RecruitingPhase 1Phase 2NCT06874010

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

A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Patients With Friedreich's Ataxia

Sponsor

Design Therapeutics, Inc.

Enrollment

20 participants

Start Date

Jun 1, 2025

Study Type

INTERVENTIONAL

Conditions

Friedreich Ataxia

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

Min Age: 18 YearsMax Age: 65 Years

Inclusion Criteria6

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 Criteria4

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.

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