A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

Part of paid clinical trials in La Jolla, California.

Sponsor
Taysha Gene Therapies, Inc.
Study ID
NCT05606614
Phase
PHASE3
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
FEMALE
Age
6 Years - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • TSHA-102 — GENETIC
    TSHA-102 is a recombinant, non-replicating, self-complementary AAV9 (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 of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the efficacy and safety of TSHA-102 at the selected dose.

Key Dates

Start date
Mar 6, 2023
Status verified
Dec 2025
Primary completion
Jun 30, 2031
Completion
Jun 30, 2031

Study Design

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

Arms

  • Experimental: Part A Cohort 1
    TSHA-102 Dose Level 1: 5.7×10¹⁴ total vector genomes (vg). Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 1 (fully enrolled, 2 participants).
  • Experimental: Part A Cohort 2
    TSHA-102 Dose Level 2: 1.0×10¹⁵ total vector genomes (vg) Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 2 (fully enrolled, 4 participants).
  • Experimental: Part B Pivotal Cohort
    TSHA-102 at Selected Dose (Dose Level 2): 1.0 × 10¹⁵ total vector genomes (vg) Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 2 (1.0 × 10¹⁵).

Primary Outcome Measure

Part A: Safety and Tolerability of TSHA-102 [ Time Frame: Baseline through Week 52 ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
UC San DiegoLa JollaCalifornia92093
Karen Ditslear, M.S.
[email protected]
858-246-2288
Rush University Medical CenterChicagoIllinois60612
Milana Milic
[email protected]
Boston Children's HospitalBostonMassachusetts02115
Washington University, St. LouisSt LouisMissouri63110-
UT Southwestern Children's Medical CenterDallasTexas75930
[email protected]
214-456-2464

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By research site
UC San Diego· La Jolla, CARush University Medical Center· Chicago, ILBoston Children's Hospital· Boston, MAWashington University, St. Louis· St Louis, MOUT Southwestern Children's Medical Center· Dallas, TX

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