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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05606614
Recruitment Status : Recruiting
First Posted : November 7, 2022
Last Update Posted : April 2, 2024
Sponsor:
Information provided by (Responsible Party):
Taysha Gene Therapies, Inc.

Study Description
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Brief 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.


Condition or disease Intervention/treatment Phase
Rett Syndrome Genetic: TSHA-102 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label, Randomized, Dose-escalation and Dose-expansion Study of the Safety and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, in the Treatment of Adult Females With Rett Syndrome
Actual Study Start Date : March 6, 2023
Estimated Primary Completion Date : November 8, 2024
Estimated Study Completion Date : January 5, 2032

Resource links provided by the National Library of Medicine


Arms and Interventions
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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
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Primary Outcome Measures :
  1. Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: Baseline through Week 52 ]
  2. 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 [ Time Frame: Baseline through Week 52 ]
  3. Change from baseline in participant's status after TSHA-102 administration as assessed by the Revised Motor Behavior Assessment (R-MBA) [ Time Frame: Baseline through Week 52 ]
  4. Change from baseline in participant's status after TSHA-102 administration as assessed by the Rett Syndrome Hand Function Scale (RSHFS) [ Time Frame: Baseline through Week 52 ]

Secondary Outcome Measures :
  1. Change from Baseline in participant's status after TSHA-102 administration as assessed by Clinical Global Impressions-Severity (CGI-S) [ Time Frame: Baseline through Week 52 ]
  2. Change from baseline in monthly seizure frequency (52 weeks) [ Time Frame: Baseline through Week 52 ]
  3. Change from baseline in adaptive behavior as assessed by Vineland-3 [ Time Frame: Baseline through Week 52 ]
  4. Change from Baseline in quantitative EEG findings with visual evoked potentials [ Time Frame: Baseline through Week 52 ]
  5. Change from Baseline in quantitative EEG findings with auditory evoked potentials [ Time Frame: Baseline through Week 52 ]

Eligibility Criteria
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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05606614


Contacts
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Contact: Taysha Gene Therapies Medical Information 833-489-8742 medinfo@tayshagtx.com

Locations
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United States, Illinois
Rush University Medical Center Not yet recruiting
Chicago, Illinois, United States, 60612
Contact: Taysha MedInfo         
Canada
Taysha Study Site Recruiting
Montréal, Canada
Contact: Taysha MedInfo         
Sponsors and Collaborators
Taysha Gene Therapies, Inc.
Investigators
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Study Director: Meredith Schultz, M.D. Taysha Gene Therapies
More Information
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Responsible Party: Taysha Gene Therapies, Inc.
ClinicalTrials.gov Identifier: NCT05606614    
Other Study ID Numbers: TSHA-102-CL-101
First Posted: November 7, 2022    Key Record Dates
Last Update Posted: April 2, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Rett
MECP2
Additional relevant MeSH terms:
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Rett Syndrome
Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
 Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System