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Condition: Hereditary Diseases Synced from ClinicalTrials.gov · July 28, 2026

Recruiting studies: Hereditary Diseases

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 · NCT04367246Cycling-Based Priming Coordination Training for Enhancing Ataxia Recovery and Brain Plasticity
NA · 1 location(s) · Chang Gung University · NCT06600269A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
Phase N/A · 6 location(s) · Genetix Biotherapeutics Inc. · NCT06271512Phase 2 Randomized Double-blind Study to Assess Topical Zabalafin Hydrogel Vs Vehicle in Mild to Moderate AD.
Phase 2 · 12 location(s) · Alphyn Biologics · NCT06855745Cord Blood S100B Protein Levels in Neonates Following Intrauterine Transfusions for HDFN-Associated Fetal Anemia
Phase N/A · 1 location(s) · Institute of Mother and Child, Warsaw, Poland · NCT06984445Application of a Clinical Decision Support System to Reduce Mechanical Ventilation Duration After Cardiac Surgery in Chi
Phase N/A · 1 location(s) · Boston Children's Hospital · NCT05687292Novel Genetic Disorders of the Immune System
Phase N/A · 1 location(s) · National Institute of Allergy and Infectious Diseases (NIAID · NCT02257892Food-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 · NCT03835767Topotecan and Melphalan for Retinoblastoma
Phase 3 · 1 location(s) · Sun Yat-sen University · NCT04799002Efficacy and Safety of Zelpultide Alfa in Preterm Neonates at High Risk of Developing Bronchopulmonary Dysplasia (BPD)
Phase 2/Phase 3 · 50 location(s) · Airway Therapeutics, Inc. · NCT06897839Exercise Training in Transthyretin Cardiac Amyloidosis
NA · 1 location(s) · Brigham and Women's Hospital · NCT05797857Cardiac Structure and Function in MPS
Phase N/A · 3 location(s) · Children's Hospital of Orange County · NCT07361536AnovaOS Network Powered Patient Registry
Phase N/A · 1 location(s) · Anova Enterprises, Inc · NCT05013944A Phase 2 Trial of Dotinurad in Xanthine Oxidase Inhibitor (XOI) Intolerant/Uricase Failure Gout Participants
Phase 2 · 17 location(s) · Crystalys Therapeutics · NCT07535034Placental Progesterone Membrane Receptors in Fetal Growth Restriction
Phase N/A · 1 location(s) · Samsun University · NCT07701837Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI
Phase 2 · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT06887777Mirdametinib in Patients With Advanced NF1-mutant Melanoma
Phase 2 · 1 location(s) · Kevin Kim, MD · NCT07237100A Gaucher Disease Gene Therapy Trial With FLT201
Phase 3 · 33 location(s) · Spur Therapeutics · NCT07223944Phase 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 · NCT07161037Tolerance and Efficacy Open-label Study With RV4421B, in Various Populations With Atopic Dermatitis for 12 Weeks
NA · 1 location(s) · Pierre Fabre Dermo Cosmetique · NCT07014007Natural 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 · NCT07416201Modified Orbital Decompression in the Treatment of Moderate-to-severe Grave's Ophthalmopathy
NA · 1 location(s) · Sun Yat-sen University · NCT06588764Ventripoint Single Ventricle Study
NA · 1 location(s) · Duke University · NCT05262907Optimizing Low Vision Rehabilitation in Emotionally Distressed Patients With Inherited Retinal Diseases
NA · 1 location(s) · University of Michigan · NCT06651736Natural History of Craniofacial Anomalies and Developmental Growth Variants
Phase N/A · 1 location(s) · National Institute of Dental and Craniofacial Research (NIDC · NCT02639312Utility of PCD Diagnostics to Improve Clinical Care
NA · 1 location(s) · Vanderbilt University Medical Center · NCT03704207The Severe Von Willebrand Disease (sVWD) Patient Registry
Phase N/A · 1 location(s) · VWD Connect Foundation · NCT05437536A 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 · NCT04678011Identification 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 · NCT06599411Clinical Study of Curcumin in Preventing Postoperative Adhesion of Bilateral Vocal Cords
Phase 1/Phase 2 · 1 location(s) · Eye & ENT Hospital of Fudan University · NCT05688488
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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.