Status: Recruiting
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of CTI-1601 as well as children (age 2 years and older), adolescents and adults with FRDA who have not participated in a prior clinical study of CTI-1601.
Participants needed: 85
Trial details
Phase: Phase 2Age: 2-60Biological sex: AllType: InterventionalSponsor: Larimar Therapeutics, Inc.Updated: May 13, 2026Locations: 8
Eligibility criteria
Subjects with FRDA who have or have not previously completed participation in a... [+2]
Subjects who are confirmed as compound heterozygous (GAA repeat expansion on onl... [+10]