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Clinical trial condition

Genetic Diseases, X-Linked

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

33 public trials 8 recruiting studies
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Genetic Diseases, X-Linked trial results

Active, not recruiting

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

NCT07480564

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +12 more
Locations
  • Boston Children's Hospital Boston, Massachusetts, United States
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Rush University Medical Center Chicago, Illinois, United States
View Trial Details
Active, not recruiting

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

NCT05606614

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +12 more
Locations
  • City of Hope Duarte, California, United States
  • UC San Diego La Jolla, California, United States
  • UC San Francisco Benioff Children's Hospital Oakland, California, United States
  • Rush University Medical Center Chicago, Illinois, United States
View Trial Details
Active, not recruiting

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

NCT05898620

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +12 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Nicklaus Children's Hospital Research Institute Miami, Florida, United States
View all 16 locations
View Trial Details
Completed

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +8 more
Locations
  • Phoenix Childrens Hospital Phoenix, Arizona, United States
  • Children's Hospital of Los Angeles Los Angeles, California, United States
  • University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland Oakland, California, United States
  • Stanford University Medical Center Palo Alto, California, United States
View all 64 locations
View Trial Details
Completed

Mackenzie's Mission: The Australian Reproductive Carrier Screening Project

NCT04157595

Conditions
Autosomal Recessive Disorder, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, +6 more
Locations
  • Forster Community Health Service Forster, New South Wales, Australia
  • Liverpool Hospital Liverpool, New South Wales, Australia
  • Hunter Genetics Newcastle, New South Wales, Australia
  • Royal Hospital for Women Sydney, New South Wales, Australia
View all 24 locations
View Trial Details
Completed

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT03406780

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • University of California, Davis Sacramento, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Nemours Children's Hospital Orlando, Florida, United States
  • University of Iowa Iowa City, Iowa, United States
View Trial Details
Completed

Neural Biomarkers in Fragile X Syndrome

NCT06957054

Conditions
Central Nervous System Diseases, Chromosome Disorders, Congenital Abnormalities, +20 more
Locations
  • Hospital del Mar Research Institute (HMRI) Barcelona, Spain
  • Consorci Corporació Sanitaria Parc Taulí. Institut Investigació i Innovació Parc Taulí (I3PT) Sabadell, Barcelona, Spain
View Trial Details
Completed

To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome

NCT06849973

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • Biomed Testing Facility # BIO-04-92093 La Jolla, California, United States
  • Biomed Testing Facility # BIO-03-90095 Los Angeles, California, United States
  • Biomed Testing Facility # BIO-02-95817 Sacramento, California, United States
  • Biomed Testing Facility # BIO-01-94104 San Francisco, California, United States
View all 16 locations
View Trial Details

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