DT-216P2 is a small molecule therapy designed to increase the body’s natural production of frataxin, the protein that is deficient in people with Friedrich ataxia. It works by targeting the root cause of the disease – a GAA repeat expansion in the FXN gene that disrupts how the gene is read resulting in reduced frataxin protein. DT-216P2 selectively targets this expanded sequence to recruit the body’s own machinery, restore proper gene activity and increase natural frataxin protein levels.
A Phase 1 study of multiple increasing doses of DT-216P2 in people with FA is underway. Participants will be enrolled for up to 12 weeks and administered doses of DT-216P2 once a week, with regular safety assessments after dosing.
Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.
Study Details:
Who may be eligible:
- Age 18 to 65 with genetically confirmed Friedreich ataxia and symptom onset before age 25
- Able to stand unassisted for at least 10 seconds
- Ability to transfer independently
- Not currently enrolled in another FA therapeutic trial
- No prior experimental gene therapy
- Not taking blood thinners
- Willing to undergo muscle biopsy before and after treatment
- Willing to undergo enclosed MRI procedures before and after treatment
Additional information:
- If you take Skyclarys (omaveloxolone): Please discuss with your physician whether stopping at least 30 days before the study start date and remaining off the medication during study participation is appropriate for you.
- Contraception is required throughout the study and for a period after completion.
- Participation requires approximately a 14-week stay in Australia near the study site and adherence to protocol requirements.
- Both participants and caregivers must be fluent in English and able to provide adequate consent in English.
- Travel, lodging, and reasonable expenses are covered for the participant and a caregiver.