🏛️ Verified NIH Record • Updated 2026-06-08
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome (NCT05898620)
A Baseline-Controlled, Open-Label, Multicenter, Single-Arm, Pivotal Study to Evaluate the Efficacy, Safety, and Tolerability of NGN-401 in Subjects With Rett Syndrome (Embolden)
🔵 Active, Not Recruiting Phase3 📋 Clinical Research Protocol Sponsor: Neurogene Inc.
🩺 Plain-English Summary (8th-Grade Level)
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
Who can join: Review the eligibility criteria below.
Location: University of Alabama at Birmingham — Birmingham, Alabama
Age range: 3 Years
🧪 Interventions in This Study
- NGN-401 (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 (16 Sites)
- University of Alabama at Birmingham — Birmingham, Alabama
- UCSF Benioff Children's Hospital Oakland — Oakland, California
- Children's Hospital Colorado — Aurora, Colorado
- Nicklaus Children's Hospital Research Institute — Miami, Florida
- Rush University Medical Center — Chicago, Illinois
- Kennedy Krieger Institute — Baltimore, Maryland
- Boston Children's Hospital — Boston, Massachusetts
- Montefiore Medical Center — New York, New York
- UNC at Chapel Hill — Chapel Hill, North Carolina
- Nationwide Children's Hospital — Columbus, Ohio
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Vanderbilt University Medical Center — Nashville, Tennessee
- Texas Children's Hospital — Houston, Texas
- The Children's Hospital at Westmead — Sydney, New South Wales
- Royal Hospital for Children and Young People — Edinburgh,
- Manchester University NHS Foundation Trust — Manchester,
💰 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 Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) — Chicago, Illinois
- 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 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.