title: An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

summary: Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials

lead_sponsor: Global Blood Therapeutics

location_country:
Kenya
United Kingdom
Italy
Oman
Lebanon
Canada
United States
France
Turkey
Netherlands
Egypt

location_facility: Indiana Hemophilia and Thrombosis Center

location_facility: University of North Carolina at Chapel Hill

location_facility: UCSF Benioff Children's Hospital Oakland

location_facility: University of Pittsburgh Medical Center Health System

location_facility: KEMRI - Centre for Respiratory Disease Research (CRDR)

location_facility: American University of Beirut Medical Center

location_facility: University of Illinois College of Medicine

location_facility: KEMRI (Kenya Medical Research Institute) Kisumu

location_facility: Brigham and Women's Hospital Department of Neurology


status: Completed

primary_purpose: Treatment

acronym: Marigold

intervention_type: Drug

title: A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) Followed by Long-term Open-label Treatment

study_completion_date_type: Actual

lead_sponsor: Marinus Pharmaceuticals

type: EudraCT Number

source: euctr

design_allocation: Randomized

study_start_date_type: Actual

study_type: Interventional

ReferenceType: derived

ReferenceCitation: Knight EMP, Amin S, Bahi-Buisson N, Benke TA, Cross JH, Demarest ST, Olson HE, Specchio N, Fleming TR, Aimetti AA, Gasior M, Devinsky O; Marigold Trial Group. Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2022 May;21(5):417-427. doi: 10.1016/S1474-4422(22)00077-1. Erratum in: Lancet Neurol. 2022 Jul;21(7):e7.

enrollment_type: Actual

location_country:
Poland
United Kingdom
Italy
Russian Federation
Australia
United States
Israel
France

location_facility:
Marinus Research Facility
Marinus Research Site

summary: A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone therapy compared to placebo for the treatment of seizures in children and young adults with genetically confirmed CDKL5 gene mutation.

lead_sponsor_type: industriel

status_simplified: Completed


title: Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease

summary: The objective of PB-102-F60 is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F03, PB-102-F20 or PB-102-F30.

status: Enrolling by invitation

study_completion_date_type: Anticipated

enrollment_type: Anticipated

ipd_sharing: Undecided

lead_sponsor: Protalix

location_country:
Slovenia
Italy
Spain
Australia
United Kingdom
Finland
Canada
Hungary
United States
France
Norway
Netherlands
Czechia

location_facility: University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital

location_facility: Azienda Ospedaliera Universitaria "Federico II"

location_facility: University of Iowa Hospitals and Clinics

location_facility: Eccles Primary Children's Outpatient Services Building

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Renal Disease Research Institute, LLC - Dallas

location_facility: Haukeland University Hospital Klinisk Forskningspost

intervention_type: Biological

status_simplified: Unknown


title: Cangrelor Versus Ticagrelor In Patients With Acute Myocardial Infarction Complicated With Initial Cardiogenic Shock

acronym: DAPT-SHOCK-AMI

summary: Multicenter randomized double blind trial comparing intravenous cangrelor and oral ticagrelor in patients with acute myocardial infarction complicated by initial cardiogenic shock and treated with primary angioplasty.

status: Recruiting

ipd_sharing: Yes

lead_sponsor: Faculty Hospital Kralovske Vinohrady

location_country: Germany France Slovakia Czechia

location_facility: Department of Cardiology, Regional Hospital,

location_facility: Institute of Clinical and Experimental Medicine

location_facility: Cardiology department, Regional hospital

location_facility: Cardiology Department, Regional Hospital

location_facility: Department of Cardiology, University Hospital Brno-Bohunice

location_facility: Middle-Slovak Institute of Cardiovascular Diseases

location_facility: Center of Interventional Neuroradiology and Endovascular Treatment

lead_sponsor_type: academique

status_simplified: Ongoing


ipd_sharing: No

status: Active, not recruiting

acronym: Alliance

title: A Phase 3, Randomized, Double-Blind Study to Evaluate the Safety and Efficacy of Fixed Dose Combination of Bictegravir/Emtricitabine/Tenofovir Alafenamide Versus Dolutegravir + Emtricitabine/Tenofovir Disoproxil Fumarate in Treatment Naïve, HIV-1 and Hepatitis B Co-Infected Adults

lead_sponsor: Gilead Sciences

location_country:
Taiwan
Hong Kong
Spain
Greece
Dominican Republic
Thailand
Korea, Republic of
United States
France
China
Puerto Rico
Turkey
Japan
Malaysia

location_facility: Instituto Dominicano de Estudios Virologicos (IDEV)

location_facility: The Catholic University of Korea, Seoul St. Mary's Hospital

location_facility: Hospital General Universitario Santa Lucia

location_facility: Hospital General Universitario de Valencia

location_facility: Ministry of Health and Welfare Taoyuan General Hospital

location_facility: Center Hospital of the National Center for Global Health and Medicine

location_facility: Beijing Ditan Hospital Capital Medical University

location_facility: Korgialenio-Benakio Greek Red Cross General Hospital

location_facility: Taipei City Hospital Linsen, Chinese Medicine and Kunming Branch

location_facility: National Hospital Organization Osaka National Hospital

location_facility: The Crofoot Research Center, INC (DBA: Gordon E. Crofoot MD PA)

location_facility: Faculty of Medicine, Chiang Mai University

location_facility: AHEPA University Hospital of Thessaloniki

location_facility: Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

location_facility: 1st Affiliated Hospital of Zhejiang University

location_facility: Istanbul University Cerrahpasa Medical Faculty

location_facility: Thai Red Cross AIDS Research Centre (HIV-NAT)

location_facility: Beijing YouAn Hospital, Capital Medical University

location_facility: Bamrasnaradura Infectious Diseases Institute

location_facility: The Fifth Medical Center of PLA General Hospital

location_facility: National Hospital Organization Nagoya Medical Center

location_facility: Marmara University Pendik Training and Research Hospital

location_facility: Kaohsiung Medical University Chung-Ho Memorial Hospital

location_facility: Faculty of Medicine Ramathibodi Hospital, Mahidol University

location_facility: CHUVI - Hospital Universitario Alvaro Cunqueiro

summary: The primary objective of this study is to evaluate the efficacy of fixed-dose combination (FDC) of bictegravir/emtricitabine/ tenofovir alafenamide (B/F/TAF) versus dolutegravir (DTG) + emtricitabine/tenofovir disoproxil fumarate (F/TDF) in HIV and hepatitis B virus (HBV) treatment naive, HIV-1 and HBV co-infected adults.


title: A Multicenter, Randomized, Double-Blind, Secukinumab-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Bimekizumab in Adult Subjects With Moderate to Severe Chronic Plaque Psoriasis

acronym: BE RADIANT

summary: This is a study to compare the efficacy of bimekizumab versus secukinumab in subjects with moderate to severe chronic plaque psoriasis (PSO).

ReferenceCitation: Gottlieb AB, Warren RB, Augustin M, Garcia L, Cioffi C, Peterson L, Pelligra C, Ciaravino V. Psychometric Validation of the Psoriasis Symptoms and Impacts Measure (P-SIM): A Novel Patient-Reported Outcome Instrument for Patients with Plaque Psoriasis, Using Reported Data from the BE RADIANT Phase 3b Trial. Adv Ther. 2021 Oct;38(10):5253-5269. doi: 10.1007/s12325-021-01836-1. Epub 2021 Sep 2.

ReferenceCitation: Yeremenko N. Out of the shadow of interleukin-17A: the role of interleukin-17F and other interleukin-17 family cytokines in spondyloarthritis. Curr Opin Rheumatol. 2021 Jul 1;33(4):333-340. doi: 10.1097/BOR.0000000000000805. Review.

ReferenceCitation: Reich K, Warren RB, Lebwohl M, Gooderham M, Strober B, Langley RG, Paul C, De Cuyper D, Vanvoorden V, Madden C, Cioffi C, Peterson L, Blauvelt A. Bimekizumab versus Secukinumab in Plaque Psoriasis. N Engl J Med. 2021 Jul 8;385(2):142-152. doi: 10.1056/NEJMoa2102383. Epub 2021 Apr 23.

ipd_sharing_description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available

lead_sponsor: UCB Biopharma SRL

location_country:
Poland
Spain
United Kingdom
Australia
Netherlands
Canada
United States
France
Turkey
Belgium
Germany

intervention_type: Other


title: A Prospective Post Market Study of Patients With Symptomatic Benign Prostatic Hyperplasia Treated by Prostatic Artery Embolization With Embosphere® Microspheres

acronym: PROstate

summary: Prostatic artery embolization with Embosphere Microspheres is a relatively new procedure. The goal of this post market study is to evaluate long-term safety and effectiveness in a 'real world' setting.

study_type: Observational

time_perspective: Prospective

lead_sponsor: Merit Medical Systems, Inc.

location_country:
United States
Italy
United Kingdom
France

location_facility: Royal Bournemouth and Christchurch Hospital

location_facility: University of Miami- Miller School of Medicine

location_facility: Azienda Ospedaliera S. Croce e Carle Cuneo

intervention_type: Device


title: PeriOperative ISchemic Evaluation-3 Trial

summary: This study is a multicentre, international, randomized controlled trial of tranexamic acid (TXA) versus placebo and, using a partial factorial design, of a perioperative hypotension-avoidance versus hypertension-avoidance strategy.

ReferenceCitation: Devereaux PJ, Marcucci M, Painter TW, Conen D, Lomivorotov V, Sessler DI, Chan MTV, Borges FK, Martínez-Zapata MJ, Wang CY, Xavier D, Ofori SN, Wang MK, Efremov S, Landoni G, Kleinlugtenbelt YV, Szczeklik W, Schmartz D, Garg AX, Short TG, Wittmann M, Meyhoff CS, Amir M, Torres D, Patel A, Duceppe E, Ruetzler K, Parlow JL, Tandon V, Fleischmann E, Polanczyk CA, Lamy A, Astrakov SV, Rao M, Wu WKK, Bhatt K, de Nadal M, Likhvantsev VV, Paniagua P, Aguado HJ, Whitlock RP, McGillion MH, Prystajecky M, Vincent J, Eikelboom J, Copland I, Balasubramanian K, Turan A, Bangdiwala SI, Stillo D, Gross PL, Cafaro T, Alfonsi P, Roshanov PS, Belley-Côté EP, Spence J, Richards T, VanHelder T, McIntyre W, Guyatt G, Yusuf S, Leslie K; POISE-3 Investigators. Tranexamic Acid in Patients Undergoing Noncardiac Surgery. N Engl J Med. 2022 May 26;386(21):1986-1997. doi: 10.1056/NEJMoa2201171. Epub 2022 Apr 2.

ReferenceCitation: Marcucci M, Painter TW, Conen D, Leslie K, Lomivorotov VV, Sessler D, Chan MTV, Borges FK, Martínez Zapata MJ, Wang CY, Xavier D, Ofori SN, Landoni G, Efremov S, Kleinlugtenbelt YV, Szczeklik W, Schmartz D, Garg AX, Short TG, Wittmann M, Meyhoff CS, Amir M, Torres D, Patel A, Duceppe E, Ruetzler K, Parlow JL, Tandon V, Wang MK, Fleischmann E, Polanczyk CA, Jayaram R, Astrakov SV, Rao M, VanHelder T, Wu WKK, Cheong CC, Ayad S, Abubakirov M, Kirov M, Bhatt K, de Nadal M, Likhvantsev V, Iglesisas PP, Aguado HJ, McGillion M, Lamy A, Whitlock RP, Roshanov P, Stillo D, Copland I, Vincent J, Balasubramanian K, Bangdiwala SI, Biccard B, Kurz A, Srinathan S, Petit S, Eikelboom J, Richards T, Gross PL, Alfonsi P, Guyatt G, Belley-Cote E, Spence J, McIntyre W, Yusuf S, Devereaux PJ. Rationale and design of the PeriOperative ISchemic Evaluation-3 (POISE-3): a randomized controlled trial evaluating tranexamic acid and a strategy to minimize hypotension in noncardiac surgery. Trials. 2022 Jan 31;23(1):101. doi: 10.1186/s13063-021-05992-1.

lead_sponsor: Population Health Research Institute

location_country:
Spain
Italy
United Kingdom
United States
Belgium
South Africa
Poland
Hong Kong
Austria
Australia
Russian Federation
India
Malaysia
Brazil
Canada
China
Germany
Denmark
New Zealand
France
Chile
Netherlands
Pakistan

location_facility: University of North Carolina at Chapel Hill

location_facility: Specialistyczny Szpital im. E. Szczeklika w Tarnowie

location_facility: Hospital of Saint-Petersburg State University

location_facility: Hospital Clínico Universitario in Valladolid

location_facility: Hospital Universitario Fundacion Alcorcon

location_facility: Steve Biko Academic Hospital - University of Pretoria

location_facility: Second Hospital of Anhui Medical University

location_facility: Sefako Makgatho Health Sciences University (SMU)

location_facility: E. Meshalkin National Medical Research Center

location_facility: Moscow Regional Research & Clinical Institute (MONIKI)

location_facility: NH Rabindranath Tagore International Inst. of Cardiac Sciences

location_facility: The Fourth Affiliated Hospital of Harbin Medical University

location_facility: University of Calgary, O'Brien Institute for Public Health

location_facility: University Health Network (Toronto General Hospital)

location_facility: The Ohio State University Wexner Medical Center

location_facility: West China Hospital of Sichuan University

location_facility: Sumandeep Vidyapeeth & Dhiraj General Hospital

location_facility: Szpital Uniwersytecki im Karola Marcinkowskiego w Zielonej Górze

location_facility: Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow

location_facility: V. Negovskiy Reanimatology Research Institute


title: A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, STUDY OF THE EFFICACY AND SAFETY OF TOFACITINIB IN SUBJECTS WITH ACTIVE ANKYLOSING SPONDYLITIS (AS)

lead_sponsor: Pfizer

type: Other Identifier

ReferenceCitation: Cella D, Lenderking WR, Chongpinitchai P, Bushmakin AG, Dina O, Wang L, Cappelleri JC, Navarro-Compán V. Functional Assessment of Chronic Illness Therapy-Fatigue is a reliable and valid measure in patients with active ankylosing spondylitis. J Patient Rep Outcomes. 2022 Sep 23;6(1):100. doi: 10.1186/s41687-022-00508-0.

ReferenceCitation: Navarro-Compán V, Wei JC, Van den Bosch F, Magrey M, Wang L, Fleishaker D, Cappelleri JC, Wang C, Wu J, Dina O, Fallon L, Strand V. Effect of tofacitinib on pain, fatigue, health-related quality of life and work productivity in patients with active ankylosing spondylitis: results from a phase III, randomised, double-blind, placebo-controlled trial. RMD Open. 2022 Jun;8(2). pii: e002253. doi: 10.1136/rmdopen-2022-002253.

ReferenceCitation: Deodhar A, Sliwinska-Stanczyk P, Xu H, Baraliakos X, Gensler LS, Fleishaker D, Wang L, Wu J, Menon S, Wang C, Dina O, Fallon L, Kanik KS, van der Heijde D. Tofacitinib for the treatment of ankylosing spondylitis: a phase III, randomised, double-blind, placebo-controlled study. Ann Rheum Dis. 2021 Aug;80(8):1004-1013. doi: 10.1136/annrheumdis-2020-219601. Epub 2021 Apr 27.

ipd_sharing_description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

location_country:
Poland
Russian Federation
Australia
Canada
Ukraine
Hungary
Korea, Republic of
United States
France
China
Israel
Turkey
Bulgaria
Czechia

location_facility: State Healthcare Institution "Regional Clinical Hospital"

location_facility: The Second Affiliated Hospital of Zhejiang University School of Medicine

location_facility: Centre de Recherche Musculo-Squelettique

location_facility: VITAL MEDICAL CENTER (VITÁL-MEDICINA Kft.)

location_facility: Paramount Medical Research and Consulting, LLC

location_facility: Military-Medical Clinical Center (Clinical Hospital with 200 beds) of State Border Guard Service of

location_facility: Ankara Universitesi Tip Fakultesi Ibn-i Sina Hastanesi

location_facility: Ternopil University Hospital, Department of Rheumatology,

location_facility: Hacettepe Universitesi Tip Fakultesi, Ic Hastaliklari Anabilim

location_facility: Rheumatology Associates of North Alabama, PC

location_facility: Rheumatology Research Unit Sunshine Coast

location_facility: Trinity Universal Research Associates, Inc.

location_facility: Zespol Poradni Specjalistycznych REUMED, Wallenroda Filia nr 1

location_facility: Rheumatology and Immunology Department, Shanghai Changzheng Hospital

location_facility: Prywatna Praktyka Lekarska Prof. UM dr hab. med. Pawel Hrycaj

location_facility: University of California, San Francisco Medical Center

location_facility: Szpital Uniwersytecki w Krakowie, Zaklad Radiologii

location_facility: Szpital Sw. Elzbiety, Mokotowskie Centrum Medyczne, Zaklad Diagnostyki

location_facility: Limited Liability Company Medical Center "Rheuma-Med"

location_facility: Malopolskie Badania Kliniczne Sp. z o.o. s.k.

location_facility: Center for Rheumatology and Bone Research

location_facility: Pest megyei Flór Ferenc Kórház, Reumatológia-és Fizioterápiás Osztály

location_facility: Samodzielny Publiczny Zaklad Opieki Zdrowotnej MSWiA

location_facility: NZOZ JAMED Pracownia Rentgenowska Jerema Antoszewski

location_facility: The First Affiliated Hosptial of Wenzhou Medical University

location_facility: Izmir Katip Celebi Univ. Ataturk Egitim ve Arastirma Hastanesi

location_facility: RSBHI "Smolensk Regional Clinical Hospital"

location_facility: Vinnytsia Regional Clinical Hospital n.a. M.I.Pyrohov, rheumatology department,

location_facility: Precision Comprehensive Clinical Research Solutions

location_facility: Istanbul Universitesi Istanbul Tip Fakultesi, Ic Hastaliklari

location_facility: Lviv Communal Clinical Hospital #4, Rheumatology department

location_facility: Geo Medical Sp. z.o.o Szpital Geo Medical

location_facility: The Third Affiliated Hospital, Sun Yat-Sen University

location_facility: Arizona Arthritis & Rheumatology Associates, P.C.

location_facility: Multiprofile Hospital for active treatment "Kaspela" EOOD

location_facility: Diagnostic-Consulting Center XVII - Sofia

location_facility: Izmir Katip Celebi Univ Ataturk Egitim ve Arastirma Hastanesi

location_facility: Articularis Healthcare Group, Inc d/b/a Low Country Rheumatology

location_facility: G.R.M.O. (Groupe de recherche en maladies osseuses) Inc.

location_facility: State Budgetary Healthcare Institution "Orenburg Regional Clinical Hospital"

location_facility: Ankara Universitesi Tip Fakultesi, Ibn-i Sina Hastanesi

location_facility: Marmara Univ. Istanbul Pendik Egitim ve Arastirma Hastanesi

location_facility: Kyiv City Clinical Hospital # 3, Rheumatology Department

summary: The purpose of this study is to determine if tofacitinib is safe and effective in subjects with active ankylosing spondylitis.


title: A Phase II Open-label, Randomized, Three-arm, Multicenter Study of LAG525 Given in Combination With Spartalizumab (PDR001), or With Spartalizumab and Carboplatin, or With Carboplatin, as First or Second Line Therapy in Patients With Advanced Triple-negative Breast Cancer

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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com.

location_country:
Taiwan
Spain
Italy
Australia
Lebanon
Argentina
Canada
Thailand
Hungary
Korea, Republic of
United States
France
Israel
Singapore
Belgium
Japan
Germany

location_facility:
Highlands Oncology Group
Novartis Investigative Site
Ironwood Cancer and Research Centers

summary: The main purpose of this study was to assess the antitumor activity of three combinations: i) LAG525 + spartalizumab; ii) LAG525 + spartalizumab + carboplatin, and iii) LAG525 + carboplatin in participants with advanced triple-negative breast cancer (TNBC) in first or second line therapy.


title: A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

acronym: STAR

summary: The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM).

ReferenceCitation: Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5. Review.

lead_sponsor: NightstaRx Ltd, a Biogen Company

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

intervention_type: Genetic


status: Terminated

acronym: CREAD OLE

title: A Multicenter, Open-Label, Long-Term Extension Of Phase III Studies (BN29552/BN29553) Of Crenezumab In Patients With Alzheimer's Disease

lead_sponsor: Hoffmann-La Roche

location_country:
Poland
Hong Kong
Spain
Italy
Russian Federation
Australia
United Kingdom
Finland
Canada
Lithuania
Korea, Republic of
United States
France
Turkey
Mexico
Germany

location_facility: The Cognitive and Research Center of New Jersey

location_facility: Heidelberg Repatriation Hospital; Medical and Cognitive Research Centre

location_facility: Hospital Virgen del Puerto. Servicio de Neurología

location_facility: Ondokuz Mayis Univ. Med. Fac.; Neurology

location_facility: Hospital General De Catalunya; Servicio de Neurologia

location_facility: Kawartha Centre - Redefining Healthy Aging

location_facility: SHI City Psychoneurological Dispensary #7 (with Hospital)

location_facility: Neurodegenerative Disorders Research; Neurology

location_facility: Hospital Universitario 12 de Octubre; Servicio de Neurologia

location_facility: National Clinical Research Inc.-Richmond

location_facility: State autonomous institution of healthcare Inter-regional clinical and diagnostic center

location_facility: Clinica Universitaria de Navarra; Servicio de Neurología

location_facility: Hospital Mutua De Terrasa; Servicio de Neurologia

location_facility: Vilnius University Hospital Santariskiu Clinic

location_facility: Alzheimer's Research and Treatment Center

location_facility: Surrey and Borders NHS Foundation Trust; Research and Development Departmant; Abraham Cowley Unit

location_facility: Ospedale San Giovanni Calibita Fatebenefratell;Neurologia

location_facility: University of California, Davis; Alzheimers Disease Center, Department of Neurology

location_facility: Advanced Memory Research Institute of NJ

location_facility: Institute for Neurodegenerative Disorders

location_facility: Prince of Wales Hospital; Dept. of Medicine & Therapeutics

location_facility: State Autonomous Healthcare Institution "Republican Clinical Neurological Center

location_facility: Southern Illinois University, School of Medicine

location_facility: Klinikum rechts der Isar der TU München; Klinik für Psychiatrie und Psychotherapie

location_facility: Hospital Angeles de Culiacán, Neurociencias Estudios Clínicos SC

location_facility: Research Center for Clinical Studies, Inc.

summary: In the BN40031 OLE study, a dose of crenezumab of 60 mg/kg intravenous (IV) every 4 weeks (Q4W) will be offered to all participants who complete Study BN29552 or BN29553 and who meet eligibility criteria in order to evaluate safety in participants on long-term crenezumab treatment and to investigate the effect of crenezumab on the underlying disease process and disease course as an exploratory efficacy objective.


title: FLExAbility Sensor Enabled Substrate Targeted Ablation for the Reduction of Ventricular Tachycardia (LESS-VT) Study

acronym: LESS-VT

summary: This clinical investigation is intended to demonstrate the safety and effectiveness of ventricular ablation therapy using the FlexAbility Sensor Enabled Ablation Catheter in patients with drug-refractory monomorphic ventricular tachycardia in whom ventricular tachycardia recurs despite antiarrhythmic drug therapy or when antiarrhythmic drugs are not tolerated or desired.

lead_sponsor: Abbott Medical Devices

location_country:
Spain
Italy
United Kingdom
United States
France
Germany
Czechia

location_facility: University Hospital - Univ. of Alabama at Birmingham (UAB)

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: Hospital of the University of Pennsylvania

location_facility: Affinity Cardiovascular Specialists, LLC

location_facility: University of California at San Diego (UCSD) Medical Center


title: Pyruvate Kinase Deficiency Global Longitudinal Registry

acronym: PEAK Registry

summary:
This study is an observational (ie, noninterventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare nonspherocytic hemolytic anemia.

This Registry will be open for enrollment for 7 years and all enrolled participants will be followed prospectively for a minimum of 2 years, and up to 9 years.

Data will be collected from participating Registry Physicians, participants, and, where appropriate, parents/guardians who have provided informed consent or assent (where relevant) and authorization pursuant to applicable laws and regulations.

Data should include demographic, clinical, and treatment data; and other data of relevance to the management of patients with PK deficiency. Annual chart review and data entry are expected in order to enhance longitudinal understanding of PK deficiency; however, no specific protocol schedule of assessment is required by this Registry protocol.

lead_sponsor: Agios Pharmaceuticals, Inc.

location_country:
Denmark
Portugal
Spain
Ireland
Italy
Netherlands
United Kingdom
Canada
Thailand
Switzerland
Korea, Republic of
United States
France
Turkey
Germany
Czechia

location_facility: AOU dell'Universita degli Studi della Campania Luigi Vanvitelli

location_facility: The Catholic University of Korea, Seoul St. Mary's Hospital

location_facility: Hospital Universitario Vall d'Hebron - PPDS

location_facility: Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Evangelisches Krankenhaus Bielefeld gGmbH

location_facility: Centro Hospitalar E Universitario de Coimbra EPE

location_facility: University of Arkansas for Medical Sciences

location_facility: Centro Hospitalar de Vila Nova de Gaia / Espinho E.P.E

location_facility: The Newcastle Upon Tyne Hospitals NHS Foundation Trust

location_facility: Centro Hospitalar Lisboa Central- Hospital Dona Estefania

location_facility: Hospital Universitario Germans Trias i Pujol

location_facility: Hospital Infantil Universitario Nino Jesus


title: A Phase 2,Multicenter,Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab,With or Without Hydroxyurea/Hydroxycarbamide,in Sequential,Descending Age Groups of Pediatric Sickle Cell Disease Patients With Vaso-Occlusive Crisis

ReferenceCitation: Del Pozo Martín Y. 2021 ASH annual meeting. Lancet Haematol. 2022 Feb;9(2):e92-e93. doi: 10.1016/S2352-3026(21)00384-7. Epub 2021 Dec 16.

location_country:
Brazil
Belgium
Spain
Italy
Oman
Lebanon
United Kingdom
Canada
Switzerland
United States
France
India
Turkey
Colombia
Germany

summary: The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 6 months to <18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years. The efficacy and safety of crizanlizumab was already demonstrated in adults with sickle cell disease. The approach is to extrapolate from the PK/pharmacodynamics (PD) already established in the adult population. The study is designed as a Phase II, multicenter, open-label study.


title: Evaluate the Safety and Efficacy of ALLN-177 in Patients With Enteric Hyperoxaluria: A Phase III Randomized, Placebo-Controlled Study

summary: The purpose of this study is to determine the efficacy and safety of ALLN-177 in patients with enteric hyperoxaluria.

ReferenceType: background

ReferenceCitation: Langman CB, Grujic D, Pease RM, Easter L, Nezzer J, Margolin A, Brettman L. A Double-Blind, Placebo Controlled, Randomized Phase 1 Cross-Over Study with ALLN-177, an Orally Administered Oxalate Degrading Enzyme. Am J Nephrol. 2016;44(2):150-8. doi: 10.1159/000448766. Epub 2016 Aug 17.

lead_sponsor: Allena Pharmaceuticals

location_country:
Spain
Italy
United Kingdom
Canada
United States
France
Germany

location_facility: University of North Carolina Chapel Hill

location_facility: Universitatsklinikum Freiburg, Klinik for Urologie

location_facility: Research center of CHU de Québec - Université Laval (CHUL)

location_facility: Centre de Recherche du Centre Hospitalier de l'Université de Montréal (CRCHUM)

location_facility: Washington University School of Medicine

location_facility: Urological Associates of Southern Arizona

location_facility: University of Alabama, Department of Urology

location_facility: Hospital Universitari de Girona Doctor Josep Trueta

location_facility: Universitaetsklinikum Bonn; Klinik und Poliklinik für Urologie und Kinderurologie

location_facility: PSHI GmbH; PFÜTZNER Science & Health Institute GmbH

location_facility: Albert Einstein College of Medicine Montefiore Medical Center

location_facility: IRCCS Azienda Ospedaliera Universitaria San Martino IST

location_facility: University of Alberta, Division of Urology

location_facility: Toronto Digestive Disease Associates, Inc.

location_facility: Azienda Socio-Sanitaria Territoriale di Lecco (ASST di Lecco)

location_facility: Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore

location_facility: Universitat Bonn - Zentrum für Kinderheilkunde


title: Randomized, Double-blind, Placebo-controlled, Two-Part, Adaptive Design Study of Safety, Tolerability, Preliminary Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Oral Doses of MYK-491 in Patients With Stable Heart Failure With Reduced Ejection Fraction

summary: The purpose of this Phase 1b/2a study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of MYK-491 in patients with DCM and stable heart failure.

lead_sponsor: MyoKardia, Inc.

location_country:
Poland
United Kingdom
Netherlands
Sweden
United States
France
Germany

location_facility: Wojewodzki Szpital Specjalistyczny we Wroclawiu, Oddzial Kardiologiczny z Pododdzialem Intensywnego Nadzoru Kardiologicznego i Pododdzialem Leczenia Zaburzen Rytmu Serca

location_facility: Jacksonville Center for Clinical Research

location_facility: Wojewodzki Szpital Specjalistyczny Im M Kopernika

location_facility: University of Pennsylvania Heart and Vascular Center


title: A Phase 1 Study of AG-270 in the Treatment of Subjects With Advanced Solid Tumors or Lymphoma With Homozygous Deletion of MTAP

summary: This study will evaluate the safety, pharmacokinetics, pharmacodynamics, and clinical activity of AG-270 in participants with advanced solid tumors or lymphoma with homozygous MTAP deletion.

design_allocation: Non-Randomized

ReferenceCitation: Konteatis Z, Travins J, Gross S, Marjon K, Barnett A, Mandley E, Nicolay B, Nagaraja R, Chen Y, Sun Y, Liu Z, Yu J, Ye Z, Jiang F, Wei W, Fang C, Gao Y, Kalev P, Hyer ML, DeLaBarre B, Jin L, Padyana AK, Dang L, Murtie J, Biller SA, Sui Z, Marks KM. Discovery of AG-270, a First-in-Class Oral MAT2A Inhibitor for the Treatment of Tumors with Homozygous MTAP Deletion. J Med Chem. 2021 Apr 22;64(8):4430-4449. doi: 10.1021/acs.jmedchem.0c01895. Epub 2021 Apr 8.

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

Access can be requested for all interventional clinical studies:

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

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

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

lead_sponsor: Institut de Recherches Internationales Servier

location_country:
United States
Spain
France

location_facility:
Dana Farber Cancer Institute
Beth Israel
Sarah Cannon Cancer Center
Hospital Universitario Vall d'Hebron
Institut Gustave Roussy
Memorial Sloan Kettering Cance Center
Yale University
Massachusetts General Hospital


acronym:
Phase 2 Study of PT2977 for Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma

 Etude de Phase 2 du PT2977 pour le traitement  du cancer du rein associé à la maladie de VHL

title: An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma

lead_sponsor: Peloton Therapeutics, Inc.

ReferenceCitation: Jonasch E, Donskov F, Iliopoulos O, Rathmell WK, Narayan VK, Maughan BL, Oudard S, Else T, Maranchie JK, Welsh SJ, Thamake S, Park EK, Perini RF, Linehan WM, Srinivasan R; MK-6482-004 Investigators. Belzutifan for Renal Cell Carcinoma in von Hippel-Lindau Disease. N Engl J Med. 2021 Nov 25;385(22):2036-2046. doi: 10.1056/NEJMoa2103425.

location_country:
Denmark
United States
United Kingdom
France

location_facility: University of Pennsylvania Medical Center

location_facility: National Institutes of Health Clinical Center

summary: This study is designed to investigate belzutifan as a treatment for VHL disease associated RCC.


type: Other Grant/Funding Number

title: Biology of Juvenile Myoclonic Epilepsy

acronym: BIOJUME

summary: The investigators are collecting genetic information through blood samples as well as clinical and EEG data from over 1000 people with Juvenile Myoclonic Epilepsy (JME) across the UK, Europe and North America. This study will draw on both existing and new samples from JME patients. These will be compared to anonymised data from samples for 2000 controls. The goal of this study is to find the genetic cause of JME. Finding the cause will help create better treatments for JME, as well as improve patient outcomes by allowing us to detect it earlier.

time_perspective: Other

lead_sponsor: King's College London

location_country:
Denmark
United Kingdom
Italy
Estonia
Canada
United States
France
Norway
Czechia

location_facility: Walton Centre for Neurology and Neurosurgery

location_facility: Commissione Genetica Lega Italiana contro l'Epilepssia


title: An Adaptive Seamless Randomized, Double-blind, Placebo-controlled, Dose Ranging Study to Investigate the Efficacy and Safety of LNP023 in Primary IgA Nephropathy Patients

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
Belgium
Japan
Czechia
Hong Kong
Australia
Argentina
India
Israel
Turkey
Norway
Sweden
Malaysia
Singapore
Brazil
China
Germany
Denmark
Taiwan
Finland
Thailand
Korea, Republic of
France
Colombia
Netherlands

summary: Efficacy and safety of LNP023 in IgAN patients


title: A Pivotal Phase 2 Trial of Antibody Naxitamab (hu3F8) and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow

summary:
Children and adults diagnosed with high-risk neuroblastoma patients with primary refractory disease or incomplete response to salvage treatment in bone and/or bone marrow will be treated for up to 101 weeks with naxitamab and granulocyte-macrophage colony stimulating factor (GM-CSF). Patients will be followed for up to five years after first dose.

Naxitamab, also known as hu3F8 is a humanised monoclonal antibody targeting GD2

lead_sponsor: Y-mAbs Therapeutics

location_country:
Denmark
Hong Kong
Spain
Italy
United Kingdom
Canada
United States
France
Germany

location_facility: University Medical Center Hamburg-Eppendorf

location_facility: Hospital Universitario y Politécnico La Fe

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: Hospital Infantil Universitario Niño Jesús


title: Natural History of the Progression of Choroideremia Study

acronym: NIGHT

summary: The objective of this natural history study is to gain a better understanding of the progression of choroideremia (CHM) and add to the knowledge base for this rare disease.

ReferenceCitation: Hariri AH, Velaga SB, Girach A, Ip MS, Le PV, Lam BL, Fischer MD, Sankila EM, Pennesi ME, Holz FG, MacLaren RE, Birch DG, Hoyng CB, MacDonald IM, Black GC, Tsang SH, Bressler NM, Larsen M, Gorin MB, Webster AR, Sadda SR; Natural History of the Progression of Choroideremia (NIGHT) Study Group. Measurement and Reproducibility of Preserved Ellipsoid Zone Area and Preserved Retinal Pigment Epithelium Area in Eyes With Choroideremia. Am J Ophthalmol. 2017 Jul;179:110-117. doi: 10.1016/j.ajo.2017.05.002. Epub 2017 May 10.

ipd_sharing_description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/

location_country:
Denmark
Brazil
United Kingdom
Netherlands
Finland
Canada
United States
France
Germany


title: A Phase 1a/1b Study of an Anti-IDO-1 Agent (LY3381916) Administered Alone or in Combination With Anti- PD-L1 Checkpoint Antibody (LY3300054) in Solid Tumors

summary: The purpose of this study is to evaluate the safety of the study drug LY3381916 administered alone or in combination with anti-programmed cell death ligand 1 (PD-L1) checkpoint antibody (LY3300054).

lead_sponsor: Eli Lilly and Company

location_country:
Denmark
Spain
Italy
United States
France
Belgium

location_facility: Hospital Clinico Universitario Virgen de la Victoria


acronym: ENVISION

title: ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias

lead_sponsor: Alnylam Pharmaceuticals

ReferenceCitation: Wang B, Ventura P, Takase KI, Thapar M, Cassiman D, Kubisch I, Liu S, Sweetser MT, Balwani M. Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study. Orphanet J Rare Dis. 2022 Aug 26;17(1):327. doi: 10.1186/s13023-022-02463-x.

ReferenceCitation: Balwani M, Sardh E, Ventura P, Peiró PA, Rees DC, Stölzel U, Bissell DM, Bonkovsky HL, Windyga J, Anderson KE, Parker C, Silver SM, Keel SB, Wang JD, Stein PE, Harper P, Vassiliou D, Wang B, Phillips J, Ivanova A, Langendonk JG, Kauppinen R, Minder E, Horie Y, Penz C, Chen J, Liu S, Ko JJ, Sweetser MT, Garg P, Vaishnaw A, Kim JB, Simon AR, Gouya L; ENVISION Investigators. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. N Engl J Med. 2020 Jun 11;382(24):2289-2301. doi: 10.1056/NEJMoa1913147.

location_country:
Denmark
Poland
Taiwan
Spain
Italy
United Kingdom
Australia
Netherlands
Canada
Finland
Sweden
Korea, Republic of
United States
France
Mexico
Japan
Germany
Bulgaria

summary: The purpose of this study is to evaluate the effect of subcutaneous givosiran (ALN-AS1), compared to placebo, on the rate of porphyria attacks in patients with Acute Hepatic Porphyrias (AHP).


title: InterStim® Amplitude Study

summary: This feasibility study will explore the effects of three different InterStim amplitude settings on overactive bladder symptoms (OAB).

primary_purpose: Basic Science

lead_sponsor: MedtronicNeuro

location_country:
United Kingdom
Italy
Canada
United States
France
Belgium
Netherlands

location_facility: Azienda Ospedaliera Universitaria Integrata Verona

location_facility: University College London Hospitals NHS Foundation Trust - University College Hospital

location_facility: Chattanooga Hamilton County Hospital Authority d/b/a Erlanger Health System

location_facility: University Urology Associates, Toronto Western Hospital

location_facility: CHU Hôpitaux de Rouen - Hôpital Charles Nicolle

location_facility: Aurora Research Institute - Aurora West Allis Women's Pavilion

location_facility: UZ Leuven - Campus Gasthuisberg - Department Urology

location_facility: Academic Urology & Urogynecology of Arizona

location_facility: Adult Pediatric Urology and Urogynecology


title: A Phase 1 Study Evaluating the Safety, Tolerability and Pharmacokinetics of AMG 757 in Subjects With Small Cell Lung Cancer

summary: A study to assess the safety, tolerability, and pharmacokinetics of AMG 757 in Subjects with Small Cell Lung Cancer

ReferenceCitation: Owen DH, Giffin MJ, Bailis JM, Smit MD, Carbone DP, He K. DLL3: an emerging target in small cell lung cancer. J Hematol Oncol. 2019 Jun 18;12(1):61. doi: 10.1186/s13045-019-0745-2.

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

lead_sponsor: Amgen

location_country:
Poland
Taiwan
Spain
United Kingdom
Austria
Australia
Netherlands
Switzerland
United States
France
Japan
Germany

location_facility: The Ohio State University Wexner Medical Center - Thoracic Oncology Clinic

location_facility: John Hopkins Sidney Kimmel Comprehensive Cancer Center

location_facility: Europejskie Centrum Zdrowia Otwock Szpital imienia Fryderyka Chopina

location_facility: Hospital Clinic i Provincial de Barcelona

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: University Hospitals Cleveland Medical Center

location_facility: Nederlands Kanker Instituut, Antoni van Leeuwenhoek Ziekenhuis

location_facility: Linkou Chang Gung Memorial Hospital of Chang Gung Medical Foundation

location_facility: Kaohsiung Medical University Chung-Ho Memorial Hospital


title: Contrast-Enhanced Ultrasound Evaluation of Focal Liver Lesions in Patients With Cirrhosis or Other Risk Factors for Developing HCC

summary: This clinical trial studies how well contrast-enhanced ultrasound imaging works in diagnosing liver cancer in patients with cirrhosis. Diagnostic procedures, such as contrast-enhanced ultrasound imaging, may help find and diagnose liver cancer.

primary_purpose: Diagnostic

lead_sponsor: Sidney Kimmel Cancer Center at Thomas Jefferson University

location_country:
United Kingdom
Italy
Canada
Switzerland
United States
France

location_facility: University of Texas Southwestern Medical Center

intervention_type: Procedure


title: A Phase III Multicenter Randomized, Sham Controlled, Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2

summary: This study is a phase 3, randomized, multi-center study to evaluate the efficacy and safety of the NT-501 implants in participants with macular telangiectasia type 2.

lead_sponsor: Neurotech Pharmaceuticals

location_country:
Australia
United States
United Kingdom
France

location_facility: Scripps Clinic Carmel Valley Ambulatory Surgery Center

location_facility: University of Utah John A. Moran Eye Center

location_facility: Jules Stein Eye Institute / David Geffen School of Medicine

location_facility: Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust

location_facility: Retina Consultants of Southern Colorado, P.C.

intervention_type: Combination Product


acronym: MOVE

title: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)

lead_sponsor: Clementia Pharmaceuticals Inc.

ReferenceCitation: Shimono K, Tung WE, Macolino C, Chi AH, Didizian JH, Mundy C, Chandraratna RA, Mishina Y, Enomoto-Iwamoto M, Pacifici M, Iwamoto M. Potent inhibition of heterotopic ossification by nuclear retinoic acid receptor-γ agonists. Nat Med. 2011 Apr;17(4):454-60. doi: 10.1038/nm.2334. Epub 2011 Apr 3. Erratum in: Nat Med. 2012 Oct;18(10):1592.

location_country:
Brazil
Spain
Italy
United Kingdom
Australia
Argentina
Canada
United States
France
Japan
Sweden

location_facility: Hospital Italiano de Buenos Aires, Tte General Juan Domingo Peron 4190

location_facility: Hospital for Sick Children, 555 University Avenue

location_facility: Royal National Orthopaedic Hospital, Brockely Hill

location_facility: University of California San Francisco, Division of Endocrinology and Metabolism

location_facility: Istituto Giannina Gaslini, Department of Pediatrics, Unit of Rare Diseases

location_facility: Hospital Universitari i Politècnic La Fe, Unidad de Reumatología Pediatrica

location_facility: Groupe Hospitalier Necker Enfants Malades

summary: Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by heterotopic ossification (HO) often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to ankyloses of major joints with cumulative and irreversible loss of movement and disability.


title: ASSURE: An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Seladelpar in Subjects With Primary Biliary Cholangitis (PBC)

summary: An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Seladelpar in Subjects with Primary Biliary Cholangitis (PBC)

lead_sponsor: CymaBay Therapeutics, Inc.

location_country:
Poland
Romania
Spain
Greece
Italy
Australia
Mexico
Argentina
Canada
Netherlands
Russian Federation
United Kingdom
Hungary
Korea, Republic of
United States
Chile
France
Israel
Belgium
Germany

location_facility: The Catholic University of Korea, Seoul St. Mary's Hospital

location_facility: Schiff Center for Liver Diseases/University of Miami

location_facility: SPbGU Medical and diagnostic center of the clinic of high medical technology n.a. N.I. Pirogov

location_facility: Federal state budget educational institution of high education "Stavropol state medical university"

location_facility: Gastroenterologisch - Hepatologisches Zentrum Kiel

location_facility: Richmond Community Hospital LLC., d/b/a Bon Secours Liver Institute of Richmond

location_facility: CINME (Centro de Investigaciones Metabolicas

location_facility: Severance Hospital, Yonsei University Health System

location_facility: Hospital Universitario Germans Trias i Pujol

location_facility: Universitatsklinik Tubingen, Medizinische Klinik, Innere Medizin I

location_facility: Uniwersyteckie Centrum Kliniczne im. Prof. K. Gibinskiego Slaskiego Uniwersytetu Medycznego w Katowicach, Oddzial Gastroenterologii i Hepatologii

location_facility: Universitatsklinikum Erlangen, Medizinische Klink I, Gastroenterologie

location_facility: U.T. Southwestern Medical Center Investigation Drug Service

location_facility: Soon Chun Hyang University Hospital Bucheon

location_facility: Federal State Autonomous Educational Institution of Higher Education "Peoples' Friendship University of Russia", Centre of Liver Studies

location_facility: Consultorio de la Doctora Maria Sarai Gonzalez Huezo

location_facility: University of Rochester Medical Center - PPDS

location_facility: Charite Universitatsmedizin Berlin, Medizinische Poliklinik

location_facility: Portsmouth Hospitals University NHS Trust

location_facility: University of California, Davis Medical Center

location_facility: Southern Therapy and Advanced Research (STAR), LLC

location_facility: Northwell Health Center for Liver Diseases

location_facility: Penn State Milton S Hershey Medical Center

location_facility: General University Hospital of Larissa Department of Medicine and Research Laboratory of Internal Medicine

location_facility: California Pacific Medical Center- Sutter Pacific Medical Foundation

location_facility: The Liver Institute at Methodist Dallas Medical Center

location_facility: American Research Corporation at the Texas Liver Institute

location_facility: The Newcastle Upon Tyne Hospital NHS Foundation Trust

location_facility: Hospital Universitario Virgen de la Victoria

location_facility: Hull University Teaching Hospitals NHS Trust


title: A Double-blind, Placebo-controlled, Randomized Trial to Determine the Safety and Efficacy of EMA401 in Reducing 24-hour Average Pain Intensity Score in Patients With Painful Diabetic Neuropathy (EMPADINE)

acronym: EMPADINE

summary: The purpose of this study is to evaluate safety and efficacy of EMA401 compared to placebo in patients with painful diabetic neuropathy (PDN).

ReferenceCitation: Rice ASC, Dworkin RH, Finnerup NB, Attal N, Anand P, Freeman R, Piaia A, Callegari F, Doerr C, Mondal S, Narayanan N, Ecochard L, Flossbach Y, Pandhi S. Efficacy and safety of EMA401 in peripheral neuropathic pain: results of 2 randomised, double-blind, phase 2 studies in patients with postherpetic neuralgia and painful diabetic neuropathy. Pain. 2021 Oct 1;162(10):2578-2589. doi: 10.1097/j.pain.0000000000002252.

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.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Denmark
Poland
Portugal
Spain
United Kingdom
Austria
Australia
Canada
Finland
Hungary
France
Belgium
Norway
Germany
Bulgaria
Slovakia


title: A TheraSphere® Advanced Dosimetry Retrospective Global Study Evaluation in Hepatocellular Carcinoma Treatment

acronym: TARGET

summary: This retrospective, multinational, single-arm study will be conducted in at least 8 sites. An interim analysis will be conducted with data from 100 patients with up to 10 well defined HCC tumor(s) and with at least one tumor ≥3 cm. Normal tissue absorbed dose using pre-procedural 99mTc MAA SPECT or SPECT/CT imaging will be measured to allow the mean absorbed normal tissue dose corresponding to a ≤15% probability of CTCAE grade 3 or higher hyperbilirubinemia (in the absence of disease progression) to be calculated. Total bilirubin will be recorded and graded according to CTCAE version 4.02. All dose-related SAEs at 3 months follow-up will be followed until resolution, death or lost-to-follow-up. AEs related to disease progression will not be considered related to TheraSphere.

time_perspective: Retrospective

ReferenceCitation: Lam M, Garin E, Maccauro M, Kappadath SC, Sze DY, Turkmen C, Cantasdemir M, Haste P, Herrmann K, Alsuhaibani HS, Dreher M, Fowers KD, Salem R. A global evaluation of advanced dosimetry in transarterial radioembolization of hepatocellular carcinoma with Yttrium-90: the TARGET study. Eur J Nucl Med Mol Imaging. 2022 Aug;49(10):3340-3352. doi: 10.1007/s00259-022-05774-0. Epub 2022 Apr 8.

lead_sponsor: Boston Scientific Corporation

location_country:
Saudi Arabia
Italy
Netherlands
Switzerland
United States
France
Turkey
Germany

location_facility: Northwestern Memorial Hospital, Robert H Lurie Comprehensive Cancer Center

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: University of Florida College of Medicine

location_facility: Istanbul university Istanbul medical school

location_facility: Washington University in St. Louis, School of Medicine

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Foundation IRCCS Istituto Nazionale Tumori


acronym: REFLECT

title: Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected With G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year

lead_sponsor: GenSight Biologics

location_country:
Taiwan
Spain
Italy
United Kingdom
United States
France
Belgium

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

location_facility: University of Colorado Health Eye Center

location_facility: Wills Eye Institute - Ocular Oncology Service

summary: The goal of this clinical trial is to assess the safety and efficacy of GS010, a gene therapy, in improving the retina functional & structural outcomes in subjects with LHON due to the G11778A ND4 mitochondrial mutation when vision loss duration is present up to one year.


type: Registry Identifier

title: REFINE: Regorafenib Observational Study in Hepatocellular Carcinoma

summary: Observational study to evaluate, under real-world practice conditions, the safety and effectiveness of regorafenib in patients diagnosed with unresectable hepatocellular carcinoma (uHCC)

ipd_sharing_description: Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

lead_sponsor: Bayer

location_country:
Saudi Arabia
Italy
Spain
United States
Belgium
Japan
Austria
Russian Federation
Argentina
Turkey
Sweden
Egypt
Canada
China
Denmark
Taiwan
Greece
Thailand
Korea, Republic of
France
Netherlands

location_facility: St. Joseph´s Hospital and Medical Center

location_facility: University of Louisville - Clinical Trials Unit

location_facility: C.R.Wood Cancer Center, Glen Falls Hospital

location_facility: Corporal Michael J. Crescenz VA Medical Center


title: A Phase 3 Long-term Safety Extension Study of SHP647 in Subjects With Moderate to Severe Ulcerative Colitis or Crohn's Disease (AIDA)

summary: The purpose of this study is to evaluate the safety and tolerability of long-term treatment with ontamalimab in participants with moderate to severe Ulcerative Colitis (UC) or Crohn's disease (CD)

lead_sponsor: Shire

location_country:
Croatia
Serbia
Spain
Italy
United Kingdom
Lithuania
Ukraine
United States
Belgium
Japan
Romania
South Africa
Czechia
Poland
Portugal
Austria
Australia
Russian Federation
Argentina
Israel
Turkey
Mexico
Slovakia
Bosnia and Herzegovina
Lebanon
Canada
Switzerland
Hungary
Germany
Bulgaria
Greece
Ireland
Estonia
New Zealand
Korea, Republic of
France
Colombia
Netherlands

location_facility: Washington University School of Medicine

location_facility: Szpital Uniwersytecki Nr 2 im. Dr Jana Biziela w Bydgoszczy

location_facility: Chiinkai Dojima General & Gastroenterology Clinic

location_facility: Azienda Ospedaliera Universitaria Careggi

location_facility: Fejer Megyei Szent Gyorgy Egyetemi Oktato Korhaz

location_facility: Communal Nonprofit Enterprise Vinnytsia Regional Clinical Hospital named after N.I. Pirogov VRC

location_facility: Medical Center of LLC Medical Center Dopomoga-Plus

location_facility: Centrum Medyczne Czestochowa - PRATIA - PPDS

location_facility: Szpital Zakonu Bonifratrow pw. Aniolow Strozow w Katowicach

location_facility: Treatment and Diagnostic Center "Healthy and Happy" of LLC "Healthy and Happy"

location_facility: Instituto de Investigaciones Aplicadas a la Neurociencia A.C.

location_facility: VA Long Beach Healthcare System - NAVREF - PPDS

location_facility: University Multiprofile Hospital for Active Treatment Sveta Anna

location_facility: Regional Municipal Non-profit Enterprise "Chernivtsi Regional Clinical Hospital"

location_facility: Medical Center of LLC Medical Clinic Blagomed

location_facility: Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - Panstwowy Instytut Badawczy

location_facility: Atlanta Gastroenterology Specialists, PC

location_facility: Second Multiprofile Hospital for Active Treatment Sofia

location_facility: General County Hospital Vukovar and Croatian Veterans Hospital

location_facility: Colo-Proctology Center Matsushima Clinic

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

location_facility: Azienda Ospedaliero Universitaria Di Bologna - Policlinico S Orsola Malpighi

location_facility: Omega Research Consultants LLC - Clinedge - PPDS

location_facility: Centro de Investigación Médica Aguascalientes

location_facility: Municipal Enterprise Kryvyi Rih City Clinical Hospital #2 of Kryvyi Rih City Council

location_facility: Municipal Non-profit Enterprise of Kyiv Regional Council Kyiv Regional Clinical Hospital

location_facility: Uniwersytecki Szpital Kliniczny im. Wojskowej Akademii Medycznej Centralny Szpital Weteranow

location_facility: Medizinische Universitat Wien (Medical University of Vienna)

location_facility: Municipal Nonprofit Enterprise Lviv Clinical Emergency Care Hospital

location_facility: Istanbul Universitesi Cerrahpasa Tip Fakultesi

location_facility: Niepubliczny Zakład Opieki Zdrowotnej VIVAMED

location_facility: First St. Petersburg State Medical University n.a. I.P Pavlov

location_facility: Kindred Medical Institute for Clinical Trials, LLC

location_facility: Gastromed Kopon Zmudzinski i Wspolnicy Sp.j.Specjalistyczne Centrum Gastrologii i Endoskopii Specj

location_facility: Cluj-Napoca Emergency Clinical County Hospital

location_facility: St. Elizabeth Municipal Clinical Hospital

location_facility: OM Research LLC - Lancaster - ClinEdge - PPDS

location_facility: Phylasis Clinicas Research S. de R.L. de C.V.

location_facility: Klinikum rechts der Isa der Technischen Universitaet Muenchen

location_facility: Veterans Research Foundation of Pittsburgh - NAVREF - PPDS

location_facility: Moscow Regional Research Clinical Institute Na Mfvladimirskiy

location_facility: Azienda Ospedaliero Universitaria Di Modena Policlinico

location_facility: Diagnostic and Consulting Center Aleksandrovska EOOD

location_facility: Prof. Dr. Matei Bals Institute of Infectious Diseases

location_facility: DelRicht Clinical Research, LLC - ClinEdge - PPDS

location_facility: Hospital Senhora da Oliveira - Guimaraes, E.P.E

location_facility: Ospedale Casa Sollievo Della Sofferenza IRCCS

location_facility: Krajska zdravotni, a.s. - Masarykova nemocnice v Usti nad Labem, o.z.

location_facility: Miedzyleski Szpital Specjalistyczny w Warszawie

location_facility: Centrum Medyczne A-Z Clinic Mateusz Sidor, Piotr Puc-Lekarze Spolka Partnerska

location_facility: Acibadem City Clinic University Multiprofile Hospital for Active Treatment EOOD

location_facility: Ishida Clinic of IBD and Gastroenterology

location_facility: Accel Research Sites - St. Petersburg - ERN - PPDS

location_facility: MNPE of Kharkiv Regional Council Regional Clinical Specialised Dispensary of Radiation Protection

location_facility: Twoja Przychodnia - Szczecińskie Centrum Medyczne

location_facility: The Catholic University of Korea, St. Vincent's Hospital

location_facility: University Multiprofile Hospital for Active Treatment Sv Ivan Rilski EAD

location_facility: Hospital Universitari i Politecnic La Fe de Valencia

location_facility: Hospital Privado Centro Médico de Córdoba

location_facility: Digestive Health Associates of Texas, P.A.dba DHAT Research Institute

location_facility: Niepubliczny Zaklad Opieki Zdrowotnej CENTRUM MEDYCZNE Szpital Swietej Rodziny

location_facility: Communal Non-profit Enterprise of Kyiv Regional Council Kyiv Regional Hospital

location_facility: University Multiprofile Hospital for Active Treatment - Dr. Georgi Stranski EAD

location_facility: Regional Consulting and Diagnostics Centre

location_facility: Municipal Nonprofit Enterprise of Kharkiv Regional Council Regional Clinical Hospital

location_facility: Hospital Universitario Juan Ramon Jimenez

location_facility: Elite Clinical Studies - Phoenix - Clinedge - PPDS

location_facility: Unidad de Atencion Medica e Investigacion en Salud

location_facility: Laporte County Institute For Clinical Research

location_facility: Gastrointestinal Diseases, Inc Research - IACT- HyperCore - PPDS

location_facility: Health Pharma Professional Research S.A de C.V.

location_facility: Centro de Investigacion Clinica Acelerada, S.C.

location_facility: NYU Langone Long Island Clinical Research Associates

location_facility: Communal Non-Commercial Enterprise Vinnytsia City Clinical Hospital 1

location_facility: Yonsei University Wonju Severance Christian Hospital

location_facility: Theageneio Anticancer Oncology Hospital of Thessaloniki

location_facility: Hospital of Lithuanian University of Health Sciences Kaunas Clinics

location_facility: ME Dnipropetrovsk Regional Clinical Hospital n.a. I.I Mechnykov Dnipropetrovsk Regional Council

location_facility: Nemocnice Pardubickeho kraje, a.s. Orlickoustecka nemocnice

location_facility: Inquest Clinical Research/Coastal Gastroenterology Associates, PA - TDDC - PPDS

location_facility: Multiprofile Hospital for Active Treatment Eurohospital

location_facility: CHI Franciscan Digestive Care Associates

location_facility: Hospital Universitario Fundacion Jimenez Diaz

location_facility: Veteran's Research and Education Foundation - NAVREF - PPDS

location_facility: Fondazione Policlinico Universitario A Gemelli

location_facility: MNPE City Hospital No. 6 of Zaporizhzhia City Council

location_facility: Centro Hospitalar do Algarve - Hospital de Portimao

location_facility: Specialized Hospital for Active Treatment of Pneumophthisiatric Diseases Dr.D.Gramatikov- Ruse- PPDS

location_facility: Centrum Opieki Zdrowotnej Orkan-Med Stec-Michalska Sp. J.

location_facility: Medical Center OK!Clinic+LLC International Institute of Clinical Research

location_facility: MNPE Central City Clinical Hospital of Uzhhorod City Council

location_facility: Centro Medico Teknon - Grupo Quironsalud

location_facility: Municipal Non-profit Enterprise City Emergency Care Hospital of Zaporizhzhia Regional Council

location_facility: Fondazione IRCCS Policlinico San Matteo di Pavia

location_facility: CATS Research Center - University of Arizona

location_facility: Alliance Clinical Research-(Vestavia Hills)

location_facility: Dr.Carol Davila Emergency University Central Military Hospital

location_facility: Gastroenterologische Facharztpraxis am Mexikoplatz

location_facility: Hammoud Hospital University Medical Center

location_facility: Azienda Ospedaliera Ordine Mauriziano di Torino

location_facility: Investigacion Biomedica para el Desarrollo de Farmacos S.A. de C.V.

location_facility: Shiga University of Medical Science Hospital

location_facility: LLC Medical Center Family Medicine Clinic

location_facility: Azienda Ospedaliera Mater Domini Di Catanzaro

location_facility: Edward Hines Jr VA Hospital - NAVREF - PPDS

location_facility: Hospital Universitario Virgen del Rocio - PPDS

location_facility: University Multiprofile Hospital for Active Treatment Tsaritsa Yoanna - ISUL EAD

location_facility: University General Hospital of Heraklion

location_facility: Private Healthcare Institution Clinical Hospital RZD-Medicina of Samara city

location_facility: Western General Hospital Edinburgh - PPDS

location_facility: Vilnius University Hospital Santaros Klinikos

location_facility: Diagnostic Consultative Centre Mladost - M OOD

location_facility: Szpital Specjalistyczny sw Lukasza - Oddzial Gastroenterologii

location_facility: Twoja Przychodnia - Centrum Medyczne Nowa Sol

location_facility: ENCORE Borland-Groover Clinical Research - ERN - PPDS

location_facility: La Sapienza-Università di Roma-Policlinico Umberto I

location_facility: Atria Clinical Research - Clinedge - PPDS

location_facility: IPS Centro Médico Julián Coronel S.A.S. - PPDS

location_facility: Uniwersytecki Szpital Kliniczny w Bialymstoku

location_facility: Clinic of SI National Institute of Therapy n.a. L.T. Mala of NAMS of Ukraine

location_facility: Medical Corporation Shoyu-kai Fujita Gastroenterology Hospital

location_facility: Centrum Medyczne Warszawa - PRATIA - PPDS

location_facility: Research Institute of Physiology and Basic Medicine

location_facility: Samodzielny Publiczny Szpital Wojewodzki im. Papieza Jana Pawla II

location_facility: Asthma and Allergy Associates PC - CRN - PPDS

location_facility: Municipal Non-profit Enterprise Kherson City Clinical Hospital named after Ye.Ye. Karabelesh

location_facility: State Institution "Institute of Gastroenterology of National Academy of Medical Sciences of Ukraine"

location_facility: Azienda Ospedaliera Città della Salute e della Scienza di Torino

location_facility: Bayside Clinical Research - New Port Richey

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Azienda Ospedale Università Padova - Dipartimento Salute della Donna e del Bambino

location_facility: Kitasato University Kitasato Institute Hospital

location_facility: Municipal Non-profit Enterprise Odessa Regional Clinical Hospital of Odessa Regional Council

location_facility: Kyungpook National University Chilgok Hospital

location_facility: Clinica de Higado y Gastroenterologia Integral, S.C.

location_facility: Hakodate Koseiin Hakodate Goryoukaku Hospital

location_facility: Russian Medical Military Academy n.a. S.M. Kirov

location_facility: Azienda Ospedaliera San Camillo Forlanini

location_facility: Clinical Research Center Spółka z Ograniczoną Odpowiedzialnością, Medic-R Spółka Komandytowa

location_facility: Municipal Nonprofit Enterprise CCH #2 n.a. prof. O.O. Shalimov of Kharkiv City Council

location_facility: Gastroenterology Associates of Western Michigan, PLC

location_facility: Clinical Hospital Center Bezanijska Kosa

location_facility: Fundación Cardioinfantil Instituto Cardiológico

location_facility: Toronto Digestive Disease Associates Inc

location_facility: Lviv Railway Clinical Hospital of branch Health Center of Joint Stock Co. Ukrainian Railway

location_facility: Dokkyo Medical University Saitama Medical Center

location_facility: Severance Hospital Yonsei University Health System - PPDS

location_facility: Institut Klinicke A Experimentalni Mediciny

location_facility: CHA Bundang Medical Center, CHA University

location_facility: Nizhegorodskaya Regional Clinical Hospital n.a. Semashko

location_facility: A.ö. Krankenhaus der Barmherzigen Brüder

location_facility: Centrum Diagnostyczno - Lecznicze Barska sp. z o.o.


title: A Phase Ib/II, Open-Label, Multicenter, Randomized, Umbrella Study Evaluating the Efficacy and Safety of Multiple Immunotherapy-Based Treatment Combinations in Patients With Locally Advanced Unresectable or Metastatic Gastric or Gastroesophageal Junction Cancer or Esophageal Cancer (Morpheus-Gastric and Esophageal Cancer)

summary: A Phase Ib/II, open label, multi-center, randomized study designed to assess the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of immunotherapy-based treatment combinations in patients with locally advanced unresectable or metastatic G/GEJ cancer (hereafter referred to as gastric cancer) and esophageal cancer. Two cohorts of patients with gastric cancer have been enrolled in parallel in this study: the second-line (2L) Gastric Cancer Cohort consists of patients with gastric cancer who have progressed after receiving a platinum-containing or fluoropyrimide-containing chemotherapy regimen in the first-line setting, and the first-line (1L) Gastric Cancer Cohort consists of patients with gastric cancer who have not received prior chemotherapy in this setting. In each cohort, eligible patients will be assigned to one of several treatment arms. Additionally, a cohort of patients with esophageal cancer who have not received prior systemic treatment for their disease will be enrolled in this study. Eligible patients will be randomized to chemotherapy or the combination of chemotherapy with checkpoint inhibitor immunotherapy.

lead_sponsor: Hoffmann-La Roche

location_country:
Taiwan
Spain
United Kingdom
Australia
Netherlands
Korea, Republic of
United States
Israel
France
Germany

location_facility: Mayo Clinic - Rochester; Breast Cancer Center

location_facility: Rabin MC; Davidof Center - Oncology Institute

location_facility: Hospital Universitari Vall dHebron; Oncology

location_facility: Uni of Southern California; Norris Comprehensive Cancer Ctr

location_facility: National Taiwan University Hospital (NTUH) - Cancer Research Center

location_facility: UCLA Jonsson Comprehensive Cancer Center

location_facility: Robert H. Lurie Comprehensive Cancer Center of Northwestern University

location_facility: Universidad de Navarra - Clinica Universitaria de Navarra (CUN)

location_facility: Peter MacCallum Cancer Centre; Medical Oncology

location_facility: Krankenhaus Nordwest GmbH - Institut Fuer Klinisch-Onkologische Forschung (IKF)

location_facility: Barts and The London School of Medicine and Dentistry - Barts Cancer Institute (BCI)-CECM

location_facility: Universitatsklinik Heidelberg; Universitätshautklinik und Nationales Centrum für Tumorerkrankungen

location_facility: University of Ulsan College of Medicine - Asan Medical Center (AMC) - Asan Cancer Center (ACC)

location_facility: The Catholic University of Korea St. Vincent's Hospital

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Dana-Farber Cancer Institute - Gastrointestinal Cancer Treatment Center

location_facility: Ben-Gurion University of the Negev - Soroka University Medical Center

location_facility: Seoul National University Hospital (SNUH) - Medical Oncology Center

location_facility: Froedtert and The Medical College of Wisconsin

location_facility: Universitätsklinikum Essen; Innere Klinik (Tumorforschung)

location_facility: Yonsei University College of Medicine (YUCM)-Yonsei Cancer Center; Cancer Metastasis Research Center

location_facility: Het Nederlands Kanker Instituut Antoni Van Leeuwenhoek Ziekenhuis

location_facility: The Royal Marsden NHS Foundation Trust - Royal Marsden Hospital (RMH) - Sutton

location_facility: Seoul National University Bundang Hospital


title: A Double-blind, Placebo-Controlled, Randomized Withdrawal Following Open Label Therapy Study to Assess the Safety and Efficacy of Levoketoconazole (2S, 4R-ketoconazole) in the Treatment of Endogenous Cushing's Syndrome

lead_sponsor: Cortendo AB

location_country:
Denmark
Poland
Spain
Greece
Italy
Hungary
United States
France
Israel
Romania
Netherlands
Bulgaria

location_facility: Allegheny Neuroendocrinology Center, West Penn Allegheny Health System

location_facility: General Hospital of Athens 'G. Gennimatas', Department of Endocrinology-Diabetes Centre

location_facility: Hospital Universitario Ramón y Cajal, Endocrinology and Nutrition

location_facility: General Hospital of Athens 'Laiko', "National and Kapodistrian University of Athens Medical School Pathophysiology - Endocrinology"

location_facility: University General Hospital of Ioannina, Department of Endocrinology

location_facility: AOU Policlinico G. Martino Sezione di Endocrinologia

location_facility: University Specialized Hospital for Active Treatment in Endocrinology (USHATE) I Klinika

location_facility: Policlinico Universitario Sant'Andrea, Scienze Mediche

location_facility: Maria Sklodowska-Curie Memorial Institute - Cancer Center Gliwice Branch, Nuclear Medicine and Endocrine Oncology Department

location_facility: University Multiprofile Hospital for Active Treatment "Aleksandrovska" EAD Clinic of Endocrinology and Metabolic Diseases

location_facility: Alexandovska University Hospital, Clinic of Endocrinology & Metabolic Diseases

location_facility: Oregon Health & Science University, Neurological Surgery

location_facility: Columbia University, College of P&S Medicine/Neuro-endocrine Unit

location_facility: Wojskowy Instytut Medyczny, Klinika Gastroenterologii, Endokrynologii I Chorob Wewnetrznych

location_facility: APHM Hôpital de la Conception, Service d'Endocrinologie, Diabète et Maladies Métaboliques

location_facility: Memorial Sloan Kettering Cancer Center, Endocrinology

location_facility: Hippokration General Hospital, "Endocrinology and Diabetes Department

location_facility: Tel-Aviv Sourasky Medical Center Institute of Endocrinology, Metabolism and Hypertension

location_facility: Northwestern University, Medicine - Endocrinology

location_facility: Spitalul Clinic Judetean Mures, Compartimentul de Endocrinologie

location_facility: University of Genova, Department of Internal Medicine & Medical Specialties (DiMI)

location_facility: UCLA School of Medicine, Medicine/Endocrinology Department

location_facility: Cedars-Sinai Medical Center, Cedars-Sinai Pituitary Center

location_facility: Bnai Zion Medical Center, Institute of Endocrinology

location_facility: Institutul National de Endocrinologie C.I. Parhon, Sectia Clinica de Endocrinologie II, Patologia tiroidei de corelatie

location_facility: Policlinico Agostino Gemelli, Università Cattolica del Sacro Cuore, Endocrinology

location_facility: Keck Medical Center University of Southern California HCII, Internal Medicine - Keck Hospital

location_facility: Spitalul Clinic Judetean de Urgenta Cluj-Napoca, Sectia Clinica Endocrinologie

location_facility: Leiden University, Leiden University Medical Center, Department of Endocrinology

location_facility: Johns Hopkins University, Endocrinology Department

location_facility: Clinica di Endocrinologia e malattie del Metabolismo, Dipartimento Specialità Mediche

location_facility: Spitalul Clinic Judetean de Urgenta "Pius Brinzeu", Departamentul de Endocrinologie

location_facility: Hospital Universidad De La Ribera, Endocrinologia

location_facility: Instytut Centrum Zdrowia Matki Polki, Oddział Endokrynologii i Chorób Metabolicznych

location_facility: Rabin Medical Center- Beilinson Hospital, "Institute of Endocrinology & Metabolism, Beilinson Campus"

location_facility: Rigshospitalet, Copenhagen University Hospital, Endocrinology Department

location_facility: Semmelweis Egyetem II. Belgyógyászati Klinika A.épület I.emelet ODM-labor

location_facility: University of Michigan, Comprehensive Endocrine Research, Internal Medicine - MEND

location_facility: Evangelismos Athens General Hospital, Department of Endocrinology

location_facility: AOU Federico II, Sezione di Endocrinologia (Edificio 1, Secondo Piano)

location_facility: AOU Citta della Salure e della Scienza SC Endocrinologia, Diabetologia e Metabolismo

location_facility: The Center for Diabetes and Endocrine Care

location_facility: Washington University School of Medicine, Endocrinology

location_facility: Erasmus Medical Center, Department of Internal Medicine

summary: This is a double-blind, randomized, placebo-controlled withdrawal and rescue/restoration study in subjects with endogenous Cushing's Syndrome (CS) previously treated with single-arm, open-label levoketoconazole that will assess efficacy, safety, tolerability, and pharmacokinetics of levoketoconazole.


title: A Phase 1 Dose Escalation and Cohort Expansion Study of TSR-033, an Anti-LAG-3 Monoclonal Antibody, Alone and in Combination With an Anti-PD-1 in Patients With Advanced Solid Tumors

acronym: CITRINO

summary: This is a multicenter, open-label, first-in-human Phase 1 study evaluating the anti-lymphocyte activation gene-3 (LAG-3) antibody TSR-033 alone, in combination with the anti-PD-1 antibody dostarlimab, and in combination with dostarlimab, modified folinic acid (FOL)/leucovorin, 5-fluorouracil and oxaliplatin (OX) (mFOLFOX6) or FOL/leucovorin, 5-fluorouracil and irinotecan (IRI) (FOLFIRI), and bevacizumab in participants with advanced solid tumors in a broad range of solid tumors. Participants with disease types selected for evaluation in this study are expected to derive clinical benefit with addition of an anti-PD-1. The study will be conducted in two parts with Part 1 consisting of dose escalation to determine the recommended phase 2 dose (RP2D) of TSR-033 as a single agent (Part 1a) and in combination with dostarlimab (Part 1c). RP2D decisions will be based on the occurrence of dose-limiting toxicities (DLTs), pharmacokinetics (PK), as well as pharmacodynamics (PDy) data. Part 2A of the study will investigate the anti-tumor activity of TSR-033 and dostarlimab in combination in participants with advanced or metastatic microsatellite stable colorectal cancer (MSS-CRC). Part 2B of the study will investigate the safety and anti-tumor activity of TSR-033 and dostarlimab in combination with chemotherapy (Cohort B1: mFOLFOX6 and Cohort B2: FOLFIRI) and bevacizumab in participants with advanced or metastatic MSS-CRC.

lead_sponsor: Tesaro, Inc.

location_country:
United States
France


title: OsteoCool Tumor Ablation Post-Market Study (OPuS One)

acronym: OPuS One

summary: This study evaluates the effectiveness of the Medtronic OsteoCool™ RF Ablation System.

ReferenceType: result

ReferenceCitation: Levy J, Hopkins T, Morris J, Tran ND, David E, Massari F, Farid H, Vogel A, O'Connell WG, Sunenshine P, Dixon R, Gangi A, von der Höh N, Bagla S. Radiofrequency Ablation for the Palliative Treatment of Bone Metastases: Outcomes from the Multicenter OsteoCool Tumor Ablation Post-Market Study (OPuS One Study) in 100 Patients. J Vasc Interv Radiol. 2020 Nov;31(11):1745-1752. doi: 10.1016/j.jvir.2020.07.014.

location_country:
Luxembourg
Canada
United States
France
Germany

location_facility: Hôpitaux Universitaires de Strasbourg - Nouvel Hôpital Civil

location_facility: University of Massachusetts Memorial Medical Center

location_facility: University of Texas (UT) Southwestern Medical Center

location_facility: University of North Carolina at Chapel Hill

location_facility: Banner - University Medical Center Phoenix


title: A Phase 2, Multi-center, Open Label Study of NIR178 in Combination With PDR001 in Patients With Selected Advanced Solid Tumors and Non-Hodgkin Lymphoma

location_country:
Taiwan
Spain
Italy
Austria
Australia
Netherlands
Argentina
Switzerland
United States
France
Singapore
Belgium
Japan
Germany
Czechia

location_facility: H Lee Moffitt Cancer Center and Research Institute Inc

location_facility: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

location_facility: MD Anderson Cancer Center/University of Texas

summary: The purpose of this phase 2 study is to evaluate the efficacy and safety of NIR178 in combination with PDR001 in multiple solid tumors and diffuse large B-cell lymphoma (DLBCL) and further explore schedule variations of NIR178 to optimize immune activation through inhibition of A2aR.


title: Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice

summary: The study addresses the safety, utilisation and effectiveness of Obizur in the treatment of bleeding episodes in real-life clinical practice in Europe and the United States.

ipd_sharing_description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

lead_sponsor: Baxalta now part of Shire

location_country:
United Kingdom
Italy
Austria
Netherlands
United States
France
Germany

location_facility: Azienda Ospedaliera Nazionale Santi Antonio e Biagio e Cesare Arrigo

location_facility: Universitaetsklinikum Carl Gustav Carus TU Dresden

location_facility: Policlinico Universitario Agostino Gemelli

location_facility: University Hospitals Cleveland Medical Center

location_facility: Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone

location_facility: Umberto I Pol. di Roma-Università di Roma La Sapienza

location_facility: The University of Texas Health Science Center at Houston

location_facility: Klinikum der Johann Wolfgang Goethe-Universitaet


title: Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Childhood Cerebral Adrenoleukodystrophy (CCALD)

summary: This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.

study_start_date_type: Anticipated

status: Withdrawn

lead_sponsor: NeuroVia, Inc.

location_country:
United Kingdom
Russian Federation
Australia
Argentina
Ukraine
France
Chile
Colombia

location_facility: Saint Petersburg State Pediatric Medical University

location_facility: Great Ormond Street Hospital for Children

location_facility: National Children's Specialized Hospital 'OKHMATDET'

location_facility: Moscow Morozov's Children Clinical Hospital

location_facility: Hospital General de ninos Pedro de Elizalde


title: Clinical and Basic Investigations Into Phosphomannomutase Deficiency (PMM2-CDG)

summary:
Clinical and Basic Investigations into Phosphomannomutase deficiency (PMM2-CDG)

This is a natural history (observational) protocol designed to collect clinical and biological information in patients with PMM2-CDG (CDG-Ia).

ReferenceCitation: Čechová A, Honzík T, Edmondson AC, Ficicioglu C, Serrano M, Barone R, De Lonlay P, Schiff M, Witters P, Lam C, Patterson M, Janssen MCH, Correia J, Quelhas D, Sykut-Cegielska J, Plotkin H, Morava E, Sarafoglou K. Should patients with Phosphomannomutase 2-CDG (PMM2-CDG) be screened for adrenal insufficiency? Mol Genet Metab. 2021 Aug;133(4):397-399. doi: 10.1016/j.ymgme.2021.06.003. Epub 2021 Jun 11.

lead_sponsor: Glycomine, Inc.

location_country:
Poland
Portugal
Spain
Italy
France
United States
Belgium
Netherlands
Czechia

location_facility: Children's Hospital of Philadelphia (CHOP)

location_facility: Mother and Child Institute (Instytut Matki i Dziecka)

location_facility: Radboud University Nejmegen Medical Center


title: Micra Atrial Tracking Using a Ventricular Accelerometer Study

acronym: MARVEL

summary:
The purpose of this study is to characterize performance of the MARVEL algorithm downloaded into the MicraTM TPS to provide atrial synchronous ventricular pacing in subjects with AV block.

A sub-study will be conducted within the Marvel study, the purpose of this study is to characterize the MARVEL algorithm in patients who were enrolled in the MARVEL study, and to collect and compare the accelerometer signals and AV synchrony at a second point in time.

primary_purpose: Device Feasibility

ReferenceCitation: Chinitz L, Ritter P, Khelae SK, Iacopino S, Garweg C, Grazia-Bongiorni M, Neuzil P, Johansen JB, Mont L, Gonzalez E, Sagi V, Duray GZ, Clementy N, Sheldon T, Splett V, Stromberg K, Wood N, Steinwender C. Accelerometer-based atrioventricular synchronous pacing with a ventricular leadless pacemaker: Results from the Micra atrioventricular feasibility studies. Heart Rhythm. 2018 Sep;15(9):1363-1371. doi: 10.1016/j.hrthm.2018.05.004. Epub 2018 May 11.

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
Denmark
Spain
Italy
Austria
United States
France
Belgium
Malaysia
Czechia

location_facility: Azienda Ospedaliero-Universitaria Pisana - Stabilimento di Cisanello

location_facility: Kepler Universitätsklinikum Med Campus III

location_facility: Hospital Universitari Clínic de Barcelona


title: Multi-Center Clinical Evaluation of the ATTUNE Revision System in Complex Primary Total Knee Arthroplasty

summary: Five-year, Prospective, multi-center, non-randomized, non-controlled study of the ATTUNE® Revision total knee prostheses, utilizing the fixed bearing (FB) and rotating platform (RP) tibial component with the posterior stabilizing (PS) femoral component in complex primary total knee arthroplasty.

lead_sponsor: DePuy Orthopaedics

location_country:
United Kingdom
Ireland
Austria
Australia
Italy
Canada
Netherlands
New Zealand
Switzerland
United States
France
Belgium
Germany

location_facility: Kantonsspital Baselland (Bruderholz, Liestal, Laufen), Department of Orthopaedic Surgery and Traumatology

location_facility: Asklepios Orthopädische Klinik Lindenlohe

location_facility: Klinik und Poliklinik für Orthopädie und Sportorthopädie am Klinikum rechts der Isar der Technischen Universität Münche

location_facility: London Health Sciences Centre University Hospital

location_facility: The Ohio State University Wexner Medical Center

location_facility: University of Mississippi Medical Center

location_facility: Krankenhaus der Barmherzigen Schwestern Linz Betriebsgesellschaft m.b.H.

location_facility: The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust

location_facility: Hampshire Hospitals NHS Foundation Trust

location_facility: Centre Hospitalier Universitaire de Rennes


title: Rate of Progression in USH2A-related Retinal Degeneration

summary: The overall goal of this project funded by the Foundation Fighting Blindness is to characterize the natural history of disease progression in patients with USH2A related retinal degeneration associated with congenital hearing loss (Usher syndrome type 2a) or non-syndromic retinitis pigmentosa (RP39).

ipd_sharing_description: A de-identified database is available upon request through the public domain on the FFB/Jaeb public website.

lead_sponsor: Jaeb Center for Health Research

location_country:
United Kingdom
Netherlands
Canada
United States
France
Germany

location_facility: Kellogg Eye Center, University of Michigan

location_facility: Centre hospitalier National d'Ophtalmologie des Quinze-Vingts


title: Assessment of the Carillon® Mitral Contour System® in Treating Heart Failure With at Least Mild Functional Mitral Regurgitation

summary: The objective of this prospective, randomized, blinded clinical trial is to assess the safety and efficacy of the CARILLON Mitral Contour System in treating heart failure with at least mild functional regurgitation.

ReferenceCitation: Schofer J, Siminiak T, Haude M, Herrman JP, Vainer J, Wu JC, Levy WC, Mauri L, Feldman T, Kwong RY, Kaye DM, Duffy SJ, Tübler T, Degen H, Brandt MC, Van Bibber R, Goldberg S, Reuter DG, Hoppe UC. Percutaneous mitral annuloplasty for functional mitral regurgitation: results of the CARILLON Mitral Annuloplasty Device European Union Study. Circulation. 2009 Jul 28;120(4):326-33. doi: 10.1161/CIRCULATIONAHA.109.849885. Epub 2009 Jul 13.

ReferenceCitation: Siminiak T, Wu JC, Haude M, Hoppe UC, Sadowski J, Lipiecki J, Fajadet J, Shah AM, Feldman T, Kaye DM, Goldberg SL, Levy WC, Solomon SD, Reuter DG. Treatment of functional mitral regurgitation by percutaneous annuloplasty: results of the TITAN Trial. Eur J Heart Fail. 2012 Aug;14(8):931-8. doi: 10.1093/eurjhf/hfs076. Epub 2012 May 21.

ReferenceCitation: Lipiecki J, Siminiak T, Sievert H, Müller-Ehmsen J, Degen H, Wu JC, Schandrin C, Kalmucki P, Hofmann I, Reuter D, Goldberg SL, Haude M. Coronary sinus-based percutaneous annuloplasty as treatment for functional mitral regurgitation: the TITAN II trial. Open Heart. 2016 Jul 8;3(2):e000411. doi: 10.1136/openhrt-2016-000411. eCollection 2016.

lead_sponsor: Cardiac Dimensions, Inc.

location_country:
Poland
United Kingdom
Greece
United States
France

location_facility: Lindner Research Center at the Christ Hospital

location_facility: New York Presbyterian- Weill Cornell Medical College

location_facility: Dignity Health Research Institute at Mercy Gilbert and Chandler Regional Medical Centers

location_facility: Vassar Brothers- Hudson Valley Cardiovascular Practice


title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate the Efficacy and Safety of Elafibranor at Doses of 80 mg and 120mg After 12 Weeks of Treatment in Patients With Primary Biliary Cholangitis and Inadequate Response to Ursodeoxycholic Acid

lead_sponsor: Genfit

ReferenceCitation: Schattenberg JM, Pares A, Kowdley KV, Heneghan MA, Caldwell S, Pratt D, Bonder A, Hirschfield GM, Levy C, Vierling J, Jones D, Tailleux A, Staels B, Megnien S, Hanf R, Magrez D, Birman P, Luketic V. A randomized placebo-controlled trial of elafibranor in patients with primary biliary cholangitis and incomplete response to UDCA. J Hepatol. 2021 Jun;74(6):1344-1354. doi: 10.1016/j.jhep.2021.01.013. Epub 2021 Jan 21.

location_country:
United Kingdom
Spain
United States
France
Germany

location_facility: The Newcastle Upon Tyne Hosptials NHS Foundation Trust

location_facility: University of Birmingham Centre for Liver Research

location_facility: Clinic for Gastroenterology and Hepatology

summary: The primary objective of the study is to compare the effect of daily oral administration of elafibranor 80mg and 120 mg on change in serum alkaline phosphatase (ALP) to that of placebo in patients with PBC and inadequate response to Ursodeoxycholic acid (UDCA).


title: A 52-Week Open-Label Extension Study of Pimavanserin for the Treatment of Agitation and Aggression in Subjects With Alzheimer's Disease

summary: To evaluate the safety and tolerability of pimavanserin over 52 weeks of treatment in subjects with probable AD who have symptoms of agitation and aggression

lead_sponsor: ACADIA Pharmaceuticals Inc.

location_country:
United Kingdom
Spain
United States
Chile
France

location_facility: Greater Manchester Mental Health NHS Foundation Trust

location_facility: Parkinson's Disease and Movement Disorders Center of Boca Raton

location_facility: ANI Neurology, PLLC dba Alzheimer's Memory Center

location_facility: Memory Enhancement Center of America, Inc.

location_facility: West London Cognitive Disorders Treatment & Research Unit, Lakeside Mental Health Unit, West Middlesex University Hosp. Site

location_facility: CHU de Toulouse - Cite de la sante - Gerontople

location_facility: RICE-The Research Institute for the Care of Older People, The RICE Centre, The Royal United Hospital

location_facility: Pharmaceutical Research Associates, Inc.


title: An Open-Label Study to Evaluate the Safety And Efficacy of Sofosbuvir/Velpatasvir/Voxilaprevir Fixed-Dose Combination for 12 Weeks in Subjects Who Participated in a Prior Gilead-Sponsored HCV Treatment Study

summary: The primary objectives of this study are to determine the efficacy, safety, and tolerability of treatment with sofosbuvir/velpatasvir/voxilaprevir (SOF/VEL/VOX) fixed-dose combination (FDC) for 12 weeks in participants with chronic hepatitis C virus (HCV) infection with or without cirrhosis, who did not achieve sustained viral response (SVR) after receiving prior treatment in a Gilead-sponsored HCV treatment study of direct-acting antiviral (DAA)-containing regimens.

ReferenceCitation: Ruane P, Strasser SJ, Gane EJ, Hyland RH, Shao J, Dvory-Sobol H, et al. Retreatment with Sofosbuvir/Velpatasvir/Voxilaprevir for 12 weeks is safe and effective for patients who have previously received Sofosbuvir/Velpatasvir or Sofosbuvir/Velpatasvir/Voxilaprevir [Abstract LBO-06]. 16th International Symposium on Viral Hepatitis and Liver Diseases (ISVHLD); 2018 14-17 June; Toronto, Canada

ipd_sharing_description: Qualified external researchers may request IPD for this study after study completion. For more information, please visit our website at https://www.gilead.com/about/ethics-and-code-of-conduct/policies.

lead_sponsor: Gilead Sciences

location_country:
United Kingdom
Australia
New Zealand
Canada
United States
France
Germany

location_facility: New Orleans Center for Clinical Research

location_facility: Toronto Centre for Liver Disease (TCLD), Toronto General Hospital

location_facility: Centre Hospitalier Universitaire de Rouen

location_facility: Saint Louis University, Gastroenterology & Hepatology, Clinical Research Unit

location_facility: Emory Hospital Midtown Infectious Disease Clinic

location_facility: Columbia University Medical Center/ New York Presbyterian

location_facility: University of Washington/Harborview Medical Center

location_facility: Christchurch Clinical Studies Trust, Ltd

location_facility: University of Colorado Denver (Leprino Building)


title: TAME Cardiac Arrest Trial: Targeted Therapeutic Mild Hypercapnia After Resuscitated Cardiac Arrest: A Phase III Multi-Centre Randomised Controlled Trial

acronym: TAME

summary: The TAME Cardiac Arrest trial will study the ability of higher arterial carbon dioxide (PaCO2) levels to reduce brain damage, comparing giving patients 'normal' to 'slightly higher than normal' blood PaCO2 levels and assessing their ability to return to normal life-tasks. It will be the largest trial ever conducted in heart attack patients in the intensive care unit. This therapy is cost free and, if shown to be effective, will improve thousands of lives, transform clinical practice, and yield major savings.

ReferenceCitation: Eastwood GM, Schneider AG, Suzuki S, Peck L, Young H, Tanaka A, Mårtensson J, Warrillow S, McGuinness S, Parke R, Gilder E, Mccarthy L, Galt P, Taori G, Eliott S, Lamac T, Bailey M, Harley N, Barge D, Hodgson CL, Morganti-Kossmann MC, Pébay A, Conquest A, Archer JS, Bernard S, Stub D, Hart GK, Bellomo R. Targeted therapeutic mild hypercapnia after cardiac arrest: A phase II multi-centre randomised controlled trial (the CCC trial). Resuscitation. 2016 Jul;104:83-90. doi: 10.1016/j.resuscitation.2016.03.023. Epub 2016 Apr 7.

lead_sponsor: Australian and New Zealand Intensive Care Research Centre

location_country:
Denmark
Saudi Arabia
Ireland
Italy
Australia
New Zealand
Finland
Slovenia
United Kingdom
Sweden
France
Belgium
Norway
Netherlands

location_facility: Cliniques Universitaires de Bruxelles Hospital Erasme


title: Phase 1 Study of Lorlatinib (PF-06463922), an Oral Small Molecule Inhibitor of ALK/ROS1, for Patients With ALK-Driven Relapsed or Refractory Neuroblastoma

summary: Lorlatinib is a novel inhibitor across ALK variants, including those resistant to crizotinib. In this first pediatric phase 1 trial of lorlatinib, the drug will be utilized as a single agent and in combination with chemotherapy in patients with relapsed/refractory neuroblastoma. The dose escalation phase of this study (Cohort A1) uses a traditional Phase I 3+3 design. Once a recommended phase 2 pediatric dose is identified, an expansion cohort of 6 patients (Cohort B1), within which ALKi naïve patients will be prioritized, will be initiated. Parallel cohorts will be initiated in adults or patients with large BSA (Cohort A2) and in combination with chemotherapy upon establishing RP2D (Cohort B2).

ReferenceCitation: Baranowska-Kortylewicz J, Kortylewicz ZP, McIntyre EM, Sharp JG, Coulter DW. Multifarious Functions of Butyrylcholinesterase in Neuroblastoma: Impact of BCHE Deletion on the Neuroblastoma Growth In Vitro and In Vivo. J Pediatr Hematol Oncol. 2022 Aug 1;44(6):293-304. doi: 10.1097/MPH.0000000000002285. Epub 2021 Sep 6.

lead_sponsor: New Approaches to Neuroblastoma Therapy Consortium

location_country:
United Kingdom
United States
Canada
France

location_facility: Children's Hospital and Regional Medical Center - Seattle

location_facility: Childrens Hospital Boston, Dana-Farber Cancer Institute.

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: UCSF Helen Diller Family Comprehensive Cancer Center

location_facility: University of Chicago, Comer Children's Hospital