TSHA-102 for Rett Syndrome

Not currently recruiting at 13 trial locations

Overseen By Taysha Gene Therapies Medical Information

Age: < 18

Sex: Female

Trial Phase: Phase 1 & 2

Sponsor: Taysha Gene Therapies, Inc.

Disqualifiers: Other neurodevelopmental disorders, Brain injury, Atypical Rett, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy called TSHA-102 for girls with Rett Syndrome, a severe neurological disorder caused by a specific gene mutation. The research aims to assess the safety and effectiveness of two different dose levels of this treatment. Girls with a confirmed diagnosis of typical Rett Syndrome, who have had their condition for at least 5 years, might be suitable candidates for this trial. The study will last up to 6 years and will help determine if TSHA-102 can improve symptoms and overall health. As a Phase 1/Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that TSHA-102 is likely to be safe for humans?

Research shows that TSHA-102, a gene therapy tested for Rett Syndrome, has promising safety results. Earlier studies found that patients did not experience severe side effects, indicating the treatment is well-tolerated. Since the therapy is in Phase 1/2 trials, there is initial evidence of safety in humans. The therapy uses a method applied in other treatments, adding confidence to its safety. However, more data from ongoing trials will provide a clearer picture of its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Rett Syndrome?

Unlike the standard treatments for Rett Syndrome, which mainly focus on managing symptoms through medications like anticonvulsants or therapies to improve mobility, TSHA-102 is unique because it targets the genetic root of the disorder. Researchers are excited about TSHA-102 because it uses gene therapy to potentially correct the underlying MECP2 gene mutation responsible for Rett Syndrome. This innovative approach offers hope for more significant and lasting improvements in symptoms, moving beyond just managing the condition to possibly altering its course.

What evidence suggests that TSHA-102 might be an effective treatment for Rett Syndrome?

Research has shown that TSHA-102, a gene therapy for Rett Syndrome, shows promising results in early studies. This therapy uses a harmless virus to deliver a healthy version of the MECP2 gene directly to cells, aiming to correct issues caused by the faulty gene. In earlier studies, patients who received TSHA-102 experienced improvements in posture and stability just 25 weeks after treatment. Importantly, no serious side effects were reported, indicating a positive safety profile. This suggests that TSHA-102 could be an effective new treatment option for people with Rett Syndrome. Participants in this trial will receive TSHA-102 at different dose levels to further evaluate its effectiveness and safety. ^{3 5 6 7 8}

Who Is on the Research Team?

Laura Pisani, M.D.

Principal Investigator

Taysha Gene Therapies

Are You a Good Fit for This Trial?

This trial is for young girls aged 5 to 8 with Rett Syndrome, a neurological disorder. They must have a specific gene mutation (MECP2) and be up-to-date on vaccinations. Their caregiver must agree to potential blood product treatments for side effects.

Inclusion Criteria

The person taking care of you must be okay with you getting a blood transfusion if the doctor thinks it's necessary.

I am up to date with all my vaccinations, with the last dose received over 42 days ago.

I have Rett Syndrome with a confirmed MECP2 gene mutation.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose-Escalation

Participants receive a single intrathecal administration of TSHA-102 at varying dose levels to evaluate safety and tolerability

52 weeks

Regular visits for monitoring

Dose-Expansion

Further evaluation of safety, tolerability, and preliminary efficacy at selected dose levels

52 weeks

Regular visits for monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 years

What Are the Treatments Tested in This Trial?

Interventions

• TSHA-102

Trial Overview The REVEAL Pediatric Study tests TSHA-102, a gene therapy, in two different doses to see how safe it is and if it works for treating Rett Syndrome in pediatric females over the course of up to six years.

How Is the Trial Designed?

Group I: Cohort 2 Experimental Treatment 1 Intervention

Dose Level 2

Group II: Cohort 1 Experimental Treatment 1 Intervention

Dose Level 1

TSHA-102 is already approved in United States, Canada, United Kingdom for the following indications:

Approved in United States as TSHA-102 for:

• Rett syndrome (investigational)

Approved in Canada as TSHA-102 for:

• Rett syndrome (investigational)

Approved in United Kingdom as TSHA-102 for:

• Rett syndrome (investigational)

Who Is Running the Clinical Trial?

Taysha Gene Therapies, Inc.

Lead Sponsor

Trials

5

Recruited

60+

Citations

1. ir.tayshagtx.com ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-presents-new-supplemental-data-analysis

Taysha Gene Therapies Presents New Supplemental Data ... TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome.

2. ir.tayshagtx.com ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-announces-fda-breakthrough-therapy

Taysha Gene Therapies Announces FDA Breakthrough ... TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06152237

Safety and Efficacy of TSHA-102 in Pediatric Females With ... Participant has a confirmed diagnosis of classical/typical Rett Syndrome with a documented mutation of the MECP2 gene that results in loss of function.

4. reverserett.org reverserett.org/news/articles/a-review-of-gene-therapy-clinical-trials/

A Review of Gene Therapy Clinical Trials The patients experienced no severe adverse events, and, as of the last data reported by Taysha, the patients showed increasing improvements in ...

5. ir.tayshagtx.com ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-announces-positive-clinical-data-across

Taysha Gene Therapies Announces Positive Clinical Data ... Additionally, the patient showed improvement in posture and stability at week 25 post-treatment. We believe these longer-term clinical data ...

6. ir.tayshagtx.com ir.tayshagtx.com/static-files/13d0b9a1-1b34-42d8-9231-79c861547884

Safety and Efficacy Data on TSHA-102 AAV9 Investigational clinical outcomes in Rett syndrome. Responder Rate: Time to Response. Pediatric, adolescent, and adult participants in Part A of the REVEAL.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT06152237

NCT06152237 | Safety and Efficacy of TSHA-102 in ... Participant has a confirmed diagnosis of classical/typical Rett Syndrome with a documented mutation of the MECP2 gene that results in loss of function.

8. clinicaltrials.gov clinicaltrials.gov/study/NCT05606614

NCT05606614 | Safety and Efficacy of TSHA-102 in ... The REVEAL Adult Study is a multi-center, Phase 1/2 open-label, dose-escalation study of TSHA-102, an investigational gene therapy, in adult females with Rett ...