Clinical Trials on Familial Hemophagocytic Lymphohistiocytosis

Total 1233 results

Novartis Pharmaceuticals

Completed

Evaluate the Safety and Efficacy of Canakinumab in Pediatric Patients With Colchicine Intolerant or Colchicine Resistant Familial Mediterranean Fever (FMF) (CONTROL FMF)

Colchicine Resistant/Intolerant Familial Mediterranean Fever

Israel
Immunovia, Inc.

Completed

A Study of IMMray™ PanCan-d Test for Early Detection of Pancreatic Cancer in High-risk Groups

Pancreatic Ductal Adenocarcinoma | Familial Pancreatic Cancer | FAMMM - Familial Atypical Mole Malignant Melanoma Syndrome

United States, Sweden, Canada, Spain, United Kingdom
Yale University
National Institute on Alcohol Abuse and Alcoholism (NIAAA)

Recruiting

Functional Neuroimaging of Alcoholism Vulnerability: Probing Glutamate and Reward, Using the mGluR5 Inhibitor Mavoglurant

Familial Alcoholism Vulnerability

United States
Albireo

Approved for marketing

Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis

Progressive Familial Intrahepatic Cholestasis

United States
Novartis Pharmaceuticals

Completed

A Pilot Study to Assess the Efficacy and Safety of LCQ908 Alone and in Combination With Fenofibrate or Lovaza® in Patients With Severe Hypertriglyceridemia

Non Familial Chylocmicronemia Syndrome (Non-FCS)

United States, Canada, Russian Federation
National Liver Institute, Egypt

Unknown

Gastrostomy-Biliary Diversion: Innovative Management for Bile Canalicular Transport Disorders

Progressive Familial Intrahepatic Cholestasis

Egypt
Duke University

Completed

Collaboration to Promote Early Childhood Well Being in Families Experiencing Homelessness

Families Experiencing Homelessness With Children Under 48 Months of Age

United States
Lev-Hasharon Mental Healtlh Center

Unknown

Family Involvement in The Treatment Of a Mentally Ill Relative

Families of Mentally Ill

Israel
Massachusetts General Hospital
Broad Institute

Recruiting

Biomarker Profiling in Individuals at Risk for Prion Disease

Prion Diseases | Familial Fatal Insomnia | CJD (Creutzfeldt Jakob Disease) | GSS | FFI

United States
Alnylam Pharmaceuticals

Active, not recruiting

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Australia, Brazil, Bulgaria, France, Italy, Japan, Mexico, Netherlands, Taiwan, United Kingdom, Belgium, Czechia, Denmark, New Zealand, Sweden, Korea, Republic of, Argentina, Chile, Hong Kong, Portugal
Alnylam Pharmaceuticals

Active, not recruiting

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Poland, Argentina, Australia, Belgium, France, Germany, Japan, Netherlands, Portugal, Spain, Sweden, United Kingdom, Canada, Czechia, Denmark, Hungary, Ireland, Norway, Korea, Republic of, Austria, Croatia, Israel, Latvia, Lithuan... and more
Kastle Therapeutics, LLC
Ionis Pharmaceuticals, Inc.

Completed

Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease (RADICHOL II)

Coronary Artery Disease | Heterozygous Familial Hypercholesterolemia

United States, Canada
Memorial Sloan Kettering Cancer Center
University of Southern California; Weill Medical College of Cornell University and other collaborators

Active, not recruiting

Early Onset and Familial Gastric Cancer Registry

Gastric Cancer

United States, Nigeria
University of Chicago

Terminated

The Effects of Estrogen Replacement Therapy in Postmenopausal Women With Hypercalciuria and Low Bone Mass

Osteoporosis | Osteopenia | Hypercalciuria | Hypercalciuria, Familial Idiopathic

United States
Intellia Therapeutics
Regeneron Pharmaceuticals

Recruiting

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Australia, United Kingdom, New Zealand, Canada
Massachusetts General Hospital
Pfizer; Akcea Therapeutics; Alnylam Pharmaceuticals; Eidos Therapeutics, a BridgeBio...

Recruiting

Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF (TTRinHFpEF)

Heart Failure | Amyloidosis | Heart Failure, Diastolic | Transthyretin Amyloidosis

United States
Elif Oral

Completed

Study of Gemcabene in Adults With FPLD

Fatty Liver | Hypertriglyceridemia | NASH - Nonalcoholic Steatohepatitis | Familial Partial Lipodystrophy

United States
NYU Langone Health

Withdrawn

The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia

Nausea | Vomiting | Familial Dysautonomia

United States
NYU Langone Health

Completed

Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia

Familial Dysautonomia
Daiichi Sankyo, Inc.

Terminated

Efficacy and Safety of the ACAT Inhibitor CS-505 (Pactimibe) for Reducing the Progression of Carotid Artery Disease. This Study is Also Known as CAPTIVATE.

Atherosclerosis | Heterozygous Familial Hypercholesterolemia

United States, United Kingdom, Spain, South Africa, Israel, Canada, Netherlands, Norway, Sweden
Virginia Commonwealth University

Not yet recruiting

Southeastern ATTR Amyloidosis Consortium: SEATTRAC Family Registry

Amyloidosis, Hereditary

United States
Arizona State University
Arizona Health Care Cost Containment System

Enrolling by invitation

Family Check-Up Online (FCO)

Families of Middle School Students at Title 1 Schools

United States
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Cardiovascular Evaluation of Patients With High Cholesterol and Normal Volunteers

Homozygous Familial Hypercholesterolemic

United States
Eidos Therapeutics, a BridgeBio company

Completed

Study of AG10 in Amyloid Cardiomyopathy

Familial ATTR-CM (ATTRm-CM, or FAC) | Wild-type ATTR-CM (ATTRwt-CM)

United States
Centre Medico-Chirurgicale et Obstetrique Cote...

Unknown

Place of Ultrasound Guidance of Surgery Site in Surgery for Lumbar Canal Stenosis and Lumbar Disc Herniations

Lumbar Stenosis, Familial

France
Assistance Publique - Hôpitaux de Paris

Completed

Identification of Genes and Pathogenesis Involved in Familial Thoracic Aortic Aneurysm (TAA)

Thoracic Aortic Aneurysm

France
National Human Genome Research Institute (NHGRI)

Completed

Genetic Analysis of Familial Melanoma

Melanoma

United States
AstraZeneca

Recruiting

Transthyretin Amyloidosis Cardiomyopathy in Patients With HFpEF in Russia (TETRAMER)

Transthyretin Amyloidosis Cardiomyopathy, Heart Failure With Preserved Ejection Fraction

Russian Federation
NYU Langone Health

Completed

Carbidopa for the Treatment of Excessive Blood Pressure Variability (CarbiFD)

Dysautonomia, Familial | Baroreflex Failure Syndrome

United States
Ionis Pharmaceuticals, Inc.

Approved for marketing

ATTR Expanded Access Program (EAP) by Ionis

Amyloidosis, Hereditary
University Hospital, Montpellier

Completed

Better Understand Children and Adolescents' Intrafamilial Oppositional Defiant Disorder (TOPi)

Oppositional Defiant Disorder With Familial Setting

France
Fondazione I.R.C.C.S. Istituto Neurologico Carlo...

Recruiting

SEarchiNg biomarkErs Cerebral Amyloid Angiopathy (SENECA) (SENECA)

Cerebral Amyloid Angiopathy

Italy
Pfizer

Completed

This Study Will Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis

Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype

United States
Pfizer

Completed

Study Evaluating the Safety,Tolerability and Efficacy of PF-04360365 in Adults With Probable Cerebral Amyloid Angiopathy

Cerebral Amyloid Angiopathy

Canada, United States, Netherlands, France, United Kingdom
University Hospital, Montpellier

Terminated

Prevalence of POU4F3 and SLC17A8 Mutations

Familial Deafness

France
University Hospital, Montpellier
Soroptimist association of Monaco

Recruiting

Analyse of Tumour and Constitutional DNA for the Study of the Determinism in Child Neoplasia (ALADIN)

Solid Tumor, Childhood | Pediatric Tumor | Familial Cancer | Hematological Tumor

France
Alnylam Pharmaceuticals

Recruiting

A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy (cAPPricorn-1)

Cerebral Amyloid Angiopathy

Canada
Shenzhen Second People's Hospital

Not yet recruiting

the Safety and Efficacy of Antiplatelet Therapy in Patients of CAA

Cerebral Amyloid Angiopathy
Centre Hospitalier Universitaire de Nīmes

Completed

Periventricular White Matter Hyperintensities in Cerebral Amyloid Angiopathy and Hypertensive Arteriopathy (PVWMH)

Cerebral Amyloid Angiopathy | Hypertensive Arteriopathy

France
Anna Olczak

Completed

Balance Eyesight and Muscle Tension in the Cervical Spine in Cerebral Amyloid Angiopathy

Cerebral Amyloid Angiopathy

Poland
Fondazione IRCCS Istituto Nazionale dei Tumori,...

Completed

Preventive Anti-inflammatory Diet to Reduce Gastro-intestinal Inflammation in FAP Patients: a Prospective Pilot Study (FAPPER)

Familial Adenomatous Polyposis (FAP)

Italy
University Hospital, Strasbourg, France

Unknown

Identification of Mutations Responsible for Rare Familial Skin Diseases by Next Generation Sequencing (DERMA-SEQ)

Familial Lipomatosis | Very Rare Dermatologic Diseases

France
Mayo Clinic

Not yet recruiting

Sotatercept on Central Cardiopulmonary Performance and Peripheral Oxygen Transport During Exercise in Pulmonary Arterial Hypertension

Pulmonary Arterial Hypertension

United States
International University of Health and Welfare
Japan Agency for Medical Research and Development; Chugai Pharmaceutical; Keio...

Recruiting

Satralizumab in the Treatment of Pulmonary Arterial Hypertension (SATISFY-JP Trial)

Pulmonary Arterial Hypertension

Japan
Central Hospital, Nancy, France

Not yet recruiting

Assessment of Right Ventricular Function by Cardiac MRI in Patients With Pulmonary Arterial Hypertension (IRMA) (IRMA)

Pulmonary Arterial Hypertension
Vanderbilt University Medical Center
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

The MObile Health InterVEntion in Pulmonary Arterial Hypertension (MOVE PAH) Study (MOVE PAH))

Pulmonary Arterial Hypertension

United States
Stephen Y. Chan
National Heart, Lung, and Blood Institute (NHLBI); Bayer

Recruiting

PET Image in PAH Patients

Pulmonary Arterial Hypertension

United States
Acceleron Pharma, Inc., a wholly-owned subsidiary...

Recruiting

Study of Sotatercept in Newly Diagnosed Intermediate- and High-Risk PAH Participants (MK-7962-005/A011-13) (HYPERION)

Pulmonary Arterial Hypertension

United States, Belgium, France, Germany, Spain, Argentina, Australia, Austria, Colombia, Croatia, Denmark, Greece, Israel, Korea, Republic of, Netherlands, Portugal, Serbia, Taiwan, Brazil, Canada, Czechia, Italy, New Zealand, Sweden, S... and more
SoniVie Inc.

Not yet recruiting

TROPHY PAH Pivotal Study - TReatment of Pulmonary HYpertension for PAH Pivotal Study

Pulmonary Arterial Hypertension
Gmax Biopharm LLC.

Recruiting

A Study of GMA301 in Subjects With Pulmonary Arterial Hypertension

Pulmonary Arterial Hypertension

United States, China

Clinical Trials on Familial Hemophagocytic Lymphohistiocytosis

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Latvia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations