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)
Neurogene Inc.
Summary
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
Description
The pivotal study, Embolden (TM), is a conversion of the phase 1/2 study and is an open-label, baseline-controlled, multicenter, single-arm study designed to assess the efficacy, safety, and tolerability of administration of NGN401, an adeno-associated viral vector serotype 9 (AAV9) using Neurogene's proprietary transgene regulation technology. NGN-401 contains a full-length human MECP2 gene and is designed to express therapeutic levels of the MeCP2 protein while avoiding overexpression. The study treatment will be given as a single administration under general anesthesia via intracerebrovent…
Eligibility
- Age range
- 3+ years
- Sex
- Female
- Healthy volunteers
- No
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 trea…
Interventions
- GeneticNGN-401
NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.
Locations (16)
- University of Alabama at BirminghamBirmingham, Alabama
- UCSF Benioff Children's Hospital OaklandOakland, California
- Children's Hospital ColoradoAurora, Colorado
- Nicklaus Children's Hospital Research InstituteMiami, Florida
- Rush University Medical CenterChicago, Illinois
- Kennedy Krieger InstituteBaltimore, Maryland