Back to resultsA Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome
Primary Purpose
Rett Syndrome
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
NGN-401
Sponsored by
About this trial
This is an interventional treatment trial for Rett Syndrome focused on measuring Typical Rett Syndrome, MECP2, Rett Disorder, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Neurodevelopmental Disorders, Neurobehavioral Manifestations, Neurologic Manifestations, Intellectual Disability, Nervous System Diseases, Heredodegenerative Disorders, Nervous System, Pathologic Processes, RTT
Eligibility Criteria
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 must reside within a 2-hour drive of the study center for at least 3 months following treatment 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.
Sites / Locations
- Children's Hospital Colorado
- Boston Children's Hospital
- Texas Children's HospitalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Cohort 1
Arm Description
Participants will receive a single intracerebroventricular (ICV) administration of the study treatment.
Outcomes
Primary Outcome Measures
Incidence of Treatment Emergent Adverse Events (TEAEs)
Incidence, type, severity, and frequency of TEAEs
Incidence of Serious Adverse Events (SAEs)
Incidence, type, severity, and frequency of SAEs
Incidence of Adverse Events of Special Interest (AESIs)
Incidence, type, severity, and frequency of AESIs
Incidence of clinical laboratory abnormalities
Incidence, type, severity, and frequency of clinical laboratory abnormalities
Incidence of new physical and neurologic exam abnormalities
Incidence, type, severity, and frequency of new physical and neurologic exam abnormalities
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05898620
Brief Title
A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome
Official Title
A Phase 1/2, Open-Label Clinical Study to Evaluate Safety, Tolerability, and Efficacy of NGN-401 in Pediatric Subjects With Rett Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 2023 (Anticipated)
Primary Completion Date
October 2029 (Anticipated)
Study Completion Date
October 2029 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurogene Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in female children with typical Rett syndrome.
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Rett Syndrome
Keywords
Typical Rett Syndrome, MECP2, Rett Disorder, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Neurodevelopmental Disorders, Neurobehavioral Manifestations, Neurologic Manifestations, Intellectual Disability, Nervous System Diseases, Heredodegenerative Disorders, Nervous System, Pathologic Processes, RTT
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
Participants will receive a single intracerebroventricular (ICV) administration of the study treatment.
Intervention Type
Genetic
Intervention Name(s)
NGN-401
Intervention Description
NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.
Primary Outcome Measure Information:
Title
Incidence of Treatment Emergent Adverse Events (TEAEs)
Description
Incidence, type, severity, and frequency of TEAEs
Time Frame
5 years
Title
Incidence of Serious Adverse Events (SAEs)
Description
Incidence, type, severity, and frequency of SAEs
Time Frame
5 years
Title
Incidence of Adverse Events of Special Interest (AESIs)
Description
Incidence, type, severity, and frequency of AESIs
Time Frame
5 years
Title
Incidence of clinical laboratory abnormalities
Description
Incidence, type, severity, and frequency of clinical laboratory abnormalities
Time Frame
5 years
Title
Incidence of new physical and neurologic exam abnormalities
Description
Incidence, type, severity, and frequency of new physical and neurologic exam abnormalities
Time Frame
5 years
10. Eligibility
Sex
Female
Gender Based
Yes
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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 must reside within a 2-hour drive of the study center for at least 3 months following treatment
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Contact Center
Phone
+1 877-237-5020
Email
medicalinfo@neurogene.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Albena Patroneva, MD, MBA
Organizational Affiliation
Neurogene Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Email
NeurogeneRettStudy@childrenscolorado.org
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Phone
617-355-5230
Email
rettresearch@childrens.harvard.edu
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Phone
832-824-7729
Email
Julie.Thompson@bcm.edu
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome
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