Study Overview:

This is a double-blind, placebo-controlled, study evaluating two doses (25 mg and 50 mg) of CTI-1601. This study will consist of at least 2 cohorts with 12 to 15 subjects participating in each cohort. Subjects will be dosed once daily (QD) for 14 days followed by dosing every other day (QOD) through Day 28.

Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.

Study Details:

Study visits will take place at Clinilabs in Eatontown, NJ. Participation requires about 30 days of overnight stays at Clinilabs and a nearby hotel and several home visits by a nurse. Compensation for time and travel will be provided for qualified participants.

Key Inclusion Criteria:

  • Subject is 18 years old or older
  • Genetically confirmed diagnosis of FA (homozygous GAA repeat expansion)
  • Subject can travel 25 feet with or without the use of assistive devices
  • Subject can transfer from bed to chair either independetly or with assistance while still physically contributing to the transfer in some way (e.g., partially able to support weight)
  • Subject can perform basic daily care, such as feeding or personal hygeine, with minimal assistance

Key Exclusion Criteria:

  • Previous participation in a CTI-1601 study if it resulted in an adverse event
  • Use of an investigational drug or device within 90 days prior to screening
  • Use of certain medications and supplements
  • Poor heart function (LVEF <45%)

Additional inclusion and exclusion criteria may apply and will be evaluated by a study doctor.

Length of Study Commitment:

  • One month

Participating Study Locations

Institution Name and LocationInvestigatorStudy Coordinator Contact Information

Clinilabs Drug Development Corporation
Eatontown, New Jersey

Magdy Shenouda, M.D.,
Clinilabs, Inc.

Michele Sapoff
(212) 994-4569

Explore the FA Drug Development Pipeline

FARA believes that there are many different approaches to treating Friedreich’s ataxia, and that it will require a cocktail approach of two or more treatments to slow, stop, reverse, and cure FA. Learn more about the approach behind this potential treatment and explore the other approaches that are in the FA Drug Development pipeline.