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

Phase 1

Recruiting

• Conditions: Friedreich Ataxia
• Interventions: Drug: DT-216P2

• Registration Number: NCT06874010
• Lead Sponsor: Design Therapeutics, Inc.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-02-25
• Study First Submitted QC: 2025-03-11
• Study First Posted: 2025-03-13
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-14
• Last Update Posted: 2025-05-18
• Study Start: 2025-06
• Primary Completion: 2026-12
• Study Completion: 2027-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
DT-216P2	DT-216P2	-

Primary Outcome Measures

Name	Time	Method
Frequency of treatment-emergent adverse events (TEAE)	Up to Week 12

Secondary Outcome Measures

Name	Time	Method
Area under the curve (AUClast, AUCinf)	Up to Week 12
Maximum concentration (Cmax)	Up to Week 12
Time to Cmax (Tmax)	Up to Week 12
Half-life (t1/2)	Up to Week 12
Frataxin expression at baseline and after treatment will be measured	Up to Week 12

Trial Locations

Locations (3)

Scientia Clinical Research Ltd; 🇦🇺 Sydney, New South Wales, Australia

Doherty Clinical Trials; 🇦🇺 East Melbourne, Victoria, Australia

Nucleus Network; 🇦🇺 Melbourne, Victoria, Australia