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Clinical trial condition

Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

12,584 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT06547216

Conditions
Atrophies, Facioscapulohumeral, Atrophy, Facioscapulohumeral, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +33 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • Stanford University Palo Alto, California, United States
  • University of California San Diego San Diego, California, United States
  • University of Colorado Denver, Colorado, United States
View all 17 locations
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Active, not recruiting

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

NCT05222906

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Yale University School of Medicine - Investigational Site Number: 8400003 New Haven, Connecticut, United States
  • University of Iowa - Investigational Site Number: 8400002 Iowa City, Iowa, United States
  • Texas Oncology - Medical City Dallas Site Number : 8400008 Dallas, Texas, United States
  • Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001 Fairfax, Virginia, United States
View all 22 locations
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Active, not recruiting

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

NCT06181136

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • University of Iowa Stead Family Children's Hospital Iowa City, Iowa, United States
  • University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States
  • Baylor College of Medicine and Texas Children's Hospital Houston, Texas, United States
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Active, not recruiting

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

NCT02843035

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • Yale University School of Medicine Site Number : 840002 New Haven, Connecticut, United States
  • Baylor Institute of Metabolic Diseases Site Number : 840001 Dallas, Texas, United States
  • Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 840003 Fairfax, Virginia, United States
  • Investigational Site Number : 276001 Mainz, Germany
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Active, not recruiting

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

NCT03962543

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +18 more
Locations
  • University of Alabama at Birmingham/Children's of Alabama Birmingham, Alabama, United States
  • Mayo Clinic Arizona Phoenix, Arizona, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
View all 43 locations
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Active, not recruiting

Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)

NCT03213652

Conditions
Advanced Malignant Solid Neoplasm, Central Nervous System Neoplasms, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +82 more
Locations
  • Children's Hospital of Alabama Birmingham, Alabama, United States
  • Providence Alaska Medical Center Anchorage, Alaska, United States
  • Banner Children's at Desert Mesa, Arizona, United States
  • Banner University Medical Center - Tucson Tucson, Arizona, United States
View all 126 locations
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Active, not recruiting

A Study to Evaluate the Treatment Response and Safety of Two Dose Regimens of Subcutaneous Amlitelimab Compared With Treatment Withdrawal in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis

NCT06407934

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, Dermatitis Atopic, +9 more
Locations
  • Cahaba Dermatology & Skin Health Center- Site Number : 8401066 Birmingham, Alabama, United States
  • Center for Dermatology and Plastic Surgery- Site Number : 8401119 Scottsdale, Arizona, United States
  • Scottsdale Clinical Trials- Site Number : 8401149 Scottsdale, Arizona, United States
  • Eclipse Clinical Research- Site Number : 8401158 Tucson, Arizona, United States
View all 324 locations
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