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

Launched by DESIGN THERAPEUTICS, INC. · Mar 11, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new medication called DT-216P2 to see how safe it is and how well it works in people with Friedreich's Ataxia (FA), a rare genetic condition that affects movement and coordination. The study will involve giving participants increasing doses of the medication to understand how their bodies process it and to monitor any side effects. The trial is currently not recruiting participants, but when it begins, it will be open to adults aged 18 to 55 who have been genetically confirmed to have FA and meet certain health criteria.

To participate, individuals must have a specific genetic marker for FA and be in a certain stage of the disease, which is measured using a scale. They also need to have a healthy body weight and be able to give informed consent, meaning they understand the study and agree to take part. Participants will be closely monitored throughout the study, and there are specific rules about other medications and health conditions that could affect their participation. This trial represents an important step in finding new treatments for FA, and participants will contribute valuable information that could help others in the future.

Gender

ALL

Eligibility 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.