This study is in progress, not accepting new patients

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

a study on Rett Syndrome

Summary

Eligibility
for females ages 3 years and up (full criteria)
Location
at UCSF
Dates
study started
study ends around
Principal Investigator
by Alexander Fay (ucsf)

Description

Summary

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

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)

Details

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 intracerebroventricular (ICV) delivery. Each participant will be followed for efficacy and safety for 3 years after treatment, and is expected to enroll in a long-term follow-up study for 12 years.

Keywords

Rett Syndrome, Typical Rett Syndrome, MECP2, Rett Disorder, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Neurodevelopmental Disorders, Neurobehavioral Manifestations, Neurologic Manifestations, Intellectual Disability, Nervous System Diseases, Pathologic Processes, RTT, Inborn Genetic Diseases, X-Linked Genetic Diseases, NGN-401

Eligibility

You can join if…

Open to females ages 3 years and up

  • 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 CAN'T join 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.

Locations

  • UCSF Benioff Children's Hospital Oakland
    Oakland California 94609 United States
  • Children's Hospital Colorado
    Aurora Colorado 80045 United States

Lead Scientist at University of California Health

  • Alexander Fay (ucsf)
    Associate Professor, Neurology, School of Medicine. Authored (or co-authored) 20 research publications

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Neurogene Inc.
ID
NCT05898620
Phase
Phase 3 Rett Syndrome Research Study
Study Type
Interventional
Participants
Expecting 33 study participants
Last Updated