Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

A Phase 1b, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose and Multi-Dose Study of DT-216 in Adult Patients With Friedreich Ataxia

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.

Inclusion Criteria:

• Genetically confirmed diagnosis of Friedreich Ataxia with homozygous GAA repeat expansions.
• Able and willing to sign informed consent form prior to study enrollment.
• Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening.

Exclusion Criteria:

• Has any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study.
• Has clinically significant abnormal laboratory results.
• Has significant cardiac disease.
• Received an investigational drug within 3 months of screening.