC. Betibeglogene Autotemcel Gene Therapy for Non-β(0)/β(0) Genotype β-Thalassemia. N Engl J Med. 2022 Feb 3;386(5):415-427. doi: 10.1056/NEJMoa2113206. Epub 2021 Dec 11.

ReferenceCitation: Hamed EM, Meabed MH, Aly UF, Hussein RRS. Recent Progress in Gene Therapy and Other Targeted Therapeutic Approaches for Beta Thalassemia. Curr Drug Targets. 2019;20(16):1603-1623. doi: 10.2174/1389450120666190726155733. Review.

location_country:
United Kingdom
Italy
Thailand
United States
France
Germany

summary: This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 23 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0 mutation at both alleles of the hemoglobin β (HBB) gene. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.


title: An Open Label, 1-Year Trial, Including a Double-Blind Placebo-Controlled Withdrawal Period, of RM-493, a MC4R Agonist, in Early Onset POMC Deficiency Obesity Due to Bi-Allelic Loss-of-Function POMC or PCSK1 Genetic Mutation

lead_sponsor: Rhythm Pharmaceuticals, Inc.

ReferenceCitation: Clement K, van den Akker E, Argente J, Bahm A, Chung WK, Connors H, De Waele K, Farooqi IS, Gonneau-Lejeune J, Gordon G, Kohlsdorf K, Poitou C, Puder L, Swain J, Stewart M, Yuan G, Wabitsch M, Kuhnen P; Setmelanotide POMC and LEPR Phase 3 Trial Investigators. Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials. Lancet Diabetes Endocrinol. 2020 Dec;8(12):960-970. doi: 10.1016/S2213-8587(20)30364-8. Epub 2020 Oct 30.

location_country:
Spain
United Kingdom
Canada
United States
France
Belgium
Germany

location_facility: University of Cambridge Metabolic Research Laboratories

location_facility: Charité Campus Virchow-Klinikum / Institute for Experimental Pediatric Endocrinology

location_facility: Institute of Cardiometabolism and Nutrition / Hopital de la Pitié-Salpêtrière

summary: The purpose of the study is to determine the effect of setmelanotide (RM-493) on weight and other factors in patients with pro-opiomelanocortin (POMC) deficiency obesity due to rare bi-allelic loss-of function POMC or PCSK1 genetic mutations.


title: Molecular Reclassification to Find Clinically Useful Biomarkers for Systemic Autoimmune Diseases: Cross Sectional Cohort

acronym: PRECISESADS

summary: Connective tissue diseases (CTD) or systemic autoimmune diseases (SADs) as they are known today are a group of chronic inflammatory conditions with autoimmune aetiology with few treatment options and difficult diagnosis.Brest team contribute to perform a new classification of the following systemic autoimmune diseases in a European Union's Seventh Framework Programme. The aim of this research is to reclassify the individuals affected by SADs into molecular clusters instead of clinical entities through the determination of molecular profiles using several "Omics" techniques.

lead_sponsor: Fundación Pública Andaluza Progreso y Salud

location_country: Portugal Spain Italy Austria Switzerland Hungary France Belgium Germany

location_facility: Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico (IRCCS)

location_facility: Deutsches Rheuma-Forschungszentrum Berlin (DRFZ)

location_facility: Hospital Universitario San Cecilio Servicio Andaluz de Salud

location_facility: Hospital Clinic I Provicia- Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS)

location_facility: Hospital Regional de Málaga Servicio Andaluz de Salud

location_facility: Hospital Universitario Marqués de Valdecilla, Servicio Cántabro de Salud

location_facility: Hospital Universitario Reina Sofía Andaluz de Salud

location_facility: UZ Leuven - KU Leuven, Department of Rheumatology (KU LEUVEN)

location_facility: Université catholique de Louvain - Cliniques Universitaires Saint-Luc


title: A Multi-Center, Open-Label, Single-Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency

lead_sponsor: Grifols Therapeutics LLC

location_country:
Poland
Spain
United Kingdom
Australia
Sweden
Hungary
France
Germany
Czechia

location_facility: The Children's Memorial Health Institute

location_facility: Children's Hospital at Municipal Hospital St. Georg

summary: Approximately 60 subjects will be enrolled in order to have approximately 20 adult subjects and 20 pediatric subjects treated with subcutaneously administered Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) who complete the entire study. This study will include 3 study stages: Screening/Previous Regimen Phase, IGSC 20% Treatment Stage 1 (13 IGSC 20% weekly doses), and IGSC 20% Treatment Stage 2 (39 IGSC 20% weekly doses). A total of 52 doses of IGSC 20% will be administered with a final follow-up visit 1 week after the last dose at Week 53. Subjects/caregivers will be trained on self-administration of IGSC 20% by the clinical site personnel.


title: Pipeline Flex With SHield Technology Embolization - An International MulticEnter ObservationaL Post Market StuDy of Treated Intra Cranial Aneurysms (SHIELD)

acronym: SHIELD

summary: This is a prospective, single-arm, multi-center post-market observational study assessing the performance of the Pipeline™ Flex Embolization Device with Shield Technology™ in subjects undergoing treatment for intracranial aneurysms in a large real-world, post-market setting.

ReferenceCitation: Nelson PK, Lylyk P, Szikora I, Wetzel SG, Wanke I, Fiorella D. The pipeline embolization device for the intracranial treatment of aneurysms trial. AJNR Am J Neuroradiol. 2011 Jan;32(1):34-40. doi: 10.3174/ajnr.A2421. Epub 2010 Dec 9.

ReferenceCitation: Becske T, Kallmes DF, Saatci I, McDougall CG, Szikora I, Lanzino G, Moran CJ, Woo HH, Lopes DK, Berez AL, Cher DJ, Siddiqui AH, Levy EI, Albuquerque FC, Fiorella DJ, Berentei Z, Marosfoi M, Cekirge SH, Nelson PK. Pipeline for uncoilable or failed aneurysms: results from a multicenter clinical trial. Radiology. 2013 Jun;267(3):858-68. doi: 10.1148/radiol.13120099. Epub 2013 Feb 15.

ReferenceCitation: Kallmes DF, Hanel R, Lopes D, Boccardi E, Bonafé A, Cekirge S, Fiorella D, Jabbour P, Levy E, McDougall C, Siddiqui A, Szikora I, Woo H, Albuquerque F, Bozorgchami H, Dashti SR, Delgado Almandoz JE, Kelly ME, Turner R 4th, Woodward BK, Brinjikji W, Lanzino G, Lylyk P. International retrospective study of the pipeline embolization device: a multicenter aneurysm treatment study. AJNR Am J Neuroradiol. 2015 Jan;36(1):108-15. doi: 10.3174/ajnr.A4111. Epub 2014 Oct 29. Erratum in: AJNR Am J Neuroradiol. 2015 May;36(5):E39-40.

ReferenceCitation: Chitale R, Gonzalez LF, Randazzo C, Dumont AS, Tjoumakaris S, Rosenwasser R, Chalouhi N, Gordon D, Jabbour P. Single center experience with pipeline stent: feasibility, technique, and complications. Neurosurgery. 2012 Sep;71(3):679-91; discussion 691. doi: 10.1227/NEU.0b013e318260fe86.

ReferenceCitation: Saatci I, Yavuz K, Ozer C, Geyik S, Cekirge HS. Treatment of intracranial aneurysms using the pipeline flow-diverter embolization device: a single-center experience with long-term follow-up results. AJNR Am J Neuroradiol. 2012 Sep;33(8):1436-46. doi: 10.3174/ajnr.A3246. Epub 2012 Jul 19.

ReferenceCitation: Kan P, Siddiqui AH, Veznedaroglu E, Liebman KM, Binning MJ, Dumont TM, Ogilvy CS, Gaughen JR Jr, Mocco J, Velat GJ, Ringer AJ, Welch BG, Horowitz MB, Snyder KV, Hopkins LN, Levy EI. Early postmarket results after treatment of intracranial aneurysms with the pipeline embolization device: a U.S. multicenter experience. Neurosurgery. 2012 Dec;71(6):1080-7; discussion 1087-8. doi: 10.1227/NEU.0b013e31827060d9.

ReferenceCitation: Yu SC, Kwok CK, Cheng PW, Chan KY, Lau SS, Lui WM, Leung KM, Lee R, Cheng HK, Cheung YL, Chan CM, Wong GK, Hui JW, Wong YC, Tan CB, Poon WL, Pang KY, Wong AK, Fung KH. Intracranial aneurysms: midterm outcome of pipeline embolization device--a prospective study in 143 patients with 178 aneurysms. Radiology. 2012 Dec;265(3):893-901. doi: 10.1148/radiol.12120422. Epub 2012 Sep 20.

ReferenceCitation: McAuliffe W, Wycoco V, Rice H, Phatouros C, Singh TJ, Wenderoth J. Immediate and midterm results following treatment of unruptured intracranial aneurysms with the pipeline embolization device. AJNR Am J Neuroradiol. 2012 Jan;33(1):164-70. doi: 10.3174/ajnr.A2727. Epub 2011 Oct 6.

ReferenceCitation: Skukalek SL, Winkler AM, Kang J, Dion JE, Cawley CM, Webb A, Dannenbaum MJ, Schuette AJ, Asbury B, Tong FC. Effect of antiplatelet therapy and platelet function testing on hemorrhagic and thrombotic complications in patients with cerebral aneurysms treated with the pipeline embolization device: a review and meta-analysis. J Neurointerv Surg. 2016 Jan;8(1):58-65. doi: 10.1136/neurintsurg-2014-011145. Epub 2014 Nov 10. Review.

ReferenceCitation: Rice H, Martínez Galdámez M, Holtmannspötter M, Spelle L, Lagios K, Ruggiero M, Vega P, Sonwalkar H, Chapot R, Lamin S. Periprocedural to 1-year safety and efficacy outcomes with the Pipeline Embolization Device with Shield technology for intracranial aneurysms: a prospective, post-market, multi-center study. J Neurointerv Surg. 2020 Nov;12(11):1107-1112. doi: 10.1136/neurintsurg-2020-015943. Epub 2020 Jun 1.

ipd_sharing_description: No individual participant data will be available

lead_sponsor: Medtronic Neurovascular Clinical Affairs

location_country:
Denmark
Spain
Greece
Italy
Australia
United Kingdom
Finland
Hungary
France
Israel
Germany

location_facility: Hospital Universitario Central de Asturias


title: STA®-Liatest®D-Di-Exclusion of Venous Thromboembolism

acronym: DiET

summary: The purpose of this study is to demonstrate the ability of STA® Liatest® D-Di combined with a clinical pretest probability (PTP) to safely exclude pulmonary embolism (PE) or Deep Venous Thrombosis (DVT) in a 3 month follow-up.

ReferenceCitation: Keeling DM, Mackie IJ, Moody A, Watson HG; Haemostasis and Thrombosis Task Force of the British Committee for Standards in Haematology. The diagnosis of deep vein thrombosis in symptomatic outpatients and the potential for clinical assessment and D-dimer assays to reduce the need for diagnostic imaging. Br J Haematol. 2004 Jan;124(1):15-25.

ReferenceCitation: Goekoop RJ, Steeghs N, Niessen RW, Jonkers GJ, Dik H, Castel A, Werker-van Gelder L, Vlasveld LT, van Klink RC, Planken EV, Huisman MV. Simple and safe exclusion of pulmonary embolism in outpatients using quantitative D-dimer and Wells' simplified decision rule. Thromb Haemost. 2007 Jan;97(1):146-50.

ReferenceCitation: Kelly J, Hunt BJ. A clinical probability assessment and D-dimer measurement should be the initial step in the investigation of suspected venous thromboembolism. Chest. 2003 Sep;124(3):1116-9. Review.

ReferenceCitation: Pasha SM, Klok FA, Snoep JD, Mos IC, Goekoop RJ, Rodger MA, Huisman MV. Safety of excluding acute pulmonary embolism based on an unlikely clinical probability by the Wells rule and normal D-dimer concentration: a meta-analysis. Thromb Res. 2010 Apr;125(4):e123-7. doi: 10.1016/j.thromres.2009.11.009. Epub 2009 Nov 26.

ReferenceCitation: Perrier A, Desmarais S, Goehring C, de Moerloose P, Morabia A, Unger PF, Slosman D, Junod A, Bounameaux H. D-dimer testing for suspected pulmonary embolism in outpatients. Am J Respir Crit Care Med. 1997 Aug;156(2 Pt 1):492-6.

ReferenceCitation: Wells PS, Anderson DR, Rodger M, Forgie M, Kearon C, Dreyer J, Kovacs G, Mitchell M, Lewandowski B, Kovacs MJ. Evaluation of D-dimer in the diagnosis of suspected deep-vein thrombosis. N Engl J Med. 2003 Sep 25;349(13):1227-35.

ReferenceCitation: Pernod G, Caterino J, Maignan M, Tissier C, Kassis J, Lazarchick J; DIET study group. D-Dimer Use and Pulmonary Embolism Diagnosis in Emergency Units: Why Is There Such a Difference in Pulmonary Embolism Prevalence between the United States of America and Countries Outside USA? PLoS One. 2017 Jan 13;12(1):e0169268. doi: 10.1371/journal.pone.0169268. eCollection 2017.

lead_sponsor: Diagnostica Stago

location_country:
Spain
Italy
Canada
United States
France

location_facility:
Medical Center of South Carolina
General Hospital
Lehigh Valley Hospital Health Network
University Hospital
IRCCS H San Raffaele
University of Iowa
Brigham and Woman's Hosp
Oklahoma Health Sciences Center
Moses Cone Memorial Hospital
Indiana University Health
Northwestern Medical Center
Maisonneuve Rosemont Hospital
Univeristy Medical Center
Texas Health Fort Worth Hospital
Massachusetts General Hospital


title: A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene

acronym: RESCUE

summary: The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for six months or less.

ReferenceCitation: Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison. Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021.

ReferenceCitation: Newman NJ, Yu-Wai-Man P, Carelli V, Moster ML, Biousse V, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Barboni P, DeBusk AA, Girmens JF, Rudolph G, Karanjia R, Taiel M, Blouin L, Smits G, Katz B, Sahel JA; LHON Study Group. Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset. Ophthalmology. 2021 May;128(5):649-660. doi: 10.1016/j.ophtha.2020.12.012. Epub 2021 Jan 12.

location_facility: Department of Neurology, University of Munich, Friedrich-Baur-Institute

location_facility: Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University

location_facility: Moorfields Eye Hospital NHS Foundation Trust

location_facility: IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria

location_facility: Doheny Eye Center, University of California, Los Angeles

location_facility: Centre National Hospitalier d'Ophtalmologie des Quinze-VingtCentre National Hospitalier d'Ophtalmologie des Quinze-Vingt

location_facility: Department of Ophthalmology, Emory University School of Medicine