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

PHASE1PHASE2RECRUITING

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

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Study details:

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.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

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
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    Eligibility

    Age eligible for study : 4 and older

    Healthy volunteers accepted : No

    Gender eligible for study: Female

    Things to know

    Study dates

    Study start: 2023-06-13

    Primary completion: 2029-10-01

    Study completion finish: 2029-10-01

    study type

    Study type

    TREATMENT

    phase

    Phase

      PHASE1

      PHASE2

    trial

    Trial ID

    NCT05898620

    Intervention or treatment

    GENETIC: NGN-401

    Conditions

    • Rett Syndrome

    Find a site

    Closest Location:

    The Children's Hospital at Westmead

    Research sites nearby

    Select from list below to view details:

    • The Children's Hospital at Westmead

      Sydney, New South Wales, Australia

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    Study Plan

    This section provides details of the study plan, including how the study is designed and what the study is measuring.

    How is the study designed?

    Participant Group/ArmIntervention/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.

    What is the study measuring?

    Primary outcome

    Primary Outcome MeasurePrimary Outcome DescriptionPrimary Outcome Time Frame
    Incidence of Treatment Emergent Adverse Events (TEAEs)Incidence, type, severity, and frequency of TEAEs5 years
    Incidence of Serious Adverse Events (SAEs)Incidence, type, severity, and frequency of SAEs5 years
    Incidence of Adverse Events of Special Interest (AESIs)Incidence, type, severity, and frequency of AESIs5 years
    Incidence of clinical laboratory abnormalitiesIncidence, type, severity, and frequency of clinical laboratory abnormalities5 years
    Incidence of new physical and neurologic exam abnormalitiesIncidence, type, severity, and frequency of new physical and neurologic exam abnormalities5 years

    Secondary outcome

    Frequently Asked Questions

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    References

    Clinical Trials Gov: A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children with Rett Syndrome

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