Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome
Recruiting
Phase 3
Interventional Study
Rett Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 2 – 3
- Sex
- Female
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 3 (estimated)
- Sponsor
- Taysha Gene Therapies, Inc. · Industry
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About This Trial
The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Pediatric females between the ages of 2 and less than 4 years old.
* Participant has a clinical diagnosis of classic/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of gene function.
* Participan…
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