A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
NCT ID: NCT06874010
Last Updated: 2025-05-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
20 participants
INTERVENTIONAL
2025-06-30
2027-03-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
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DT-216P2
DT-216P2
Active
Interventions
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DT-216P2
Active
Eligibility Criteria
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Inclusion Criteria
* 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 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.
18 Years
65 Years
ALL
No
Sponsors
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Design Therapeutics, Inc.
INDUSTRY
Responsible Party
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Locations
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Scientia Clinical Research Ltd
Sydney, New South Wales, Australia
Doherty Clinical Trials
East Melbourne, Victoria, Australia
Nucleus Network
Melbourne, Victoria, Australia
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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DTX-216P2-101
Identifier Type: -
Identifier Source: org_study_id
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