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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
Inclusion Criteria:
* 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.
Exclusion Criteria:
* 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.
Primary outcome measure(s)
Frequency of treatment-emergent adverse events (TEAE) — Up to Week 12
Trial sites (3)
Facility
City
Region
Status
Scientia Clinical Research Ltd
Sydney
New South Wales
Recruiting
Doherty Clinical Trials
East Melbourne
Victoria
Recruiting
Nucleus Network
Melbourne
Victoria
Recruiting
More Design Therapeutics, Inc. trials in Australia
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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