Clinical Trials on Hereditary Diseases

Total 379801 results

Maastricht University Medical Center
ZonMw: The Netherlands Organisation for Health Research and Development

Completed

DMEK Versus DSAEK Study (DMEK)

Fuchs' Endothelial Dystrophy

Netherlands
Oregon Health and Science University

Terminated

OCT-guided DSAEK Graft Shaping and Smoothing (DSAEK)

Fuchs' Endothelial Dystrophy

United States
Santhera Pharmaceuticals

No longer available

Expanded Access Program for Idebenone in Patients With Leber's Hereditary Optic Neuropathy Who Completed the LEROS Study (US EAP LHON)

Leber's Hereditary Optic Neuropathy
Stealth BioTherapeutics Inc.

Completed

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

Leber's Hereditary Optic Neuropathy

United States
Massachusetts General Hospital

Completed

L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1

Hereditary Sensory and Autonomic Neuropathy Type I

United States
Brigham and Women's Hospital
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Genomic Sequencing for Childhood Risk and Newborn Illness

Genetic Predisposition to Disease | Hereditary Disease

United States
Rutgers, The State University of New Jersey
National Cancer Institute (NCI); Georgetown University

Recruiting

Personalized Oncology Promoting Equity for Black Lives (PROPEL)

Hereditary Cancer | Genetic Testing

United States
Instituto Nacional de Ciencias Medicas y Nutricion...

Completed

Educational Video Versus In-person Genetic Counseling for Hereditary Cancer

Hereditary Cancer | Genetic Counseling

Mexico
Medical University of Vienna

Recruiting

Correlation Between In-vivo Anatomy of Corneal Dystrophies as Assessed by High- Resolution Optical Coherence Tomography (OCT) Measurement and Histological Examination

Corneal Dystrophy

Austria
Pro-Change Behavior Systems

Recruiting

Previvors Recharge: A Resilience Program for Cancer Previvors (PreCharge)

Mental Health Issue | Hereditary Cancer

United States
Argo Sante
GenSight Biologics

Completed

Evaluation of Impact of Disease and Visual Disability on Quality of Life and Loss of Independence of Patients Living in France With Leber's Hereditary Optic Neuropathy (LHON) Through Qualitative and Quantitative Data Collection

Leber's Hereditary Optic Neuropathy (LHON)

France
Institut National de la Santé Et de la Recherche...

Unknown

Immunome Project Consortium for Autoinflammatory Disorders (ImmunAID)

Autoinflammatory Diseases, Hereditary
Massachusetts General Hospital
Broad Institute; Laboratory for Molecular Medicine

Completed

Investigating the Feasibility and Implementation of Whole Genome Sequencing in Patients With Suspected Genetic Disorder

Genetic Predisposition to Disease | Hereditary Disease

United States
Edison Pharmaceuticals Inc

No longer available

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

Leber's Hereditary Optic Neuropathy

United States
Takeda

Recruiting

A Study to Review the Treatment and Outcomes of Teenagers and Adults With Non-histaminergic Angioedema With Normal C1 Inhibitor in Canada (PROSPECT)

Angioedema | Hereditary Angioedema (HAE)

Canada
Takeda

Recruiting

A Study in Adults With Hereditary Angioedema (HAE) Who Currently Receive Icatibant at Home

Hereditary Angioedema (HAE)

United Kingdom
Shire
Takeda Development Center Americas, Inc.

No longer available

Expanded Access for the Prevention of Acute Attacks of 1) Hereditary Angioedema (HAE) in Children and 2) Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH) in Teenagers and Adults

Angioedema | Hereditary Angioedema (HAE)

United States, Canada, Germany, Hungary, Spain
Shire

Completed

Efficacy and Safety of Lanadelumab (SHP643) in Japanese Participants With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

Japan
Shire

Completed

A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema

Hereditary Angioedema (HAE)

United States, Canada, Australia, Austria, Colombia, Germany, Hungary, Israel, Italy, Spain
Shire

Completed

Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

United States, Canada, Jordan
Shire

Completed

Observational Safety Study for KALBITOR (Ecallantide) in the Treatment of Acute Attacks of Hereditary Angioedema

Hereditary Angioedema (HAE)

United States
Shire

Completed

Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects

Hereditary Angioedema (HAE)

United States
Shire

Completed

Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

Japan
Shire

Completed

EDEMA2: Evaluation of DX-88's Effect in Mitigating Angioedema

Hereditary Angioedema (HAE)
Shire
Dyax Corp.

Completed

A Single Increasing Dose Study to Assess Safety and Tolerability of DX-2930 in Healthy Subjects

Hereditary Angioedema (HAE)

United States
Johns Hopkins University

Recruiting

Neodymium-doped Yttrium Aluminum Garnet (Nd:YAG) Laser Treatment for Granular Corneal Dystrophy

Granular Dystrophy, Corneal

United States
University Eye Hospital, Freiburg

Completed

Supine Positioning for Graft Attachment After Descemet Membrane Endothelial Keratoplasty (SUPER-DMEK)

Fuchs' Endothelial Dystrophy

Germany
Byron Lam
National Eye Institute (NEI)

Active, not recruiting

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON)

Leber's Hereditary Optic Neuropathy

United States
Santhera Pharmaceuticals

Completed

RHODOS Follow-up Single-visit Study (RHODOS-OFU)

Leber's Hereditary Optic Neuropathy

Canada, Germany, United Kingdom
Universitaire Ziekenhuizen KU Leuven
Fund for Scientific Research, Flanders, Belgium; Funds for Research in Ophthalmology... and other collaborators

Completed

The Molecular Pathogenesis of Late-onset Fuchs' Endothelial Corneal Dystrophy

Fuchs' Endothelial Dystrophy

Belgium
Boston Children's Hospital
CureAP4 Foundation

Recruiting

Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP)

Hereditary Spastic Paraplegia | SPG50 | SPG47 | SPG51 | SPG52 | AP4-related Hereditary Spastic Paraplegia | Early Onset Hereditary Spastic Paraplegia | SPG4 | SPG3A | SPG15 | SPG11

United States
Takeda

Not yet recruiting

A Study of Lanadelumab (Takhzyro) and Icatibant (Firazyr®) in Persons With HAE in China

Hereditary Angioedema (HAE)

China
CSL Behring

Recruiting

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

Hereditary Angioedema (HAE)

United States, Israel
Takeda

Recruiting

A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

United Kingdom
Takeda

Recruiting

A Survey of Lanadelumab in Participants With Hereditary Angioedema

Hereditary Angioedema (HAE)

Japan
Takeda

Recruiting

A Study With Lanadelumab in Persons With Hereditary Angioedema (HAE) in Poland (CHOPIN)

Hereditary Angioedema (HAE)

Poland
Shire
Takeda Development Center Americas, Inc.

Recruiting

Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)

Hereditary Angioedema (HAE)

France, Austria, Australia, Spain, Czechia, Italy, United Kingdom, Germany, Israel, Ireland, Brazil, Denmark, Greece, Sweden
Shire
Takeda Development Center Americas, Inc.

Completed

A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II in North America (EMPOWER)

Hereditary Angioedema (HAE)

Canada, United States, Puerto Rico
Takeda

Completed

A Study in Teenagers and Adults With Hereditary Angioedema (HAE) Type I or Type II Who Use Lanadelumab as Long-Term Prophylaxis (INTEGRATED)

Hereditary Angioedema (HAE)

France, Germany, Austria, Greece
Shire

Completed

Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

United States, Jordan, Canada
Shire
Dyax Corp.

Completed

Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

Hereditary Angioedema (HAE)

United States, Canada, Germany, Italy, Jordan, Puerto Rico, United Kingdom
Shire

Withdrawn

Study to Evaluate Ecallantide in Paediatric Patients With Acute Attacks of Hereditary Angioedema

Hereditary Angioedema (HAE)
BioCryst Pharmaceuticals

Completed

Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

Hereditary Angioedema (HAE)

Austria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Romania, Switzerland, United Kingdom
Shire

Completed

A European Post-Authorisation Observational Study Of Patients With Hereditary Angioedema

Hereditary Angioedema (HAE)

Germany, Spain, United Kingdom, France, Italy, Belgium
CSL Behring
Chiltern International Inc.

Completed

Patient Registry Study of Berinert® in Normal Clinical Practice

Includes: Hereditary Angioedema

United States, Denmark, Germany, Switzerland
Eye Hospital Pristina Kosovo

Enrolling by invitation

Fresh Corneal Lenticule Implantation in Macular Corneal Distrophy With Relex Smile Surgery

Macular Corneal Dystrophy

Kosovo
Cornea Research Foundation of America

Completed

Trial of Netarsudil for Prevention of Corticosteroid-induced Intraocular Pressure Elevation

Fuchs' Endothelial Dystrophy | Bullous Keratopathy

United States
Glostrup University Hospital, Copenhagen

Unknown

Air Versus SF6 for Descemet's Membrane Endothelial Keratoplasty (DMEK)

Fuchs' Endothelial Dystrophy | Bullous Keratopathy

Denmark
National Cancer Institute (NCI)

Completed

Phase I-II Multiple-Dose Safety and Efficacy Study of a Selective Inhibitor of Cyclooxygenase - 2 (SC-58635) in Hereditary Non-Polyposis Colorectal Cancer (HNPCC) Patients and Carriers

Colorectal Neoplasm | Hereditary Nonpolyposis

United States
Avellino Labs USA, Inc.

Unknown

Study of the Prevalence of TGFBI Corneal Dystrophies

Corneal Dystrophy

Clinical Trials on Hereditary Diseases

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Bahamas

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

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