Clinical Trials on Familial Hyperinsulinism

Total 4360 results

University Hospital, Basel, Switzerland

Completed

Effects of FXR Activation on Hepatic Lipid and Glucose Metabolism

Metabolic Syndrome | Familial Combined Hyperlipidemia | Familial Hypertriglyceridemia

Switzerland
Assistance Publique Hopitaux De Marseille

Not yet recruiting

Familial Aortopathies and Cellular Exploration (FACE)

Familial Aortopathies

France
National Medical Research Center for Therapy and...
Moscow State University of Medicine and Dentistry

Recruiting

Genetic Testing and Motivational Counseling for FH (GENMOTIV-FH)

Medication Adherence | Adherence, Medication | Treatment Adherence | Familial Hypercholesterolemia | Motivational Interviewing | Adherence, Patient | Treatment Adherence and Compliance | Patient Compliance | Adherence | Hypercholesterolemia, Familial | Patient Adherence | Hypercholesterolemia, Autosomal Dominant and other conditions

Russian Federation
Alnylam Pharmaceuticals

Completed

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

ATTR Amyloidosis | Transthyretin (TTR)-Mediated Amyloidosis | Familial Amyloidotic Polyneuropathy (FAP) | Familial Amyloid Neuropathies

France, Germany, Portugal, United Kingdom, Sweden, Spain
Leiden University Medical Center
Jacobijn Gussekloo, Prof. dr.; Simon P. Mooijaart, Prof. dr.

Recruiting

Longevity Families of the Netherlands (LOF-NL)

Aging | Extreme Longevity | Longevity | Familial Longevity

Netherlands
CENTOGENE GmbH Rostock
Alnylam Pharmaceuticals

Recruiting

TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects (TRAMmoniTTR)

Cardiomyopathies | Polyneuropathies | Transthyretin Amyloidosis | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

Germany, Austria, Switzerland
Regeneron Pharmaceuticals
Sanofi

Terminated

Long Term Safety Study of PRALUENT

Heterozygous Familial Hypercholesterolemia | Non-familial Hypercholesterolemia

United States, Bulgaria, Estonia, Russian Federation, South Africa, Ukraine
Merck Sharp & Dohme LLC

Terminated

A Study of Extended-release (ER) Niacin/Laropiprant in Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0524A-158)

Hypercholesterolemia, Familial | Heterozygous Familial Hypercholesterolemia
The Cleveland Clinic
National Heart, Lung, and Blood Institute (NHLBI)

Active, not recruiting

Pulmonary Arterial Hypertension Improvement With Nutrition and Exercise (PHINE)

Insulin Resistance | Pulmonary Arterial Hypertension

United States
Eidos Therapeutics, a BridgeBio company

Withdrawn

Efficacy and Safety of Acoramidis (AG10) in Subjects With Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Boston University
National Institute of Neurological Disorders and Stroke (NINDS); Food and Drug...

Completed

The Effect of Diflunisal on Familial Amyloidosis

Familial Amyloid Polyneuropathy | Familial Amyloidosis

United States, Italy, Japan, Sweden, United Kingdom
Pfizer

Completed

The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin

Transthyretin Familial Amyloid Polyneuropathy

Japan
Marcello Arca
Pfizer; Göteborg University; University of Helsinki

Not yet recruiting

In Vivo Metabolism of apoB-containing Lipoproteins in ANGPTL3 Deficient Subjects

Familial Hypobetalipoproteinemia | Familial Hypobetalipoproteinemia - Heterozygous Form
Institut Investigacio Sanitaria Pere Virgili

Recruiting

Early Detection of Familial Hypercholesterolemia in Children (DECOPIN)

Familial Hypercholesterolemia | Familial Hypercholesterolemia - Homozygous | Familial Hypercholesterolemia - Heterozygous

Spain
Novartis Pharmaceuticals

Active, not recruiting

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia (ORION-13)

Familial Hypercholesterolemia - Homozygous

Greece, Lebanon, Turkey, France, Canada, Malaysia, Netherlands, United States
Alnylam Pharmaceuticals

Approved for marketing

Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies
Eidos Therapeutics, a BridgeBio company

Withdrawn

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

United States
SOM Innovation Biotech SA
Hospital Vall d'Hebron

Completed

Study of SOM0226 in Familial Amyloid Polyneuropathy

Familial Amyloid Polyneuropathy (FAP)

Spain
University Hospital, Lille

Recruiting

Study of Cortisol Metabolism in Familial Partial Lipodystrophy Type 2 (LIPOCORT)

Familial Partial Lipodystrophy Type 2

France
Alnylam Pharmaceuticals

Completed

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
Novartis Pharmaceuticals

Recruiting

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies (V-PEDS-OLE)

Heterozygous or Homozygous Familial Hypercholesterolemia

Netherlands, Israel, Hungary, Italy, Germany, Turkey, Spain, United States, France, Norway, Russian Federation, South Africa, United Kingdom, Canada, Switzerland, Brazil, Lebanon, Slovenia, Poland, Taiwan, Malaysia
Amgen

Completed

Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia (RAMAN)

Homozygous Familial Hypercholesterolemia HoFH

India
Intellia Therapeutics

Recruiting

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

United Kingdom, New Zealand, Sweden
Intellia Therapeutics

Active, not recruiting

Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

France, United Kingdom, New Zealand, Sweden
Novartis Pharmaceuticals

Completed

Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C (ORION-8)

Elevated Cholesterol | Homozygous Familial Hypercholesterolemia | Heterozygous Familial Hypercholesterolemia | ASCVD

United States, Canada, Czechia, Denmark, Germany, Hungary, Netherlands, Poland, South Africa, Spain, Sweden, Ukraine, United Kingdom
Mirum Pharmaceuticals, Inc.

Active, not recruiting

An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

Progressive Familial Intrahepatic Cholestasis (PFIC)

United States, Argentina, France, Singapore, United Kingdom, Belgium, Turkey, Austria, Brazil, Canada, Colombia, Germany, Italy, Lebanon, Mexico, Poland
Mirum Pharmaceuticals, Inc.

Completed

A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC) (MARCH-PFIC)

Progressive Familial Intrahepatic Cholestasis (PFIC)

Italy, United States, Argentina, France, Singapore, United Kingdom, Austria, Brazil, Mexico, Lebanon, Germany, Turkey, Poland, Belgium, Canada, Colombia, Hungary
Takeda

Active, not recruiting

A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)

Progressive Familial Intrahepatic Cholestasis (PFIC)

Japan
Mirum Pharmaceuticals, Inc.

Withdrawn

A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)

Progressive Familial Intrahepatic Cholestasis (PFIC)
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP) (BioTRAP)

Transthyretin Amyloidosis | Transthyretin Amyloid Cardiopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

India, Germany
University of British Columbia
Vancouver Coastal Health Research Institute; Genome British Columbia

Recruiting

Advancing Cardiac Care Unit-based Rapid Assessment and Treatment of hypErcholesterolemia (ACCURATE)

Acute Coronary Syndrome | Familial Hypercholesterolemia | STEMI | NSTEMI - Non-ST Segment Elevation MI | Familial Hypercholesterolemia - Heterozygous | Familial Hypercholesterolemia Due to Genetic Defect of Apolipoprotein B | Familial Hypercholesterolemia Due to Heterozygous LDL Receptor Mutation and other conditions

Canada
REGENXBIO Inc.
National Heart, Lung, and Blood Institute (NHLBI)

Terminated

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

Homozygous Familial Hypercholesterolemia (HoFH)

United States, Canada, Italy, Netherlands
Seoul National University Hospital

Recruiting

Bromfenac Sodium Hydrate Eye Drops in Familial Exudative Vitreoretinopathy

Familial Exudative Vitreoretinopathies

Korea, Republic of
Centre Hospitalier Universitaire de la Réunion

Completed

Exploratory Study of the Relationships Between the Biomarkers of Inflammation, Lipidome and Insulin Resistance and Disorders of Glycemic Regulation in a Cohort of Insulin-resistant Subjects Due to Excess Weight or Dunnigan's Lipodystrophy (IRAP-DUN 2)

Lipodystrophy | Dunnigan Syndrome

Réunion
Pfizer

Completed

Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers

Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) | Transthyretin Cardiomyopathy (TTR-CM) | Familial Amyloid Cardiomyopathy | Senile Systemic Amyloidosis (SSA)
Alnylam Pharmaceuticals

Completed

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Amyloid Neuropathies | Amyloid Neuropathies, Familial | Amyloidosis, Hereditary | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Amyloidosis, Hereditary, Transthyretin-Related | Familial Transthyretin Cardiac Amyloidosis

United States, France, Italy, Spain, Canada, Germany, Belgium, Sweden, United Kingdom
Washington University School of Medicine
Incyte Corporation

No longer available

Ruxolitinib for Chuvash Polycythemia

Erythrocytosis, Familial, 2

United States
Novartis Pharmaceuticals

Completed

A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome

Familial Chylomicronemia Syndrome (FCS)

South Africa, Spain, France, Germany, United Kingdom, United States, Canada, Netherlands
Instituto Aragones de Ciencias de la Salud
Hospital Clinic of Barcelona; Instituto de Salud Carlos III

Completed

Effect of Weight Loss on Cholesterol Metabolism in Hereditary Hypercholesterolemias and Overweight or Obesity.

Obesity | Weight Loss | Familial Hypercholesterolemias | Familial Combined Hypercholesterolemia
Pfizer

Completed

Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

Familial Amyloid Polyneuropathy

United Kingdom, Spain, United States, Argentina, Brazil, France, Germany, Portugal, Sweden
Ionis Pharmaceuticals, Inc.

Completed

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Ionis Pharmaceuticals, Inc.

Completed

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

New Zealand, United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Novartis Pharmaceuticals

Terminated

Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.

Familial Chylomicronemia Syndrome (FCS) (HLP Type I)

South Africa, Germany, United Kingdom, France, United States, Canada, Netherlands
Assistance Publique Hopitaux De Marseille

Completed

Expanding the Biomarkers in Familial Amyloid Neuropathy: MRI and Motor Unit Estimation by Electrophysiological Study

Amyloid Neuropathies | Transthyretin Amyloidosis

France
Nantes University Hospital
Pfizer

Completed

Assessment of the Prevalence of TTR Amyloid Neuropathy in a Population of Patients With Neuropathy of Unknown Aetiology (PRE-TRANS)

Transthyretin Amyloidosis | Familial Amyloid Neuropathy

France
Centre Hospitalier Universitaire de Nice

Withdrawn

Effect of Psyllium (Plantago Ovata) on Digestive Disorders in Familial Amyloidosis (Psyllium)

Familial Amyloidosis

France
University of Illinois at Chicago

Withdrawn

Digoxin for Congenital Erythrocytosis Due to Up-Regulated Hypoxia Sensing

VHL Gene Mutation | Polycythemia; Familial | Erythrocytosis; Familial | HIF-2alpha Erythrocytosis | PHD2 Erythrocytosis | Chuvash Erythrocytosis
Capital Medical University
Peking University First Hospital; The First Affiliated Hospital of Anhui Medical... and other collaborators

Recruiting

The Chinese Familial Alzheimer's Network (CFAN)

Alzheimer Disease | Familial Alzheimer Disease (FAD)

China
Pfizer

Completed

An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy

Familial Amyloid Polyneuropathy | ATTR-PN

Sweden, Brazil, Germany, Argentina, France, Portugal
University Hospital, Montpellier

Completed

Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells (DMDstem) (DMDstem)

Cardiomyopathy, Familial

France

Clinical Trials on Familial Hyperinsulinism

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Ecuador

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Drug Interventions

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