Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

• Status: Recruiting
• Trial end date: 2022-12-31
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.

• 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

• Body mass index (BMI) between 17 and 32 kg/m2

• Stage 5.5 or less on the Functional Staging for Ataxia (FSA)

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

Note: Other protocol defined Inclusion/Exclusion criteria may apply.