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

Study Details

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)

Conditions: Rett Syndrome

Study ID:

NEU24062 (NGN RTT-200)

Study Description

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

Eligibility Requirements

Inclusion Criteria

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 Criteria

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