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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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05898620
The ClinicalTrials.gov registry entry for NCT05898620 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 33 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 524-participant average among 18 other Rett Syndrome trials with a reported enrollment target (94% lower). The listed sponsor is Neurogene, which has 6 total studies on file at ClinicalTrials.gov.
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 reports 16 study locations spanning 15 distinct geographic areas - top geographies include 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
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-
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-
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Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.