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A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Patients With Friedreich's Ataxia
Led by Design Therapeutics, Inc. · Updated on 2025-05-18
20
Participants Needed
3
Research Sites
12 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying Friedreich's Ataxia (FA), a genetic condition, to assess the safety, tolerability, and how the body processes a drug called DT-216P2. This phase 1/2 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 treatment's 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 drug's 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
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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 weeks
Participants receive multiple ascending doses of the study drug DT-216P2 to assess safety, tolerability, and pharmacokinetics.
Weekly visits for up to 12 weeks
Trial Site Locations
Total: 3 locations
1
Scientia Clinical Research Ltd
Sydney, New South Wales, Australia, 2031
Actively Recruiting
2
Doherty Clinical Trials
East Melbourne, Victoria, Australia, 3002
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3
Nucleus Network
Melbourne, Victoria, Australia, 3004
Actively Recruiting
Research Team
V
Varlli Scott
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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