A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
Public ClinicalTrials.gov record NCT05898620. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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)
Study identification
- NCT ID
- NCT05898620
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Neurogene Inc.
- Industry
- Enrollment
- 33 participants
Conditions and interventions
Conditions
Interventions
- NGN-401 Genetic
Genetic
Eligibility (public fields only)
- Age range
- 3 Years and older
- Sex
- Female
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 12, 2023
- Primary completion
- Nov 30, 2029
- Completion
- Nov 30, 2029
- Last update posted
- Jun 9, 2026
2023 – 2029
United States locations
- U.S. sites
- 13
- U.S. states
- 13
- U.S. cities
- 13
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | — |
| UCSF Benioff Children's Hospital Oakland | Oakland | California | 94609 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Nicklaus Children's Hospital Research Institute | Miami | Florida | 33155 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Montefiore Medical Center | New York | New York | 10467 | — |
| UNC at Chapel Hill | Chapel Hill | North Carolina | 27514 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05898620, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 9, 2026 · Synced Jul 23, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05898620 live on ClinicalTrials.gov.