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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05898620




Registration number
NCT05898620
Ethics application status
Date submitted
1/06/2023
Date registered
12/06/2023
Date last updated
13/08/2024

Titles & IDs
Public title
A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome
Scientific title
A Phase 1/2, Open-Label Clinical Study to Evaluate Safety, Tolerability, and Efficacy of NGN-401 in Pediatric Subjects With Rett Syndrome
Secondary ID [1] 0 0
RTT-200
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - NGN-401

Experimental: Low Dose - Dose Level 1

Experimental: High Dose - Dose Level 2


Treatment: Other: NGN-401
NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of Treatment Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
5 years
Primary outcome [2] 0 0
Incidence of Serious Adverse Events (SAEs)
Timepoint [2] 0 0
5 years
Primary outcome [3] 0 0
Incidence of Adverse Events of Special Interest (AESIs)
Timepoint [3] 0 0
5 years
Primary outcome [4] 0 0
Incidence of clinical laboratory abnormalities
Timepoint [4] 0 0
5 years
Primary outcome [5] 0 0
Incidence of new physical and neurologic exam abnormalities
Timepoint [5] 0 0
5 years

Eligibility
Key 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
Minimum age
4 Years
Maximum age
10 Years
Sex
Females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/06/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2029
Actual
Sample size
Target
16
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Sydney
Recruitment postcode(s) [1] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
Texas

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Neurogene Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in female children with typical Rett syndrome.
Trial website
https://clinicaltrials.gov/study/NCT05898620
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Julie Jordan, MD
Address 0 0
Neurogene Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Contact Center
Address 0 0
Country 0 0
Phone 0 0
+1 877-237-5020
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05898620