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.
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…
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