Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

Phase 1

Completed

• Conditions: Friedreich Ataxia
• Interventions: Drug: DT-216; Drug: Placebo

• Registration Number: NCT05573698
• Lead Sponsor: Design Therapeutics, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2022-09-27
• Study First Submitted: 2022-10-06
• Study First Submitted QC: 2022-10-06
• Study First Posted: 2022-10-10
• Primary Completion: 2023-08-25
• Study Completion: 2023-08-25
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-27
• Last Update Posted: 2024-02-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 32

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.

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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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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Multiple Dose: DT-216	DT-216	Participants will be administered multiple doses of DT-216
Multiple Dose: DT-216 matching placebo	Placebo	Participants will be administered multiple doses of placebo

Primary Outcome Measures

Name	Time	Method
Safety Measure	Up to approximately 60 days	Frequency of treatment emergent adverse events (TEAEs)

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetic parameters	Up to approximately 60 days	Area Under the Concentration-time Curve (AUC) of DT-216

Trial Locations

Locations (1)

Clinilabs; 🇺🇸 Eatontown, New Jersey, United States