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
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
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• 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.
Locations
Other Locations
Australia
Doherty Clinical Trials
RECRUITING
East Melbourne
Nucleus Network
RECRUITING
Melbourne
Scientia Clinical Research Ltd
RECRUITING
Sydney
Contact Information
Primary
Varlli Scott
varlli.scott@mcri.edu.au
+613 8341 6228
Time Frame
Start Date: 2025-06
Estimated Completion Date: 2027-03
Participants
Target number of participants: 20
Treatments
Experimental: DT-216P2
Related Therapeutic Areas
Sponsors
Leads: Design Therapeutics, Inc.