Testing DT-216P2 in patients with Friedreich's Ataxia
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
This study is testing a new drug called DT-216P2 in adults with Friedreich's Ataxia to see how safe it is and how well it works in their bodies.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Design Therapeutics, Inc. Industry-sponsored |
| Locations | 3 sites (Sydney, New South Wales and 2 other locations) |
| Trial ID | NCT06874010 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety, tolerability, and pharmacokinetics of multiple ascending doses of DT-216P2 in patients diagnosed with Friedreich's Ataxia (FA). Participants aged 18-65 with a confirmed genetic diagnosis of FA will receive the treatment to assess its effects. The study aims to gather data on how the drug is processed in the body and its potential therapeutic benefits for this condition.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18-65 with a genetically confirmed diagnosis of Friedreich's Ataxia and specific clinical criteria.
Not a fit: Patients with significant comorbid conditions or psychiatric disorders that may interfere with the study are unlikely to benefit.
Why it matters
Potential benefit: If successful, this treatment could improve the management of Friedreich's Ataxia and enhance the quality of life for patients.
How similar studies have performed: While this approach is novel, similar studies targeting Friedreich's Ataxia have shown promise in exploring new treatments.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Participants must be 18-65 years of age inclusive, at the time of signing the informed consent. * Genetically confirmed diagnosis of FA, with homozygous GAA repeat expansions in the frataxin gene. * Stage 5.5 or less on the FSA at screening. * BMI between 16 and 32 kg/m2 at screening; weight should be \<= 100 kg at screening. * Male and/or female using protocol defined and regulatory approved contraception. * Capable of giving signed informed consent. Exclusion Criteria: * Any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study protocol. * Any clinically significant nonmedical conditions and psychiatric disorders that could put the participant at higher risk for participation in the study, influence the participant's ability to participate in the study, or interfere with interpretation of the participant's study results, in the opinion of the investigator. * Received an investigational agent within the last 30 days or 5 half-lives, whichever longer, prior to the first dose of study drug, or are in follow-up of another clinical study prior to study enrollment. Exception: Potential participants who are currently on Omaveloxolone must be on stable doses for at least 3 months. * Is not willing to comply with the contraceptive requirements during the study period, as per protocol.
Where this trial is running
Sydney, New South Wales and 2 other locations
- Scientia Clinical Research Ltd — Sydney, New South Wales, Australia (Recruiting)
- Doherty Clinical Trials — East Melbourne, Victoria, Australia (Recruiting)
- Nucleus Network — Melbourne, Victoria, Australia (Recruiting)
Study contacts
- Study coordinator: Varlli Scott
- Email: varlli.scott@mcri.edu.au
- Phone: +613 8341 6228
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.