A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Neurogene Inc.
Study ID
NCT05898620
Phase
PHASE3
Status
Active Not Recruiting

Conditions

Eligibility Criteria

Sex
FEMALE
Age
3 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • NGN-401 — GENETIC
    NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.

Study Details

This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.

Key Dates

Start date
Jun 13, 2023
Status verified
Jun 2026
Primary completion
Dec 31, 2029
Completion
Dec 31, 2029

Study Design

Enrollment
33 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Pediatric 1e15 vg dose (fully enrolled)
    Dose Level 1 for ages 4-10 years
  • Experimental: Adolescent/Adult 1e15 vg Dose (fully enrolled)
    Dose Level 1 for ages 11 years \& above
  • Experimental: Pediatric 3e15 vg dose (discontinued)
    Dose Level 2 for ages 4-10 years (discontinued)
  • Experimental: Pivotal Cohort
    Dose Level 1 for ages 3 and above

Primary Outcome Measure

Efficacy of NGN-401 [ Time Frame: 52 Weeks ]

Locations (13)

FacilityCityStateZIPSite coordinators
University of Alabama at BirminghamBirminghamAlabama35233-
UCSF Benioff Children's Hospital OaklandOaklandCalifornia94609-
Children's Hospital ColoradoAuroraColorado80045-
Nicklaus Children's Hospital Research InstituteMiamiFlorida33155-
Rush University Medical CenterChicagoIllinois60612-
Kennedy Krieger InstituteBaltimoreMaryland21205-
Boston Children's HospitalBostonMassachusetts02115-
Montefiore Medical CenterNew YorkNew York10467-
UNC at Chapel HillChapel HillNorth Carolina27514-
Nationwide Children's HospitalColumbusOhio43205-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
Vanderbilt University Medical CenterNashvilleTennessee37232-
Texas Children's HospitalHoustonTexas77030-

Find similar trials in Birmingham, AL

By research site
University of Alabama at Birmingham· Birmingham, ALUCSF Benioff Children's Hospital Oakland· Oakland, CAChildren's Hospital Colorado· Aurora, CONicklaus Children's Hospital Research Institute· Miami, FLRush University Medical Center· Chicago, ILKennedy Krieger Institute· Baltimore, MD

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