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NCT05898620 · ClinicalTrials.gov registry record · Phase 3
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
A Phase 3 study of Rett Syndrome, sponsored by Neurogene.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 33
- Enrollment target
- 16
- Study locations
NCT05898620: Recruiting Phase 3 study of Rett Syndrome, sponsored by Neurogene.
NCT05898620 is a Phase 3 study of Rett Syndrome that is actively recruiting participants, run by Neurogene. The registered enrollment target is 33 participants, below the 524-participant average among 18 other Rett Syndrome trials with a reported enrollment target (94% lower). The trial reports 16 study locations across 15 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05898620, a Phase 3 study of Rett Syndrome, is actively recruiting participants, sponsored by Neurogene.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 33 participants
- Enrollment target
- 16
- Study locations
Study Summary
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
Primary Outcome
Responders will be defined as participants who: * Attain a CGI-I score of ≤ 3 ("minimally improved"); * and gain any one developmental milestone/skill from a list of 28, as captured through standardized video recordings and independently verified by blinded central raters.
Conditions Studied
Interventions
- GENETIC NGN-401
Study Locations (16)
Other
- Royal Hospital for Children and Young People - Edinburgh
- Manchester University NHS Foundation Trust - Manchester
Alabama
- University of Alabama at Birmingham - Birmingham
California
- UCSF Benioff Children's Hospital Oakland - Oakland
Colorado
- Children's Hospital Colorado - Aurora
Florida
- Nicklaus Children's Hospital Research Institute - Miami
Illinois
- Rush University Medical Center - Chicago
Maryland
- Kennedy Krieger Institute - Baltimore
Massachusetts
- Boston Children's Hospital - Boston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 33 participants |
| Start Date | 2023-06-13 |
| Est. Completion | 2029-12 |
| Phase | Phase 3 |
What NCT05898620 shows while recruiting
NCT05898620 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 33 participants, a relatively small participant target, below the 524-participant average among 18 other Rett Syndrome trials with a reported enrollment target (94% lower).
The record links to 1 condition, with Rett Syndrome appearing as the primary indexed condition, and to 1 intervention - of which NGN-401 is the first listed.
NCT05898620 lists 16 locations in 15 states (Other, Alabama, California).
Frequently Asked Questions
What is clinical trial NCT05898620 about?
NCT05898620 is a clinical study titled "A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome". This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
What is the current status of trial NCT05898620?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 33 participants. The study started on 2023-06-13. Estimated completion is 2029-12.
What conditions does trial NCT05898620 study?
This clinical trial studies the following conditions: Rett Syndrome.
What interventions are being tested in trial NCT05898620?
The interventions under investigation include: NGN-401 (GENETIC).
Who is sponsoring clinical trial NCT05898620?
This trial is sponsored by Neurogene, which has 6 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05898620 being conducted?
This trial has 16 study locations across Alabama, California, Colorado, Florida, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Rett Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05898620's enrollment target sits among peer trials
33 11th of 18 higher than 8 of 18 other Rett Syndrome trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Rett Syndrome trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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