Tests DT-216P2 safety and dosing in people with Friedreich's ataxia
Tests the drug DT-216P2 in adults with Friedreich's ataxia, a rare nerve disease.
Plain-language title generated from the registry record
Registry title: A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
Key facts
- Where
- East Melbourne, Melbourne, and Sydney, AustraliaSee the 3 sites
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 65 years old?
Source: NCT06874010, who can take part
Where in Australia
- Doherty Clinical TrialsEast Melbourne, AustraliaTaking people
- Nucleus NetworkMelbourne, AustraliaTaking people
- Scientia Clinical Research LtdSydney, AustraliaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Design Therapeutics, Inc.
- Trial code
- NCT06874010
- Record updated
- 18 May 2025
- Original description
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.Read full original descriptionShow less
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
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.
Inclusion: 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: 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; 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; Is not willing to comply with the contraceptive requirements during the study period, as per protocol.