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Clinical Trials in Australia / NCT06874010
Recruiting Phase 1/2

A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia

NCT06874010 · tracked via the Priya Life Science Australia tracker
Phase
Phase 1/2
Started
2025-06
Last updated
2025-05-18

Condition(s) studied

Friedreich Ataxia

Investigational drug(s) / intervention(s)

DT-216P2 →

DT-216P2: Active

Study summary

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)

Trial sites (3)

FacilityCityRegionStatus
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

Other trials for the same condition

Official registry record

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.

View NCT06874010 on ClinicalTrials.gov ↗ ← All trials in Australia