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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Exploration Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 in Adult Subjects With Friedreich's Ataxia

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Trial Profile

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Exploration Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 in Adult Subjects With Friedreich's Ataxia

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 30 Jun 2025

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At a glance

  • Drugs Nomlabofusp (Primary)
  • Indications Friedreich's ataxia
  • Focus Adverse reactions; Registrational
  • Sponsors Larimar Therapeutics

Most Recent Events

  • 23 Jun 2025 According to a Larimar Therapeutics media release, FDA safety database recommendations and refined timeline for Biologics License Application (BLA) submission to allow for the inclusion of the recommended safety data from adults and children with Friedreich's Ataxia (FA). This comes following written responses from the U.S. Food and Drug Administration (FDA) based on discussions under the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program
  • 24 Mar 2025 According to a Larimar Therapeutics media release, the company is continuing discussions with FDA on the adequacy of the safety data set to support BLA submission and seeking accelerated approval targeted for submission by year-end 2025.
  • 18 Nov 2024 According to a Larimar Therapeutics media release, the company presented data from the Company's Phase 1 studies and the Phase 2 dose exploration study of nomlabofusp at the International Congress for Ataxia Research (ICAR) held from Nov 12-15, 2024 in London, U.K. Data from 61 adults, who participated in these studies, evaluating short-term (up to 28 days) SC doses of 25, 50, 75, and 100 mg nomlabofusp were further evaluated and presented in 3 posters.

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