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Open-label, Randomized, Dose-escalation and Dose-expansion Study of the Safety and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, in the Treatment of Adult Females With Rett Syndrome

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Trial Profile

Open-label, Randomized, Dose-escalation and Dose-expansion Study of the Safety and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, in the Treatment of Adult Females With Rett Syndrome

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 17 May 2024

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At a glance

  • Drugs TSHA 102 (Primary)
  • Indications Rett syndrome
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Acronyms REVEAL; REVEAL Adult Study
  • Sponsors Taysha Gene Therapies
  • Most Recent Events

    • 02 May 2024 According to a Taysha Gene Therapies media release, the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to TSHA-102 in clinical evaluation for Rett syndrome. RMAT designation was granted following the FDA's review of safety and efficacy data from the first three patients dosed with the low dose of TSHA-102 across both REVEAL Phase 1/2 trials (adolescent/adult and pediatric).
    • 19 Mar 2024 According to a Taysha Gene Therapies media release, company will hold a conference call and webcast today at 4:30 p.m. ET to review its financial and operating results and to provide corporate and clinical updates of this study.
    • 19 Mar 2024 According to a Taysha Gene Therapies media release, study completed dosing in cohort one (low dose, n=2) of 5.7x1014 total vg. Received IDMC approval of the Companys request to dose escalate immediately, enabling early advancement to cohort two (high dose, n=3) of 1x1015 total vg. Company also announced expansion of ongoing trial in Canada into the U.S. and initiated site activation.
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