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Condition: Genetic Disease Synced from ClinicalTrials.gov · July 28, 2026

Recruiting studies: Genetic Disease

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Li-Fraumeni Syndrome/TP53 Biobank
Phase N/A · 2 location(s) · Abramson Cancer Center at Penn Medicine · NCT04367246
Cycling-Based Priming Coordination Training for Enhancing Ataxia Recovery and Brain Plasticity
NA · 1 location(s) · Chang Gung University · NCT06600269
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
Phase N/A · 6 location(s) · Genetix Biotherapeutics Inc. · NCT06271512
Phase 2 Randomized Double-blind Study to Assess Topical Zabalafin Hydrogel Vs Vehicle in Mild to Moderate AD.
Phase 2 · 12 location(s) · Alphyn Biologics · NCT06855745
Novel Genetic Disorders of the Immune System
Phase N/A · 1 location(s) · National Institute of Allergy and Infectious Diseases (NIAID · NCT02257892
Food-Specific and Component IgE Threshold Levels That Predict Food Allergy in People With Elevated Total Serum IgE Level
Phase 2 · 1 location(s) · National Institute of Allergy and Infectious Diseases (NIAID · NCT03835767
Topotecan and Melphalan for Retinoblastoma
Phase 3 · 1 location(s) · Sun Yat-sen University · NCT04799002
Exercise Training in Transthyretin Cardiac Amyloidosis
NA · 1 location(s) · Brigham and Women's Hospital · NCT05797857
Genetic Variants and Susceptibility to Diseases of Prematurity in Very Low Birth-Weight Infants
Phase N/A · 1 location(s) · Medical College of Wisconsin · NCT00710112
Cardiac Structure and Function in MPS
Phase N/A · 3 location(s) · Children's Hospital of Orange County · NCT07361536
A Phase 2 Trial of Dotinurad in Xanthine Oxidase Inhibitor (XOI) Intolerant/Uricase Failure Gout Participants
Phase 2 · 17 location(s) · Crystalys Therapeutics · NCT07535034
A Bilingual Virtually-based Intervention (PEDALL) for the Prevention of Weight Gain in Childhood ALL Patients Considerin
NA · 1 location(s) · Columbia University · NCT05963971
Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI
Phase 2 · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT06887777
Clinical Research in ALS Study
Phase N/A · 1 location(s) · University of Miami · NCT00136500
Mirdametinib in Patients With Advanced NF1-mutant Melanoma
Phase 2 · 1 location(s) · Kevin Kim, MD · NCT07237100
A Gaucher Disease Gene Therapy Trial With FLT201
Phase 3 · 33 location(s) · Spur Therapeutics · NCT07223944
Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)
Phase 2 · 43 location(s) · Vertex Pharmaceuticals Incorporated · NCT07161037
Genetic and Environmental Risk Factors for Hemorrhagic Stroke
Phase N/A · 7 location(s) · State University of New York at Buffalo · NCT00682695
Tolerance and Efficacy Open-label Study With RV4421B, in Various Populations With Atopic Dermatitis for 12 Weeks
NA · 1 location(s) · Pierre Fabre Dermo Cosmetique · NCT07014007
Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy
Phase N/A · 1 location(s) · National Institute of Allergy and Infectious Diseases (NIAID · NCT07416201
Modified Orbital Decompression in the Treatment of Moderate-to-severe Grave's Ophthalmopathy
NA · 1 location(s) · Sun Yat-sen University · NCT06588764
Utility of PCD Diagnostics to Improve Clinical Care
NA · 1 location(s) · Vanderbilt University Medical Center · NCT03704207
The Severe Von Willebrand Disease (sVWD) Patient Registry
Phase N/A · 1 location(s) · VWD Connect Foundation · NCT05437536
A Personalized Surveillance and Intervention Protocol for Patients With Familial Adenomatous Polyposis That Have Undergo
Phase N/A · 2 location(s) · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC · NCT04678011
Identification of Cutaneous and Blood Biomarkers Predictive of Response to Systemic Treatments During Chronic Inflammato
Phase N/A · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT06599411
Boston Early-Onset Chronic Obstructive Pulmonary Disease (COPD) Study
Phase N/A · 1 location(s) · Brigham and Women's Hospital · NCT01177618
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
Phase N/A · 10 location(s) · Astellas Gene Therapies · NCT06581146
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Pr
Phase 4 · 2 location(s) · Sanofi · NCT06145373
The Application of T1 Mapping in Real-World
Phase N/A · 1 location(s) · Chinese Academy of Medical Sciences, Fuwai Hospital · NCT07354646
A Study of JNJ-95597528 in Participants With Moderate to Severe Atopic Dermatitis
Phase 2 · 62 location(s) · Janssen Research & Development, LLC · NCT07230860
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.