Status:
COMPLETED
Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia
Lead Sponsor:
Design Therapeutics, Inc.
Conditions:
Friedreich Ataxia
Eligibility:
All Genders
18-55 years
Phase:
PHASE1
Brief 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 dos...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
March 11 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 19 2022
Estimated Enrollment :
39 Patients enrolled
Trial Details
Trial ID
NCT05285540
Start Date
March 11 2022
End Date
December 19 2022
Last Update
March 28 2023
Active Locations (1)
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1
Clinilabs
Eatontown, New Jersey, United States, 07724