Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia
A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in Adult Patients With Friedreich Ataxia
1 other identifier
interventional
39
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Mar 2022
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 9, 2022
CompletedStudy Start
First participant enrolled
March 11, 2022
CompletedFirst Posted
Study publicly available on registry
March 17, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 19, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
December 19, 2022
CompletedMarch 28, 2023
March 1, 2023
9 months
March 9, 2022
March 27, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
Frequency of treatment adverse events (TEAEs)
Frequency of treatment emergent adverse events (TEAEs)
Up to approximately 30 days
Secondary Outcomes (3)
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
Other Outcomes (1)
Frataxin expression
Up to approximately 30 days
Study Arms (2)
Single Dose: DT-216
EXPERIMENTALDT-216 will be administered once
Single Dose: DT-216 matching placebo
EXPERIMENTALPlacebo will be administered once
Interventions
Placebo will be administered by intravenous (IV) injection
Eligibility Criteria
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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Clinilabs
Eatontown, New Jersey, 07724, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 9, 2022
First Posted
March 17, 2022
Study Start
March 11, 2022
Primary Completion
December 19, 2022
Study Completion
December 19, 2022
Last Updated
March 28, 2023
Record last verified: 2023-03
Data Sharing
- IPD Sharing
- Will not share