Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

Phase 1

Completed

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

• Registration Number: NCT05285540
• Lead Sponsor: Design Therapeutics, Inc.

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-03-09
• Study First Submitted QC: 2022-03-09
• Study Start: 2022-03-11
• Study First Posted: 2022-03-17
• Primary Completion: 2022-12-19
• Study Completion: 2022-12-19
• Status Verified: 2023-03
• Last Update Submitted: 2023-03-27
• Last Update Posted: 2023-03-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 39

Inclusion Criteria

• Genetically confirmed diagnosis of Friedreich Ataxia with homozygous GAA repeat expansions
• Body mass index (BMI) between 17 and 32 kg/m2
• Stage 5.5 or less on the Functional Staging for Ataxia (FSA)

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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

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Single Dose: DT-216	DT-216	DT-216 will be administered once
Single Dose: DT-216 matching placebo	DT-216 matching Placebo	Placebo will be administered once

Primary Outcome Measures

Name	Time	Method
Frequency of treatment adverse events (TEAEs)	Up to approximately 30 days	Frequency of treatment emergent adverse events (TEAEs)

Secondary Outcome Measures

Name	Time	Method
Maximum Plasma Concentration (Cmax) of DT-216	Up to approximately 30 days	Maximum Plasma Concentration (Cmax) of DT-216
Time to Maximum Plasma Concentration (Tmax) of DT-216	Up to approximately 30 days	Time to Maximum Plasma Concentration (Tmax) of DT-216
Area Under the Concentration-time Curve (AUC) of DT-216	Up to approximately 30 days	Area Under the Concentration-time Curve (AUC) of DT-216

Trial Locations

Locations (1)

Clinilabs; 🇺🇸 Eatontown, New Jersey, United States