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Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

Sponsor
Taysha Gene Therapies, Inc. (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT06152237
Collaborator
(none)
6
Enrollment
2
Arms
93.3
Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome.

The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.

Condition or Disease Intervention/Treatment Phase
  • Genetic: TSHA-102
 Phase 1

Study Design

Study Type:
Interventional
Anticipated Enrollment :
6 participants
Allocation:
Randomized
Intervention Model:
Sequential Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Multicenter, Open Label, Randomized, Dose-Escalation and Dose-Expansion Study of the Safety, Tolerability, and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females With Rett Syndrome
Anticipated Study Start Date :
Jan 22, 2024
Anticipated Primary Completion Date :
Nov 2, 2028
Anticipated Study Completion Date :
Nov 2, 2031

Arms and Interventions

Arm Intervention/Treatment
Experimental: Cohort 1

Dose Level 1

Genetic: TSHA-102
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.
Experimental: Cohort 2

Dose Level 2

Genetic: TSHA-102
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.

Outcome Measures

Primary Outcome Measures

  1. Primary Safety [Baseline through week 52]

    Proportion of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)

Secondary Outcome Measures

  1. Exploratory Efficacy [Baseline through week 52]

    Change from baseline in participant's status after TSHA-102 administration as assessed by Clinical Global Impressions Improvement (CGI-I).

  2. Exploratory Efficacy [Baseline through week 52]

    Change from baseline in participant's status after TSHA-102 administration as assessed by Revised Motor Behavior Assessment (R-MBA).

  3. Exploratory Efficacy [Baseline through week 52]

    Change from baseline in participant's status after TSHA-102 administration as assessed by Rett Syndrome Behavior Questionnaire (RSBQ).

  4. Exploratory Efficacy [Baseline through week 52]

    Change from baseline in participant's status after TSHA-102 administration as assessed by Clinical Global Impressions-Severity (CGI-S).

  5. Exploratory Efficacy [Baseline through week 52]

    Change from baseline in quantitative EEG findings with auditory and visual evoked potentials (AEP and VEP).

  6. Exploratory Efficacy [Baseline through week 52]

    Change from the steroid-free baseline period in monthly countable seizure frequency (MCSF).

Eligibility Criteria

Criteria

Ages Eligible for Study:
5 Years to 8 Years
Sexes Eligible for Study:
Female
Accepts Healthy Volunteers:
No
Inclusion Criteria:

  • Participant has a confirmed diagnosis of classical/typical Rett Syndrome with a documented mutation of the MECP2 gene that results in loss of function.

  • Participant is between ≥5 to ≤8 years of age at the time of consent.

  • Participant must be up to date with all relevant local vaccination requirements, with last vaccination dose received at least 42 days prior to the start of the immunosuppression regimen.

  • Participant's parent/caregiver must be willing to allow participant to receive blood or blood products for the treatment of an AE if medically needed.

Exclusion Criteria:

  • Participant has another neurodevelopmental disorder independent of the MECP2 gene loss of function mutation, or any other genetic syndrome with a progressive course.

  • Participant has a history of brain injury that causes neurological problems.

  • Participant had grossly abnormal psychomotor development in the first 6 months of life.

  • Participant has a diagnosis of atypical Rett syndrome.

  • Participant has an MECP2 mutation that does not cause Rett syndrome.

  • Participant requires non-invasive and invasive ventilatory support.

  • Participant has contraindications for IT administration of TSHA-102 or lumbar puncture procedure, other medical conditions, or contraindications to any medications required for IT administration.

  • Participant has acute or chronic hepatitis B or C infections.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • Taysha Gene Therapies, Inc.

Investigators

  • Study Director: Benit Maru, Bsc, MB ChB, MSc, PhD, Taysha Gene Therapies

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Taysha Gene Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT06152237
Other Study ID Numbers:
  • TSHA-102-CL-102
First Posted:
Nov 30, 2023
Last Update Posted:
Nov 30, 2023
Last Verified:
Nov 1, 2023
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Product Manufactured in and Exported from the U.S.:
Yes
Keywords provided by Taysha Gene Therapies, Inc.
Rett Syndrome
Neurodevelopmental Disorder
MECP2
Additional relevant MeSH terms:
Rett Syndrome
Syndrome

Study Results

No Results Posted as of Nov 30, 2023