A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
Public ClinicalTrials.gov record NCT05606614. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label Phase 1/2/3 Study Consisting of a Phase 1/2 Safety and Dose-escalation and Phase 3 Dose-expansion Study to Evaluate Safety and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy in Females With Rett Syndrome
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the efficacy and safety of TSHA-102 at the selected dose.
Study identification
- NCT ID
- NCT05606614
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 17 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 6 Years to 21 Years
- Sex
- Female
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 5, 2023
- Primary completion
- May 31, 2031
- Completion
- May 31, 2031
- Last update posted
- Jul 6, 2026
2023 – 2031
United States locations
- U.S. sites
- 8
- U.S. states
- 6
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| City of Hope | Duarte | California | 91010 | — |
| UC San Diego | La Jolla | California | 92093 | — |
| UC San Francisco Benioff Children's Hospital | Oakland | California | 94609 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Washington University, St. Louis | St Louis | Missouri | 63110 | — |
| UT Southwestern Children's Medical Center | Dallas | Texas | 75930 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05606614, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 6, 2026 · Synced Sep 9, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05606614 live on ClinicalTrials.gov.