Finding studies
Finding studies
Take this into the appointment.
Saves the questions and what to expect into your notes, next to the visit they belong to.
Lead
Children's Hospital of Philadelphia
With
To evaluate the effect of high dose (40-60mg) versus low dose (20-30mg) Elamipretide on high contrast visual acuity in FRDA compared to baseline at 52 weeks with the option to extend for an additional 52 weeks if there are objective signs of clinical improvement on primary or secondary endpoints. The interim analysis will be based on data from a 36-week visit. For subjects worse than 20/800 at study start, they will be followed using low vision alternatives only.
Age
16–any
Sex
ALL
Healthy volunteers
Not accepted