Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia
Completed · Phase 1 · Has a placebo group
Conditions studied: Friedreich Ataxia
In brief
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.
Key facts
- Study ID
- NCT05573698
- Run by
- Design Therapeutics, Inc.
- People needed
- 32
- Starts
- 2022-09-27
- Expected to finish
- 2023-08-25
- Last updated by the study team
- 2024-02-29
Who can join
Age: 18 and older, up to 55. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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.
Where it is running
- Clinilabs — Eatontown, New Jersey, United States
Full record on ClinicalTrials.gov
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