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

Recruiting now · Phase 1/Phase 2

Conditions studied: Friedreich Ataxia

In brief

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.

Key facts

Study ID

NCT06874010

Run by

Design Therapeutics, Inc.

People needed

20

Starts

2025-06-01

Expected to finish

2027-03-01

Last updated by the study team

2025-05-18

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 may not qualify if…

• 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.

Where it is running

• Scientia Clinical Research Ltd — Sydney, New South Wales, Australia (enrolling)
• Doherty Clinical Trials — East Melbourne, Victoria, Australia (enrolling)
• Nucleus Network — Melbourne, Victoria, Australia (enrolling)

Full record on ClinicalTrials.gov

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