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

• Status: Recruiting
• Trial end date: 2023-09-13
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Design Therapeutics

Criteria

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.