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A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children with Rett Syndrome
This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in female children with typical Rett syndrome.
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.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
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
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
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
| GENETIC: NGN-401
|
EXPERIMENTAL: High Dose
| GENETIC: NGN-401
|
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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