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

Recruiting Phase 1 Phase 2 Interventional Study
Friedreich Ataxia
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
18 – 65
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
20 (estimated)
Sponsor
Design Therapeutics, Inc. · Industry
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About This Trial

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.

Trial Locations
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Eligibility Criteria
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 sc…
Contacts

Varlli Scott

+613 8341 6228

varlli.scott@mcri.edu.au

CONTACT