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

Design Therapeutics

Status and phase: Completed; Phase 1

Conditions: Friedreich Ataxia

Treatments: Drug: DT-216 matching Placebo; Drug: DT-216

Study type: Interventional

Funder types: Industry

Identifiers

NCT05285540

DTX-216-101

Details and patient eligibility

About

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.

Enrollment: 39 patients

Sex: All

Ages: 18 to 55 years old

Volunteers: No Healthy Volunteers

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)

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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39 participants in 2 patient groups

Single Dose: DT-216

Experimental group

Description:

DT-216 will be administered once

Treatment:

Drug: DT-216

Single Dose: DT-216 matching placebo

Experimental group

Description:

Placebo will be administered once

Treatment:

Drug: DT-216 matching Placebo