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NCT07480564: ASPIRE
ASPIRE Study: A Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females Aged >2 to <4 Years With Rett Syndrome
Phase 3 trial testing TSHA-102 in Rett Syndrome in 3 participants. Currently enrolling.
1 June 2031
Quick facts
| Lead sponsor | Taysha Gene Therapies, Inc. |
|---|---|
| Phase | Phase 3 |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 3 |
| Start date | 8 May 2026 |
| Primary completion | 1 June 2031 |
| Estimated completion | 1 June 2031 |
| Sites | 1 location across United States |
Drugs / interventions tested
- TSHA-102
Conditions studied
- Rett Syndrome — all drugs for Rett Syndrome →
Sponsor
Taysha Gene Therapies, Inc. — full company profile →
Who can join
Adults 2 to 3, female only, with Rett Syndrome. Patients with the condition only — healthy volunteers not accepted.
What's being measured
Primary outcomes are the specific endpoints the trial is designed to prove or disprove.
-
Primary Safety
Time frame: Baseline through Week 25
Safety and Tolerability of TSHA-102 Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
Sponsor's own description
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.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT07480564
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of TSHA-102
Trials testing the same drug.
- NCT06152237 — Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) · Phase 1, PHASE2 · active not recruiting
Other recruiting trials for Rett Syndrome
Currently open trials in the same condition.
- NCT06840496 — To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome · Phase 3 · recruiting
- NCT07151950 — Obi Medical Robot: Evaluating Effectiveness Related to Usability · active not recruiting
- NCT06856759 — Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome · EARLY_PHASE1 · recruiting
- NCT07418905 — Technology-supported Motor Rehabilitation for People With Rett Syndrome · NA · active not recruiting
- NCT06152237 — Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) · Phase 1, PHASE2 · active not recruiting
Other Taysha Gene Therapies, Inc. trials
Trials by the same sponsor.
- NCT06152237 — Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) · Phase 1, PHASE2 · active not recruiting
- NCT05277363 — A Study of the Natural Course of SURF1 Deficiency · withdrawn
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT07480564 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Taysha Gene Therapies, Inc.
- Last refreshed: 20 May 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT07480564.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing