A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

Running, not enrolling · Phase 3

Conditions studied: Rett Syndrome

In brief

This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.

Key facts

Study ID

NCT05898620

Run by

Neurogene Inc.

People needed

33

Starts

2023-06-13

Expected to finish

2029-12-01

Last updated by the study team

2026-06-10

Who can join

Age: 3 and older. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• Females who are between the ages of ≥4 and ≤10 years for Arms 1 and 2 (Arms closed). Females who are ≥11 years of age or older for Arm 3 (Arm closed). Females who are ≥3 for Arm 4, the pivotal cohort.
• Diagnosis of typical Rett syndrome with a documented disease-causing mutation in the methyl-CpG-binding protein 2 (MECP2) gene
• Current anti-epileptic drug regimen has been stable for at least 12 weeks
• Participant must be in the post-regression stage
• Participant and caregiver should reside within a 2-hour drive of the study center for at least 3 months following treatment
• Participant must have never taken trofinetide or have taken trofinetide and discontinued due to tolerability, lack of efficacy, or other reasons. Following NGN-401 dosing, trofinetide may be initiated after a specified time period and with the support of the treating clinician.

You may not qualify if…

• Normal or near normal hand function
• Has a current clinically significant condition other than Rett syndrome
• Presence of a concomitant medical condition that precludes intracerebroventricular administration, or use of anesthetics or immune suppression needed for study related procedures
• Other inclusion and exclusion criteria apply.

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Nicklaus Children's Hospital Research Institute — Miami, Florida, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Montefiore Medical Center — New York, New York, United States
• UNC at Chapel Hill — Chapel Hill, North Carolina, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• Texas Children's Hospital — Houston, Texas, United States
• The Children's Hospital at Westmead — Sydney, New South Wales, Australia
• Royal Hospital for Children and Young People — Edinburgh, United Kingdom
• Manchester University NHS Foundation Trust — Manchester, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.