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

Description

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

Conditions

Rett Syndrome

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Study Overview

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Study Details

Study overview

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

A Phase 1/2, Open-Label Clinical Study to Evaluate Safety, Tolerability, and Efficacy of NGN-401 in Subjects With Rett Syndrome

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

Condition
Rett Syndrome
Intervention / Treatment

-

Contacts and Locations

Aurora

Children's Hospital Colorado, Aurora, Colorado, United States, 80045

Chicago

Rush University Medical Center, Chicago, Illinois, United States, 60612

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

New York

Montefiore Medical Center, New York, New York, United States, 10467

Houston

Texas Children's Hospital, Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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 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 lack of 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
  • * 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
  • * Grossly abnormal psychomotor development in the first 6 months of life
  • * A history of other genetic disorders or neurological conditions, such as stroke, brain tumor, or autoimmune processes affecting the central nervous system

Ages Eligible for Study

4 Years to

Sexes Eligible for Study

FEMALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Neurogene Inc.,

Julie Jordan, MD, STUDY_DIRECTOR, Neurogene Inc.

Study Record Dates

2029-10