Clinical Trials Logo

Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05606614
Other study ID # TSHA-102-CL-101
Secondary ID
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date March 6, 2023
Est. completion date January 5, 2032

Study information

Verified date March 2024
Source Taysha Gene Therapies, Inc.
Contact Taysha Gene Therapies Medical Information
Phone 833-489-8742
Email medinfo@tayshagtx.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The REVEAL Adult Study is a multi-center, Phase 1/2 open-label, dose-escalation study of TSHA-102, an investigational gene therapy, in adult females with Rett syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is estimated to be up to 63 months.


Recruitment information / eligibility

Status Recruiting
Enrollment 18
Est. completion date January 5, 2032
Est. primary completion date November 8, 2024
Accepts healthy volunteers No
Gender Female
Age group 12 Years and older
Eligibility Inclusion Criteria: - Participant has a clinical diagnosis of classical/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of function. - Participants must be willing 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 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 a MECP2 mutation that does not cause Rett syndrome. - Participant requires invasive ventilatory support. - Participant has contraindications for IT administration of TSHA-102 or lumbar puncture procedure, or other medical conditions, or contraindications to any medications required for IT administration. - Participant has uncontrolled seizures or a history of status epilepticus within the 3 months prior to enrollment.

Study Design


Related Conditions & MeSH terms


Intervention

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.

Locations

Country Name City State
Canada Taysha Study Site Montréal
United States Rush University Medical Center Chicago Illinois

Sponsors (1)

Lead Sponsor Collaborator
Taysha Gene Therapies, Inc.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type Measure Description Time frame Safety issue
Primary Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs) Baseline through Week 52
Primary Change from baseline in participant's status after TSHA-102 administration as assessed by Clinical Global Impressions-Improvement (CGI-I) scale, adapted to Rett syndrome Baseline through Week 52
Primary Change from baseline in participant's status after TSHA-102 administration as assessed by the Revised Motor Behavior Assessment (R-MBA) Baseline through Week 52
Primary Change from baseline in participant's status after TSHA-102 administration as assessed by the Rett Syndrome Hand Function Scale (RSHFS) Baseline through Week 52
Secondary Change from Baseline in participant's status after TSHA-102 administration as assessed by Clinical Global Impressions-Severity (CGI-S) Baseline through Week 52
Secondary Change from baseline in monthly seizure frequency (52 weeks) Baseline through Week 52
Secondary Change from baseline in adaptive behavior as assessed by Vineland-3 Baseline through Week 52
Secondary Change from Baseline in quantitative EEG findings with visual evoked potentials Baseline through Week 52
Secondary Change from Baseline in quantitative EEG findings with auditory evoked potentials Baseline through Week 52
See also
  Status Clinical Trial Phase
Completed NCT04988867 - An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome Phase 2/Phase 3
Recruiting NCT00069550 - Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome Phase 3
Enrolling by invitation NCT06139172 - Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities N/A
Not yet recruiting NCT04014985 - Patients With RETT Syndrome N/A
Not yet recruiting NCT04041713 - A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome Phase 2
Completed NCT02705677 - Biobanking of Rett Syndrome and Related Disorders
Terminated NCT02790034 - Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms Phase 2/Phase 3
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Recruiting NCT05932589 - Neurophysiologic Biomarkers in Rett Syndrome
Recruiting NCT04463316 - GROWing Up With Rare GENEtic Syndromes
Completed NCT04776746 - Open-Label Extension Study of Trofinetide for Rett Syndrome Phase 3
Completed NCT04181723 - Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDERâ„¢) Phase 3
Enrolling by invitation NCT03836300 - Parent and Infant Inter(X)Action Intervention (PIXI) N/A
Completed NCT04514549 - ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME
Completed NCT02738281 - Natural History of Rett Syndrome & Related Disorders
Terminated NCT02562820 - An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome Phase 1
Completed NCT05687214 - Osteopathic Manipulative Treatment for Constipation in People With Rett Syndrome N/A
Recruiting NCT06199700 - Esketamine for the Treatment of Rett Syndrome Early Phase 1
Completed NCT03941444 - ANAVEX2-73 Study in Patients With Rett Syndrome Phase 3
Recruiting NCT06346106 - The Diagnostic Experience of Male Rett Syndrome