NCT06874010RecruitingPhase 1/2
A Multiple Ascending Dose Study of 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
Source: ClinicalTrials.gov · Last updated 18 May 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 20 (estimated)
- First submitted
- 25 Feb 2025
- Start date
- Jun 2025
- Primary completion date
- Dec 2026
- Lead sponsor
- Design Therapeutics, Inc.
- Diseases
- Friedreich ataxia
- NCT number
- NCT06874010
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 20
- From Jun 2025 to the primary completion date, Dec 2026
- 3 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study protocol.
- 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.
Other illness
- 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.
Other
- Is not willing to comply with the contraceptive requirements during the study period, as per protocol.
Who the study is looking for
- 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.
Age in the registry: 18 years to 65 years · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Doherty Clinical Trials Coordinator of this site? Confirm its status | East Melbourne | Australia | Recruiting |
| Nucleus Network Coordinator of this site? Confirm its status | Melbourne | Australia | Recruiting |
| Scientia Clinical Research Ltd Coordinator of this site? Confirm its status | Sydney | Australia | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.