What is DT-216P2 studying for Friedreich's Ataxia?

DT-216P2 is being studied to evaluate its safety, tolerability, and how it is processed in the body for people with Friedreich's Ataxia. Researchers want to understand these factors to guide future treatments. The study is sponsored by Design Therapeutics, Inc. and focuses on patients with a confirmed genetic diagnosis of this condition.

Who may be eligible for this Friedreich's Ataxia clinical trial?

Adults aged 18 to 65 with a confirmed genetic diagnosis of Friedreich's Ataxia may be eligible. Both males and females can participate if they use approved contraception and meet health criteria like body weight and disease stage. Please review eligibility criteria for more details.

Is there any cost to participate in this Friedreich's Ataxia clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal expenses such as travel to and from the study site may vary and are not guaranteed to be covered.

Actively Recruiting

Phase 1

Phase 2

Age: 18Years - 65Years

All Genders

ID06874010

Phase 12 Study of Safety and Pharmacokinetics of DT-216P2 Given by Injection in Adults with Friedreichs Ataxia

Led by Design Therapeutics, Inc. · Updated on 2025-05-18

Participants Needed

Research Sites

12 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are studying Friedreichs Ataxia FA, a genetic condition, to assess the safety, tolerability, and how the body processes a drug called DT-216P2. This phase 12 open-label trial focuses on understanding these factors when the drug is given in multiple increasing doses to people with FA. The study is sponsored by Design Therapeutics, Inc. and aims to gather important information about the treatments effects in this patient group. Participants will receive DT-216P2 through subcutaneous under the skin or intravenous into a vein administration in varying doses. The study includes multiple dose levels to observe the drugs behavior and side effects up to 12 weeks. There is no placebo group, and all participants will be treated with DT-216P2 during the study period, which starts in June 2025 and is planned to conclude by March 2027. During the study, participants will be closely monitored for any side effects or adverse events, with particular attention to treatment-emergent adverse events up to week 12. Researchers will also measure drug levels in the body, including maximum concentration and how long it stays in the system. Frataxin protein levels, important in FA, will be checked before and after treatment. Participants will be regularly assessed for safety and treatment effects throughout the study duration.

CONDITIONS

Brief Title

A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia

Who Can Participate

Age: 18Years - 65Years

All Genders

Eligibility Criteria

You may qualify if you...

Participants must be 18-65 years of age inclusive at the time of signing informed consent
Genetically confirmed diagnosis of Friedreich's Ataxia with homozygous GAA repeat expansions in the frataxin gene
Stage 5.5 or less on the Friedreich's Ataxia Staging Assessment (FSA) at screening
Body mass index (BMI) between 16 and 32 kg/m2 at screening; weight must be 100 kg or less
Male and/or female using protocol-defined and regulatory approved contraception
Capable of giving signed informed consent

You will not qualify if you...

Any medical condition that puts the participant at risk or prevents completion of the study as judged by the investigator
Significant nonmedical conditions or psychiatric disorders that increase risk or interfere with study participation or results
Received an investigational agent within the last 30 days or 5 half-lives before first dose, except those on stable Omaveloxolone doses for at least 3 months
Unwillingness to comply with contraceptive requirements during the study period as per protocol

Research Team

Varlli Scott

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