NCT05285540
Completed
Phase 1
A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in Adult Patients With Friedreich Ataxia
ConditionsFriedreich Ataxia
Overview
- Phase
- Phase 1
- Intervention
- DT-216
- Conditions
- Friedreich Ataxia
- Sponsor
- Design Therapeutics, Inc.
- Enrollment
- 39
- Locations
- 1
- Primary Endpoint
- Frequency of treatment adverse events (TEAEs)
- Status
- Completed
- Last Updated
- 3 years ago
Overview
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 dose study is randomized, double-blind, placebo-controlled.
Investigators
Eligibility 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.
Arms & Interventions
Single Dose: DT-216
DT-216 will be administered once
Intervention: DT-216
Single Dose: DT-216 matching placebo
Placebo will be administered once
Intervention: DT-216 matching Placebo
Outcomes
Primary Outcomes
Frequency of treatment adverse events (TEAEs)
Time Frame: Up to approximately 30 days
Frequency of treatment emergent adverse events (TEAEs)
Secondary Outcomes
- Maximum Plasma Concentration (Cmax) of DT-216(Up to approximately 30 days)
- Time to Maximum Plasma Concentration (Tmax) of DT-216(Up to approximately 30 days)
- Area Under the Concentration-time Curve (AUC) of DT-216(Up to approximately 30 days)
Study Sites (1)
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