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Not recruiting
Phase 3

NGN-401

Sponsor:

Neurogene Inc.

Code:

NCT05898620

Conditions

Rett Syndrome

Eligibility Criteria

Sex: Female

Age: 3+

Healthy Volunteers: Not accepted

Interventions

NGN-401

Study Details

Brief summary:

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

Conditions

Rett Syndrome

Study ID

NCT05898620

Start date

Jun 13, 2023

Status verified date

Jun, 2026

Completion date

Dec, 2029

Anticipated

Primary completion date

Dec, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Female

Age: 3+

Healthy Volunteers: Not accepted

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

Other inclusion and exclusion criteria apply.

Study Design

Enrollment

33 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Pediatric 1e15 vg dose (fully enrolled)

Dose Level 1 for ages 4-10 years

experimental: Adolescent/Adult 1e15 vg Dose (fully enrolled)

Dose Level 1 for ages 11 years \& above

experimental: Pediatric 3e15 vg dose (discontinued)

Dose Level 2 for ages 4-10 years (discontinued)

experimental: Pivotal Cohort

Dose Level 1 for ages 3 and above

Interventions

NGN-401

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

Primary outcome measure

  • Efficacy of NGN-401 [ Time Frame: 52 Weeks ]

Central Contacts and Locations

More Information

Sponsor

Neurogene Inc.

Last update posted

Jun 10, 2026

Last verified

Jun, 2026

Keywords

  • 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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-20. This information was provided to ClinicalTrials.gov by Neurogene Inc. on 2026-06-10.