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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05898620
Recruitment Status : Recruiting
First Posted : June 12, 2023
Last Update Posted : March 13, 2024
Sponsor:
Information provided by (Responsible Party):
Neurogene Inc.

Study Description
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Brief Summary:
This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in female children with typical Rett syndrome.

Condition or disease Intervention/treatment Phase
Rett Syndrome Genetic: NGN-401 Phase 1 Phase 2

Detailed Description:

The study is a phase 1/2, open-label study designed to assess the safety, tolerability, and efficacy of administration of an adeno-associated viral vector serotype 9 (AAV9), using Neurogene's proprietary transgene regulation technology. NGN-401 contains a full-length human MECP2 gene which is designed to express therapeutic levels of the MECP2 protein while avoiding overexpression.

The study treatment will be administered under general anesthesia via intracerebroventricular (ICV) delivery. Each participant will be followed for safety and preliminary efficacy for 5 years after treatment and is expected to enroll in a long-term follow-up study for 10 years.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2, Open-Label Clinical Study to Evaluate Safety, Tolerability, and Efficacy of NGN-401 in Pediatric Subjects With Rett Syndrome
Actual Study Start Date : June 13, 2023
Estimated Primary Completion Date : October 2029
Estimated Study Completion Date : October 2029

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arms and Interventions
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Arm Intervention/treatment
Experimental: Low Dose
Dose Level 1
 Genetic: NGN-401
NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.
Experimental: High Dose
Dose Level 2
 Genetic: NGN-401
NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: 5 years ]
    Incidence, type, severity, and frequency of TEAEs

  2. Incidence of Serious Adverse Events (SAEs) [ Time Frame: 5 years ]
    Incidence, type, severity, and frequency of SAEs

  3. Incidence of Adverse Events of Special Interest (AESIs) [ Time Frame: 5 years ]
    Incidence, type, severity, and frequency of AESIs

  4. Incidence of clinical laboratory abnormalities [ Time Frame: 5 years ]
    Incidence, type, severity, and frequency of clinical laboratory abnormalities

  5. Incidence of new physical and neurologic exam abnormalities [ Time Frame: 5 years ]
    Incidence, type, severity, and frequency of new physical and neurologic exam abnormalities


Eligibility Criteria
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Ages Eligible for Study:   4 Years to 10 Years   (Child)
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion 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 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 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

Other inclusion or exclusion criteria apply.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05898620


Contacts
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Contact: Contact Center +1 877-237-5020 medicalinfo@neurogene.com

Locations
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United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact       NeurogeneRettStudy@childrenscolorado.org   
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact    617-355-5230    rettresearch@childrens.harvard.edu   
United States, Texas
Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Contact    832-824-7729    NeurogeneRettStudy@BCM.edu   
Sponsors and Collaborators
Neurogene Inc.
Investigators
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Study Director: Julie Jordan, MD Neurogene Inc.
More Information
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Responsible Party: Neurogene Inc.
ClinicalTrials.gov Identifier: NCT05898620    
Other Study ID Numbers: RTT-200
First Posted: June 12, 2023    Key Record Dates
Last Update Posted: March 13, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Neurogene Inc.:
Typical Rett Syndrome
MECP2
Rett Disorder
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Neurodevelopmental Disorders
Neurobehavioral Manifestations
 Neurologic Manifestations
Intellectual Disability
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Pathologic Processes
RTT
Additional relevant MeSH terms:
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Rett Syndrome
Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
 Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System