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 GeneTAC™ gene targeted chimera small molecule designed to specifically target the GAA repeat expansion mutation and restore FXN gene expression.
This is a first-in-human, Phase 1, study of DT-216 which means that trial will begin with single dose cohorts to assess safety. 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. After the single dose study, there will be a multiple dose study, which is also a safety study. Multiple dose studies typically assess safety over a period of several weeks to a month. More information on the multiple dose study will be available once the single dose study further along.
- 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
- Must have completed full COVID-19 vaccination at least 4 weeks before treatment
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.
Clinical Research Unit Location:
4 Industrial Way, 2nd Floor
Eatontown, New Jersey 07724
(212) 994-4567 or visit clinilabs.com/volunteers/
View the study flyer
Additional information can be found on the clinicaltrials.gov website (NCT05285540) https://www.clinicaltrials.gov/ct2/show/NCT05285540
For more information about Design Therapeutics and DT-216, view the webinar hosted by FARA with Design, Oct 2021.
Webinar link: https://www.youtube.com/watch?v=uSdWGp3cK4k