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A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Patients With Friedreich's Ataxia

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Trial Profile

A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Patients With Friedreich's Ataxia

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 10 Jun 2025

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At a glance

  • Drugs DT-216 (Primary)
  • Indications Friedreich's ataxia
  • Focus Adverse reactions
  • Acronyms RESTORE-FA
  • Sponsors Design Therapeutics

Most Recent Events

  • 04 Jun 2025 According to a Design Therapeutics media release, company has submitted an investigational new drug (IND) application for DT-216P2 with the U.S. Food and Drug Administration (FDA). The company received a clinical hold notice on the IND application from FDA, noting nonclinical deficiencies. Further details will be provided in an official letter from FDA within 30 days and the company plans to address their questions, once received.
  • 04 Jun 2025 According to a Design Therapeutics media release, company anticipates reporting data from the MAD trial, including levels of frataxin (FXN) expression based on 12 weeks of dosing, in 2026.
  • 04 Jun 2025 According to a Design Therapeutics media release, company announced that the first Friedreich ataxia (FA) patient has been dosed via intravenous (IV) infusion in this trial.

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