title: A Double-blind, Placebo-controlled, Relapse Prevention Study of Pimavanserin for the Treatment of Hallucinations and Delusions Associated With Dementia-related Psychosis

lead_sponsor: ACADIA Pharmaceuticals Inc.

ReferenceCitation: Cummings JL, Ismail Z, Dickerson BC, Ballard C, Grossberg G, McEvoy B, Foff E, Atri A. Development and assessment of a brief screening tool for psychosis in dementia. Alzheimers Dement (Amst). 2021 Dec 7;13(1):e12254. doi: 10.1002/dad2.12254. eCollection 2021. Erratum in: Alzheimers Dement (Amst). 2022 Feb 09;14(1):e12290.

ReferenceCitation: Tariot PN, Cummings JL, Soto-Martin ME, Ballard C, Erten-Lyons D, Sultzer DL, Devanand DP, Weintraub D, McEvoy B, Youakim JM, Stankovic S, Foff EP. Trial of Pimavanserin in Dementia-Related Psychosis. N Engl J Med. 2021 Jul 22;385(4):309-319. doi: 10.1056/NEJMoa2034634.

location_country:
Poland
Serbia
Spain
Italy
United Kingdom
Ukraine
France
United States
Chile
Germany
Bulgaria
Slovakia
Czechia

location_facility: IRCCS Fondazione Santa Lucia, Dipartimento di Neurologia e Psichiatria

location_facility: Clinical center of Serbia, Clinic for Neurology

location_facility: NZOZ Neuro-Kard Ilkowski i Partnerzy Spółka Partnerska Lekarzy

location_facility: Psychiatric Clinic, Military Medical Academy

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

location_facility: Poltava Regional Clinical Psychiatric Hospital named after O.F. Maltsev

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

location_facility: Azienda Ospedaliera di Padova Clinica Neurologica

location_facility: Clinical Hospital Center Dr Dragisa Misovic-Dedinje

location_facility: Municipal Institution "Odesa Regional Medical Center of Mental Health", Department #18

location_facility: Memory Enhancement Center of America, Inc.

location_facility: Premier Clinical Research Institute, Inc.

location_facility: Neuro-Behavioral Clinical Research, Inc.

location_facility: Assistance Publique Hopitaux de Marseille (AP-HM) - Hopital de La Timone - Service de Neurologie et Pathologie du Mouvement du Pr Azulay

location_facility: Klinik für Psychiatrie und Psychotherapie der Universität Tübingen

location_facility: Clinic for Psychiatry, Clinical Center Kragujevac

location_facility: Communal Institution "Dnipropetrovsk Regional Clinical Hospital n.a. I. I. Mechnikov"

location_facility: Lviv Regional State Clinical Psychiatric Hospital

location_facility: Military Medical Academy, Clinic for Neurology

location_facility: Municipal Institution of Health Care "Kharkiv Regional Clinical Psychiatric Hospital #3"

location_facility: Universita degli Studi di ROMA "La Sapienza" Dipartimento di NEUROLOGIA E PSICHIATRIA

location_facility: Millennium Psychiatric Associates, LLC; DBA Millennium Center for Clinical Research

location_facility: Department of addictive disorders of the Clinic for Psychiatry, Clinical center Kragujevac

location_facility: Municipal Institution "Odesa Regional Psychiatric Hospital #2", Female Gerontological Department # 5, Male Gerontological Department #1

location_facility: Municipal Institution "Zaporizhzhya Regional Clinical Hospital of Zaporizhzhya Regional Council"

location_facility: Visionary Investigators Network (Aventura Neurologic Associates)

location_facility: Visionary Investigators Network (First Choice Neurology Group)

location_facility: University of Kansas Medical Center Research Institute, Inc.

location_facility: Centre de Recherche du Gerontopole - CHU de Toulouse

location_facility: MUDr. Beata Dupejova, neurologicka ambulancia s.r.o

location_facility: Municipal Institution "Vinnytsya Regional Psychoneurological Hospital n.a. Acad. O.I.Yushchenko", Male Department #14, Female Department #15, Vinnytsya National Medical University n.a. M.I.Pyrogov, Department of Psychiatry, Narcology and Psychotherapeutic

location_facility: Azienda Ospedaliero-Universitaria Citta della Salute a della Scienza di Torino - c/o Presidio Ospedaliero Molinette Clinica Neurologica I

location_facility: State Institution "Institute of Neurology, Psychiatry, and Narcology of the National Academy of Medical Sciences of Ukraine", Department of Clinical, Social, and Paediatric Psychiatry

summary: The purpose of this study is to evaluate the efficacy of pimavanserin compared to placebo in preventing relapse of psychotic symptoms in subjects with dementia-related psychosis who responded to 12 weeks of open label pimavanserin treatment.


title: A Phase 2 Study of Poziotinib in Patients With Non-Small Cell Lung Cancer (NSCLC), Locally Advanced or Metastatic, With EGFR or HER2 Exon 20 Insertion Mutation (ZENITH20)

summary: This is a Phase 2, open-label, multi-center study to evaluate the efficacy and the safety/tolerability of poziotinib in seven patient cohorts for up to 603 previously treated and treatment-naïve NSCLC patients. Cohorts 3 and 4 were added with Amendment 1 and three additional cohorts were added with Amendment 2 (Cohorts 5, 6 and 7).

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

study_type: Interventional

primary_purpose: Treatment

enrollment_type: Anticipated

ReferenceType: derived

ReferenceCitation: Le X, Cornelissen R, Garassino M, Clarke JM, Tchekmedyian N, Goldman JW, Leu SY, Bhat G, Lebel F, Heymach JV, Socinski MA. Poziotinib in Non-Small-Cell Lung Cancer Harboring HER2 Exon 20 Insertion Mutations After Prior Therapies: ZENITH20-2 Trial. J Clin Oncol. 2022 Mar 1;40(7):710-718. doi: 10.1200/JCO.21.01323. Epub 2021 Nov 29.

ipd_sharing: No

lead_sponsor: Spectrum Pharmaceuticals, Inc

location_country:
Spain
Italy
Canada
United States
France
Israel
Belgium
Netherlands

location_facility: The Bond & Steele Clinic, P.A. dba Bond Clinic, P.A.

location_facility: Montefiore Einstein Medical Center for Cancer Care

location_facility: Johns Hopkins Sidney Kimmel Comprehensive Cancer Center

location_facility: Oncology Physician's Network Inc./OPN Healthcare

location_facility: Yale University, Yale Cancer Center Smilow Cancer Hospital at Yale

location_facility: Oklahoma Cancer Specialists and Research Institute, LLC

location_facility: CTCA - Midwestern Regional Medical Center

location_facility: H. Lee Moffitt Cancer Center & Research Institute

location_facility: University Hospital Germans Trias i Pujol, Department of Medical Oncology

location_facility: UCSF Helen Diller Comprehensive Cancer Center at Mt Zion

location_facility: CTCA - Southeastern Regional Medical Center

location_facility: North Shore Hematology Oncology Associates P.C. DBA NY Cancer and Blood Specialists

location_facility: Gustave Roussy Oncology Institute, Department of Medical Oncology

location_facility: The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

location_facility: Los Angeles Hematology Oncology Medical Group

location_facility: National Cancer Institute, IRCCS, Department of Medical Oncology

location_facility: National Cancer Institute Regina Elena, IRCCS, Operative Unit of Medical Oncology A 1

location_facility: Ambroise Pare University Hospital Center

location_facility: North Shore Hematology Oncology Associates DBA New York Cancer and Blood Specialists

location_facility: Hopital Larrey, CHU Toulouse, Unité d'Oncologie des Voies Respiratoires

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Ongoing


title: An Open-label, Non-randomised, Multicentre Study to Allow Continued Access to and Assess the Safety and Tolerability of AZD1775 for Patients Enrolled in AZD1775 Clinical Pharmacology Studies

summary: Open-label, non-randomised study to provide continued access to AZD1775 for patients with advanced solid tumours who have previously completed an AZD1775 clinical pharmacology study and to investigate the safety of AZD1775.

status: Completed

study_completion_date_type: Actual

enrollment_type: Actual

lead_sponsor: AstraZeneca

location_country:
Netherlands
United States
United Kingdom
France

status_simplified: Completed


title: A Phase 3, Randomized, Open-Label, Crossover Study of ASTX727 (Cedazuridine and Decitabine Fixed-Dose Combination) Versus IV Decitabine in Subjects With Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), and Acute Myeloid Leukemia (AML)

summary: Multicenter, randomized, open-label, crossover PK study of ASTX727 versus IV decitabine. Adult subjects who are candidates to receive IV decitabine will be randomized 1:1 to receive the ASTX727 tablet Daily×5 in Cycle 1 followed by IV decitabine 20 mg/m^2 Daily×5 in Cycle 2, or the converse order. After completion of PK studies during the first 2 treatment cycles, subjects will continue to receive treatment with ASTX727 from Cycle 3 onward (in 28-day cycles) until disease progression, unacceptable toxicity, or the subject discontinues treatment or withdraws from the study.

status: Active, not recruiting

design_allocation: Randomized

lead_sponsor: Astex Pharmaceuticals, Inc.

location_country:
Spain
Italy
Austria
United Kingdom
Canada
Hungary
United States
France
Germany
Czechia

location_facility: Hospital Universitario Virgen de las Nieves

location_facility: Charleston Hematology Oncology Associates

location_facility: Azienda Ospedaliera Nazionale SS. Antonio e Biagio e C. Arrigo

location_facility: Azienda Ospedaliero-Universitaria Maggiore della Carità Novara

location_facility: Fondazione IRCCS C Granda OM Policlinico

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: Hospital Universitari I Politècnic La Fe

location_facility: Fakultni Nemocnice Kralovske Vinohrady FNKV

location_facility: University of Pecs, 1st Department of Internal Medicine

location_facility: Queen Elizabeth II (QEII) Health Sciences Center

location_facility: University of Pittsburgh Hillman Cancer Center

location_facility: Princess Margaret Cancer Center - University Health Network

location_facility: Oberärztin für Innere Medizin, Hämato-/Onkologie und Palliativmedizin

location_facility: AOUC Azienda Ospedaliero-Universitaria Careggi

location_facility: Cancer & Hematology Centers of Western Michigan

location_facility: Hospital Universitario Central de Asturias

location_facility: Centre Intégré Universitaire de Santé et de Services Sociaux (CIUSSS) de l'Est-de-l'Ile-de-Montréal - Hôpital Maisonneuve Rosemont

location_facility: University of Texas Southwestern Medical Center

location_facility: Baylor Scott & White University Medical Center

location_facility: ULSS 8 Vicenza - Ospedale San Bortolo di Vicenza

location_facility: Indiana Blood and Marrow Transplantation

location_facility: Philipps-Universität Marburg, Klinik für Innere medizin, Hämatologie, Onkologie und Immunologie

location_facility: Centre de lutte contre le Cancer Leon Berard

location_facility: Compassionate Cancer Care Research Group


ipd_sharing: Yes

primary_purpose: Supportive Care

title: Long-term Safety Follow-up of Subjects With Giant Cell Tumor of Bone Treated With Denosumab in Study 20062004

lead_sponsor: Amgen

type: EudraCT Number

source: euctr

design_allocation: Non-Randomized

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

location_country:
Poland
Spain
Italy
United Kingdom
Australia
United States
France
Sweden

location_facility: University of Minnesota Medical Center Fairview

location_facility: Washington Cancer Institute at MedStar Washington Hospital

location_facility: Narodowy Instytut Onkologii im Marii Sklodowskiej-Curie â€" Panstwowy Instytut Badawczy

location_facility: Abramson Cancer Center at Pennsylvania Hospital

summary: Study 20140114 will continue to follow subjects with GCTB who were treated in Study 20062004 and remained on the study at the completion of Study 20062004 for an additional 5 years on long-term safety follow up.


title: A Randomized, Subject- and Investigator-blinded, Placebo Controlled, Multi-center, Multiple Dose Study to Assess the Efficacy and Safety of CJM112 in Patients With Inadequately Controlled Moderate to Severe Asthma

summary: An unmet medical need exists for patients with moderate and severe asthma who continue to demonstrate symptoms despite being on standard of care medications, and are not eligible for other biologic therapies developed or in development for T2-high(allergic/eosinophilic) asthma. The purpose of this study was to determine if CJM112, an anti-IL-17A antibody, displayed the clinical efficacy and safety profile to support further development in patients with inadequately controlled moderate to severe asthma with low IgE and low circulating eosinophil levels.

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
Argentina
United States
France
Israel
Belgium
Germany
Slovakia

intervention_type: Other


status: Terminated

acronym:
Diabetic Gastroparesis Study 1

 Étude de la gastroparésie diabétique 1

title: A 12-week, Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Safety and Efficacy of Relamorelin in Patients With Diabetic Gastroparesis

lead_sponsor: Allergan

location_country:
Poland
Spain
Australia
Thailand
Ukraine
Korea, Republic of
United States
India
France
Israel
Singapore
Philippines
Malaysia
Bulgaria

location_facility: M.V. Hospital for Diabetes & Diabetes Research Centre

location_facility: Indiana University Health University Hospital

location_facility: University of North Carolina at Chapel Hill

location_facility: Limited Liability Company "Medical Center "Salutem"

location_facility: Kyiv Railway hospital on the railway transport #2 of the branch "health

location_facility: Clinical Endocrinology of SI "V.Danilevsky Institute for endocrine pathology problems National Academy of Medical sciences of Ukraine"

location_facility: Centrum Badan Klinicznych Piotr Napora Lekarze Spolka Partnerska

location_facility: Vinnytsia Regional Clinical highly specialized Endocrinology Centre

location_facility: Kumudini Devi Diabetes Research Center; Ramdevrao Hospital

location_facility: West Visayas State University Medical Center

location_facility: Sanggye Paik Hospital, Inje University College of Medicine

location_facility: Family Practice Center of Wadsworth, Inc.

location_facility: Kyiv municipal clinical endocrinological center

location_facility: Municipal nonprofit entity of Kharkiv municipal council

location_facility: B. J. Government Medical College and Sassoon General Hospitals

location_facility: NZOZ Vita Diabetica - Malgorzata Buraczyk

location_facility: Royal Adelaide Hospital - Central Adelaide Local Health Network Incorporated

location_facility: Gastroenterology Associates of Western Michigan

location_facility: Polyclinic of medical services and rehabilitation department of State Joint-Stock Holding Company Artem, day-patient unit

location_facility: State Institution Ukrainian State Research Institute of Medical and Social Problems of Disability of the Ministry of Health of Ukraine

location_facility: Hospital Universitario Principe de Asturias

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

location_facility: Limited Liability Company "Medical and Diagnostic Center "ADONIS Plus", outpatient department, c. Kyiv

location_facility: Regional Public Organization Chernivtsi Regional Endocrinology Center

location_facility: Municipal nonprofit entity "Vinnytsia's city clinical hospital #1", c. Vinnytsia

location_facility: Synexus Polska Sp. z o.o.Oddzial w Poznaniu

location_facility: Odessa Railway Clinical Hospital of Branch of HC JSC Ukrzaliznytsia, Odessa National Medical University

location_facility: Long Island Gastrointestinal Research Group LLP

location_facility: Specjalistyczny Gabinet Neurologiczny Marta Banach

location_facility: Digestive Health Specialist of the South East

location_facility: Ivano-Frankivsk Central City Clinical Hospital

location_facility: Synexus Polska sp.z.o.o oddzial Czestochowa

location_facility: Regional municipal institution "Chernivtsi's regional clinical hospital", gastroenterological department, Higher state educational establishment of Ukraine "Bukovinian state medical university", department of internal medicine and infectious diseases, c.C

location_facility: Synexus Clinical Research, US, Santa Rosa

location_facility: S R Kalla (SRK) Memorial Gastro & General Hospital

location_facility: Communal Institution "Odesa Regional Clinical Hospital", Out-patient department

location_facility: Wojewodzki Szpital Specjalistyczny w Olsztynie

location_facility: Frederick Gastroenterology Associates, PA an Elligo Health Research Site

location_facility: 6th City Clinical Hospital, c. Zaporizhzhia

location_facility: Centrum Medyczne Pratia S.A. Warsaw, Poland

location_facility: Ivano-Frankivsk National Medical University

location_facility: Dr. Jivraj Mehta Smarak Health Foundation Bakeri Medical Research Centre - Gasterentrology

location_facility: Medical Center Asklepion - Humane Medicine Research EOOD

location_facility: Municipal Institution City Hospital No. 7

location_facility: New Orleans Research Institute - Metropolitan Gastroenterology Associates

location_facility: Premier Clinical Research d.b.a. STAT Research

location_facility: University of Iowa Hospitals and Clinics

location_facility: Communal Institution Kherson City Clinical Hospital

location_facility: Kindred Medical Institute for Clinical Trials, LLC

location_facility: Private Small-Scale Enterprise Medical Centre Pulse

location_facility: Connecticut Clinical Research Foundation

location_facility: Diacon Hospital and research Center - Diabetology

location_facility: Family Medical Associates, Research Department

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

location_facility: San Juan De Dios Educational Foundation, Inc.

location_facility: Synexus Polska Sp. z o.o. Oddzial w Warszawie

location_facility: Faculty of Medicine, Siriraj Hospital, Mahidol University

location_facility: Clinical Applications Laboratories, Inc.

location_facility: Synexus Polska Sp. z o.o. Oddzial w Lodzi

location_facility: Medical Research Center of Connecticut, LLC

summary: This study will evaluate the safety and efficacy of relamorelin compared to placebo in participants with diabetic gastroparesis. Participants will report daily severity scores of their diabetic gastroparesis symptoms.

status_simplified: Unknown


title: International BPA Registry

summary: The International Balloon Pulmonary Angioplasty (BPA) Registry is a prospective, multi-center, long-term observational project. Scheduled to start data collection in Q4 2017, the registry will run for approximately four years with a follow-up time for each patient of at least two years. Its primary objective is to investigate the efficacy and safety of BPA intervention in patients with chronic thromboembolic pulmonary hypertension (CTEPH) not amenable to pulmonary endarterectomy (PEA).

study_type: Observational

time_perspective: Prospective

lead_sponsor: International CTEPH Association

location_country:
Poland
United Kingdom
Austria
Netherlands
France
United States
Belgium
Japan
Germany
Czechia

location_facility: National Cerebral and Cardiovascular Center

location_facility: Centre Hospitalier Universitaire Grenoble-Alpes

location_facility: National Hospital Organization Okayama Medical Center

intervention_type: Procedure


title: Multiple Cardiac Sensors for the Management of Heart Failure

acronym: MANAGE-HF

summary: The MANAGE-HF study is a multi-center, global, prospective, open label, multi-phase trial intended to evaluate the clinical efficacy of the HeartLogic heart failure diagnostic feature.

primary_purpose: Diagnostic

ReferenceCitation: Hernandez AF, Albert NM, Allen LA, Ahmed R, Averina V, Boehmer JP, Cowie MR, Chien CV, Galvao M, Klein L, Kwan B, Lam CSP, Ruble SB, Stolen CM, Stein K; MANAGE-HF Study. Multiple cArdiac seNsors for mAnaGEment of Heart Failure (MANAGE-HF) - Phase I Evaluation of the Integration and Safety of the HeartLogic Multisensor Algorithm in Patients With Heart Failure. J Card Fail. 2022 Aug;28(8):1245-1254. doi: 10.1016/j.cardfail.2022.03.349. Epub 2022 Apr 21.

ReferenceCitation: Heggermont WA, Van Bockstal K. Heartlogic(TM): ready for prime time? Expert Rev Med Devices. 2022 Feb;19(2):107-111. doi: 10.1080/17434440.2022.2038133. Epub 2022 Feb 12.

lead_sponsor: Boston Scientific Corporation

location_country:
Germany
United States
France

location_facility: University of Southern California Hospital

location_facility: Southeast Texas Clinical Research Center

location_facility: Lindner Center for Research and Education at Christ Hosp

location_facility: Penn State Milton S Hershey Medical Center

location_facility: Cardiology Associates of Northeast Arkansas

location_facility: Strong Memorial Hospital of the University of Rochester

intervention_type: Device


type: Other Identifier

title: A Pivotal Study of Derazantinib in Patients With Inoperable or Advanced Intrahepatic Cholangiocarcinoma and FGFR2 Gene Fusions or FGFR2 Gene Mutations or Amplifications

summary: This pivotal, open-label, single-arm study will evaluate the anti-cancer activity of derazantinib by Objective Response Rate (ORR) by central radiology review as per RECIST v1.1 in subjects with inoperable or advanced intrahepatic cholangiocarcinoma (iCCA) whose tumors harbor FGFR2 gene fusions (by FISH performed by the central laboratory) or FGFR2 gene mutations or amplifications (based on NGS testing performed or commissioned by the respective study center) and who received at least one prior regimen of systemic therapy. Subjects will be dosed orally once per day at 300 mg of derazantinib capsules.

ReferenceCitation: Mazzaferro V, El-Rayes BF, Droz Dit Busset M, Cotsoglou C, Harris WP, Damjanov N, Masi G, Rimassa L, Personeni N, Braiteh F, Zagonel V, Papadopoulos KP, Hall T, Wang Y, Schwartz B, Kazakin J, Bhoori S, de Braud F, Shaib WL. Derazantinib (ARQ 087) in advanced or inoperable FGFR2 gene fusion-positive intrahepatic cholangiocarcinoma. Br J Cancer. 2019 Jan;120(2):165-171. doi: 10.1038/s41416-018-0334-0. Epub 2018 Nov 13.

lead_sponsor: Basilea Pharmaceutica

location_country:
Spain
Ireland
Italy
United Kingdom
Canada
Switzerland
Korea, Republic of
United States
France
Belgium
Germany

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

location_facility: Abramson Cancer Center of the University of Pennsylvania

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: University of Texas MD Anderson Cancer Center

location_facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz

location_facility: The University of Texas Southwestern Medical Center

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: Humanitas Cancer Center, Istituto Clinico Humanitas IRCCS

location_facility: Winship Cancer Institute of Emory University

location_facility: Sant'Orsola-Malpighi Hospital, University of Bologna

location_facility: University College London Hospitals NHS Foundation Trust

location_facility: Memorial Sloan Kettering Cancer Center - Sidney Kimmel Center for Prostate and Urologic Cancers


title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120102 Administered Intrathecally in Patients With Huntington's Disease

lead_sponsor: Wave Life Sciences Ltd.

ReferenceCitation: Rodrigues FB, Wild EJ. Huntington's Disease Clinical Trials Corner: April 2020. J Huntingtons Dis. 2020;9(2):185-197. doi: 10.3233/JHD-200002.

ReferenceCitation: Rodrigues FB, Wild EJ. Huntington's Disease Clinical Trials Corner: February 2018. J Huntingtons Dis. 2018;7(1):89-98. doi: 10.3233/JHD-189001. Review.

location_country:
Denmark
Poland
United Kingdom
Australia
Canada
United States
France
Germany

location_facility: Institut du Cerveau et de la Moelle Epinière

location_facility: Queen Elizabeth University Hospital - PPDS

location_facility: Centre Hospitalier de l-Universite de Montreal

location_facility: Odense University Hospital and University of Southern Denmark

location_facility: University of California Davis Medical Center

location_facility: Royal Devon and Exeter Hospital NHS Trust

summary: PRECISION-HD2 is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of WVE-120102 in adult patients with early manifest Huntington's disease (HD) who carry a targeted single nucleotide polymorphism (SNP) rs362331 (SNP2).


title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120101 Administered Intrathecally in Patients With Huntington's Disease

location_country:
Denmark
Poland
United Kingdom
Australia
Canada
France
Germany

location_facility: Center Hospitalier de l'Universite de Montreal

summary: PRECISION-HD1 is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of WVE-120101 in adult patients with early manifest Huntington's disease (HD) who carry a targeted single nucleotide polymorphism (SNP) rs362307 (SNP1).


title: A Phase I/II Safety and Exploratory Pharmacogenomic/Pharmacodynamic Study of Intravenous Temsirolimus (CCI-779) in Pediatric Subjects With Relapsed/Refractory Solid Tumors

lead_sponsor: Pfizer

ReferenceCitation: Geoerger B, Kieran MW, Grupp S, Perek D, Clancy J, Krygowski M, Ananthakrishnan R, Boni JP, Berkenblit A, Spunt SL. Phase II trial of temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma. Eur J Cancer. 2012 Jan;48(2):253-62. doi: 10.1016/j.ejca.2011.09.021. Epub 2011 Oct 25.

location_country:
Poland
Russian Federation
Canada
United States
France
Mexico
Germany

summary:
This is an open label, two-part study of temsirolimus given as a 60-minute intravenous (IV) infusion once weekly to pediatric subjects with advanced solid tumors.

Part 1 is an ascending-dose study to evaluate the safety of IV temsirolimus given once weekly to subjects ages 1 to 21 years with advanced solid tumors disease that is recurrent or refractory to standard therapy or for whom standard therapy is not available. (enrollment completed) Part 2 will be conducted in three groups of children with refractory or relapsed pediatric solid tumors. Subjects with the following tumor types will be enrolled: neuroblastoma, rhabdomyosarcoma, and high-grade gliomas. Subjects will receive IV temsirolimus once weekly until disease progression or unacceptable toxicity. (recruiting)


title: A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Tolerability of Licarbazepine 1000-2000mg/Day in the Treatment of Manic Episodes of Bipolar I Disorder Over 3 Weeks

summary: This purpose of this study is to evaluate the safety and efficacy of Licarbazepine for the treatment of manic episodes of bipolar disorder.

location_country:
Russian Federation
United States
France


title: Longitudinal Study of Young Patients With Embolic Stroke of Undetermined Source (ESUS)

acronym: Y-ESUS

summary: This study seeks to determine the clinical characteristics of young ESUS patients using diagnostic criteria of the Cryptogenic Stroke / ESUS International Working Group, and to determine the rates of stroke recurrence, death, and hospital readmission in a contemporary cohort of young ESUS patients during follow-up of up to 18 months.

lead_sponsor: Population Health Research Institute

location_country:
Poland
Spain
United Kingdom
Australia
Argentina
Canada
Switzerland
United States
Chile
France
South Africa
Mexico
Germany

location_facility: Texas Tech University Health Sciences Center El Paso

location_facility: East Kent Hospitals - Queen Elizabeth the Queen Mother Hospital

location_facility: University of Calgary/Foothills Medical Center

location_facility: Asklepios Klinik Nord, Department of Neurology

location_facility: Stroke Center Inselspital University Hospital Bern

location_facility: University of South Carolina - School of Medicine - Neurology

location_facility: Fundacion para la Lucha vs. Enf. Neurologicas de la Infancia

location_facility: Royal Stoke University Hospital, University Hospital of North Midlands NHS Trust

location_facility: Institute Psychiatry and Neurology, 2 Department of Neurology

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: Yoevil District Hospital Foundation Trust

location_facility: Cambridge University Foundation Trust Hospitals, Addenbrookes site

location_facility: Southend University Hospital NHS Foundation Trust

location_facility: Szpital Specjalistyczny im. Sw. Lukasza, Oddzial Neurologii z Oddzialem Udarowym

location_facility: National Institute of Neurology and Neurosurgery

location_facility: Complejo Médico de la PFA Churruca Visca

location_facility: Vancouver Stroke Program - Research Offices at Vancouver General Hospital

location_facility: University of Texas Health Center - San Antonio


title: A Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Varying Doses and Dose Regimens of Evinacumab in Patients With Persistent Hypercholesterolemia Despite Maximally Tolerated Lipid Modifying Therapy

summary: The primary objective of the study is to evaluate the reduction of LDL-C by evinacumab in comparison to placebo after 16 weeks in patients with primary hypercholesterolemia (HeFH, or non-HeFH with a history of clinical ASCVD) with persistent hypercholesterolemia despite receiving maximally-tolerated LMT. Persistent hypercholesterolemia is defined as LDL-C ≥70 mg/dL (1.81 mmol/L) for those patients with clinical ASCVD and LDL-C ≥100 mg/dL (2.59 mmol/L) for those patients without clinical ASCVD.

ReferenceCitation: Rosenson RS, Burgess LJ, Ebenbichler CF, Baum SJ, Stroes ESG, Ali S, Khilla N, Hamlin R, Pordy R, Dong Y, Son V, Gaudet D. Evinacumab in Patients with Refractory Hypercholesterolemia. N Engl J Med. 2020 Dec 10;383(24):2307-2319. doi: 10.1056/NEJMoa2031049. Epub 2020 Nov 15.

lead_sponsor: Regeneron Pharmaceuticals

location_country:
Denmark
Poland
Jordan
Spain
Italy
Austria
Australia
New Zealand
Canada
Russian Federation
United Kingdom
Sweden
United States
France
Israel
Norway
South Africa
Japan
Netherlands
Czechia

location_facility: Federal State Institution of Healthcare Clinical Hospital #122 N.A.L.G Sokolov

location_facility: Washington University School of Medicine

location_facility: Federal State Budget Institution Out-patient Clinic 3

location_facility: Sourasky Medical Center, Cardiovascular Research Center

location_facility: The Charlotte Maxeke Johannesburg Academic Hospital

location_facility: Hospital Universitario Virgen de las Nieves (HUVN)

location_facility: Slaskie Centrum Chorob Serca, III Katedra i Oddzial Kliniczny Kardiologii SUM- Oddzial Chorob Serca i Naczyn

location_facility: Univerzita Karlova v Praze 1 Lekarska Fakulta

location_facility: Ausl Della Romagna-Ospedale Degli Infermi

location_facility: Samara Regional Clinical Cardiologic Dispensary

location_facility: National Research Center For Preventative Medicine of Federal Agency of High Technology Medical Aid

location_facility: Dipartimento di Medicina Traslazionale e di Precisione dell'Università degli Studi di

location_facility: Universitair Medisch Centrum Utrech - Locatie AZU

location_facility: Ikem Institut Klinicke A Experimentalni Mediciny

location_facility: Royal Victoria Infirmary - The Newcastle Upon Tyne Hospitals NHS Foundation Trust

location_facility: University of Maryland School of Medicine

location_facility: The University Of Texas Health Science Center Houston

location_facility: Limmited Liability Company International Medical Centre SOGAZ

location_facility: Clinique des maladies lipidiques de Quebec

location_facility: Federal State Budgetary Institution Clinical-Diagnostic Center with Polyclinic

location_facility: Azienda Ospedaliero Universitaria Federico II di Napoli

location_facility: Municipal Medical and Prophylactic Institution - City Hospital for Emergency Care No.2

location_facility: Federal State Budgetary Institution Research Institute for Complex Issues of Cardiovacsular Disease

location_facility: Halina Serafin NZOZ Centrum Medyczne SERAFIN-MED

location_facility: Fondazione Toscana Gabriele Monasterio Per La Ricerca Medica E Di Sanita Pubblica

location_facility: Royal Free Hospital-Royal Free London NHS Foundation Trust

location_facility: University Hospital Innsbruck - Tyrolean Hospital


title: A Multi-centre, Double-blind, Parallel-group, Randomised, Placebo Controlled Phase II a Study to Investigate Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Different Doses of Orally Administered BI 1467335 During a 12-week Treatment Period Compared to Placebo in Patients With Clinical Evidence of NASH.

summary:
The primary objective of this study is the proof of mechanism and support of dose finding, together with the safety evaluation in patients with clinical evidence of NASH.

To gain further insight into clinical effects of AOC3 inhibition on NASH further exploratory analyses of biomarkers related to NASH and liver fibrosis will be performed. This will include the effect of BI 1467335 on reduction of secondary biomarker endpoints (ALT, AST, AP, γ-GT and CK18 fragments). Safety will be assessed throughout the study to provide key information regarding the use of BI 1467335 in patients with NASH.

ipd_sharing: Undecided

lead_sponsor: Boehringer Ingelheim

location_country:
Spain
Ireland
United Kingdom
Netherlands
Canada
United States
France
Belgium
Germany

location_facility: Hospital General Universitario de Valencia

location_facility: The University of North Carolina at Chapel Hill

location_facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz

location_facility: Rutgers Robert Wood Johnson Medical School

location_facility: Diabetes and Endocrinology Consultants, PC

location_facility: Centre Hospitalier Universitaire de Liège

location_facility: American Research Corporation at the Texas Liver Institute

location_facility: Hospital Universitario Marqués de Valdecilla


acronym: ODYSSEY HoFH

title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia

ReferenceCitation: Blom DJ, Harada-Shiba M, Rubba P, Gaudet D, Kastelein JJP, Charng MJ, Pordy R, Donahue S, Ali S, Dong Y, Khilla N, Banerjee P, Baccara-Dinet M, Rosenson RS. Efficacy and Safety of Alirocumab in Adults With Homozygous Familial Hypercholesterolemia: The ODYSSEY HoFH Trial. J Am Coll Cardiol. 2020 Jul 14;76(2):131-142. doi: 10.1016/j.jacc.2020.05.027.

location_country:
Taiwan
Greece
Austria
Italy
Canada
Ukraine
United States
France
Turkey
South Africa
Japan
Germany
Czechia

location_facility:
Regeneron Research Site
Regeneron Study Site

summary:
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) with alirocumab subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment.

The secondary objectives of the study are:

To evaluate the effect of alirocumab Q2W on other lipid parameters (ie, apolipoprotein [Apo] A-1 and B, non-high-density lipoprotein cholesterol [non-HDL-C], total-cholesterol [TC], proportion of participants with 15%, 30%, and 50% LDL-C reductions, Lp(a), HDL-C, triglycerides [TG]) in participants with HoFH
To evaluate the safety and tolerability of alirocumab SC Q2W in participants with HoFH
To assess the pharmacokinetics of alirocumab SC Q2W in participants with HoFH
To assess the potential development of anti-drug (alirocumab) antibodies


title: A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects With Light Chain (AL) Amyloidosis Who Were Previously Enrolled in Study NEOD001-201 (PRONTO)

summary: The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who have completed Study NEOD001-201.

lead_sponsor: Prothena Biosciences Ltd.

location_country:
Spain
Greece
Austria
Australia
Italy
United Kingdom
United States
France
Israel
Germany

location_facility: Hospital of the University of Pennsylvania

location_facility: Hospital Universitario Puerta de Hierro - Majadahonda

location_facility: The University of Texas MD Anderson Cancer Center


title: Randomized, Double-Blind, Double-Dummy, Placebo-Controlled, Parallel-Group, Multi-Center Trial Comparing the Effects of Orally Administered Xyrem (Sodium Oxybate) and Modafinil With Placebo in Treatment of Daytime Sleepiness in Narcolepsy

summary: This study will be conducted as a randomized, double blind, double-dummy, placebo-controlled, parallel-group trial in patients diagnosed with narcolepsy. Volunteers for this trial will be required to make 5 visits over up to 14 weeks to a participating expert physician practitioner for various sleep and narcolepsy evaluations and diaries will also be collected. Participants will take assigned medications during the course of the trial. Subjects will have a 25% probability of receiving placebo for both drugs (modafinil and Xyrem). All subject volunteers must meet criteria for narcolepsy and have evidence of daytime sleepiness. Patients will not incur any personal medical expenses due to participation in this trial. The sponsor is covering all visit costs not covered by insurance and there are some funds for patient expenses such as travel.

ReferenceType: background

ReferenceCitation: A randomized, double blind, placebo-controlled multicenter trial comparing the effects of three doses of orally administered sodium oxybate with placebo for the treatment of narcolepsy. Sleep. 2002 Feb 1;25(1):42-9.

ReferenceCitation: Scrima L, Hartman PG, Johnson FH Jr, Thomas EE, Hiller FC. The effects of gamma-hydroxybutyrate on the sleep of narcolepsy patients: a double-blind study. Sleep. 1990 Dec;13(6):479-90.

ReferenceCitation: Lammers GJ, Arends J, Declerck AC, Ferrari MD, Schouwink G, Troost J. Gammahydroxybutyrate and narcolepsy: a double-blind placebo-controlled study. Sleep. 1993 Apr;16(3):216-20.

ReferenceCitation: A 12-month, open-label, multicenter extension trial of orally administered sodium oxybate for the treatment of narcolepsy. Sleep. 2003 Feb 1;26(1):31-5.

ReferenceType: result

ReferenceCitation: Black J, Houghton WC. Sodium oxybate improves excessive daytime sleepiness in narcolepsy. Sleep. 2006 Jul;29(7):939-46.

ReferenceCitation: Black J, Pardi D, Hornfeldt CS, Inhaber N. The nightly use of sodium oxybate is associated with a reduction in nocturnal sleep disruption: a double-blind, placebo-controlled study in patients with narcolepsy. J Clin Sleep Med. 2010 Dec 15;6(6):596-602.

ReferenceCitation: Husain AM, Bujanover S, Ryan R, Scheckner B, Black J, Profant J. Incidence and duration of common, early-onset adverse events occurring during 2 randomized, placebo-controlled, phase 3 studies of sodium oxybate in participants with narcolepsy. J Clin Sleep Med. 2020 Sep 15;16(9):1469-1474. doi: 10.5664/jcsm.8530.

ReferenceCitation: Dauvilliers Y, Roth T, Guinta D, Alvarez-Horine S, Dynin E, Black J. Effect of sodium oxybate, modafinil, and their combination on disrupted nighttime sleep in narcolepsy. Sleep Med. 2017 Dec;40:53-57. doi: 10.1016/j.sleep.2017.07.030. Epub 2017 Oct 7.

lead_sponsor: Jazz Pharmaceuticals

location_country:
Germany
United States
Switzerland
France

location_facility: Sleep Disorders Center -- Union Hospital of Cecil County

location_facility: ALL-TRIALS Clinical Research, LLC -- Summit Sleep Disorder

location_facility: Community Research Management Associates,Inc.

location_facility: SLEEP WALKER -- Sleep Disorders Center & Neurodiagnostics--Lung Diagnostics, Ltd.

location_facility: Centre du sommeil -- Hopital Gui de Chauliac

location_facility: Lehigh Valley Hospital Sleep Disorders Center

location_facility: Vermont Medical Sleep Disorders Center, Inc.

location_facility: CSC Research -- Grove City Sleep Diagnostic Center

location_facility: Clinical Research Group of St. Petersburg, Inc.

location_facility: Hopital Pitie Salpetriere -- Federation des Pathologies du sommeil

location_facility: Chest Medicine Associates DBA -- Sleep Medicine Specialists

location_facility: Neurologische Poliklinik -- Universitats Spital Zurich

location_facility: The Center for Sleep and Wake Disorders/Midwest Neurology

location_facility: Cincinnati Clinic & Sleep Management Institute

location_facility: New Jersey Neuroscience Institute at JFK Medical Center

location_facility: Community Research & Sleep Management Institute

location_facility: St. Jude Medical Center -- Sleep Disorders Institute


title: Comparison of the Efficacy and Safety of Entecavir Versus Adefovir in Subjects Chronically Infected With Hepatitis B Virus and Evidence of Hepatic Decompensation

summary: This is a phase IIIb comparative study of entecavir 1.0 mg once daily (QD) vs. adefovir 10 mg QD in patients who have chronic hepatitis B infection and hepatic decompensation. The patients are treated for 96 weeks after the last subject is randomized.

lead_sponsor: Bristol-Myers Squibb

location_country:
Poland
Taiwan
Brazil
Hong Kong
United Kingdom
Greece
Russian Federation
Canada
Thailand
Indonesia
United States
France
India
Turkey
South Africa
Philippines
Singapore

location_facility: Columbia Presbyterian Medical Center (Cpmc)


title: A Multi-Center, Open-Label Phase 1b/2 Study of a Novel FGFR3 Inhibitor (B-701) Combined With Pembrolizumab in Subjects With Locally Advanced or Metastatic Urothelial Carcinoma Who Have Progressed Following Platinum-based Chemotherapy

lead_sponsor: Rainier Therapeutics

location_country:
Denmark
Poland
Serbia
Spain
Italy
Russian Federation
Netherlands
Ukraine
Sweden
Hungary
Korea, Republic of
United States
France
Turkey
Belgium
Germany
Moldova, Republic of

location_facility: Washington University School of Medicine

summary: This is a Phase 1b/2 multi-center, open-label study to establish the initial safety and to determine a recommended Phase 2 dose of B-701 in combination with pembrolizumab, and to determine safety, tolerability and efficacy of B-701 (vofatamab) plus pembrolizumab in the treatment of subjects with locally advanced or metastatic UCC, who have progressed following platinum-based chemotherapy and who have not received prior immune checkpoint inhibitor therapy.


title: A Randomized Single-Blind Study of Extracorporeal Photoimmune Therapy With UVADEX in Conjunction With Standard Therapy Alone for the Treatment of Patients With Corticosteroid-Refractory, Corticosteroid-Dependent, or Corticosteroid-Intolerant Chronic Graft-versus-Host Disease

summary: The purpose of this study is to determine whether extracorporeal photoimmune therapy with UVADEX (ECP) added to standard therapy is effective in the treatment of chronic graft-versus-host disease (GvHD).

ReferenceCitation: Smith EP, Sniecinski I, Dagis AC, Parker PM, Snyder DS, Stein AS, Nademanee A, O'Donnell MR, Molina A, Schmidt GM, Stepan DE, Kapoor N, Niland JC, Forman SJ. Extracorporeal photochemotherapy for treatment of drug-resistant graft-vs.-host disease. Biol Blood Marrow Transplant. 1998;4(1):27-37.

ReferenceCitation: Greinix HT, Volc-Platzer B, Knobler RM. Extracorporeal photochemotherapy in the treatment of severe graft-versus-host disease. Leuk Lymphoma. 2000 Feb;36(5-6):425-34. Review.

ReferenceCitation: Flowers ME, Apperley JF, van Besien K, Elmaagacli A, Grigg A, Reddy V, Bacigalupo A, Kolb HJ, Bouzas L, Michallet M, Prince HM, Knobler R, Parenti D, Gallo J, Greinix HT. A multicenter prospective phase 2 randomized study of extracorporeal photopheresis for treatment of chronic graft-versus-host disease. Blood. 2008 Oct 1;112(7):2667-74. doi: 10.1182/blood-2008-03-141481. Epub 2008 Jul 11. Erratum in: Blood. 2009 Apr 30;113(18):4478.

lead_sponsor: Mallinckrodt

location_country:
Portugal
Spain
Italy
Austria
Australia
United Kingdom
Switzerland
United States
France
Turkey
Germany
Slovakia

location_facility: Instituto Portugues de Oncologia de Francisco Gentil

location_facility: Klinika hematologie a transfuziologie FN

location_facility: Hospital dela Santa Creu i Sant Pau. Universitat Autonoma de Barcelona

location_facility: University of Arkansas for Medical Sciences

location_facility: Rush Presbyterian - St. Lukes Medical Center


title: Randomized Trial Of Adriamycin (A) Cisplatin (P) Chemotherapy Versus Paclitaxel (T) Adriamycin (A) And Cisplatin (P) In Patients With Metastatic/Relapsed Or Locally Advanced Inoperable Endometrial Cancer

summary:
RATIONALE: Drugs used in chemotherapy work in different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. It is not yet known whether doxorubicin and cisplatin are more effective with or without paclitaxel in treating endometrial cancer.

PURPOSE: Randomized phase II trial to compare the effectiveness of combining doxorubicin and cisplatin with or without paclitaxel in treating patients who have locally advanced, metastatic, and/or relapsed endometrial cancer.

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

location_country:
Portugal
Spain
Italy
Austria
United Kingdom
France
Belgium
Netherlands

location_facility: Centre for Cancer Research and Cell Biology at Belfast City Hospital

location_facility: Allgemeines Krankenhaus - Universitatskliniken

location_facility: Northern Centre for Cancer Treatment at Newcastle General Hospital

location_facility: Academisch Medisch Centrum at University of Amsterdam

location_facility: Hospitais da Universidade de Coimbra (HUC)

location_facility: Azienda Sanitaria Ospedaliera Ordine Mauriziano

location_facility: Universitair Medisch Centrum St. Radboud - Nijmegen

location_facility: Clatterbridge Centre for Oncology NHS Trust

location_facility: Cazk Groeninghe - Campus Maria's Voorzienigheid

location_facility: Centro di Riferimento Oncologico - Aviano

location_facility: Leeds Cancer Centre at St. James's University Hospital

location_facility: Mount Vernon Cancer Centre at Mount Vernon Hospital


title: A Phase II Study of Oral Fludarabine Phosphate in Patients With Previously Untreated B-Cell Chronic Lymphocytic Leukemia

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die.

PURPOSE: Phase II trial to study the effectiveness of fludarabine in treating patients who have chronic lymphocytic leukemia that has not been previously treated.

ReferenceCitation: Shustik C, Turner AR, Desjardins P, Fernandez L, Rubin S, Larratt L, Duncan AM, Rizi D, Sadura A, Shepherd L, Li D, Rassenti L, Kipps T. Oral fludarabine in untreated patients with B-cell chronic lymphocytic leukemia. Leukemia. 2010 Jan;24(1):237-9. doi: 10.1038/leu.2009.205. Epub 2009 Oct 8.

lead_sponsor: NCIC Clinical Trials Group

location_country: Canada France

location_facility: Newfoundland Cancer Treatment and Research Foundation

location_facility: Toronto Sunnybrook Regional Cancer Centre

location_facility: Northwestern Ontario Regional Cancer Centre, Thunder Bay

location_facility: Cancer Care Ontario - Windsor Regional Cancer Centre

location_facility: Algoma Reginal Cancer Program at Sault Area Hospital

location_facility: Hotel Dieu Health Sciences Hospital - Niagara

location_facility: Northeastern Ontario Regional Cancer Centre, Sudbury

location_facility: Margaret and Charles Juravinski Cancer Centre

location_facility: Cancer Care Ontario-London Regional Cancer Centre

location_facility: Durham Regional Cancer Centre at Lakeridge Health Oshawa

location_facility: Centre Hospitalier de l'Universite de Montreal


summary: The purpose of this study is to determine the safety and efficacy of Pegasys + placebo + lamivudine versus lamivudine alone in patients with lamivudine versus lamivudine alone in patients with hepatitis B antigen CHB.

lead_sponsor: Hoffmann-La Roche

location_country:
Poland
Taiwan
Brazil
United Kingdom
Australia
New Zealand
Canada
Thailand
Switzerland
Korea, Republic of
United States
France
China
Israel
Puerto Rico
Singapore
Germany


title: Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers

summary: This is an open-label, single arm, multi-center, pilot study of Nivolumab in pediatric patients with recurrent or refractory hypermutant malignancies aged 12 months to 18 years of age. This study is to assess clinical and radiological benefits of treatment with Nivolumab in children with hypermutated cancers, including those with bMMRD syndrome. It is our expectation that patients with bMMRD syndrome will account for the majority of patients enrolled on this study.

ReferenceCitation: Henderson JJ, Das A, Morgenstern DA, Sudhaman S, Bianchi V, Chung J, Negm L, Edwards M, Kram DE, Osborn M, Hawkins C, Bouffet E, Cho YJ, Tabori U. Immune Checkpoint Inhibition as Single Therapy for Synchronous Cancers Exhibiting Hypermutation: An IRRDC Study. JCO Precis Oncol. 2022 Mar;6:e2100286. doi: 10.1200/PO.21.00286.

ReferenceCitation: Rittberg R, Harlos C, Rothenmund H, Das A, Tabori U, Sinha N, Singh H, Chodirker B, Kim CA. Immune Checkpoint Inhibition as Primary Adjuvant Therapy for an IDH1-Mutant Anaplastic Astrocytoma in a Patient with CMMRD: A Case Report-Usage of Immune Checkpoint Inhibition in CMMRD. Curr Oncol. 2021 Feb 1;28(1):757-766. doi: 10.3390/curroncol28010074.

lead_sponsor: The Hospital for Sick Children

location_country:
Australia
Canada
United States
Israel
France

location_facility: Children's & Women's Health Centre of British Columbia

lead_sponsor_type: academique


acronym: STELARA

title: A Randomized Double-blind Pharmacokinetic Study of Ustekinumab in Pediatric Subjects With Moderately to Severely Active Crohn's Disease

lead_sponsor: Janssen Research & Development, LLC

location_country:
Poland
Canada
United States
France
Belgium
Germany

location_facility: Center For Digestive Health Systems-Greenville

location_facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu

location_facility: Children's Center For Digestive Healthcare, Llc

location_facility: Universitair Kinderziekenhuis Koningin Fabiola

summary: The purpose of this study is to evaluate the pharmacokinetics (PK) of ustekinumab in subjects from 2 through less than (<) 18 years old in the USA, or 6 through less than (<) 18 years old in other countries and determine if it is similar to that observed in adults with moderately to severely active Crohn's disease (CD). Also to assess the safety, immunogenicity and efficacy of ustekinumab in the treatment of moderately to severely active CD. The main part of the study continues to Week 16, at which point all subjects who are receiving benefit from ustekinumab maintenance therapy (as determined by the investigator) are eligible to enter the long-term extension (LTE) and continue to receive ustekinumab. The study extension ends at Week 268 or upon availability of the LTE basket study (CNTO1275ISD3001) whichever occurs first. If participants do not consent/assent to the LTE basket study, they will continue safety follow-up for approximately 20 weeks after the last study agent administration.


acronym: AWARD-PEDS

title: A Randomized, Double-Blind Study With an Open-Label Extension Comparing the Effect of Once-Weekly Dulaglutide With Placebo in Pediatric Patients With Type 2 Diabetes Mellitus (AWARD-PEDS: Assessment of Weekly AdministRation of LY2189265 in Diabetes-PEDiatric Study)

lead_sponsor: Eli Lilly and Company

ReferenceCitation: Arslanian SA, Hannon T, Zeitler P, Chao LC, Boucher-Berry C, Barrientos-Pérez M, Bismuth E, Dib S, Cho JI, Cox D; AWARD-PEDS Investigators. Once-Weekly Dulaglutide for the Treatment of Youths with Type 2 Diabetes. N Engl J Med. 2022 Aug 4;387(5):433-443. doi: 10.1056/NEJMoa2204601. Epub 2022 Jun 4.

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

location_country:
Brazil
Saudi Arabia
United Kingdom
Hungary
Puerto Rico
United States
France
India
Turkey
Mexico
Germany

location_facility: Hôpitaux Universitaires Paris Sud - Hôpital Bicêtre

location_facility: Division of Endocrinology, Diabetes, and Metabolism

location_facility: Seattle Children's Hospital Research Foundation

location_facility: Cincinnati Childrens Hospital Medical Center

location_facility: Florida Center for Endocrinology & Metabolism

location_facility: Dr Jivraj Mehta Smarak Health Foundation Bakeri Medical Research Centre

location_facility: Post Graduate Institute of Medical Education & Research

location_facility: Hospital da Clinicas da Faculdade de Medicina da USP

location_facility: Ondokuz Mayis University Medical Faculty

location_facility: Centro de Diabetes y Endocrinologia Pediatrica de PR

location_facility: Deenanath Mangeshkar Hospital & Research Centre

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

location_facility: Center of Excellence in Diabetes & Endocrinology

location_facility: Instituto Estadual de Diabetes e Endocrinologia

location_facility: King Salman bin Abdulaziz Hospital - Diabetic Center

summary: The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of the study drug dulaglutide compared to placebo in pediatric participants with type 2 diabetes. The study duration is approximately 60 weeks.


acronym: HORIZON

title: A Single Arm, Open-Label, Phase 2 Study of Melflufen in Combination With Dexamethasone in Patients With Relapsed Refractory Multiple Myeloma Who Are Refractory to Pomalidomide and/or an Anti-CD38 Monoclonal Antibody

lead_sponsor: Oncopeptides AB

ReferenceCitation: Richardson PG, Oriol A, Larocca A, Bladé J, Cavo M, Rodriguez-Otero P, Leleu X, Nadeem O, Hiemenz JW, Hassoun H, Touzeau C, Alegre A, Paner A, Maisel C, Mazumder A, Raptis A, Moreb JS, Anderson KC, Laubach JP, Thuresson S, Thuresson M, Byrne C, Harmenberg J, Bakker NA, Mateos MV; HORIZON (OP-106) Investigators. Melflufen and Dexamethasone in Heavily Pretreated Relapsed and Refractory Multiple Myeloma. J Clin Oncol. 2021 Mar 1;39(7):757-767. doi: 10.1200/JCO.20.02259. Epub 2020 Dec 9.

ReferenceCitation: Larocca A, Leleu X, Touzeau C, Bladé J, Paner A, Mateos MV, Cavo M, Maisel C, Alegre A, Oriol A, Raptis A, Rodriguez-Otero P, Mazumder A, Laubach J, Nadeem O, Sandberg A, Orre M, Torrång A, Bakker NA, Richardson PG. Patient-reported outcomes in relapsed/refractory multiple myeloma treated with melflufen plus dexamethasone: analyses from the Phase II HORIZON study. Br J Haematol. 2022 Feb;196(3):639-648. doi: 10.1111/bjh.17887. Epub 2021 Oct 21. Erratum in: Br J Haematol. 2022 Aug;198(3):609.

location_country:
United States
Italy
Spain
France

location_facility: Hospital Clinic i Provincial de Barcelona

location_facility: Innovative Clinical Research Institute (ICRI)

location_facility: Hospital Universitario Fundación Jiménez Díaz

location_facility: Institut Català d'Oncología (ICO) Badalona

summary: This study will evaluate melflufen in combination with dexamethasone in adult patients with relapsed or refractory multiple myeloma in whose disease is refractory to pomalidomide and/or an anti-CD38 monoclonal antibody. All patients in the study will be treated with melflufen on Day 1 and dexamethasone on Days 1, 8, 15 and 22 of each 28-day cycle.


title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study to Determine the Safety and Efficacy of Natalizumab, When Added to Avonex® (Interferon Beta-1a), in Subjects With Relapsing-Remitting Multiple Sclerosis

summary: The purpose of this study is to determine if natalizumab in combination with AVONEX is safe and effective in delaying progression of individuals diagnosed with relapsing remitting Multiple Sclerosis (MS).

ReferenceCitation: Rudick RA, Stuart WH, Calabresi PA, Confavreux C, Galetta SL, Radue EW, Lublin FD, Weinstock-Guttman B, Wynn DR, Lynn F, Panzara MA, Sandrock AW; SENTINEL Investigators. Natalizumab plus interferon beta-1a for relapsing multiple sclerosis. N Engl J Med. 2006 Mar 2;354(9):911-23.

lead_sponsor: Biogen

location_country:
Italy
Austria
United States
France
Israel
Belgium
Germany

location_facility: Maimonides Medical Center, Division of Neurology

location_facility: University of Arkansas For Medical Sciences

location_facility: The Multiple Sclerosis Center of Atlanta

location_facility: Medizinische Hochschule Hannover, Neurologische Klinik

location_facility: Fachbereich Neurologie und Klinische Neurophysiologie

location_facility: Wake Forest University School of Medicine, Department of Neurology

location_facility: Georgetown Univesity Hospital, Research Pharmacy

location_facility: Universitaetsklinikum Giessen, Oberarzt Neurologie

location_facility: University of Miami, Department of Neurology

location_facility: Hospital of the University of Pennsylvania

location_facility: St. Francis Hospital, Center for Neurological Disorders

location_facility: Rush-Presbyterian St. Luke's Medical Center

location_facility: Alexian Brothers Center for Clinical Research

location_facility: Northwestern University, Department of Neurology

location_facility: Baylor Methodist International MS Center

location_facility: University of Massachusetts Memorial Medical Center

location_facility: Michigan Institute for Neurological Disorders

location_facility: University of Maryland Hospital, Department of Neurology

location_facility: Greenstein Neurology Associates & MS Institute

location_facility: Springfield Clinic Neuroscience Institute

location_facility: Universita di Genova, Clinica Neurologica

location_facility: University of Mississippi Medical Center

location_facility: Hadassah Hebrew Uni Hospital, Department of Neurology

location_facility: St. Luke's Roosevelt Hospital, MS Research & Treatment Center

location_facility: New York Hospital - Cornell Medical Center

location_facility: Buffalo General Hospital, Department of Neurology

location_facility: University of Pittsburgh, Department of Neurology

location_facility: University of Alabama-Birmingham, Department of Neurology


title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study to Determine the Safety and Efficacy of Natalizumab in Subjects With Relapsing-Remitting Multiple Sclerosis

summary: The purpose of this study is to determine the safety and efficacy of natalizumab in the treatment of individuals who have been diagnosed with relapsing remitting multiple sclerosis (MS). It is hoped that natalizumab will prevent certain types of white blood cells from moving out of the bloodstream into organs, including the brain, that are being damaged by autoimmune disease (a disease in which the body's own immune system attacks certain organs). These white blood cells are thought to cause inflammation that can result in lesions (small areas of damage) in the brain. These lesions are thought to be the cause of relapses and disability in MS.

ReferenceCitation: Voloshyna N, Havrdová E, Hutchinson M, Nehrych T, You X, Belachew S, Hotermans C, Paes D. Natalizumab improves ambulation in relapsing-remitting multiple sclerosis: results from the prospective TIMER study and a retrospective analysis of AFFIRM. Eur J Neurol. 2015 Mar;22(3):570-7. doi: 10.1111/ene.12618. Epub 2014 Dec 15.

ReferenceCitation: Polman CH, O'Connor PW, Havrdova E, Hutchinson M, Kappos L, Miller DH, Phillips JT, Lublin FD, Giovannoni G, Wajgt A, Toal M, Lynn F, Panzara MA, Sandrock AW; AFFIRM Investigators. A randomized, placebo-controlled trial of natalizumab for relapsing multiple sclerosis. N Engl J Med. 2006 Mar 2;354(9):899-910.

ReferenceCitation: Balcer LJ, Galetta SL, Polman CH, Eggenberger E, Calabresi PA, Zhang A, Scanlon JV, Hyde R. Low-contrast acuity measures visual improvement in phase 3 trial of natalizumab in relapsing MS. J Neurol Sci. 2012 Jul 15;318(1-2):119-24. doi: 10.1016/j.jns.2012.03.009. Epub 2012 Apr 21.

location_country:
United Kingdom
Netherlands
Canada
Czech Republic
France
United States
Belgium
Germany

location_facility: Ipswich Hospital NHS Trust - Department of Clinical Neurology

location_facility: General Teaching Hospital - Neurological Department

location_facility: Lehigh Valley Hospital, Neurosciences Research

location_facility: Hospital Pardubice - Department of Neurology

location_facility: University of Toronto, MS Clinic, St. Michael's Hospital

location_facility: University of Kansas Medical Center, Department of Neurology

location_facility: Kings College Hospital, Kings Neuroscience Center

location_facility: Hopital de la Timone, Service de Neurologie

location_facility: The Radcliffe Infirmary, University Department of Clinical Neurology

location_facility: St. Josef-Hospital, Neurologische Klinik der Ruhruniversitat Bochum

location_facility: MS Research Unit, Center for Clinical Research

location_facility: Faculty Hospital Plzen - Clinic of Neurology

location_facility: University of Washington MS Research Center

location_facility: St. James University Hospital, Department of Neurology

location_facility: Faculty Hospital Motol - Neurological Clinic

location_facility: North Staffordshire Royal Infirmary - Neurology Department

location_facility: Oregon Health Sciences University, Department of Neurology

location_facility: CHRU - Hopital de Pontchaillou, Service de Neurologie

location_facility: Yale University School of Medicine, Department of Neurology

location_facility: Vancouver Hospital and Health Sciences Center UBC Pavilion, MS Clinical Research Group

location_facility: UC Davis School of Medicine, Department of Neurology

location_facility: University of Miami School of Medicine, Department of Neurology

location_facility: Sunnybrook and Women's College and Health Science Centre


title: Saline Hypertonic in Preschoolers With Cystic Fibrosis and Lung Structure as Measured by Computed Tomography (CT)

acronym: SHIP-CT

summary: The purpose of this study is to assess whether inhalation of 7% hypertonic saline (HS) twice daily for 48 weeks reduces structural lung disease as assessed by computed tomography (CT) in comparison with inhalation of 0.9% isotonic saline (IS) in preschool children (ages 3 to 6) with cystic fibrosis.

ReferenceCitation: Rosenow T, Oudraad MC, Murray CP, Turkovic L, Kuo W, de Bruijne M, Ranganathan SC, Tiddens HA, Stick SM; Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF). PRAGMA-CF. A Quantitative Structural Lung Disease Computed Tomography Outcome in Young Children with Cystic Fibrosis. Am J Respir Crit Care Med. 2015 May 15;191(10):1158-65. doi: 10.1164/rccm.201501-0061OC.

ReferenceCitation: Ramsey KA, Rosenow T, Turkovic L, Skoric B, Banton G, Adams AM, Simpson SJ, Murray C, Ranganathan SC, Stick SM, Hall GL; AREST CF. Lung Clearance Index and Structural Lung Disease on Computed Tomography in Early Cystic Fibrosis. Am J Respir Crit Care Med. 2016 Jan 1;193(1):60-7. doi: 10.1164/rccm.201507-1409OC.

ReferenceCitation: Tiddens HAWM, Chen Y, Andrinopoulou ER, Davis SD, Rosenfeld M, Ratjen F, Kronmal RA, Hinckley Stukovsky KD, Dasiewicz A, Stick SM; SHIP-CT Study Group. The effect of inhaled hypertonic saline on lung structure in children aged 3-6 years with cystic fibrosis (SHIP-CT): a multicentre, randomised, double-blind, controlled trial. Lancet Respir Med. 2022 Jul;10(7):669-678. doi: 10.1016/S2213-2600(21)00546-4. Epub 2022 Mar 11.

lead_sponsor: University of Washington, the Collaborative Health Studies Coordinating Center

location_country:
Denmark
Spain
Italy
Australia
Canada
United States
France
Belgium
Netherlands

location_facility: University of North Carolina at Chapel Hill

location_facility: Washington University School of Medicine

location_facility: Universitair Ziekenhuis Children's Hospital

location_facility: Sophia Children's Hospital at Erasmus Medical Centre

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: Copenhagen University Hospital Rigshospitalet


title: Phase III, Randomized, Multicenter Study to Assess the Efficacy and Safety of HuM195 (Recombinant Humanized Anti-CD33 Monoclonal Antibody) in Combination With Standardized Chemotherapy Compared to Standardized Chemotherapy Alone in the Treatment of Patients With Refractory or First-Relapsed Acute Myelogenous Leukemia (AML)

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. It is not yet known if chemotherapy is more effective with or without monoclonal antibody therapy for acute myelogenous leukemia.

PURPOSE: Randomized phase III trial to compare the effectiveness of combination chemotherapy with or without monoclonal antibody therapy in treating patients who have refractory or relapsed acute myelogenous leukemia.

status: Unknown status

lead_sponsor: Facet Biotech

location_country:
United Kingdom
Netherlands
Canada
United States
France
Belgium
Germany

location_facility: Centre Hospitalier Regional et Universitaire d'Angers

location_facility: Lineberger Comprehensive Cancer Center, UNC

location_facility: Queen Elizabeth II Health Science Center

location_facility: Beckman Research Institute, City of Hope

location_facility: University of Iowa Hospitals and Clinics

location_facility: University of Pennsylvania Cancer Center

location_facility: Klinikum Rechts Der Isar/Technische Universitaet Muenchen

location_facility: Louisiana State University School of Medicine

location_facility: University of California Davis Cancer Center

location_facility: North Mississippi Hematology and Oncology Associates, Ltd.

location_facility: Washington University Barnard Cancer Center

location_facility: Albert Einstein Comprehensive Cancer Center

location_facility: Jonsson Comprehensive Cancer Center, UCLA

location_facility: New York Presbyterian Hospital - Cornell Campus

location_facility: Massachusetts General Hospital Cancer Center

intervention_type: Biological


title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment With Elamipretide in Patients Hospitalized With Congestion Due to Heart Failure

summary: This is a phase 2 randomized, double-blind, placebo-controlled study to evaluate the cardiac and renal effects of short term treatment with elamipretide in patients hospitalized with congestion due to heart failure

lead_sponsor: Stealth BioTherapeutics Inc.

location_country:
Poland
Serbia
Spain
Latvia
United Kingdom
Hungary
France
Belgium
Netherlands
Bulgaria

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Clinic of Cardiology, "Second Multiprofile Hospital for Active Treatement - Sofia" EAD

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: Clinical Centre of Serbia, Emergency Centre

location_facility: Consorci Hospital General Universitari de Valencia

location_facility: Zala County Hospital Cardiology Department

location_facility: Department of Internal Diseases, "Multiprofile Hospital for Active Treatment Sveta Ekaterina - Dimitrovgrad" EOOD

location_facility: Clinic of Cardiology, "City Clinic University Multiprofile Hospital for Active Treatment" EOOD

location_facility: Clinical Hospital Centre Bezanijska kosa

location_facility: Clinic of Cardiology, Multiprofile Hospital for Active Treatment National Heart Hospital" EAD

location_facility: Pauls Stradins Clinical University Hospital, Latvian Centre of Cardiology

location_facility: Bieganski Provincial Specialist Hospital, Department of Cardiology, Clinic of Cardiology UM

location_facility: Cardinal Stefan Wyszynski, Institute of Cardiology, Clinic Heart Failure and Transplantation

location_facility: Institute for Cardiovascular Diseases of Vojvodina

location_facility: Riga East Clinical University Hospital, Clinic Gailezers

location_facility: Hospital Clínico Universitario de Valencia

location_facility: Clinical Centre of Serbia, Institute for Cardiovascular Diseases

location_facility: Clinic of Internal Diseases, "Multiprofile Hospital for Active Treatment, "Sveta Anna Sofia" AD

location_facility: Department of Cardiology, "Multiprofile Regional Hospital for Active Treatement Dr. Stefan Cherkezov" AD

location_facility: Cardinal Stefan Wyszynski, Institute of Cardiology, Clinic of Coronary Heart Disease and Structural Heart Disease

location_facility: Hungarian Institute Of Cardiology, Department of Adult Cardiology

location_facility: Semmelweis University Heart and Vascular Center

location_facility: Complexo Hospitalario Universitario de A Coruña

location_facility: Hospital Universitari i Politècnic La Fe

location_facility: County St. George University Teaching Hospital, Department of Internal Medicine Cardiology


acronym: FIREFISH

title: A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants With Type 1 Spinal Muscular Atrophy

ReferenceCitation: Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Servais L, Xiong H, Zanoteli E, Baranello G, Bruno C, Day JW, Deconinck N, Klein A, Mercuri E, Vlodavets D, Wang Y, Dodman A, El-Khairi M, Gorni K, Jaber B, Kletzl H, Gaki E, Fontoura P, Darras BT; FIREFISH Study Group. Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial. Lancet Neurol. 2022 Oct 13. pii: S1474-4422(22)00339-8. doi: 10.1016/S1474-4422(22)00339-8. [Epub ahead of print]

ReferenceCitation: Cances C, Vlodavets D, Comi GP, Masson R, Mazurkiewicz-Bełdzińska M, Saito K, Zanoteli E, Dodman A, El-Khairi M, Gorni K, Gravestock I, Hoffart J, Scalco RS, Darras BT; ANCHOVY Working Group. Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study. Orphanet J Rare Dis. 2022 Jul 29;17(1):300. doi: 10.1186/s13023-022-02455-x.

ReferenceCitation: Darras BT, Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Xiong H, Zanoteli E, Baranello G, Bruno C, Vlodavets D, Wang Y, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Fontoura P, Servais L; FIREFISH Working Group. Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls. N Engl J Med. 2021 Jul 29;385(5):427-435. doi: 10.1056/NEJMoa2102047.

ReferenceCitation: Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, Mercuri E, Rose K, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Seabrook T, Fontoura P, Servais L; FIREFISH Working Group. Risdiplam in Type 1 Spinal Muscular Atrophy. N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24.

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

location_country:
Croatia
Poland
Brazil
Serbia
Saudi Arabia
Italy
Russian Federation
Spain
Switzerland
Ukraine
United States
France
China
Turkey
Belgium
Japan

location_facility: Clinical Medical Center Zagreb; University Hospital Rebro Department of Paediatrics

location_facility: Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile

location_facility: Hospital Yeditepe University Kozyatagi; Pediatry

location_facility: Institute for Mother and Child Dr. Vukan Cupic

location_facility: Hospital das Clinicas - FMUSP_X; Neurologia

location_facility: IRCCS Istituto Giannina Gaslini; U.O.S.D. Centro di Miologia e Patologie Neurodegenerative

location_facility: Fondazione IRCCS Istituto Neurologico "Carlo Besta"; UO di Neurologia dello Sviluppo

location_facility: Ams of Ukraine; Inst. of Neurology, Psychiatry & Narcology

location_facility: King Faisal Specialist Hospital and Research Centre Building

location_facility: Columbia University Medical Center; The Neurological Institute of New York

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Unità Operativa Complessa di Neurologia

location_facility: Instituto de Puericultura E Pediatria Martagão Gesteira

location_facility: Hospital Universitari Vall d'Hebron; Area Genética Clínica y Molecular

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

location_facility: The Children's Memorial Health Institute Department of Neurology and Epileptology

location_facility: Hopital Femme Mere Enfant; Medecine Physique et Readaptation Pediatrique - L'ESCALE

location_facility: Hacettepe University, School of Medicine; Pediatrics Department; Pediatrics Child Neurology Unit

location_facility: Russian Children Neuromuscular Center of Veltischev

location_facility: IRCCS Ospedale Pediatrico Bambino Gesù; U.O. Malattie Neuromuscolari e Neurodegenerative

location_facility: Szpital Gdanskiego Uniwersytetu Medycznego; Clinic of developmental neurology

summary: Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate Risdiplam (RO7034067) for 24-months at the dose selected in Part 1.


title: A Prospective, Randomized, International, Multicenter, Double-arm, Controlled, Open-label Study of Riociguat in Patients With Pulmonary Arterial Hypertension (PAH) Who Are on a Stable Dose of Phosphodiesterase-5 Inhibitors (PDE-5i) With or Without Endothelin Receptor Antagonist (ERA), But Not at Treatment Goal

acronym: REPLACE

summary: To demonstrate the effectiveness of riociguat as replacement of phosphodiesterase-5 inhibitors (PDE-5i) therapy in pulmonary arterial hypertension (PAH) patients

ReferenceCitation: Hoeper MM, Al-Hiti H, Benza RL, Chang SA, Corris PA, Gibbs JSR, Grünig E, Jansa P, Klinger JR, Langleben D, McLaughlin VV, Meyer GMB, Ota-Arakaki J, Peacock AJ, Pulido T, Rosenkranz S, Vizza CD, Vonk-Noordegraaf A, White RJ, Chang M, Kleinjung F, Meier C, Paraschin K, Ghofrani HA, Simonneau G; REPLACE investigators. Switching to riociguat versus maintenance therapy with phosphodiesterase-5 inhibitors in patients with pulmonary arterial hypertension (REPLACE): a multicentre, open-label, randomised controlled trial. Lancet Respir Med. 2021 Jun;9(6):573-584. doi: 10.1016/S2213-2600(20)30532-4. Epub 2021 Mar 24.

lead_sponsor: Bayer

location_country:
Spain
Italy
United Kingdom
United States
Belgium
Japan
Czechia
Poland
Portugal
Austria
Turkey
Mexico
Brazil
Canada
Germany
Denmark
Taiwan
Greece
Korea, Republic of
France
Netherlands


title: A Multicenter, Randomized, Double-blind, Active-controlled Study to Evaluate the Effects of LCZ696 Compared to Valsartan on Cognitive Function in Patients With Chronic Heart Failure and Preserved Ejection Fraction

acronym: PERSPECTIVE

summary: The purpose of this study is to evaluate the effect of LCZ696 compared to valsartan on cognitive function in patients with chronic heart failure and preserved ejection fraction. Cognitive function will be assessed using a comprehensive battery of tests with an evaluation of longitudinal change of cognitive domains including memory, executive function, and attention.

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:
Croatia
Poland
Taiwan
Spain
Italy
Russian Federation
Australia
Netherlands
Argentina
Canada
Lithuania
Switzerland
United Kingdom
Korea, Republic of
United States
France
Turkey
Belgium
Germany
Bulgaria


title: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of the Safety and Efficacy of Adefovir Dipivoxil as Intensification Therapy in Combination With Highly Active Antiretroviral Therapy (HAART) in HIV Infected Patients With HIV-1 RNA > 50 and <= 400 Copies/Ml

summary: The purpose of this study is to see if it is safe and effective to give an experimental anti-HIV drug, adefovir dipivoxil (ADV), in combination with other anti-HIV drugs (HAART) to patients who have a viral load (level of HIV in the blood) between 50 and 400 copies/ml.

lead_sponsor: Gilead Sciences

location_country:
United Kingdom
Canada
United States
France
Germany

location_facility: Indiana Univ Infectious Disease Research Clinic

location_facility: Univ of Texas Southwestern Med Ctr of Dallas

location_facility: Univ of Utah Med School / Clinical Trials Ctr

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

location_facility: Ctr for AIDS Research / Education and Service (CARES)

location_facility: Rush Presbyterian - Saint Luke's Med Ctr

location_facility: San Francisco Gen Hosp / UCSF AIDS Program

location_facility: Klinikum Der Johann Wolfgang Goethe Universitat


title: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Clinical Trial to Evaluate the Anti-viral Efficacy of Masitinib in Patients With Symptomatic Mild to Moderate COVID-19

summary: The objective of the study is to evaluate the anti-viral efficacy of 3 different dosages of masitinib in patients with symptomatic mild to moderate COVID-19.

ReferenceCitation: Latham BD, Oskin DS, Crouch RD, Vergne MJ, Jackson KD. Cytochromes P450 2C8 and 3A Catalyze the Metabolic Activation of the Tyrosine Kinase Inhibitor Masitinib. Chem Res Toxicol. 2022 Sep 19;35(9):1467-1481. doi: 10.1021/acs.chemrestox.2c00057. Epub 2022 Sep 1.

ipd_sharing_description: Masitinib is under clinical investigation and has not yet been approved in any sought-after indication by any health authority worldwide. As such, there is no plan for data-sharing at this point in time.

lead_sponsor: AB Science

location_country:
Russian Federation
France
South Africa

location_facility: Intensive Care Unit, CHU Gabriel-Montpied

location_facility: Gabrichevsky Institute of Epidemiology and Microbiology


title: A Phase 2 Study of Belantamab Mafodotin in Patients With Relapsed or Refractory AL Amyloidosis

summary:
This is an open-label, multicenter, Phase 2 study in subjects with previously treated patients with light chain (AL) amyloidosis in need for therapy.

Approximately 35 subjects will receive therapy with belantamab mafodotin. Subject participation will include a Screening Phase, a Treatment Phase, a Post-Treatment Observation Phase, and a Long-term Follow-up Phase.

A safety run-in will be conducted in 6 subjects treated with belantamab mafodotin for at least 1 cycle.

According to the two-stage statistical design of the study, an interim analysis of efficacy will occur. If after 15 patients have been enrolled at least 3 complete or very good partial responses have been recorded, the accrual will continue until all planned patients have been enrolled

lead_sponsor: European Myeloma Network

location_country:
United Kingdom
Italy
Greece
Netherlands
France
Germany

location_facility: Fondazione I.R.C.C.S Policlinico "San Matteo"

location_facility: Centre hospitalier Universitaire de Limoges -


title: An Open-label, Multicenter Phase 1 Trial to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Splicing Modulator H3B-8800 for Subjects With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia

summary: A Phase 1, an Open-label, Multicenter Phase 1 Trial to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Splicing Modulator H3B-8800 (RVT-2001) for Subjects With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia

lead_sponsor: Hemavant Sciences GmbH

location_country:
Germany
United States
Spain
France

location_facility: University of North Carolina Chapel Hill

location_facility: Hospital Universitario HM Sanchinarro - CIOCC

location_facility: H Lee Moffitt Cancer Center and Research Institute

location_facility: Hospital Universitario Fundacion Jimenez Diaz

location_facility: Hospital General Universitario Gregorio Marañon


title: Basket of Baskets: A Modular, Open-label, Phase II, Multicentre Study To Evaluate Targeted Agents in Molecularly Selected Populations With Advanced Solid Tumours

acronym: BoB

summary:
The Global objective of this Basket of Basket study is to evaluate the antitumor activity of each matched therapies that will be evaluated through the study in small molecularly selected populations.

The objective of module 1 will be to determine the overall response rate by RECIST 1.1 of atezolizumab in several arms. Subjects will be separated into arms depending on the mutations of their tumour. All patients in genomically selected populations will receive atezolizumab 1200 mg IV every 3 weeks

lead_sponsor: Vall d'Hebron Institute of Oncology

location_country:
Spain
United Kingdom
Netherlands
Sweden
France
Germany


title: Multi-Center Clinical Evaluation of the ATTUNE Cementless Rotating Platform Total Knee Arthroplasty

summary: The primary objective of this study is to examine the change from pre-operative baseline to two year postoperative functional performance improvement for the ATTUNE primary, cementless TKA RP system as measured with the KOOS questionnaire (KOOS-ADL sub-score). This will be carried out for two configurations: cruciate retaining rotating platform (ATTUNE Cementless CR RP) and posterior stabilized rotating platform (ATTUNE Cementless PS RP).

lead_sponsor: DePuy Orthopaedics

location_country:
United Kingdom
Ireland
Austria
Netherlands
New Zealand
Canada
United States
France
Germany

location_facility: Texas Tech University Heath Science Center

location_facility: Sevice de Chirurgie Orthoedique et Traumatologique

location_facility: Hampton Roads Orthopaedics and Sports Medicine

location_facility: Nottingham University Hospitals NHS Trust

location_facility: Krankenhaus der Barmherzigen Schwestern Ried im Innkreis

location_facility: Foothills Medical Centre, University of Calgary

location_facility: South Infirmary Victoria University Hospital


title: A Prospective Study To Evaluate The Performance And Operation Of The Brevera™ Breast Biopsy System

summary: The Brevera Breast Biopsy System integrates tissue acquisition, real time imaging, and post biopsy handling all during the same procedure. This post-market clinical trial will be performed to obtain clinical/operational data and feedback on the Brevera Breast Biopsy System.

lead_sponsor: Hologic, Inc.

location_country:
Spain
Italy
United Kingdom
Netherlands
France
Germany

location_facility: Institut régional du Cancer de Montpellier

location_facility: Centro de Patalogia de la Mama-Fundacion Tejerina


title: Gestion du Sevrage de la Ventilation mécanique du Patient neurolésé en réanimation et Association Avec le Devenir. Etude Observationnelle Multi-centrique Internationale. Extubation Strategies in Neuro-Intensive Care Unit Patients and Associations With Outcome. The International Observational ENIO Study.

acronym: ENIO

summary:
Rationale Prolonged mechanical ventilation (MV) is common in patients with severe Brain Injury (BI). Guidelines for the management of extubation are largely lacking for patients with BI, and the role of tracheostomy is highly uncertain. More important, data on practice of management of extubation is yet underreported, as is the use of tracheotomy in this specific subset of critical care patients.

Objective The objective of this prospective observational study is to describe the management of extubation and tracheostomy in intensive care unit (ICU) patients with BI. The aim is to describe the incidence of extubation failure and the rate of tracheostomy.

Study design The "Extubation strategies in Neuro-Intensive care unit patients, and associations with Outcomes (ENIO)" is an observational multicentre international cohort study.

Study population The investigators will include patients undergoing BI, with an initial Glasgow Coma Score ≤ 12 and with a delivered duration of mechanical ventilation (MV) ≥ 24 hours at ICU admission. The inclusion period will last 6 months in total, and each centre is expected to include at least 24 patients during this period. With over 60 ICUs participating worldwide, we expect to include 1500 patients.

Main parameters Parameters to be collected include: general neurological management, ventilatory management, general ICU complications, specific data on extubation and tracheostomy, general in-ICU outcomes and in-hospital mortality.

Nature and extent of the burden and risks associated with participation Because of the observational design of the study using routinely collected data, there is no additional burden for the patient. Collection of data from ICU charts and/or (written or electronic) medical records systems bears no risk to the patients.

ReferenceCitation: Cinotti R, Pelosi P, Schultz MJ, Aikaterini I, Alvarez P, Badenes R, Mc Credie V, Elbuzidi AS, Elhadi M, Godoy DA, Gurjar M, Haenggi M, Kaye C, Mijangos-Méndez JC, Piagnerelli M, Piracchio R, Reza ST, Stevens RD, Yoshitoyo U, Asehnoune K; ENIO Study Group. Extubation strategies in neuro-intensive care unit patients and associations with outcomes: the ENIO multicentre international observational study. Ann Transl Med. 2020 Apr;8(7):503. doi: 10.21037/atm.2020.03.160.

ipd_sharing_description: All proposals for sub-studies on the main ENIO database are encouraged. Secondary studies must be approved by the steering committee. Study results will be presented and disseminated in a timely manner. Authorship will be proposed by the investigators of the sub-studies and approved by the steering committee. National groups may enter requests for data-access to analyse their national datasets. Owing to the full anonymization of data according to European Union laws and regulations, institutional datasets will not be available.

lead_sponsor: Nantes University Hospital

location_country: India France Netherlands Italy

location_facility:
Haaglanden (Medical Center)
Enschede (Medisch Spectrum Twente)
University of Genes
University of Amsterdam
Varanasi BHU Hospital
Nantes University Hospital


title: A Phase 1a/1b Study of a Novel Anti-PD-L1 Checkpoint Antibody (LY3300054) Administered Alone or in Combination With Other Agents in Advanced Refractory Solid Tumors (Phase 1a/1b Anti-PD-L1 Combinations in Tumors-PACT)

acronym: PACT

summary: The main purpose of this study is to evaluate the safety and tolerability of anti-programmed cell death ligand 1 (PD-L1) checkpoint antibody LY3300054 in participants with advanced refractory solid tumors.

ReferenceCitation: Hollebecque A, Chung HC, de Miguel MJ, Italiano A, Machiels JP, Lin CC, Dhani NC, Peeters M, Moreno V, Su WC, Chow KH, Galvao VR, Carlsen M, Yu D, Szpurka AM, Zhao Y, Schmidt SL, Gandhi L, Xu X, Bang YJ. Safety and Antitumor Activity of α-PD-L1 Antibody as Monotherapy or in Combination with α-TIM-3 Antibody in Patients with Microsatellite Instability-High/Mismatch Repair-Deficient Tumors. Clin Cancer Res. 2021 Dec 1;27(23):6393-6404. doi: 10.1158/1078-0432.CCR-21-0261. Epub 2021 Aug 31.

ReferenceCitation: Patnaik A, Yap TA, Chung HC, de Miguel MJ, Bang YJ, Lin CC, Su WC, Italiano A, Chow KH, Szpurka AM, Yu D, Zhao Y, Carlsen M, Schmidt S, Vangerow B, Gandhi L, Xu X, Bendell J. Safety and Clinical Activity of a New Anti-PD-L1 Antibody as Monotherapy or Combined with Targeted Therapy in Advanced Solid Tumors: The PACT Phase Ia/Ib Trial. Clin Cancer Res. 2021 Mar 1;27(5):1267-1277. doi: 10.1158/1078-0432.CCR-20-2821. Epub 2020 Nov 23.

location_country:
Taiwan
Spain
Canada
Korea, Republic of
United States
France
Belgium

location_facility: University of Texas MD Anderson Cancer Center

location_facility: Severance Hospital Yonsei University Health System


acronym:
POC in lupus nephrits

 Preuve de concept dans la néphropathie lupique

title: A Double-blind, Randomised, Placebo-controlled Trial Evaluating the Effect of BI 655064 Administered as Sub-cutaneous Injections, on Renal Response After One Year of Treatment, in Patients With Active Lupus Nephritis

lead_sponsor: Boehringer Ingelheim

location_country:
Poland
Hong Kong
Portugal
Serbia
Greece
Italy
Australia
Spain
Canada
Thailand
United Kingdom
Philippines
Korea, Republic of
United States
France
Malaysia
Mexico
Japan
Germany
Czechia

location_facility: Angeles University Foundation Medical Center

location_facility: University Clinical Hospital in Bialystok I

location_facility: General University Hospital Prague 2, Nephrology Clinic

location_facility: Centro Hospitalar Universitário São João,EPE

location_facility: Columbia University Medical Center-New York Presbyterian Hospital

location_facility: Feinstein Institute for Medical Research

location_facility: Instituto Nacional de Cs Médicas y Nutrición S Zubiran

location_facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz

location_facility: CHUC - Centro Hospitalar e Universitário de Coimbra, EPE

location_facility: Universitätsklinikum Schleswig-Holstein, Campus Lübeck

location_facility: Integral Rheumatology and Immunology Specialist

location_facility: Hospital of the University of Occupational and Environmental Health

location_facility: University General Hospital of Heraklion

location_facility: St. Marianna University School of Medicine Hospital

location_facility: Norbert Barlicki University Clinical Hospital No.1, Lodz

location_facility: Azienda Ospedaliera Universitaria di Padova

location_facility: Instituto Nacional de Cardiologia Ignacio Chavez

location_facility: NZOZ Centrum Medyczne AESKULAP,Private Prac, Radom

location_facility: CHU de Quebec-Universite Laval Research Centre

summary: The overall purpose of the study is to assess the efficacy of three different doses of BI 655064 against placebo as add-on therapy to standard of care (SOC) treatment for active lupus nephritis in order to characterize the dose-response relationship within the therapeutic range, and select the target dose for phase III development.


title: An Exploratory, Multi-Center, Open-Label, Single-Arm Study to Evaluate the Discontinuation Effect of Clopidogrel After Drug Eluting Stent (DECADES) on Inflammatory and Platelet Activation Markers in Subjects Who Are Receiving Low Dose Acetylsalicylic Acid (ASA)

acronym: DECADES

summary: The purpose of the study is to look at the biomarkers of inflammation and platelet activation in patients with drug eluting stents implanted approximately 12 months ago on aspirin and statin, for a 4-week period after the routine discontinuation of clopidogrel

ReferenceCitation: Wykrzykowska JJ, Warnholtz A, de Jaeger P, Curzen N, Oldroyd KG, Collet JP, Ten Berg JM, Rademaker T, Goedhart D, Lissens J, Kint PP, Serruys PW. Effect of clopidogrel discontinuation at 1 year after drug eluting stent placement on soluble CD40L, P-selectin and C-reactive protein levels: DECADES (Discontinuation Effect of Clopidogrel After Drug Eluting Stent): a multicenter, open-label study. J Thromb Thrombolysis. 2009 Nov;28(4):410-7. doi: 10.1007/s11239-009-0354-y.

lead_sponsor: Bristol-Myers Squibb

location_country:
Germany
France
Netherlands
United Kingdom


title: A Phase 4, Double-Blind, Randomized, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy, Safety, and Tolerability of Mirabegron in Men With Overactive Bladder (OAB) Symptoms While Taking the Alpha Blocker Tamsulosin Hydrochloride for Lower Urinary Tract Symptoms (LUTS) Due to Benign Prostatic Hyperplasia (BPH)

acronym: PLUS

summary: The purpose of the study was to assess the efficacy, safety, and tolerability of mirabegron versus placebo in men with overactive bladder (OAB) symptoms while taking tamsulosin hydrochloride for lower urinary tract symptoms (LUTS) due to Benign Prostatic Hyperplasia (BPH).

ipd_sharing_description: Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as compounds terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Conditions and exceptions are described under the Sponsor Specific Details for Astellas on www.clinicalstudydatarequest.com.

lead_sponsor: Astellas Pharma Global Development, Inc.

location_country:
Poland
Spain
Italy
United Kingdom
Canada
United States
France
Germany
Czechia

location_facility:
Site PL48004
Site DE49007
Site CA15008
Site US01012
Site US01081
Site US01015
Site US01009
Site US01005
Site DE49004
Site CZ42004
Site IT39010
Site US01003
Site CZ42006
Site PL48005
Site ES34003
Site GB44005
Site ES34009
Site US01056
Site PL48007
Site ES34007
Site ES34002
Site DE49002
Site ES34006
Site CZ42001
Site DE49003
Site PL48006
Site US01029
Site US01075
Site IT39002
Site IT39004
Site GB44004
Site ES34004
Site DE49006
Site US01021
Site DE49005
Site US01070
Site IT39001
Site GB44011
Site US01079
Site DE49001
Site US01027
Site US01026
Site US01041
Site CA15002
Site CA15014
Site GB44002
Site FR33002
Site US01034
Site US01031
Site US01019
Site US01097
Site CZ42003
Site IT39006
Site CA15001
Site IT39005
Site FR33004
Site US01025
Site US01060
Site ES34001
Site US01069
Site US01061
Site US01063
Site CA15009
Site CA15003
Site US01067
Site US01078
Site IT39008
Site PL48001
Site GB44006
Site US01016
Site US01064
Site CA15011
Site CZ42005
Site CA15005
Site US01094
Site IT39003
Site US01072
Site US01042
Site US01068


title: Assessment of Histologic Prognostic Factors in Peritoneal Malignant Mesothelioma

summary: Malignant peritoneal mesothelioma is a rare neoplasm. The most common type, the epithelioid type, has been further divided into histological patterns of tubulo-papillary, acinar, adenomatoid, micropapillary, or solid. Its prognosis is improved by the use of a locoregional treatment combining extensive cytoreductive surgery (CRS) and hyperthermic intraperitoneal chemotherapy (HIPEC), which increases survival up to 50 months. Histology is one of the most important prognostic variable that, forms the basis for treatment decisions. However, the prognostic of the epithelioid type varies greatly due to its tumor heterogeneity. It is therefore necessary to find prognostic factors of malignant epithelioid peritoneal mesothelioma in order to better define the therapeutic strategy. Among histological factors, solid growth, tumor necrosis, nuclear atypia, and mitotic count were found to be independent prognostic factors in epithelioid malignant pleural mesothelioma. However, in epithelioid malignant peritoneal mesothelioma (EMPM), these factors were studied in small and heterogeneous series in terms of histological growth and definitions used for histological factors. The present large study was conducted to investigate the prognostic impact of several histologic factors in EMPM. Their prognosis impacts were assessed using overall survival (OS) and progression-free survival (PFS) in EMPM.

time_perspective: Retrospective

lead_sponsor: Hospices Civils de Lyon

location_country: Portugal India France Belgium Norway Germany

location_facility: Institut f. Pathologie und Neuropathologie