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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

TREATMENT

Phase

PHASE1

PHASE2

Trial ID

NCT05898620

Intervention or treatment

GENETIC: NGN-401

Conditions

• Rett Syndrome

Condition: Rett Syndrome
GENETIC: NGN-401
Sydney, New South Wales, Australia
Sponsor: Neurogene Inc.

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

Find a site

Closest Location:

The Children's Hospital at Westmead

Research sites nearby

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The Children's Hospital at Westmead
Sydney, New South Wales, Australia

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/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.

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Incidence of Treatment Emergent Adverse Events (TEAEs)	Incidence, type, severity, and frequency of TEAEs	5 years
Incidence of Serious Adverse Events (SAEs)	Incidence, type, severity, and frequency of SAEs	5 years
Incidence of Adverse Events of Special Interest (AESIs)	Incidence, type, severity, and frequency of AESIs	5 years
Incidence of clinical laboratory abnormalities	Incidence, type, severity, and frequency of clinical laboratory abnormalities	5 years
Incidence of new physical and neurologic exam abnormalities	Incidence, type, severity, and frequency of new physical and neurologic exam abnormalities	5 years

Secondary outcome

Frequently Asked Questions

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You may be eligible to participate in this trial based on your search.Apply for study

Are you running this trial? If you're a clinic or sponsor, you can claim this study.Claim this trial

References

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