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Trial record 3 of 84 for:    Rett Syndrome
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Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

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ClinicalTrials.gov Identifier: NCT06152237
Recruitment Status : Recruiting
First Posted : November 30, 2023
Last Update Posted : February 14, 2024
Sponsor:
Information provided by (Responsible Party):
Taysha Gene Therapies, Inc.

Study Description
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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
Rett Syndrome Genetic: TSHA-102 Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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
Actual Study Start Date : December 12, 2023
Estimated Primary Completion Date : November 2, 2028
Estimated Study Completion Date : November 2, 2031

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. Primary Safety [ Time Frame: Baseline through week 52 ]
    The incidence of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)


Secondary Outcome Measures :
  1. Exploratory Efficacy [ Time Frame: Baseline through week 52 ]
    Change from baseline in participant's status after TSHA-102 administration as assessed by Clinical Global Impressions Improvement (CGI-I). This 7-point scale (1 = very much improved, 7 = very much worse, etc.) is used by the clinician to assess the participant's overall performance status; higher scores indicate increased severity.

  2. Exploratory Efficacy [ Time Frame: Baseline through week 52 ]
    Change from baseline in participant's status after TSHA-102 administration as assessed by Revised Motor Behavior Assessment (R-MBA). This 34-item questionnaire with scores of 0-4 will be administered by a cliniciant to indicate frequency of daily activities (behavioral/social, respiratory, motor/physical, etc.) in participants with Rett Syndrome. Higher scores correlate with greater clinical severity of disease.

  3. Exploratory Efficacy [ Time Frame: Baseline through week 52 ]
    Change from baseline in participant's status after TSHA-102 administration as assessed by Rett Syndrome Behavior Questionnaire (RSBQ). The RSBQ is a 45-item questionnaire and is completed by the participant's Caregiver. Scores (0 = not true, 1 = somewhat/sometimes true, or 2 = very true) are applied to subscales including General Mood, Breathing Problems, Fear/Anxiety, Walking/Standing, etc.; higher scores indicate greater severity.

  4. Exploratory Efficacy [ Time Frame: Baseline through week 52 ]
    Change from baseline in participant's status after TSHA-102 administration as assessed by Clinical Global Impressions-Severity (CGI-S). This 7-point scale (1 = normal - not I'll all all, 7 = extremely ill, etc.) will be administered by a clinician, based on their experience with patients with the same diagnosis. A higher score indicates greater severity of illness.

  5. Exploratory Efficacy [ Time Frame: Baseline through week 52 ]
    Change from baseline in quantitative EEG findings with auditory evoked potential and visual evoked potentials (AEP and VEP). This testing will provide a measure of the electrophysiologic responses of the brain to visual and auditory stimuli.

  6. Exploratory Efficacy [ Time Frame: Baseline through week 52 ]
    The percent change from the steroid-free baseline period in monthly countable seizure frequency (MCSF). This testing will provide a measure of participants with seizure freedom following administration of TSHA-102.


Eligibility Criteria
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Ages Eligible for Study:   5 Years to 8 Years   (Child)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

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
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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): NCT06152237


Contacts
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Contact: Taysha Gene Therapies Medical Information 1-833-489-8742 medinfo@tayshagtx.com

Locations
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United States, California
University of California San Diego (UCSD) Not yet recruiting
La Jolla, California, United States, 92037
United States, Illinois
Rush University Medical Center Recruiting
Chicago, Illinois, United States, 60612
Contact: Milana Milic    312-942-4670    milana_milic@rush.edu   
Contact: Samantha Dreyer    312-563-9304    samantha_l_dreyer@rush.edu   
Principal Investigator: Elizabeth Berry-Kravis, MD, PhD         
United States, Massachusetts
Boston Children's at Brookline Not yet recruiting
Boston, Massachusetts, United States, 02445
Contact: Grace Correa    617-355-5230    rettresearch@childrens.harvard.edu   
Principal Investigator: David Lieberman, MD, PhD         
United States, Minnesota
Gillette Children's Specialty Healthcare Not yet recruiting
Saint Paul, Minnesota, United States, 55101
Contact: Emily Hince, CCRC    651-229-3961    emilyahince@gillettechildrens.com   
Principal Investigator: Timothy Feyma, MD         
United States, Missouri
Washington University, St. Louis Not yet recruiting
Saint Louis, Missouri, United States, 63110
Contact: Ali Vonderheid       neuro_rettresearch@email.wustl.edu   
Principal Investigator: Robin Ryther, MD, PhD         
United States, Pennsylvania
Children's Hospital of Philadelphia Research Institute Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Jennie Minnick    267-426-1242    minnick@chop.edu   
Principal Investigator: Eric Marsh, MD, PhD         
United States, Tennessee
Vanderbilt Kennedy Center Not yet recruiting
Nashville, Tennessee, United States, 37203
Contact: Madeline Rockouski    615-322-1140    madeline.a.rockouski@vumc.org   
Principal Investigator: Jeffrey Neul, MD, PhD         
United States, Texas
University of Texas Southwestern Medical Center (UTSW) Not yet recruiting
Dallas, Texas, United States, 75390
United Kingdom
Children's Neurosciences, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust Not yet recruiting
London, United Kingdom
Contact: Lumsden         
Sponsors and Collaborators
Taysha Gene Therapies, Inc.
Investigators
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Study Director: Benit Maru, Bsc, MB ChB, MSc, PhD Taysha Gene Therapies
More Information
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Responsible Party: Taysha Gene Therapies, Inc.
ClinicalTrials.gov Identifier: NCT06152237    
Other Study ID Numbers: TSHA-102-CL-102
First Posted: November 30, 2023    Key Record Dates
Last Update Posted: February 14, 2024
Last Verified: February 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
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