A Phase 1b, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose and Multi-Dose Study of DT-216 in Adult Patients With Friedreich Ataxia

  • STATUS
    Recruiting
  • End date
    Sep 13, 2023
  • participants needed
    38
  • sponsor
    Design Therapeutics
Updated on 25 October 2022

Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.

Details
Condition Friedreich Ataxia
Treatment Placebo, DT-216
Clinical Study IdentifierNCT05573698
SponsorDesign Therapeutics
Last Modified on25 October 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Genetically confirmed diagnosis of Friedreich Ataxia with homozygous GAA repeat expansions
Able and willing to sign informed consent form prior to study enrollment
Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening

Exclusion Criteria

Has any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study
Has clinically significant abnormal laboratory results
Has significant cardiac disease
Received an investigational drug within 3 months of screening
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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