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 planned. 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:
Compensation for time, travel, and participation will be provided for eligible participants.
You May Be Eligible if You:
- Are male or female between the ages of 18 to 65 years old (inclusive) at screening
- Can perform basic daily care, such as feeding yourself and personal hygiene, with minimal assistance
- Can sit up with thighs and arms together crossed without requiring support on more than 2 sides
- Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening
Other inclusion/ exclusion criteria may apply.