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Clinical trial condition

Rett Syndrome

Explore clinical trials studying Rett Syndrome. Study availability and eligibility vary by location and protocol.

70 public trials 12 recruiting studies
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Rett Syndrome trial results

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

Early Check: Expanded Screening in Newborns

NCT03655223

Conditions
17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
Locations
  • RTI International Research Triangle Park, North Carolina, 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
Active, Not Recruiting

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

NCT06152237

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +9 more
Locations
  • Rush University Medical Center & Children's Hospital Chicago, Illinois, United States
  • Gillette Children's Specialty Healthcare Saint Paul, Minnesota, United States
  • Washington University, St. Louis St Louis, Missouri, United States
  • CHU Ste-Justine Montreal, Quebec, Canada
View Trial Details

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