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Phase 1 clinical trial of DT-216 to assess the safety, tolerability, pharmacokinetics, and frataxin (FXN) levels in Friedreich Ataxia

DT-216 is a new molecule to activate transcription of the FXN gene and restore production of the frataxin protein. More specifically, it is a GeneTACâ„¢ gene targeted chimera small molecule designed to specifically target the GAA repeat expansion mutation and restore FXN gene expression.

This is a frst-in-human, Phase 1, study of DT-216. The initial single ascending dose part of the Phase 1 has completed recruitment and we are now beginning the Multiple Ascending Dose (MAD) study. Enrollment will be in cohorts (typically 5-8 people). The first cohort will be enrolled and tested at the first dose, then there will be a review of the safety, then typically several weeks later the next cohort will be enrolled to test the next dose. In the MAD study subjects will be treated with a once a week dose over 3 weeks, with regular safety assessments after dosing. The study treatment period is approximately 21-22 days and includes 3 non-consecutive inpatient stays at the study unit for a total of 10 nights/11 days. In between the unit stays during the 3 week dosing subjects can stay at a local hotel. Maximum duration of study participation is approximately 10.5 weeks.

Inclusion criteria:

  • Individuals with FA ages 18 to 55 years old (inclusive) at screening
  • Genetically confirmed diagnosis of FA with homozygous GAA repeat expansions
  • Weight between 90 and 200lbs (approximately)
  • Ability to sit upright with thighs together and arms crossed without requiring support on more than two sides
  • Ability to perform basic daily care such as feeding yourself and basic personal hygiene with minimal assistance

This study is being conducted at Clinilabs and will require about a week long stay at the study site. Compensation will be provided for qualified participants and caregivers. Individuals and caregivers who are not local to the research facility will receive no-cost transportation and accommodations.

Contact Clinilabs for more information or to express interest in the study.
Joan Mallet 212-994-4567
Eileen Thomas 212-994-4567

View the study flyer

Additional information can be found on the website (NCT05285540)

For more information about Design Therapeutics and DT-216, view the webinar hosted by FARA with Design, Oct 2021.
Webinar link:

Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular studies.

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