A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
Study details:
The pivotal study, Embolden (TM), is a conversion of the phase 1/2 study and is an open-label, baseline-controlled, multicenter, single-arm study designed to assess the efficacy, safety, and tolerability of administration of NGN401, an adeno-associated viral vector serotype 9 (AAV9) using Neurogene's proprietary transgene regulation technology. NGN-401 contains a full-length human MECP2 gene and is designed to express therapeutic levels of the MeCP2 protein while avoiding overexpression. The study treatment will be given as a single administration under general anesthesia via intracerebroventricular (ICV) delivery.
Each participant will be followed for efficacy and safety for 3 years after treatment, and is expected to enroll in a long-term follow-up study for 12 years.
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 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
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 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
Eligibility
Age eligible for study : NA
Healthy volunteers accepted : No
Gender eligible for study: ALL
Things to know
Study dates
Study start:
Primary completion:
Study completion finish:
Study type
TREATMENT
Phase
- PHASE3
Study ID
NCT05898620
Intervention or treatment
GENETIC: NGN-401
Conditions
- Rett Syndrome
You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.
Research sites nearby
Closest Location:
The Children's Hospital at Westmead
Select from list below to view details:
The Children's Hospital at Westmead
Sydney, New South Wales, Australia
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Who is running the clinical trial?
Neurogene Inc.
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: Pediatric 1e15 vg dose (fully enrolled)
| Genetic: NGN-401 |
EXPERIMENTAL: Adolescent/Adult 1e15 vg Dose (fully enrolled)
| Genetic: NGN-401 |
EXPERIMENTAL: Pediatric 3e15 vg dose (discontinued)
| Genetic: NGN-401 |
EXPERIMENTAL: Pivotal Cohort
| Genetic: NGN-401 |
What is the study measuring?
Primary Outcome
| Measure | Description | 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
References
Clinical Trials Gov: A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett SyndromeOther trials to consider
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