Actively Recruiting
A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
Led by Design Therapeutics, Inc. · Updated on 2025-05-18
20
Participants Needed
3
Research Sites
91 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.
CONDITIONS
Official Title
A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be 18-65 years of age inclusive, at the time of signing the informed consent.
- Genetically confirmed diagnosis of FA, with homozygous GAA repeat expansions in the frataxin gene.
- Stage 5.5 or less on the FSA at screening.
- BMI between 16 and 32 kg/m2 at screening; weight should be <= 100 kg at screening.
- Male and/or female using protocol defined and regulatory approved contraception.
- Capable of giving signed informed consent.
You will not qualify if you...
- Any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study protocol.
- Any clinically significant nonmedical conditions and psychiatric disorders that could put the participant at higher risk for participation in the study, influence the participant's ability to participate in the study, or interfere with interpretation of the participant's study results, in the opinion of the investigator.
- Received an investigational agent within the last 30 days or 5 half-lives, whichever longer, prior to the first dose of study drug, or are in follow-up of another clinical study prior to study enrollment. Exception: Potential participants who are currently on Omaveloxolone must be on stable doses for at least 3 months.
- Is not willing to comply with the contraceptive requirements during the study period, as per protocol.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Scientia Clinical Research Ltd
Sydney, New South Wales, Australia, 2031
Actively Recruiting
2
Doherty Clinical Trials
East Melbourne, Victoria, Australia, 3002
Actively Recruiting
3
Nucleus Network
Melbourne, Victoria, Australia, 3004
Actively Recruiting
Research Team
V
Varlli Scott
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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