Paid Medical Trial Volunteer for Friedreich’s Ataxia (MAD)

Study Schedule:

6 clinic visits including 1 telehealth visit, 4 home visits by a visiting nurse, and 1 in-patient stay at the clinical research unit for up to 22 days/21 nights. Total study duration up to 71 days.

Individuals who have a genetically confirmed diagnosis of Friedreich’s ataxia are needed for a Phase I 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 18 years of age or older
Have genetically confirmed Friedreich’s ataxia, homozygous GAA repeat expansions, with repeat sizing.
Are able to traverse 25 feet with or without a self propelled assistive device
Are able to perform basic daily care
Are able to sit upright with thighs together and arms crossed without requiring support on more than two sides
Are able to transfer from bed to chair either independently or with assistance while still physically contributing to the transfer in some way (e.g. able to partially support weight)

Travel reimbursement available and a stipend per visit will be provided upto $5000.