Long-Term Safety and Effects of Daily Subcutaneous CTI-1601 in Children, Adolescents, and Adults With Friedreichs Ataxia

What this Trial Is About

Researchers are evaluating the long-term safety, tolerability, pharmacokinetics PK, pharmacodynamics PD, and clinical effects of CTI-1601, a recombinant fusion protein designed to deliver human frataxin, in people with Friedreichs ataxia FRDA. The study includes children aged 2 years and older, adolescents, and adults with FRDA, including those who have or have not participated in previous CTI-1601 studies. This open-label Phase 2 study aims to assess tissue frataxin levels, clinical evaluations, gene expression, and select lipids related to FRDA. Participants receive daily subcutaneous injections of CTI-1601. Adults 18 years and older receive a fixed 50 mg dose, while children and adolescents aged 2 to 17 years receive a weight-based dose of 0.8 mgkg, up to a maximum of 50 mg. The study includes long-term treatment administration and monitoring to evaluate safety and clinical effects. During the study, participants will undergo various assessments including motor function tests, neurological evaluations using the modified Friedreichs Ataxia Rating Scale mFARS, fatigue scales, daily living activities, and safety measures like electrocardiograms and echocardiograms. Researchers will also measure frataxin concentrations from cheek swabs and skin biopsies. The study will monitor treatment-emergent adverse events and other clinical outcomes over a period of up to 24 months.

An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia