Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) (NCT06152237)
A Multicenter, Open Label, Randomized, Dose-Escalation and Dose-Expansion Study of the Safety, Tolerability, and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females With Rett Syndrome
🩺 Plain-English Summary (8th-Grade Level)
The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.
Who can join: Review the eligibility criteria below.
Location: Rush University Medical Center & Children's Hospital — Chicago, Illinois
Age range: 5 Years to 8 Years
🧪 Interventions in This Study
- TSHA-102 (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (5 Sites)
- Rush University Medical Center & Children's Hospital — Chicago, Illinois
- Gillette Children's Specialty Healthcare — Saint Paul, Minnesota
- Washington University, St. Louis — St Louis, Missouri
- CHU Ste-Justine — Montreal, Quebec
- Children's Neurosciences, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust — London,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Rett Syndrome
- Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome — Birmingham, Alabama
- Technology-supported Motor Rehabilitation for People With Rett Syndrome — Bethesda, Maryland
- A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study) — Duarte, California
- A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome — Birmingham, Alabama
- A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome — Toronto, Ontario
Explore by condition: All Rett Syndrome clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.