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

NCT05898620 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 33

Last updated 2026-03-13

No results posted yet for this study

Summary

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

Conditions

Interventions

GENETIC

NGN-401

NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.

Sponsors & Collaborators

  • Neurogene Inc.

    lead INDUSTRY

Principal Investigators

  • Julie Jordan, MD · Neurogene Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
SEQUENTIAL

Eligibility

Min Age
3 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-06-13
Primary Completion
2029-12-31
Completion
2029-12-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05898620 on ClinicalTrials.gov