RecruitingPhase 3NCT05898620
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
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)
Sponsor
Neurogene Inc.
Enrollment
33 participants
Start Date
Jun 13, 2023
Study Type
INTERVENTIONAL
Conditions
Summary
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
Eligibility
Sex: FEMALEMin Age: 3 Years
Inclusion Criteria6
- 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.
Exclusion Criteria3
- 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
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Interventions
GENETICNGN-401
NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.
Locations(16)
View Full Details on ClinicalTrials.gov
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NCT05898620
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