ClinicalTrials.gg
Sign In
Active, not recruitingNCT07480564ASPIREUpdated Aug 19, 2026

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

A Phase 3 interventional study of TSHA-102 in Rett Syndrome, sponsored by Taysha Gene Therapies, Inc.. Active, not recruiting at 3 sites in United States. Open to female participants aged 2 Years to 3 Years. Per ClinicalTrials.gov, last updated 2026-08-19.

Sponsored by Taysha Gene Therapies, Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
4
Allocation
Not applicable
Ages
2 Years to 3 Years
Sex
Female
01

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.

Read the detailed description

ASPIRE is an open-label study designed to evaluate the safety, tolerability and preliminary efficacy of TSHA-102 in 3 pediatric females aged 2 to less than 4 years old with typical Rett syndrome.

TSHA-102 is designed to target the genetic root cause of Rett syndrome by regulating the expression of MECP2 in cells. Each participant will be followed for the observation period of 5 years after TSHA-102 administration.

02

Conditions studied

  • Rett Syndrome

Keywords

  • Neurodevelopmental Disorders
  • Rett
  • MECP2
  • AAV9
  • Typical Rett Syndrome
  • Classic Rett Syndrome
  • RTT
  • Rett Disorder
  • Retts
  • MECP2-Related Disorder
  • Gene Therapy
  • Intrathecal Administration
  • Genetic Diseases, X-Linked
  • Nervous System Diseases
  • Developmental Regression
  • TSHA-102
  • miRARE
  • Self-complementary Vector
  • Neurologic Manifestations
  • Intellectual Disability
  • Pathologic Process
  • X-Linked Intellectual Disability
  • Congenital, Hereditary, and Neonatal Diseases and Abnormalities
03

In context

Rett Syndrome

96 studies on the registry are indexed under Rett Syndrome; 19 are open to participants now.

This study's enrollment of 4 is below the median of 30 across 64 interventional studies indexed under Rett Syndrome.

Browse Rett Syndrome studies →

Lead sponsor

Taysha Gene Therapies, Inc. is the lead sponsor of 4 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 3 Years
Sexes eligible
Female
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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.
  • Participants must be willing to receive blood or blood products for the treatment of an AE if medically needed.
  • Participants and parent/caregiver must agree to reside within easy access to the study site prior to the baseline visit and at least 3 months after TSHA-102 treatment.

Exclusion criteria

Exclusion Criteria:

  • Participant has another neurodevelopmental disorder independent of the MECP2 loss-of-function mutation, or any other genetic syndrome with a progressive course.
  • Participant has a history of brain injury that causes neurological problems or had grossly abnormal psychomotor development in the first 6 months of life.
  • Participant has a diagnosis of atypical Rett syndrome or a MECP2 gene mutation that does not cause Rett syndrome.
  • Participant requires invasive ventilatory support.

Note: Other protocol defined inclusion/exclusion criteria may apply

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
4 participants (actual)

Study arms

  • Experimental
    Treatment

    Participants receive a single intrathecal (IT) administration of TSHA-102 at 1.0 × 10¹⁵ total vector genomes (vg) adjusted for the participant's brain volume.

    Genetic: TSHA-102

Interventions

  • GeneticTSHA-102

    TSHA-102 is a recombinant, non-replicating, self-complementary adeno-associated virus serotype 9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intrathecal (IT) administration.

06

What researchers measure

Primary outcomes

  1. Primary Safety

    Safety and Tolerability of TSHA-102 Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)

    Time frame: Baseline through Week 25

07

Study locations

3 sites
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started May 2026 (actual). Primary completion is estimated for Jun 2031, 61 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Mar 13, 2026
First posted
Mar 18, 2026
StartActual
May 8, 2026
Primary completionEst.
Jun 2031
CompletionEst.
Jun 2031
Last update
Aug 19, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Aug 19, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT07480564
Lead sponsor
Taysha Gene Therapies, Inc.
Responsible party
Sponsor
First posted
Mar 18, 2026
Start date
May 8, 2026
Primary completion
Jun 2031 (estimated)
Completion
Jun 2031 (estimated)
Last update
Aug 19, 2026

Study contacts

Medical Monitor, M.D.
study director · Taysha Gene Therapies

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Rett Syndrome →

Other studies from this sponsor

Taysha Gene Therapies, Inc.

Start the discussion