Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Taysha Gene Therapies, Inc.
Study ID
NCT07480564
Phase
PHASE3
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
FEMALE
Age
2 Years - 3 Years
Healthy Volunteers
Not accepted

Interventions

  • TSHA-102 — GENETIC
    TSHA-102 is a recombinant, non-replicating, self-complementary adeno-associated virus serotype 9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intrathecal (IT) administration.

Study Details

The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.

Key Dates

Start date
May 8, 2026
Status verified
May 2026
Primary completion
Jun 30, 2031
Completion
Jun 30, 2031

Study Design

Enrollment
3 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment
    Participants receive a single intrathecal (IT) administration of TSHA-102 at 1.0 × 10¹⁵ total vector genomes (vg) adjusted for the participant's brain volume.

Primary Outcome Measure

Primary Safety [ Time Frame: Baseline through Week 25 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Boston Children's HospitalBostonMassachusetts02115

Find similar trials in Boston, MA

By research site
Boston Children's Hospital· Boston, MA

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