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NCT07480564 · ClinicalTrials.gov registry record · Phase 3
Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome
A Phase 3 study, sponsored by Taysha Gene Therapies.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 3
- Enrollment target
NCT07480564 is a Phase 3 study that is actively recruiting participants, run by Taysha Gene Therapies. The registered enrollment target is 3 participants.
The verdict
NCT07480564, a Phase 3 study, is actively recruiting participants, sponsored by Taysha Gene Therapies.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 3 participants
- Enrollment target
Study Summary
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.
Interventions
- GENETIC TSHA-102
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2026-05-08 |
| Est. Completion | 2031-06 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07480564
The ClinicalTrials.gov registry entry for NCT07480564 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 3 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Taysha Gene Therapies, which has 3 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which TSHA-102 is the first listed.
NCT07480564 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT07480564 about?
NCT07480564 is a clinical study titled "Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome". 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.
What is the current status of trial NCT07480564?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 3 participants. The study started on 2026-05-08. Estimated completion is 2031-06.
What interventions are being tested in trial NCT07480564?
The interventions under investigation include: TSHA-102 (GENETIC).
Who is sponsoring clinical trial NCT07480564?
This trial is sponsored by Taysha Gene Therapies, which has 3 total clinical trials registered on ClinicalTrials.gov.
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