Age Range: 18 and older
Gender: Male, Female
Stipend: $7,750 for participants and $6,000 for caregivers
One telehealth visit and home nurse visit, 17 day/16 night inpatient stay, followed by 5 outpatient follow-up visits every other day, and 5 day/ 4 night inpatient stay. Upon discharge, home nurse follow-up visits 7 days and 30 days after the last dose of study drug. Total study duration up to 86 days.
Individuals who have a genetically confirmed diagnosis of Friedreich’s ataxia are needed for a Phase 2 research study of an investigational drug that is designed to replace frataxin, the protein deficient in FA, in mitochondria of cells.
You may be eligible if you:
- Are male or female 18 years of age or older
- Are able to perform basic daily care, such as feeding yourself and personal hygiene, with minimal assistance
- Can sit upright with thighs together and arms crossed without requiring support on more than 2 sides
- Have genetically confirmed Friedreich’s ataxia, homozygous GAA repeat expansions, with repeat sizing
- Weigh over 40kg (88 lbs)
Compensation for your time and travel per visit will be provided for qualified participants.
Click below to take our brief online questionnaire