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Clinical trial

A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in Adult Patients With Friedreich Ataxia

ID
Name
DTX-216-101
Description
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.
Trial arms
Trial start
2022-03-11
Estimated PCD
2022-12-19
Trial end
2022-12-19
Status
Completed
Phase
Early phase I
Treatment
DT-216
DT-216 will be administered by intravenous (IV) injection
Arms:
Single Dose: DT-216
DT-216 matching Placebo
Placebo will be administered by intravenous (IV) injection
Arms:
Single Dose: DT-216 matching placebo
Size
39
Primary endpoint
Frequency of treatment adverse events (TEAEs)
Up to approximately 30 days
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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 39, 'type': 'ACTUAL'}}
Updated at
2023-03-28

1 organization

1 product

1 indication

Organization
Design Therapeutics
Product
DT-216
Indication
Friedreich Ataxia