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

Design Therapeutics, Inc.1 site in 1 country32 target enrollmentSeptember 27, 2022

ConditionsFriedreich Ataxia

InterventionsDT-216 Placebo

DrugsDT-216 Placebo

Overview

Phase: Phase 1
Intervention: DT-216
Conditions: Friedreich Ataxia
Sponsor: Design Therapeutics, Inc.
Enrollment: 32
Locations: 1
Primary Endpoint: Safety Measure
Status: Completed
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

September 27, 2022

End Date

August 25, 2023

Last Updated

2 years ago

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

Design Therapeutics, Inc.

Responsible Party

Sponsor

Eligibility Criteria

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.