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

Design Therapeutics

Status and phase: Completed; Phase 1

Conditions: Friedreich Ataxia

Treatments: Drug: Placebo; Drug: DT-216

Study type: Interventional

Funder types: Industry

Identifiers

NCT05573698

DTX-216-102

Details and patient eligibility

About

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.

Enrollment: 32 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.
• 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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32 participants in 2 patient groups, including a placebo group

Multiple Dose: DT-216

Experimental group

Description:

Participants will be administered multiple doses of DT-216

Treatment:

Drug: DT-216

Multiple Dose: DT-216 matching placebo

Placebo Comparator group

Description:

Participants will be administered multiple doses of placebo

Treatment:

Drug: Placebo