Study to Evaluate 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 intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.
Key facts
- Study ID
- NCT05285540
- Run by
- Design Therapeutics, Inc.
- People needed
- 39
- Starts
- 2022-03-11
- Expected to finish
- 2022-12-19
- Last updated by the study team
- 2023-03-28
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
- Body mass index (BMI) between 17 and 32 kg/m2
- Stage 5.5 or less on the Functional Staging for Ataxia (FSA)
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
- Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Where it is running
- Clinilabs — Eatontown, New Jersey, United States
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.