title: An Open-label, Long-term, Safety and Efficacy Study of Aticaprant as Adjunctive Therapy in Adult and Elderly Participants With Major Depressive Disorder (MDD)

acronym: VENTURA-LT

summary: The purpose of this study is to assess the long-term safety and tolerability of aticaprant administered as adjunctive therapy to a current antidepressant (selective serotonin reuptake inhibitor [SSRI] or serotonin and norepinephrine reuptake inhibitor [SNRI]) in all participants with major depressive disorder (MDD).

ipd_sharing_description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

lead_sponsor: Janssen Research & Development, LLC

location_country:
Italy
Poland
Slovakia
Sweden
Belgium
United States
Spain
South Africa
Czechia
Bulgaria
France

location_facility: Diagnostic Consulting Center Mladost - M Varna

location_facility: Wexner Medical Center at the Ohio State University

location_facility: Cabinet Medical des Drs Prizac-Desbonnet Scottez

location_facility: Liptovska Nemocnica S Poliklinikou Mudr. Ivana Stodolu

location_facility: Medical Research Group of Central Florida

location_facility: Specjalistyczna Indywidualna Praktyka Lekarska

location_facility: University of Alabama at Birmingham - The Kirklin Clinic

location_facility: Nemocnica s poliklinikou Prievidza so sidlom v Bojniciach

location_facility: Azienda Ospedaliera Universitaria Senese Policlinico Santa Maria alle Scotte

location_facility: Psychiatricka ambulance, MUDr. Marta Holanova

location_facility: University of Pennsylvania - Perelman School of Medicine

location_facility: DCC 'Sv. Vrach and Sv. Sv. Kuzma and Damyan', OOD

location_facility: Ambulatory for Individual Practice for Specialized Medical Care in Psychiatry Dr. Ivo Natsov ET

location_facility: Mlynowamed Specjalistyczny Psychiatryczny Gabinet Lekarski Joanna Lazarczyk

location_facility: Ambulatory Group practice for specialized help in psychiary Philipopolis ODD

location_facility: Psychiatric Care and Research Center (PCRC)


study_start_date_type: Actual

intervention_type: Drug

lead_sponsor: PTC Therapeutics

summary: The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone in Study PTC743-NEU-003-FA (NCT04577352) or Study PTC743-NEU-005-FA.

location_facility: Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares

location_facility: Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)

location_facility: CBR Neurogenetic Research Clinic, University of Auckland

location_facility: Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)

location_facility: University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology

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

status: Enrolling by invitation

title: Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

primary_purpose: Treatment

type: EudraCT Number

source: euctr

enrollment_type: Anticipated

study_completion_date_type: Anticipated

location_country:
Germany
Italy
New Zealand
Australia
Brazil
United States
Spain
Canada
France

study_type: Interventional

lead_sponsor_type: industriel

status_simplified: Unknown


lead_sponsor: Fore Biotherapeutics

summary: The objective of this study is to evaluate the efficacy of FORE8394 in participants with locally advanced or metastatic solid tumors, or recurrent or progressive primary central nervous system (CNS) tumors harboring BRAF fusions, or in participants with recurrent high-grade glioma (HGG) harboring BRAF V600E mutation. This will be conducted as two single arm open-label subprotocols (F8394-201A; F8394-201B) under one master protocol.

location_facility: University of California Los Angeles Rheumatology - Westwood

location_facility: Chonnam National University Hwasun Hospital

location_facility: Nebraska Cancer Specialists - Midwest Cancer Center - Legacy

location_facility: Catholic University of Korea Saint Vincent's Hospital

location_facility: Hospital Clinico Universitarlo de Santiago

location_facility: University of Miami Hospital and Clinics

location_facility: Hospital Clinico Universitarlo de Valencia

status: Recruiting

title: A Phase 2 Master Protocol to Assess the Efficacy and Safety of FORE8394, an Inhibitor of BRAF Class 1 and Class 2 Alterations, in Participants With Cancer Harboring BRAF Alterations

ipd_sharing_description: Fore is committed to sharing with qualified external researchers access to deidentified patient-level data and related study documents (eg. study protocol) from eligible studies following publication of the study results.

design_allocation: Non-Randomized

location_country:
France
United Kingdom
United States
Spain
Korea, Republic of
Sweden

ipd_sharing: Yes

status_simplified: Ongoing


acronym: Cielo

lead_sponsor: Hoffmann-La Roche

summary: The purpose of this study is to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab in participants with anti-N-methyl-D-aspartic acid receptor (NMDAR) and anti-leucine-rich glioma-inactivated 1 (LGI1) encephalitis.

sponsor_collaborators:
Hoffmann-La Roche
Chugai Pharmaceutical Co.

location_facility: Hopital neurologique Pierre Wertheimer - CHU Lyon; Neurologie

location_facility: Guangzhou First Municipal People's Hospital

location_facility: Fakultni nemocnice v Motole; Neurologicka klinika 2. LF UK a FN Motol

location_facility: IRCCS Ospedale San Raffaele; Neurologia Neurofisiologia Neuroriabilitazione-Centro Sclerosi Multipla

location_facility: Fujian Medical University Union Hospital

location_facility: Beijing Tiantan Hospital,Capital Medical University

location_facility: Beijing Children's Hospital, Capital Medical University

location_facility: Hyogo prefectural Kobe Children's Hospital

location_facility: The Second Xiangya Hospital of Central South University

location_facility: Fondazione Istituto Neurologico Mondino IRCCS

location_facility: Kinki University Hospital, Faculty of Medicine

location_facility: A. O. U. Federico II; Dip Neuroscienze, Scienze Riproduttive ed Odontostomatologiche

location_facility: UCSF- Multiple Sclerosis Centre; Department of Neurology

location_facility: University of Colorado; Anschutz Medical Campus Department of Neurology

location_facility: The First Affiliated Hospital of Wenzhou Medical University

location_facility: University of Maryland Medical Center; Department of Neurology

location_facility: The First Hospital of Shanxi Medical University

location_facility: AOU Policlinico Giaccone; UOC Neurologia e Neurofisiopatologia-Amb Sclerosi Multipla

location_facility: Kepler Universitätsklinikum GmbH - Neuromed Campus; Innere Medizin mit Neuroonkologie

location_facility: Tongji Hospital Tongji Medical College Huazhong University of Science and Technology

location_facility: St.Marianna University School of Medicine hospital; Medical Oncology

location_facility: Instytut Psychiatrii i Neurologii II Klinika Neurologiczna

location_facility: Regionalny Szpital Specjalistyczny im. W. Bieganskiego; Oddzial Neurologiczny

location_facility: Odense Universitetshospital, Neurologisk Afdeling N

location_facility: West China Hospital - Sichuan University

location_facility: Szpital Uniwersytecki w Krakowie; Oddzia? kliniczny Neurologii

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta

location_facility: Affiliated Hospital of Jining Medical University

location_facility: AOU Seconda Università degli Studi; Dip.Assistenziale Integrato Medicina Int-I Clinica Neurologica

title: A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Basket Study To Evaluate The Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Anti-N-Methyl-D-Aspartic Acid Receptor (NMDAR) Or Anti-Leucine-Rich Glioma-Inactivated 1 (LGI1) Encephalitis

ipd_sharing_description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.clinicalstudydatarequest.com). Further details on Roche's criteria for eligible studies are available here (https://clinicalstudydatarequest.com/Study-Sponsors/Study-Sponsors-Roche.aspx). For further details on Roche's Global Policy on Sharing of Clinical Study 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)

design_allocation: Randomized

location_country:
Italy
Poland
Argentina
Denmark
Japan
Brazil
Netherlands
Austria
United States
China
Korea, Republic of
Taiwan
Czechia
France


type: Registry Identifier

title: An 18-month, Open-label, Single-arm Safety Extension Study of an age-and Bodyweight-adjusted Oral Finerenone Regimen, in Addition to an ACEI or ARB, for the Treatment of Children and Young Adults From 1 to 18 Years of Age With Chronic Kidney Disease and Proteinuria

acronym: FIONA OLE

summary:
Researchers are looking for a better way to treat children who have chronic kidney disease (CKD), which is long-term kidney disease, and proteinuria, a condition in which a person´s kidneys leak protein into the urine.

The kidneys filter waste and fluid from the blood to form urine. In children with CKD, the kidney´s filters do not work as well as they should. This can lead to accumulation of waste and fluid in the body and proteinuria. CKD can lead to other medical problems, such as high blood pressure, also known as hypertension. Vice versa, hypertension and proteinuria can also contribute to worsening of CKD. Therefore, the treatment of CKD aims to control blood pressure and proteinuria. There are treatments available for doctors to prescribe to children with CKD and hypertension and/or proteinuria. These include "angiotensin-converting enzyme inhibitors" (ACEI) and "angiotensin receptor blockers" (ARB). Both ACEI and ARB can help improve kidney function by reducing the activity of the renin-angiotensin-aldosterone system (RAAS). The RAAS is a system that works with the kidneys to control blood pressure and the balance of fluid and electrolytes in the blood. In people with CKD, the RAAS is often too active, which can impair the ability of the kidneys to work properly and cause hypertension and proteinuria. However, ACEI or ARB treatment alone does not work for all patients with CKD as they only target the angiotensin part of the renin-angiotensin-aldosterone system.

The study treatment, finerenone, is expected to help control RAAS overactivation together with an ACEI or ARB.

So, the researchers in this study want to learn more about whether finerenone given in addition to either an ACEI or ARB can help their kidney function.

The main purpose of this study is to learn how safe the treatment is when used of finerenone in addition to an ACEI or ARB in long-term.

To see how safe the treatment is, the study team will collect information on medical problems which are also known as "treatment emergent adverse events" (TEAEs). And they will also collect levels of an electrolyte called potassium in the blood by taking blood samples, and measure blood pressure during the study.

The secondary purpose of this study is to learn how well long-term use of finerenone can reduce the amount of protein in the participants' urine and benefit kidney function when taken with standard of care.

To see how the treatment works, the study team will collect participants' urine samples to assess urinary albumin-to-creatinine ratio (UACR) and urinary protein-to-creatinine ratio (UPCR), which are important assessments for calculating the level of protein in the urine. Researchers will also collect blood samples to analyze serum creatinine and calculate estimated glomerular filtration rate (eGFR). A significant decline in eGFR indicates worsening kidney function.

The study will include participants who had previously participated in FIONA study (NCT05196035). The participants will be aged from 1 year up to 18 years.

The participants will be in the study for approximately 19 months. They will take study treatment for up to 18 months and will be follow up for 1 month. During this period, at least 12 visits are planned for patients who newly start finerenone, and at least 8 visits for patients who already received finerenone.

In the visit, the study team will:

have their blood pressure, heart rate, temperature, height and weight measured
have blood and urine samples taken
have physical examinations
have their heart examined by an electrocardiogram and echocardiography (a sonogram of the heart)
answer questions about their medication and whether they have any adverse events, or have their parents or guardian's answer
answer questions about how they are feeling, or have their parents or guardian's answer
answer question about how they like the study medication, or have their parents or guardian's answer The doctors will keep track of any adverse events. An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.

The doctors will check the participants' health about 30 days after the participants take their last treatment.

ipd_sharing: No

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

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

lead_sponsor: Bayer

location_country:
Finland
Germany
Poland
Argentina
Australia
Turkey
Czechia
Canada
France
Lithuania
Portugal
Hungary
Denmark
Switzerland
Belgium
Italy
United Kingdom
Sweden
Netherlands
Austria
Korea, Republic of
Spain
Greece
Israel


title: An Open-label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients Aged 4 to 11 Years With Activated Phosphoinositide 3-Kinase Delta Syndrome Followed by an Open-label Long-term Extension

summary: This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 4 to 11 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).

lead_sponsor: Pharming Technologies B.V.

sponsor_collaborators:
Pharming Technologies B.V.
CMIC Co, Ltd. Japan
Labcorp Central Laboratory
Fortrea
Aixial Group

location_country:
United States
Japan
Netherlands
France

location_facility: Texas Children's Hospital Infusion Center

location_facility: Tokyo Medical And Dental University Hospitall


title: An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)

acronym: BOLD-EXT

summary: An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia

lead_sponsor: Albireo

location_country:
Germany
Italy
Poland
New Zealand
Australia
Netherlands
United States
Spain
Malaysia
Turkey
Canada
France

location_facility: University Children´s Hospital Tuebingen

location_facility: The Childrens Hospital at Montefiore Albert Einstein School of Medicine

location_facility: Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens Hospital

location_facility: Children's Healhcare of Atlanta- Emory University school of medicine

location_facility: Starship Child Health, Te Whatu Ora - Health New Zealand

location_facility: Hassenfeld Children's Hospital at NYU Langone


title: Microbiome-based Stratification of Individuals at Risk of HIV-1 Acquisition, Chronic Clinical Complications, Antimicrobial Drug Resistance, and Unresponsiveness to Therapeutic HIV-1 Vaccination

acronym: MISTRAL

summary:
MISTRAL (Microbiome-based stratification of individuals at risk of HIV-1 acquisition, chronic clinical complications, antimicrobial drug resistance, and unresponsiveness to therapeutic HIV-1 vaccination) is a 5-year EU Horizon 2020 project, running from 1/1/2020 - 31/12/2024.

The project is led by Fundacio Privada Institut de Recerca de la Sida-Caixa CAIXA in Barcelona and aims to explore the gut microbiota in relation to HIV-1, seeking microbiome biomarkers to support development of interventions that mitigate infection and enhance response to vaccines and therapies.

If successful, MISTRAL will benefit millions of human beings living with, or at risk of acquiring HIV-1 infection, and will produce novel concepts and technical innovations applicable to other human diseases. By doing that, MISTRAL will help to unlock the full clinical potential of the human microbiome to stratify patient outcomes and will irreversibly bring microbiome science closer to clinical practice

study_start_date_type: Anticipated

status: Not yet recruiting

study_type: Observational

time_perspective: Prospective

ReferenceType: background

ReferenceCitation: Li SX, Armstrong A, Neff CP, Shaffer M, Lozupone CA, Palmer BE. Complexities of Gut Microbiome Dysbiosis in the Context of HIV Infection and Antiretroviral Therapy. Clin Pharmacol Ther. 2016 Jun;99(6):600-11. doi: 10.1002/cpt.363. Epub 2016 Apr 16.

ReferenceCitation: Herrera S, Martinez-Sanz J, Serrano-Villar S. HIV, Cancer, and the Microbiota: Common Pathways Influencing Different Diseases. Front Immunol. 2019 Jun 27;10:1466. doi: 10.3389/fimmu.2019.01466. eCollection 2019.

ReferenceCitation: Hsu DC, Sereti I, Ananworanich J. Serious Non-AIDS events: Immunopathogenesis and interventional strategies. AIDS Res Ther. 2013 Dec 13;10(1):29. doi: 10.1186/1742-6405-10-29.

lead_sponsor: Rigshospitalet, Denmark

sponsor_collaborators:
Rigshospitalet, Denmark
IrsiCaixa
Horizon 2020 - European Commission

location_country:
Germany
Italy
Poland
Hungary
Israel
Denmark
United Kingdom
Belgium
Spain
Luxembourg
Greece
Croatia
Czechia
France

location_facility: Royal Sussex County Hospital, HIV/GUM Research

location_facility: Hosp. Universitari Germans Trias i Pujol, Hospital de dia VIH

location_facility: University College London, Mortimer Market Centre

location_facility: University Hospital of Infectious Diseases

location_facility: Hospital Clinic - IDIBAPS. University of Barcelona, C/Villarroel, 170

location_facility: Wojewodzki Szpital Specjalistyczny im. WI. Bieganskiego

location_facility: University Hospital Bonn, Klinisches StudieZentrum Immun.

lead_sponsor_type: academique


title: A Phase 2B, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Rencofilstat in Adult Subjects With Nonalcoholic Steatohepatitis and Advanced Liver Fibrosis

acronym: ASCEND

summary: This is a randomized, double-blind, placebo-controlled, parallel-dosing, multi-center study to evaluate the efficacy and safety of Rencofilstat as evidenced by histopathological improvements in fibrosis in adult NASH subjects with F2 or F3 fibrosis (NASH CRN system). Antifibrotic biomarker activity will be evaluated on an exploratory basis.

lead_sponsor: Hepion Pharmaceuticals, Inc.

location_country:
United States
Mexico
France

location_facility: Bon Secours Liver Institute of Hampton Roads

location_facility: Digestive Research Alliance of Michiana, LLC

location_facility: Gastrointestinal Specialists of Georgia, PC

location_facility: Centro de Investigacion y Gastroenterologia SC


title: Endpoint Enabling Study of Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD)

acronym: CANDID

summary: Observational, noninterventional, 3-year study to examine the presence of detectable differences in neurocognitive, developmental, motor, neurophysiologic, and quality of life measures over time in patients with cyclin-dependent kinase-like 5 deficiency disorder.

lead_sponsor: Loulou Foundation

location_country:
Germany
Italy
United Kingdom
United States
Spain
Canada
France

location_facility: Azienda Ospedaliera Universitaria Integrata di Verona

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: University Hospitals Bristol and Weston NHS Foundation Trust

location_facility: Fondazione Policlinico Universitario A Gemelli, IRCCS

location_facility: Alberta Children's Hospital, Pediatric Epilepsy and Child Neurology

location_facility: DRK Berlin - Epilepsiezentrum und Neuropädiatrie

location_facility: Hospital Infantil Universitario Niño Jesús

location_facility: Childrens Hospital of Philadlephia Division of Neurology

intervention_type: Other


title: A Phase 3b, Open-label, Multi-country, Multi-centre, Long-term Follow-up Study of ZOSTER-049 (Follow-up of ZOSTER-006/022 Studies) to Assess the Prophylactic Efficacy, Safety and Persistence of Immune Response of a Herpes Zoster Subunit Vaccine and Assessment of Persistence of Immune Response and Safety of 1 or 2 Additional Doses Administered in ZOSTER-049 in 2 Subgroups of Older Adults

summary: The purpose of the current ZOSTER-101 long-term follow-up (LTFU) study of ZOSTER-049 (NCT02723773) study, an extension of ZOSTER-006 (NCT01165177) and ZOSTER-022 (NCT01165229) primary studies, is to assess the long-term vaccine efficacy (VE) against Herpes Zoster (HZ) (approximately 11-15 years post primary vaccination in ZOSTER-006/022 studies), persistence of immunogenicity and safety of GSK's Herpes Zoster subunit (HZ/su) vaccine in older adults. The persistence of immunogenicity and safety of 1 or 2 additional doses (0, 2-month schedule) of HZ/su vaccine administered to a small group of participants in ZOSTER-049 study (approximately 5 years after the initial vaccination in ZOSTER-006/022 studies) will also be assessed.

primary_purpose: Prevention

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

lead_sponsor: GlaxoSmithKline

location_country:
Finland
Germany
Australia
Japan
Sweden
Mexico
United States
Korea, Republic of
Taiwan
Canada
Estonia
France

intervention_type: Biological


title: A Phase 3, Multicenter, Randomized, Double-Blind Study to Evaluate the Safety and Efficacy of Contezolid Acefosamil and Contezolid Compared to Linezolid Administered Intravenously and Orally to Adults With Moderate or Severe Diabetic Foot Infections (DFI)

summary: This is a Phase 3, multicenter, randomized, double-blind, safety and efficacy study of contezolid acefosamil (IV)/contezolid (PO) compared with linezolid (IV and PO) administered for a total of 14 to 28 days in adult subjects with moderate or severe DFI.

lead_sponsor: MicuRx

location_country:
Italy
Lithuania
Portugal
United States
China
Latvia
Spain
Greece
Bulgaria
Estonia
Puerto Rico
France

location_facility: Clinical Research Management Group, Inc.

location_facility: Medical Institute Ministry of Interior Central Clinical Base

location_facility: MHAT Blagoevgrad AD, Department of Surgery

location_facility: Vilnius University Hospital Santaros Klimkos

location_facility: Chu Hsien-I Memorial Hospital of Tianjin Medical University

location_facility: The Second Affiliated Hospital of Guangxi Medical University

location_facility: AORN - Ospedali dei Colli - Ospedale D. Contugno

location_facility: Hainan Medical College No. 2 Affiliated Hospital

location_facility: Multiprofile Hospital for Active Treatment - KANEV

location_facility: Hospital of Lithuanian University of Health Sciences

location_facility: The Second Affiliated Hospital of Chongqing Medical University

location_facility: The First Hospital of Hebei Medical University

location_facility: Affiliated Hospital of Jiangsu University

location_facility: The Second Affiliated Hospital of Shantou University Medical Center

location_facility: Union Hospital Tongji Medical College Huazhong University

location_facility: Xi'an Medical University First Affiliated Hospital

location_facility: Affiliated Hospital of Yunnan University

location_facility: University Medical and Research Center, LLC


title: FINE-REAL: A Non-interventional Study Providing Insights Into the Use of Finerenone in a Routine Clinical Setting

summary:
This is an observational study in people with chronic kidney disease (CKD) and type 2 diabetes (T2D) who will be receiving finerenone.

Kidneys filter extra water and waste out of the blood and make urine. CKD is a long-term, progressive, decrease in the kidneys' ability to filter the blood properly. In people with T2D, the body does not make enough of a hormone called insulin, or does not use insulin well enough, resulting in high blood sugar levels that can cause damage to the kidneys. As a result, CKD can occur as a complication of T2D.

Finerenone works by blocking certain proteins, called mineralocorticoid receptors. An increased stimulation of these proteins is thought to damage the kidneys and the heart. By lowering their stimulation, finerenone reduces the risk of kidney disease progressively getting worse.

Finerenone is available and approved for doctors to prescribe to people with CKD and T2D. Since it has only recently become available for these patients, there is a need for more information about the use of finerenone in the real-world setting.

The main purpose of the study is to learn more about treatment patterns in people with CKD and T2D who just started or will start finerenone treatment as decided and prescribed by their doctor as part of their routine medical care.

To answer this question, the researchers will collect data on:

Clinical characteristics (e.g., history of CKD and T2D, blood pressure, heart health) of the participants
Reasons for starting finerenone
Reasons for stopping finerenone early
How long participants have been taking finerenone (planned by their doctor compared to actual time it was taken)
Dosing of finerenone
Other medications used while taking finerenone

The researchers will also collect data on medical problems (called adverse events) that the participants may have during the study. All adverse events are collected, even if they might not be related to the study treatment.

Hyperkalemia, a medical term used to describe a potassium level in the blood that is higher than normal, is of special interest when finerenone is combined with some medications commonly taken to control blood pressure. Researchers want to know how often higher potassium levels occur, and when it leads to:

Stopping finerenone treatment too early
Dialysis (a medical procedure to filter the blood of extra water and waste)
Care in a hospital

All data will come from medical records or from interviews study doctors will have with the participants during visits that take place during routine medical care.

Participants in the US will be invited to provide voluntary blood and urine samples that could be analyzed later to better understand possible changes in protein or nucleic acid levels over time.

Each participant will be in the study for 12 months. This time participating in the study may be shorter if their finerenone treatment is stopped early or the study comes to an end as planned in September 2027.

location_country:
Germany
Argentina
Brazil
Thailand
Luxembourg
Chile
Canada
France
Slovenia
Colombia
Denmark
Switzerland
United States
Belgium
China
Italy
Singapore
Netherlands
Mexico
Korea, Republic of
Saudi Arabia
Spain
Taiwan
Russian Federation

location_facility: University of Maryland Midtown Professionals Group Healthcare

location_facility: Jackson Medical Mall University Home Dialysis Clinic

location_facility: Academy of Diabetes Thyroid and Endocrine

location_facility: MultiCare Endocrinology Specialists Tacoma

location_facility: University of Central Florida College of Medicine

location_facility: Kidney and Hypertension Center of Wabash Valley LLC

location_facility: Nephrology Associates Northern Illinois and Indiana

location_facility: Keck School of Medicine of University of Southern California

location_facility: University of Iowa Preventive Intervention Clinic


acronym: GYROS

summary: The Gyrate Atrophy Ocular and Systemic Study characterizes the natural history of ornithine levels and retinal degeneration (RD) associated with disease-causing OAT variants in the presence of standard care dietary treatment regimens over 4 years. The research goal is to understand the impact of OAT mutations on plasma ornithine levels and retinal degeneration.

ipd_sharing_description: After the study is completed, the de-identified, archived data will be transmitted to and stored at the FFB Consortium Coordinating Center, under the supervision of Allison Ayala, for use by other researchers including those outside of the study.

lead_sponsor: Jaeb Center for Health Research

sponsor_collaborators:
Jaeb Center for Health Research
Conquering Gyrate Atrophy Foundation
Food and Drug Administration (FDA)
Department of Health and Human Services

location_country:
Finland
Germany
United Kingdom
Brazil
United States
Canada
France

location_facility: Center for Advanced Retinal and Ocular Therapeutics

location_facility: Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, INSERM-DGOS CIC1423

location_facility: Johns Hopkins University, Wilmer Eye Institute

location_facility: Harvard Univ., Massachusetts Eye and Ear Infirmary

location_facility: University of Toronto, Hospital for Sick Children

location_facility: University of Tuebingen, Centre for Ophthalmology


title: Non-Arteritic Anterior Ischemic Optic Neuropathy Risk Factors: New Perspectives

acronym: NARROW

summary: The purpose of the study is to use new diagnostic methods (OCT and OCT-A) to shed light on risk factors for the development of NA-AION. The risk factors we are focusing on are comorbidities along with anatomical and vascular characteristics of the optic nerve.

collaborators: Moorfields Eye Hospital NHS Foundation Trust

location_country:
New Zealand
Israel
United Kingdom
Denmark
Australia
Iran, Islamic Republic of
United States
Canada
France

location_facility:
UCSF Medical Center
Research St. Joseph's
Zealand University Hospital
Moorfield's Eye Hospital
Odense University Hospital
Aalborg University Hospital
Capital and Coast DHB
John A. Moran Eye Center
Lawson Health Research Institute
Massachusetts Eye and Ear
University og Colorado
Sheba Medical Center
Stanford Medicine
University of Calgary
King's College Hospital
Sydney Eye Hospital
University of Cambridge
Bordeaux University Hospital
Aarhus University Hospital
Farabi Eye Hospital


title: An Open-label Trial of the Long-term Safety and Tolerability of Nintedanib Per os, on Top of Standard of Care, Over at Least 2 Years, in Children and Adolescents With Clinically Significant Fibrosing Interstitial Lung Disease (InPedILD®-ON)

summary:
This study is open to children and adolescents with interstitial lung disease (ILD) that causes lung fibrosis. This is a study for people who took part in a previous study (study 1199-0337, InPedILD™) and for people who are between 6 and 17 years old and have fibrosing ILD.

This study tests a medicine called nintedanib. Nintedanib is already used to treat different types of lung fibrosis in adults. The purpose of the study is to find out how well long-term treatment with nintedanib is tolerated in children and adolescents.

All participants take nintedanib capsules twice a day. Participants are in the study for at least 6 months or until nintedanib or other treatment options become available outside of this study.

During the first 2 years, they visit the study site between 9 and 11 times. Afterwards, they visit the study site every 3 months.

The doctors collect information on any health problems of the participants.

ipd_sharing_description:
Once the criteria in section "Time Frame" are fulfilled, researchers can use the following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement".

Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.

lead_sponsor: Boehringer Ingelheim

location_country:
Finland
Germany
Italy
Poland
Portugal
Norway
Argentina
United Kingdom
Brazil
Netherlands
Mexico
Belgium
United States
Spain
Greece
Czechia
Canada
France

location_facility: Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center

location_facility: Teaching Hospital Motol, Oncology Clinic

location_facility: Oslo Universitetssykehus HF, Rikshospitalet

location_facility: CHUP, EPE - Centro Materno Infantil do Norte

location_facility: Independent Public Teaching Children's Hospital

location_facility: Hamburger Zentrum für Kinder- und Jugendrheumatologie

location_facility: Hospital de Pediatría " Prof. Dr. Juan P. Garrahan"

location_facility: Serviços Medicos Respirar Sul Fluminense

location_facility: Centro Hospitalar Universitário de Lisboa Norte, EPE - Hospital Santa Maria

location_facility: Centro de Pesquisa Clinica do Instituto da Crianca - HCFMUSP

location_facility: General Hospital of Thessaloniki "Ippokrateio"


title: A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy

acronym: CARAT

summary:
This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy.

Study visits will take place approximately every 3 to 6 months
Participants who complete the randomized period may continue to the long-term extension (LTE) to receive venglustat for up to additional 34 months with the total study duration up to 4.4 years maximum.

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

lead_sponsor: Sanofi

location_country:
Germany
Poland
Japan
Turkey
Czechia
Canada
France
Denmark
United States
China
Italy
Norway
United Kingdom
Netherlands
Austria
Korea, Republic of
Spain
Greece
Taiwan

location_facility: University of Alabama at Birmingham (UAB) - The Kirklin Clin Site Number : 8400010

location_facility: Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 8400004


type: Other Identifier

title: A Phase 2, Open-label, Single-arm, Multicenter Study of SOT101 in Combination With Pembrolizumab to Evaluate the Efficacy and Safety in Patients With Selected Advanced/Refractory Solid Tumors

summary: The primary objective of the study is to estimate the antitumor efficacy of SOT101 in combination with pembrolizumab in selected tumors.

lead_sponsor: SOTIO Biotech AG

sponsor_collaborators:
SOTIO Biotech AG
Merck Sharp & Dohme LLC

location_country:
Italy
Hungary
Belgium
United States
Spain
Czechia
France

location_facility: Azienda Socio Sanitaria Territoriale (ASST) degli Spedali Civili di Brescia

location_facility: Universitair Ziekenhuis Leuven - Campus Gasthuisberg

location_facility: Clinica Universidad de Navarra - Pamplona

location_facility: Centre Hospitalier Universitaire de Liège

location_facility: Petz Aladár Egyetemi Oktató Kórház - Győr

location_facility: Hospital Clínico Universitario de Valencia

location_facility: Azienda Ospedaliera Universitaria Senese - L'ospedale Santa Maria alle Scotte

location_facility: Hospital Universitario Puerta de Hierro - Majadahonda

location_facility: Hospital Teresa Herrera - Materno Infantil


title: Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects

acronym: DRAGON

summary: The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1

status: Active, not recruiting

enrollment_type: Actual

lead_sponsor: Belite Bio, Inc

location_country:
Germany
United Kingdom
Hong Kong
Australia
Netherlands
Switzerland
Belgium
United States
China
Taiwan
France


title: Optimization of Prostate Biopsy - Micro-Ultrasound Versus MRI: A 3-arm Randomized Controlled Trial Evaluating the Role of 29MHz Micro-ultrasound in Guiding Prostate Biopsy in Men With Clinical Suspicion of Prostate Cancer.

acronym: OPTIMUM

summary: OPTIMUM is a study designed to compare the ability of ultra-high resolution transrectal micro-ultrasound (microUS) and multiparametric MRI (mpMRI)/US fusion to guide prostate biopsy.

primary_purpose: Diagnostic

lead_sponsor: Sunnybrook Health Sciences Centre

sponsor_collaborators:
Sunnybrook Health Sciences Centre
Exact Imaging

location_country:
Germany
Austria
Belgium
United States
Spain
Canada
France

location_facility: University Health Network - Princess Margaret Hospital

location_facility: Sunnybrook and Women's College Health Sciences Centre

intervention_type: Diagnostic Test


title: A First-in-Human Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, as a Single Agent in Advanced Solid Tumor Patients and in Combination With Fulvestrant in Patients With Advanced Breast Cancer

summary: This is an open-label, FIH study designed to evaluate the maximum tolerated dose, recommended Phase 2 dose, safety, tolerability, PK, pharmacodynamics, and preliminary antineoplastic activity of RLY-2608, in advanced solid tumor patients with a Phosphatidylinositol-4,5-bisphosphate-3 kinase, catalytic subunit alpha (PIK3CA) mutation in blood and/or tumor per local assessment. The study will evaluate RLY-2608 as a single agent for patients with unresectable or metastatic solid tumors, RLY-2608 + fulvestrant and RLY-2608 + fulvestrant + CDK4/6 inhibitor (palbociclib or ribociclib) combination arms for patients with HR+ HER2- locally advanced or metastatic breast cancer. The RLY-2608 single agent arm, RLY-2608 + fulvestrant combination arm, and triple combination arms will have 2 parts: a dose escalation (Part 1) and a dose expansion (Part 2).

lead_sponsor: Relay Therapeutics, Inc.

location_country:
United States
Italy
Spain
France

location_facility: The University of Texas M.D. Anderson Cancer Center

location_facility: Boca Raton Clinical Research (BRCR) Global

location_facility: START Madrid - Hospital Fundacion Jimenez Diaz

location_facility: Institut Catala D'Oncologia - Badalona (ICO Badalona)

location_facility: Columbia University Herbert Irving Comprehensive Cancer Center


title: A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months

acronym: PERIDOT

summary:
This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo controlled study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in participants ≥16 years of age with Fabry disease who are treatment-naïve or untreated for at least 6 months.

Study visits will take place approximately every 3 months.
The double-blind period will be followed by an open-label extension (OLE) during which participants who have completed the double-blind period will be treated with venglustat for up to an additional 12 months.

location_country:
Finland
Germany
Poland
Argentina
Australia
Brazil
Japan
Turkey
Canada
France
Denmark
United States
Romania
China
Italy
Norway
United Kingdom
Austria
Mexico
Greece

location_facility: No.85 South Jiefang road, Yingze District_Site Number: 1560004

location_facility: Azienda Ospedaliera Universitaria "Federico II", U.O. di Nefrologia- Diparimento di Sanità Pubblica_Investigational Site Number: 3800001

location_facility: Fondazione IRCCS San Gerardo dei Tintori, S.C. Nefrologia - Clinica Nefrologica_Investigational Site Number: 3800002

location_facility: Hospital de Clínicas de Porto Alegre_Investigational Site Number: 0760001

location_facility: Oregon Health and Sciences University_Investigational Site Number: 8400007

location_facility: Tohoku University Hospital_Investigational Site Number: 3920001

location_facility: Fondazione Policlinico Universitario_Investigational Site Number: 3800004

location_facility: Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004

location_facility: The Jikei University Hospital_Investigational Site Number: 3920003

location_facility: Advent Health Orlando_Investigational Site Number: 8400008

location_facility: Kagoshima University Hospital_Investigational Site Number: 3920002

location_facility: Instituto de Nefrologia Pergamino_Investigational Site Number: 0320001

location_facility: ISphinCS GmbH_Investigational Site Number: 2760004

location_facility: University Hospital of Ioannina_Investigational Site Number: 3000002

location_facility: Azienda Ospedaliera Universitaria_Investigational Site Number: 3800003

location_facility: Westchester Medical Center Healthcare Corporation Site Number : 8400001

location_facility: Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012

location_facility: M.A.G.I.C Calgary LTD_Investigational Site Number: 1240003

location_facility: No.197,2nd Ruijin road, Huangpu district_Site Number: 1560003

location_facility: Instituto de Investigaciones Clínicas Quilmes (IICQ) SRL_Investigational Site Number: 0320003

location_facility: Fundacion Cori para la Investigación y Prevención del Cancer_Investigational Site Number: 0320002

location_facility: UCLA Medical Center_Investigational Site Number: 8400006

location_facility: Fukuoka University Hospital_Investigational Site Number: 3920004

location_facility: Hospital Universitario "Dr. José Eleuterio González" Departamento de Genética Centro Universitario contra el cáncer_Investigational Site Number: 4840001

location_facility: No.8, Xishiku Street, Xicheng District_Site Number: 1560001

location_facility: Odette del Carmen DIAZ-AVENDAÑO Clinstile, S.A. de C.V. Durango_Investigational Site Number: 4840002

location_facility: Institutul Clinic Fundeni_Investigational Site Number: 6420001

location_facility: Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011

location_facility: University Hospital of Heraklion_Investigational Site Number: 3000001


title: The SPYRAL AFFIRM Global Clinical Study of Renal Denervation With the Symplicity Spyral Renal Denervation System in Subjects With Uncontrolled Hypertension (SPYRAL AFFIRM)

summary:
The purpose of this single-arm interventional study is to evaluate the long-term safety, efficacy, and durability of the Symplicity Spyral system in subjects treated with renal denervation.

Additionally, long-term follow-up data will also be collected from eligible subjects previously treated in the SPYRAL PIVOTAL-SPYRAL HTN-OFF MED and SPYRAL HTN-ON MED studies.

lead_sponsor: Medtronic Vascular

location_country:
Germany
United Kingdom
Netherlands
Belgium
United States
France

location_facility: University of Colorado Anschutz Medical Campus

location_facility: Ziekenhuis Oost Limburg - Campus Sint-Jan

location_facility: Metropolitan Heart and Vascular Institute (MHVI)

location_facility: Providence Regional Medical Center Everett

location_facility: Baylor Jack and Jane Hamilton Heart and Vascular Hospital

location_facility: University of Alabama at Birmingham (UAB) Hospital

location_facility: Baylor Scott & White The Heart Hospital Baylor Plano

location_facility: Hôpital Saint André -Centre Hospitalier Universitaire de Bordeaux

location_facility: Hospital of the University of Pennsylvania

location_facility: Algemeen Stedelijk Ziekenhuis - R&D Hartziekten

location_facility: Orlando Health Heart & Vascular Institute

intervention_type: Device


acronym: ACTIVATE-Kids

lead_sponsor: Agios Pharmaceuticals, Inc.

summary: Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not regularly receiving blood transfusions. Participants will be randomized 2:1 to receive either mitapivat or matching placebo. Randomization will be stratified by age (1 to < 6 years, 6 to < 12 years, 12 to < 18 years). Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 12-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat for up to 5 years in the open-label extension (OLE) period.

location_facility: Centre hospitalier Universitaire de Sainte-Justine

location_facility: Charite - UB - CVK - Medizinische Klinik

location_facility: Children's Healthcare of Atlanta - Emory

location_facility: Hospital Universitario Infantil Nino Jesus

location_facility: Ospedale Pediatrico Bambino Gesu Oncoematologia

title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

location_country:
Germany
France
Italy
Netherlands
Switzerland
United States
Spain
Canada
Israel


title: The Oxford Risk Factors And Non-Invasive Imaging Study

acronym: ORFAN

summary: ORFAN is a prospective, multi-centre, multi-ethnic cohort observational study collecting CT scans, biological material and outcomes data, to develop and validate novel biomarkers of cardiometabolic and other disease risk.

time_perspective: Other

lead_sponsor: University of Oxford

collaborators: National Consortium of Intelligent Medical Imaging

location_country:
Germany
Italy
Hungary
United Kingdom
Romania
Australia
Japan
Netherlands
Belgium
United Arab Emirates
United States
China
Korea, Republic of
Greece
France

location_facility: Royal United Hospitals Bath NHS Foundation Trust

location_facility: University Hospital of Manchester NHS Foundation Trust

location_facility: MTA-SE Cardiovascular Imaging Research Group

location_facility: Heart and Lung Centre, New Cross Hospital

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

location_facility: University of Edinburgh, Royal Infirmary

location_facility: TEDA International Cardiovascular Hospital

location_facility: University Hospitals Birmingham NHS Trust

location_facility: Oxford University Hospitals NHS Foundation Trust

location_facility: University of Medicine and Pharmacy of Targu Mures

location_facility: NIH National Heart, Lunch, and Blood Institute

location_facility: Centro Cardiologico Monzino IRCCS, University of Milan

location_facility: University Hosptials of Leicester NHS Trust

location_facility: Cleveland Clinic Heart and Vascular Institute

location_facility: Sandwell and West Birmingham Hospitals NHS Trust


title: AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF MARSTACIMAB PROPHYLAXIS IN SEVERE (COAGULATION FACTOR ACTIVITY <1%) HEMOPHILIA A PARTICIPANTS WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY ≤2%) WITH OR WITHOUT INHIBITORS

summary:
Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study B7841005. Approximately 145 adolescent and adult participants 12 to <75 years of age with severe hemophilia A or moderately severe to severe hemophilia B (defined as FVIII activity <1% or FIX activity ≤2%, respectively) with or without inhibitors are expected to be enrolled in this study during which they will receive prophylaxis (defined as treatment by SC injection of marstacimab). The dosing regimen of marstacimab is 150 mg SC once weekly.

All participants will be provided the PFP for administration of marstacimab in the study. Use of the PFS will be permitted at the investigator's discretion for those participants who have difficulty with administration of the PFP. Additionally, participants will be provided the PFS for use in this study in countries where the PFS is anticipated to be the only presentation available commercially. In addition, an optional, open-label, single arm, substudy using the PFP will be administered to the first approximately 20 participants rolling over from Study B7841005 who agree to participate in the substudy.

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

lead_sponsor: Pfizer

location_country:
Italy
India
Hong Kong
Japan
Mexico
United States
China
Korea, Republic of
Spain
Turkey
Croatia
Taiwan
Oman
Canada
Serbia
France

location_facility: Severance Hospital, Yonsei University Health System

location_facility: The Affiliated Hospital of Guizhou Medical University

location_facility: Kyung Hee University Hospital at Gangdong

location_facility: Ondokuz Mayıs University Healthcare Practice and Research Center

location_facility: Institute of hematology&blood disease hospital

location_facility: Beijing Children's Hospital, Capital Medical University

location_facility: Institute for Mother and Child healthcare "Dr Vukan Cupic"

location_facility: Hamilton Health Sciences - McMaster University Medical Centre

location_facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

location_facility: Centro Multidisciplinario para el Desarrollo Especializado de la Investigacion Clinica en Yucatan

location_facility: Nagoya University Hospital - Transfusion Medicine

location_facility: Gaziantep University Şahinbey Research and Practice Hospital

location_facility: Gazi University Health Research and Application Center Gazi Hospital

location_facility: Nanfang Hospital, Southern Medical University

location_facility: Hôpital Universitaire Necker Enfants Malades

location_facility: Hacettepe University Faculty of Medicine


title: HALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome

summary: The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.

lead_sponsor: Ionis Pharmaceuticals, Inc.

sponsor_collaborators:
Ionis Pharmaceuticals, Inc.
Biogen

location_country:
France
Italy
United Kingdom
Australia
United States
Israel

location_facility: Azienda Ospedaliera Universitaria Pisana

location_facility: Colorado Children's Hospital Research Institute

location_facility: University of North Carolina at Chapel Hill School of Medicine

location_facility: Sydney Children's Hospital, Kids Cancer Centre


acronym: Orbit

lead_sponsor: Ultragenyx Pharmaceutical Inc

summary: The primary objectives of the study are to identify a setrusumab dosing strategy in participants with OI and to evaluate the effect of setrusumab vs placebo on reduction in fracture rate.

sponsor_collaborators:
Ultragenyx Pharmaceutical Inc
Mereo BioPharma

location_facility: Atrium Health Levine Children's Hospital

location_facility: Uniwersytet Medyczny w Lodzi - Klinika Endokrynologii i Chorob Metabolicznych

location_facility: University of Toronto- The Hospital for Sick Children (SickKids)

location_facility: Nemours/ Alfred i. duPoint Hospital for Children

location_facility: Shriners Hospitals for Children - Chicago

location_facility: Shriners Hospital for Children - Northern California

location_facility: Washington University School of Medicine

location_facility: New Mexico Clinical Research & Osteoporosis Center, Inc.

location_facility: Nationwide Children's Hospital- Ohio State University College of Medicine

location_facility: Azienda Ospedaliera Universitaria Policlinico Umberto I

location_facility: Sheffield Children's NHS Foundation Trust

title: An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single-Blind, Dose-Evaluation Phase and a Phase 3 Double-Blind, Placebo-Controlled Phase to Assess the Efficacy and Safety of Setrusumab in Subjects With Osteogenesis Imperfecta

location_country:
Germany
Italy
Poland
Portugal
United Kingdom
Argentina
Australia
Netherlands
United States
Turkey
Canada
France


title: A Phase 3, Multicenter, Open-Label, Randomized Study of Nemvaleukin Alfa in Combination With Pembrolizumab Versus Investigator's Choice Chemotherapy in Patients With Platinum-Resistant Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer (ARTISTRY-7)

summary: This is a Phase 3, multicenter, open-label, randomized study of nemvaleukin in combination with pembrolizumab versus protocol-specific Investigator's choice chemotherapy in patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer.

ipd_sharing: Undecided

lead_sponsor: Alkermes, Inc.

sponsor_collaborators:
Alkermes, Inc.
Merck Sharp & Dohme LLC

location_country:
Germany
Italy
Israel
Singapore
United Kingdom
Australia
Austria
Belgium
United States
Korea, Republic of
Spain
Taiwan
Czechia
Canada
France

location_facility:
Alkermes Investigational Site
Alkermes Investigator Site
Alkermes Investigator


acronym: UNITED

lead_sponsor: Janssen Research & Development, LLC

summary: The purpose of this study is to collect long-term safety data of subcutaneous (SC) ustekinumab

location_facility: Children's Center For Digestive Healthcare, Llc

location_facility: Szabolcs Szatmar Bereg Varmegyei Oktatokorhaz

location_facility: National Center for Child Health and Development

location_facility: Borsod-Abauj-Zemplen Megyei Korhaz es Egyetemi Oktato Korhaz

location_facility: Uniwersytecki Szpital Dzieciecy w Krakowie

location_facility: Centro de Investigaciones Medicas Tucuman

location_facility: Charité - Universitätsmedizin Berlin, Campus Virchow Klinikum

title: A Phase 3, Multicenter, Open-label, Basket, Long-term Extension Study of Ustekinumab in Pediatric Clinical Study Participants (2 to <18 Years of Age)

primary_purpose: Other

location_country:
Germany
Poland
Hungary
Argentina
United Kingdom
Japan
Belgium
United States
France


title: An Open-Label, Multicenter, Phase 1b/2 Study of E7386 in Combination With Pembrolizumab in Previously Treated Subjects With Selected Solid Tumors

summary:
The Phase 1b part of this study is conducted to assess the safety and tolerability of E7386 in combination with pembrolizumab in participants with previously treated selected solid tumors, and to determine the recommended Phase 2 dose (RP2D) of E7386 in combination with pembrolizumab.

The Phase 2 part of this study is conducted to assess the objective response rate (ORR) of E7386 in combination with pembrolizumab (melanoma, colorectal cancer [CRC], hepatocellular carcinoma [HCC]) or of E7386 in combination with pembrolizumab plus lenvatinib (HCC) according to Response Evaluation Criteria in Solid Tumors (RECIST) 1.1.

ipd_sharing_description: Eisai's data sharing commitment and further information on how to request data can be found on our website http://eisaiclinicaltrials.com/.

lead_sponsor: Eisai Inc.

sponsor_collaborators:
Eisai Inc.
Merck Sharp & Dohme LLC

location_country:
United Kingdom
Japan
United States
Spain
France

location_facility: Hospital Regional Universitario de Malaga

location_facility: The Royal Marsden NHS Foundation Trust - The Royal Marsden Hospital

location_facility: Consorcio Hospital General Universitario de Valencia

location_facility: Royal Free Hospital NHS Foundation Trust

location_facility: Complejo Asistencial Universitario de Salamanca

location_facility: Hospital General Universitario Gregorio Maranon


title: A Randomized Double-Blind Placebo-Controlled Study of the LEPR Agonist Antibody REGN4461 for the Treatment of Metabolic Abnormalities in Patients With Familial Partial Lipodystrophy

acronym: LEAP

summary:
Two cohorts are being studied based on leptin levels. Cohort A is composed of patients with baseline leptin <8.0 ng/mL and Cohort B is composed of patients with baseline leptin 8.0 to ≤20.0 ng/mL

The primary objectives will be evaluated for patients in Cohort A only:

To evaluate the effect of REGN4461 on fasting triglycerides (TG) in patients with elevated baseline fasting TG
To evaluate the effect of REGN4461 on hyperglycemia in patients with elevated baseline Hemoglobin A1c (HbA1c)

The following secondary objectives of the study will be evaluated for Cohort B and for the combined set of Cohorts A plus B:

To evaluate the effect of REGN4461 on fasting TG levels in patients with hypertriglyceridemia
To evaluate the effect of REGN4461 on glycemic control in patients with hyperglycemia

The following secondary objectives of the study will be evaluated for Cohorts A and B separately, and for the combined set of Cohorts A plus B:

To evaluate the effect of REGN4461 on liver fat in patients with hepatic steatosis
To evaluate the effect of REGN4461 on hunger
To evaluate safety and tolerability of REGN4461
To characterize the concentration profile of REGN4461 over time
To assess immunogenicity to REGN4461

ipd_sharing_description: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing

lead_sponsor: Regeneron Pharmaceuticals

location_country:
United Kingdom
United States
Spain
Turkey
France

location_facility: Excel Medical Clinical Trials - A Flourish Research Site

location_facility: Complexo Hospitalario Universitario de Santiago-Hospital Médico-Cirúrxico de Conxo

location_facility: ICAN, Institute of Cardiometabolism and Nutrition

location_facility: Cambridge University Hospitals NHS Foundation Trust


title: A Phase-II Study Comparing Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation Following Autologous Transplantation to Autologous Transplantation Alone in Multiple Myeloma

summary: The purpose of this study is to determine whether autologous transplantation (using the patient's own stem cells from the blood), followed by non-myeloablative (i.e. less intense) allogeneic transplantation (where the blood stem cells from a sibling donor are used for the transplantation) improves the outcome in patients with newly diagnosed multiple myeloma.

status: Completed

study_completion_date_type: Actual

lead_sponsor: European Society for Blood and Marrow Transplantation

location_country:
Finland
Germany
Italy
Norway
United Kingdom
Denmark
Sweden
Austria
Turkey
Canada
France

location_facility:
Herlev Hospital
University of Milano
Ospedale Ferrarotto
University Hospital
Tampere University Hospital
University of Leipzig
Medizinische Universität Wien
CHU Lapeyronie
University of Heidelberg
Helsinki University Central Hospital
Rigshospitalet
Turku University
Sahlgrenska University Hospital
IRCCS, Casa Sollievo della Sofferenza
Royal Marsden Hospital
Uni. Modena, Policlinico
Rikshospitalet
University Faculty of Medicine
Huddinge University Hospital
Ospedale di Careggi

intervention_type: Procedure

status_simplified: Completed


title: HONEST-PREPS: Hospital Network Study - Preparation for a Randomized Evaluation of Anti-Pneumonia Strategies

acronym: HONEST-PREPS

summary:
Hospital Acquired and Ventilator Associated Pneumonia (HAP/VAP) pose a significant burden to patients admitted to the Intensive Care Unit (ICU). Reported incidence ranges from 10-16% in all ICU patients (including HAP and VAP) and around 20-30% in ventilated patients (VAP). Patients with HAP/VAP have a high mortality rate. The estimated attributable mortality of VAP is 6-13%.

Randomized Controlled Trials (RCTs) are the gold standard for evaluating medical interventions, but are difficult to perform in this population. Several preventive and therapeutic treatment options are being developed that will require evaluation in phase-III trials. These trials are challenging due to the relatively low incidence of the outcome (e.g. HAP/VAP) or of the domain under study (e.g. specific antibiotic resistant infections) and the requirement of informed consent in critically ill patients. There is a need for a well-organized and well-trained international RCT network that enables efficient execution of a series of RCTs in this population.

The aim of the current study is to set up an infrastructure to prospectively enroll patients at risk of HAP/VAP in ICUs in several European countries. Site personnel will be trained to obtain a GCP (Good Clinical Practice) certification (if not already done), to timely identify and enroll patients at risk of HAP/VAP, to timely identify occurrence of HAP/VAP, collect informed consent forms, collect source data, enter data into a clinical database, and use a dedicated system to reply to queries. Site sample collection, processing, identifying the causative organism, and antibiotic susceptibility testing will be validated and adapted if required where possible. Where site infrastructure and regulations allow, the possibility of automated data collection of included participants will be explored to ensure sustainability of the future platform. Furthermore, collected data will be used to inform future diagnostic, preventive and therapeutic trials. E.g. they may support assumptions in sample size calculations and expected number of enrolled participants, they may help in prioritizing interventions, or they may be used in simulations of adaptive trials to optimize decision rules.

lead_sponsor: UMC Utrecht

sponsor_collaborators:
UMC Utrecht
Universiteit Antwerpen
University Hospital, Geneva

location_country: Albania Romania Latvia Croatia Czechia Serbia France

location_facility: Central Military Emergency University Hospital "Dr. Carol Davila "

location_facility: Paul Stradins Clinical University Hospital

location_facility: Institute for Pulmonary Diseases of Vojvodina

location_facility: General Hospital Dr Josip Bencevic Slavonski Brod


title: Avelumab Master Protocol: An Open-label Continuation Study for Participants Continuing From Pfizer-sponsored Avelumab Clinical Studies.

summary: This Master Protocol for Avelumab Continuation Sub-Studies is to provide continued treatment access, safety follow-up, and when applicable, overall survival follow-up for eligible participants who continue to derive a benefit from study intervention in the Pfizer-sponsored Avelumab parent studies.

location_country:
Italy
Poland
New Zealand
Hungary
Israel
Denmark
Australia
Japan
United Kingdom
Belgium
United States
Korea, Republic of
Spain
Taiwan
Canada
Serbia
Russian Federation
France

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

location_facility: GBUZ SK Pyatigorsk Interdistrict Oncology dispensary

location_facility: Institut de Cancérologie de Lorraine Alexis Vautrin

location_facility: Kingston Health Sciences Centre-Kingston General Hospital Site

location_facility: Memorial Sloan Kettering Cancer Center - Investigational Drug Service Pharmacy

location_facility: National Hospital Organization Kyushu Cancer Center

location_facility: Private Medical Institution "Euromedservice"

location_facility: Institute for Oncology and Radiology of Serbia

location_facility: Centralny Szpital Kliniczny MSWiA w Warszawie

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: Institut Català d'Oncologia - L'Hospitalet

location_facility: Seoul National University Bundang Hospital

location_facility: Memorial Sloan Kettering Cancer Center 53rd street.

location_facility: Hospital of the University of Pennsylvania

location_facility: FBIH "Privolzhskiy Regional Medical Center" of FMBA

location_facility: Azienda Ospedaliero Universitaria Ospedali Riuniti Umberto I - G.M. Lancisi - G. Salesi

location_facility: The Newcastle Upon Tyne Hospitals NHS Foundation Trust

location_facility: Memorial Sloan Kettering Cancer Center - David H. Koch Center for Cancer Care (74th Street).

location_facility: Chang Gung Medical Foundation-Linkou Branch

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: UCLA Hematology/Oncology - Administrative Office

location_facility: Hospital Universitario Fundación Jiménez Díaz

location_facility: Ronald Reagan UCLA Medical Center - Drug Information Center, Pharmaceutical Services

location_facility: Moscow Scientific Research Oncology Institute n.a. P.A. Hertzen

location_facility: Budgetary Healthcare Institution of Omsk Region "Clinical Oncology Dispensary"

location_facility: SAHI Republican Clinical Oncology Dispensary under the Ministry of Health, Republic of Bashkortostan


acronym: PIVOINE

lead_sponsor: Ipsen

summary:
The main objective of this study is to further evaluate the safety and efficacy of palovarotene in adult and paediatric participants with FOP.

The aim of the study is also to ensure treatment continuity to participants who have completed one of the parent studies (Study PVO-1A-301, Study PVO-1A-202 and Study PVO-1A-204) and who, in the investigator's judgement, may benefit from palovarotene therapy.

location_facility: University of California San Francisco (UCSF)

location_facility: The Perelman School of Medicine - The University of Pennsylvania

location_facility: Groupe Hospitalier Necker Enfants Malades

title: Rollover Study; Multicentre, Phase III, Open-label Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed Study PVO-1A-301 or PVO-1A-202/PVO-1A-204 and May Benefit From Palovarotene Therapy.

ipd_sharing_description:
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.

Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.

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


title: A Study of Intravesical Enfortumab Vedotin For Treatment of Patients With Non-muscle Invasive Bladder Cancer (NMIBC)

summary:
This study will test a drug called enfortumab vedotin in participants with a type of bladder cancer called non-muscle invasive bladder cancer (NMIBC).

This study will also evaluate what the side effects are and if the drug works to treat NMIBC. A side effect is anything a drug does to your body besides treating your disease.

In this study enfortumab vedotin will be put into the bladder using a catheter. A catheter is a thin tube that can be put into your bladder.

lead_sponsor: Astellas Pharma Global Development, Inc.

location_country:
United Kingdom
United States
Spain
Canada
France

location_facility: University of California at San Francisco

location_facility: Laura & Isaac Perlmutter Cancer Center at NYU Langone Health

location_facility: University of Texas Southwestern Medical Center

location_facility: UCLA Department of Medicine - Hematology & Oncology


title: An Open-label, Phase 2 Basket Study of SEA-CD40 Combination Therapies in Advanced Malignancies

summary:
This trial is being done to see if an experimental drug (SEA-CD40) works when it's given with other cancer drugs to treat some types of cancer. It will also study side effects from the drug.

There are 2 parts in this trial. In one part, participants have melanoma that has come back after treatment or can't be removed by surgery. Participants in this part will get SEA-CD40 and pembrolizumab. In the other part, participants have non-small cell lung cancer (NSCLC) that has spread through their body. These participants will get SEA-CD40, pembrolizumab, carboplatin, and pemetrexed.

lead_sponsor: Seagen Inc.

sponsor_collaborators:
Seagen Inc.
Merck Sharp & Dohme LLC

location_country:
Germany
Sweden
United States
Spain
Canada
France

location_facility: Tennessee Oncology-Nashville/Sarah Cannon Research Institute

location_facility: Carbone Cancer Center / University of Wisconsin

location_facility: The Angeles Clinic and Research Institute

location_facility: University of Texas Southwestern/Simmons Cancer Center

location_facility: Florida Cancer Specialists - South Region

location_facility: California Pacific Medical Center Research Institute/Sutter Medical Centre

location_facility: Cleveland Clinic - Taussig Cancer Institute

location_facility: Morristown Medical Center/ Carol G. Simon Cancer Center

location_facility: MD Anderson Cancer Center / University of Texas

location_facility: Florida Cancer Specialists - North Region

location_facility: START Madrid-CIOCC_Hospital HM Sanchinarro

location_facility: Hospital Clinico Universitario de Valencia


title: A Phase 1b/2, Open Label Study of DAY101 Monotherapy or Combination With Other Therapies for Patients With Recurrent, Progressive, or Refractory Solid Tumors Harboring MAPK Pathway Aberrations

summary: This is a Phase 1b/2, multi-center, open label umbrella study of patients ≥12 years of age with recurrent, progressive, or refractory melanoma or other solid tumors with alterations in the key proteins of the RAS/RAF/MEK/ERK pathway, referred to as the MAPK pathway.

lead_sponsor: Day One Biopharmaceuticals, Inc.

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

location_facility: Severance Hospital, Yonsei University Health System

location_facility: Vanderbilt-Ingram Cancer Center Recruitment and Eligibility Office


summary:
The primary objective of the study is to determine the efficacy of REGN1908-1909, as compared to placebo, to reduce allergic rhinitis/conjunctivitis symptoms and allergy rescue medication use during natural cat exposure.

The Secondary Objectives are:

To assess the reduction of allergic symptoms and use of allergy rescue medications after treatment with REGN1908-1909 versus placebo, as measured by the individual components of the CSMS
To assess health-related quality of life (HRQoL) as measured by the Rhinoconjunctivitis Quality of Life Questionnaire (RQLQ[S])
To determine the efficacy of REGN1908-1909, as compared to placebo, to inhibit a wheal-and-flare response to a skin prick test with cat allergen
To assess the durability of effect in allergic rhinitis and conjunctivitis symptom and medication scores after multiple doses of REGN1908-1909 compared to placebo given every 12 weeks (Q12W)
To determine the efficacy following multiple doses of REGN1908-1909 compared to placebo at inhibiting a wheal-and-flare response to a skin prick test with cat allergen
To estimate the effect of REGN1908-1909 on lung function, as compared to placebo, in patients with asthma
To determine the efficacy of REGN1908-1909 as compared to placebo to reduce asthma symptoms in patients with asthma
To assess whether there is a difference in asthma rescue medication use in patients with asthma who are treated with REGN1908-1909 compared to placebo
To assess whether there is a difference in nighttime awakenings in patients with asthma treated with REGN1908-1909 compared to placebo
To evaluate the short-term and long-term safety and tolerability of REGN1908-1909, including the incidence of hypersensitivity reactions, local injection site reactions, and asthma exacerbations
To determine systemic exposure of total (free and antigen-bound) antibodies as measured by concentration of REGN1908 and REGN1909
To assess the immunogenicity of REGN1908 and REGN1909

location_facility: Velocity Clinical Research, Inc. (Meridan)

location_facility: Asthma and Allergy Center of Chicago Sc-Oak Park

location_facility: Centrum Nowoczesnych Terapii Dobry Lekarz Sp. z o.o.

location_facility: Peninsula Research Associates - CRN - PPDS

location_facility: Centre d'investigation Clinique Mauricie

location_facility: Colorado Allergy and Asthma Centers PC - CRN - PPDS

location_facility: LMC Manna Research - Quebec - HyperCore - PPDS

location_facility: Respiratory Medicine Research Institute of Michigan PLC

location_facility: Asthma & Allergy Physicians of Rhode Island Clinical Research Institute (AAPRI CRI)

location_facility: Seattle Allergy & Asthma Research Institute

location_facility: Riverside Medical Group - Circuit - PPDS

location_facility: Centrum Alergologii Specjalistyczna Przychodnia Alergologiczna

location_facility: Allergy and Asthma Medical Group and Research Center - CRN - PPDS

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

location_facility: Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi

location_facility: Allergy Asthma and Clinical Research Center

location_facility: Asthma, Nasal Disease and Allergy Research Center of New England

location_facility: Allergy and Clinical Immunology Associates

location_facility: Asthma and Allergy Associates PC - CRN - PPDS

location_facility: Princeton Center For Clinical Research - CRN - PPDS

location_facility: SUNY Downstate Health Science University

location_facility: Clinique Spécialisée en Allergie de la Capitale

location_facility: Joel Liem Medicine Professional Corporation

location_facility: Allergy Partners of Western North Carolina

location_facility: AES - DRS - Synexus Polska Sp. z o.o. Oddzial we Wroclawiu

location_facility: Specjalistyczna Przychodnia Lekarska Alergo-Med Sp. z.o.o

location_facility: Nebraska Medical Research Institute, Inc. - CRN - PPDS

location_facility: Alergo-Med Specjalistyczna Przychodnia Lekarska Sp. z.o.o

location_facility: SPZOZ Uniwersytecki Szpital Kliniczny nr 1 im Norberta Barlickiego Uniwersytetu Medycznego w Lodzi

location_facility: ALL-MED - Specjalistyczna Opieka Medyczna - Medyczny Instytut Badawczy

location_facility: Private Practice Dr Jean Benoit Martinot

location_facility: Allergy and Asthma Associates of Southern California - CRN - PPDS

location_facility: Clinical Research Institute, Inc - CRN - PPDS

location_facility: Universitätsklinikum Carl Gustav Carus an der TU Dresden

location_facility: Aventiv Research Inc - Columbus - HyperCore - PPDS

status: Terminated

title: A Randomized, Double-Blind, Placebo-Controlled Study in Cat-Allergic Patients With Allergic Rhinitis Who Live With a Cat to Assess the Efficacy and Safety of Anti-Fel d 1 Antibodies During Natural Cat Exposure in the Home

location_country:
Germany
Poland
Belgium
United States
Canada
France


title: A Phase 2B/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease

summary:
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here.

This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords).

In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control).

In Part C, all participants will receive study drug.

lead_sponsor: Mandos LLC

location_country:
Germany
New Zealand
United Kingdom
Singapore
Australia
Switzerland
United States
Turkey
France

location_facility: Multicare Institute for Research and Innovation

location_facility: National University Hospital (Singapore) Pte, Ltd

location_facility: Eunice Kennedy Shriver National Institute of Child Health and Human Development

location_facility: Birmingham Children's Hospital NHS Foundation Trust

location_facility: Dell Children's Medical Center of Central Texas

location_facility: Children's Hospital of Orange County: CHOC Children's

location_facility: CHU Paris Est - Hopital d'Enfants Armand-Trousseau


title: Natural History of the Progression of X-Linked Retinitis Pigmentosa

acronym: XOLARIS

summary: The objective of the study is to gain a better understanding of disease progression over time in participants with X-linked retinitis pigmentosa (XLRP).

lead_sponsor: NightstaRx Ltd, a Biogen Company

location_country:
Germany
Finland
United Kingdom
Netherlands
United States
Canada
France


title: A Dose-Escalation and Expansion Study of the Safety and Pharmacokinetics of XB002 as Single-Agent and Combination Therapy in Subjects With Inoperable Locally Advanced or Metastatic Solid Tumors

summary: This is a Phase 1, open-label, multicenter, dose-escalation and expansion study evaluating the safety, tolerability, PK, pharmacodynamics, and clinical antitumor activity of XB002 administered IV q3w alone and in combination with nivolumab or bevacizumab to subjects with advanced solid tumors.

lead_sponsor: Exelixis

location_country:
Italy
United Kingdom
Australia
Netherlands
Belgium
United States
Spain
France

location_facility:
Exelixis Clinical Site #40
Exelixis Clinical Site #34
Exelixis Clinical Site #11
Exelixis Clinical Site #29
Exelixis Clinical Site #3
Exelixis Clinical Site #19
Exelixis Clinical Site #21
Exelixis Clinical Site #38
Exelixis Clinical Site #26
Exelixis Clinical Site #8
Exelixis Clinical Site #16
Exelixis Clinical Site #1
Exelixis Clinical Site #17
Exelixis Clinical Site #39
Exelixis Clinical Site #28
Exelixis Clinical Site #18
Exelixis Clinical Site #13
Exelixis Clinical Site #43
Exelixis Clinical Site #12
Exelixis Clinical Site #14
Exelixis Clinical Site #35
Exelixis Clinical Site #25
Exelixis Clinical Site #32
Exelixis Clinical Site #42
Exelixis Clinical Site #41
Exelixis Clinical Site #22
Exelixis Clinical Site #10
Exelixis Clinical Site #23
Exelixis Clinical Site #15
Exelixis Clinical Site #36
Exelixis Clinical Site #6
Exelixis Clinical Site #37
Exelixis Clinical Site #33
Exelixis Clinical Site #9
Exelixis Clinical Site #24
Exelixis Clinical Site #4
Exelixis Clinical Site #30
Exelixis Clinical Site #31
Exelixis Clinical Site #7
Exelixis Clinical Site #5
Exelixis Clinical Site #27
Exelixis Clinical Site #20
Exelixis Clinical Site #2


acronym: Intramuscular VIR-7831 (sotrovimab) for mild/moderate COVID-19 Study Phase: Phase III

lead_sponsor: Vir Biotechnology, Inc.

summary:
The COMET-TAIL main study evaluated efficacy, safety, and tolerability of IM sotrovimab versus IV sotrovimab in high-risk patients for the treatment of mild/moderate COVID-19. In the safety substudy, the aim is the evaluation of the safety and tolerability of sotrovimab across a single ascending dose level and over different infusion times when given for the treatment of mild/moderate COVID-19 to participants at high risk of disease progression

The safety sub-study was discontinued early in the context of ongoing concerns regarding the continuous change in the SARS-CoV-2 circulating variants.

ReferenceType: derived

ReferenceCitation: Kreuzberger N, Hirsch C, Chai KL, Tomlinson E, Khosravi Z, Popp M, Neidhardt M, Piechotta V, Salomon S, Valk SJ, Monsef I, Schmaderer C, Wood EM, So-Osman C, Roberts DJ, McQuilten Z, Estcourt LJ, Skoetz N. SARS-CoV-2-neutralising monoclonal antibodies for treatment of COVID-19. Cochrane Database Syst Rev. 2021 Sep 2;9(9):CD013825. doi: 10.1002/14651858.CD013825.pub2.

oa_host_type: repository

evidence: oa repository (via OAI-PMH doi match)

host_type: repository

license_normalized: no license

repository_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

version: publishedVersion

evidence: oa repository (via OAI-PMH title and first author match)

repository_institution: University of Oxford - Oxford University Research Archive (ORA)

evidence: oa repository (via pmcid lookup)

repository_normalized: PubMed Central

repositories_concat: PubMed Central;ora.ox.ac.uk;pure.eur.nl

unpaywall_oa_status: green

publisher_dissemination: Wiley

sponsor_collaborators:
Vir Biotechnology, Inc.
GlaxoSmithKline

location_facility:
Investigative Sites
Investigative Site

title: A Phase 3 Randomized, Multi-center, Open Label Study to Assess the Efficacy, Safety, and Tolerability of Monoclonal Antibody VIR-7831 (Sotrovimab) Given Intramuscularly Versus Intravenously for the Treatment of Mild/Moderate Coronavirus Disease 2019 (COVID-19) in High-risk Non-hospitalized Patients; Safety Substudy Assessing the Safety and Tolerability of Single Ascending Dose Monoclonal Antibody VIR-7831

location_country:
United States
Ukraine
France


title: An Open-Label, Multicenter, Phase Ib Trial Evaluating the Safety, Pharmacokinetics, and Activity of Cevostamab as Monotherapy and Cevostamab Plus Pomalidomide and Dexamethasone or Cevostamab Plus Daratumumab and Dexamethasone in Patients With Relapsed or Refractory Multiple Myeloma

acronym: CAMMA 1

summary: This Phase Ib, multicenter, open-label study will evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of cevostamab monotherapy, cevostamab plus pomalidomide and dexamethasone (Pd) or cevostamab plus daratumumab and dexamethasone (Dd) which will be administered to participants with relapsed or refractory multiple myeloma (R/R MM) via intravenous (IV) infusion.

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

lead_sponsor: Genentech, Inc.

sponsor_collaborators:
Genentech, Inc.
Hoffmann-La Roche

location_country:
Italy
Poland
Israel
Denmark
Australia
United States
Korea, Republic of
Spain
Czechia
Canada
France

location_facility: Hospital General Universitario Gregorio Marañon; Servicio de Hematología

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

location_facility: Colorado Blood Cancer Institute (CBCI) at Presbyterian/ St. Luke's Medical Center

location_facility: Fakultni Nemocnice Ostrava; Klinika hematoonkologie FNO a LF OU

location_facility: City of Hope - Lennar Foundation Cancer Center

location_facility: The Alfred Hospital; Malignant Haematology & Stem Cell Transplant Service

location_facility: Peter MacCallum Cancer Centre; Department of Haematology

location_facility: Rambam Medical Center; Heamatology & Bone Marrow Transplantation

location_facility: Rigshospitalet; Hæmatologisk Klinik, Klinisk Afprøvnings Team KAT

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

location_facility: Washington University School of Medicine

location_facility: University Health Network; Princess Margaret Hospital; Medical Oncology Dept

location_facility: Uniwersyteckie Centrum Kliniczne; Kilnika Hematologii i Transplantologii, Oddzia? Wczesnych Faz

location_facility: I Interni klinika; Vseobecna fakultni nemocnice


title: Effectiveness of the SpaceOAR Vue System in Subjects With Prostate Cancer Being Treated With Stereotactic Body Radiotherapy

acronym: SABRE

summary: To demonstrate the effectiveness of the SpaceOAR Vue System in reducing late gastrointestinal (GI) toxicity in subjects undergoing Stereotactic Body Radiotherapy (SBRT) to treat prostate cancer.

lead_sponsor: Boston Scientific Corporation

location_country:
Ireland
Germany
Italy
United Kingdom
Australia
Switzerland
United States
Spain
France

location_facility: Azienda Ospedaliero Universitaria di Parma

location_facility: GenesisCare, Hospital San Francisco de Asis

location_facility: Royal Surrey County Hospital NHS Foundation Trust

location_facility: Norfolk and Norwich University Hospital NHS Trust

location_facility: New Jersey Urology, a Summit Health Company


title: A Phase I/Ib, Open-label, Multi-center Study of DFF332 as a Single Agent and in Combination With Everolimus or IO Agents in Patients With Advanced/Relapsed ccRCC and Other Malignancies With HIF2α Stabilizing Mutations

summary: This is first in human study of DFF332, a small molecule that targets a protein called HIF2α. By acting on HIF2α, DFF332 may be able to stop the growth of certain types of cancer. DFF332 will be tested at different doses as single agent and in combination with Everolimus (RAD001, an mTOR inhibitor), and also in combination with Spartalizumab (PDR001, an anti-PD1) plus Taminadenant (NIR178, an adenosine A2A receptor antagonist), in patients with advanced clear cell renal cell carcinoma and other malignancies with HIF stabilizing mutations.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
Singapore
Japan
United States
Spain
Czechia
France

location_facility: Memorial Sloane Kettering Cancer Center .

location_facility: Uni of TX MD Anderson Cancer Cntr Dept.ofMDAndersonCancerCtr(8)

location_facility: Washington Uni School of Med Main Center


lead_sponsor: Tavanta Therapeutics

summary: The purpose of this study is to investigate the safety and efficacy of a new formulation of an existing drug product called TAVT-45 in patients with metastatic prostate cancer.

title: Phase 3 Study Investigating the Efficacy and Safety of TAVT-45 (Abiraterone Acetate) Granules for Oral Suspension (Novel Abiraterone Acetate Formulation) Relative to a Reference Abiraterone Acetate Formulation in Patients With mCSPC & mCRPC

ipd_sharing_description:
According to the Informed Consent Form:

The health information that may be used and disclosed includes:

All information collected during the research described in the Informed Consent Form for the study; and
Health information in my medical records that is relevant to the study.

The Providers may disclose health information in my medical records to:

Researchers;
The Sponsor of the study and its agents and contractors; and
Representatives of government agencies, Advarra IRB, and other persons who watch over the safety, effectiveness, and conduct of research.

The Researchers may:

Use and share patient health information among themselves, with the Sponsor, and with other participating researchers and laboratories to conduct the study; and
Disclose health information to representatives of government agencies, review boards

location_country:
Poland
Hungary
United Kingdom
Sweden
United States
Spain
Puerto Rico
France


title: A Phase 1, Multicenter, Open-Label, Dose Escalation and Expansion Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of Orally Administered FHD-286 in Subjects With Metastatic Uveal Melanoma

summary: This Phase 1, multicenter, open-label, dose escalation and expansion study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of FHD-286 oral monotherapy in subjects with metastatic Uveal Melanoma (UM).

lead_sponsor: Foghorn Therapeutics Inc.

location_country:
United States
Netherlands
France

location_facility: University of Miami Health System, Sylvester Comprehensive Cancer Center

location_facility: The Angeles Clinic and Research Institute

location_facility: Sidney Kimmel Cancer Center - Jefferson Health

location_facility: Columbia University, Herbert Irving Comprehensive Cancer Center


title: Study of Four-factor Prothrombin Complex Concentrate, OCTAPLEX, in Patients With Acute Major Bleeding on Direct Oral Anticoagulant (DOAC) Therapy With Factor Xa Inhibitor

summary: This is a multicentre, prospective, randomised, double-blinded, group-sequential, parallel-group, adaptive design, phase 3 study to demonstrate the haemostatic efficacy and safety of four-factor prothrombin complex concentrate, OCTAPLEX, in patients with acute major bleeding on DOAC therapy with factor Xa inhibitor. Patients will be randomised 1:1 to either of two study groups: low-dose vs. high-dose OCTAPLEX.

lead_sponsor: Octapharma

location_country:
Germany
Italy
Poland
United Kingdom
Austria
United States
Bosnia and Herzegovina
Spain
Turkey
Croatia
Ukraine
Georgia
France

location_facility: Azienda Ospedaliero -Universitaria di Modena

location_facility: Public Non-profit Enterprise Central City Clinical Hospital of Ivano-Frankivsk City Council

location_facility: Klinikum Klagenfurt am Wörthersee Anästhesiologie und Intensivmedizin

location_facility: K.Eristavi National Center of Experimental and Clinical Surgery

location_facility: Public Non-profit Enterprise Regional Clinical Hospital of lvano-Frankivsk Regional Council

location_facility: Ospedale Maggiore - IRCCS Istituto di Scienze Neurologiche di Bologna

location_facility: Kahramanmaraş Sütçü İmam University Faculty of Medicine

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

location_facility: Istanbul University Istanbul Faculty of Medicine Department of Internal Diseases, Division of Hematology

location_facility: Health Sciences University Bursa High Specialization Training and Research Hospital

location_facility: University Clinical Centre of the Republic of Srpska

location_facility: OU Health - University of Oklahoma Medical Center

location_facility: Ondokuz Mayıs University Faculty of Medicine

location_facility: North Hampshire Hospitals NHS Foundation Trust, Basingstoke and North Hampshire Hospital

location_facility: Public Non-profit Enterprise of Lviv Regional Council Lviv Public non profit Regional Clinical Hospital

location_facility: University of Mississippi Medical Center

location_facility: Centre Hospitalier Universitaire Francois Mitterand

location_facility: Samodzielny Publiczny Zakład Opieki Zdrowotnej w Łęcznej

location_facility: Universitaetsklinikum Aachen, Klinik fuer Anaesthesiologie

location_facility: Universitaetsklinikum Frankfurt - Klinik fuer Anaesthesiologie, Intensivmedizin und Schmerztherapie

location_facility: LTS ,, Israel-Geoargian Medical Research clinic Helsicore"


title: A Modular Phase I/II, Open-Label, Multi-Centre Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of AZD0466 Monotherapy or in Combination in Patients With Advanced Haematological Malignancies

summary: The purpose of the study is to the evaluate safety, tolerability, pharmacokinetics (PK), and efficacy of AZD0466 as monotherapy in partciapants with advanced haematological malignancies and also to assess drug-drug interaction (DDI) potential between AZD0466 and the azole antifungal voriconazole.

ipd_sharing_description:
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All requests will be evaluated as per the AZ disclosure commitment:

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

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

lead_sponsor: AstraZeneca

location_country:
Germany
Italy
Australia
United States
Korea, Republic of
France


title: An Open-label, Multi-center, Phase I Study of Oral IAG933 in Adult Patients With Advanced Mesothelioma and Other Solid Tumors

summary: The purpose of this study is to characterize the safety and tolerability of IAG933 in patients with mesothelioma, NF2/LATS1/LATS2 mutated tumors and tumors with functional YAP/TAZ fusions and to identify the maximum tolerated dose and/or recommended dose.

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

location_facility: Massachusetts General Hospital Massachusetts General Hospital


acronym: ATLAS

lead_sponsor: Biogen

summary: The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).

ReferenceCitation: Benatar M, Wuu J, Andersen PM, Bucelli RC, Andrews JA, Otto M, Farahany NA, Harrington EA, Chen W, Mitchell AA, Ferguson T, Chew S, Gedney L, Oakley S, Heo J, Chary S, Fanning L, Graham D, Sun P, Liu Y, Wong J, Fradette S. Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study. Neurotherapeutics. 2022 Jul;19(4):1248-1258. doi: 10.1007/s13311-022-01237-4. Epub 2022 May 18. Erratum In: Neurotherapeutics. 2022 Sep 29;:

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: cc-by

license_normalized: cc-by

repository_institution: Umeå University - DiVA at Umeå University

unpaywall_oa_status: hybrid

publisher_dissemination: Springer-Nature

location_facility: Hospital Universitari i Politecnic La Fe

location_facility: Cresla "Rita Levi Montalcini" Department of Neuroscience

location_facility: Washington University School of Medicine

location_facility: Holy Cross Hospital Phil Smith Neuroscience Institute

title: A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

ipd_sharing_description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

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


acronym: START

lead_sponsor: Galderma R&D

summary: The purpose of this study is to evaluate the safety and efficacy of trifarotene 50 microgram per gram (mcg/g) cream compared to its vehicle on the risk of formation of atrophic acne scars after 24 weeks of treatment in facial acne participants assessed by atrophic acne scars count.

location_facility:
Galderma Investigational Site #8873
Galderma Investigational Site #8189
Galderma Investigational Site #6167
Galderma Investigational Site #9936
Galderma Investigational Site #9928
Galderma Investigational Site #9920
Galderma Investigational Site #8295
Galderma Investigational Site #8367
Galderma Investigational Site #8601
Galderma Investigational Site #8108
Galderma Investigational Site #8883
Galderma Investigational Site #8838
Galderma Investigational Site #8881
Galderma Investigational Site #8886
Galderma Investigational Site #9918
Galderma Investigational Site #9927
Galderma Investigational Site #8447
Galderma Investigational Site #8608

title: Evaluation of the Risk of Atrophic Acne Scar Formation During Treatment of Acne Vulgaris Subjects With Trifarotene 50 μg/g Cream Versus Vehicle Cream Over 24 Weeks

location_country:
United States
Canada
France


title: REVEAL-CKD: A Retrospective, Multinational Observational Study to Determine the Prevalence and Consequences of Undiagnosed Chronic Kidney Disease

acronym: REVEAL-CKD

summary: This is a retrospective, multinational, non-interventional, observational study. A series of cohort studies will be conducted to assess the prevalence of undiagnosed stage 3 CKD in each region. The study will also assess the current state of CKD management in patients with undiagnosed CKD

time_perspective: Retrospective

ipd_sharing_description: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

location_country:
Germany
Italy
United Kingdom
Australia
Brazil
Japan
United States
China
Spain
Canada
France