The Children’s Hospital of Philadelphia (CHOP) is recruiting patients with Friedreich ataxia (FRDA) in the United States for a Phase 1/2 clinical trial studying the safety and effects of Elamipretide in the treatment of advanced stage FRDA symptoms, specifically vision loss and cardiac disease in both adults and older children.
This study will include up to 18 patients to be enrolled at the Children’s Hospital of Philadelphia.
Eligibility criteria include:
- Be 16 years of age or older
- Have genetic confirmation of your FRDA (point mutations allowed)
- Have been diagnosed with FRDA at age 18 years old or younger
- Have vision loss and/or cardiac disease
- Be willing to administer a daily injection (shot)
- Not be actively enrolled in any clinical trial
- Have any clinically relevant medical or surgical condition that could interfere with the administration of study drug, or compromise your safety or well-being
- Be pregnant, planning a pregnancy, or breastfeeding
- Have a history of substance abuse
- Have any other active cause of optic neuropathy or cardiac disease outside of FRDA
- Have a history of an uncontrolled arrhythmia
Additional requirements apply and will be discussed with you by the study doctor.
If you agree to take part, your participation will require a phone pre-screening, as well as up to 6 in-person visits over the course of 1 year. As a subject in the research study you will:
- Complete a phone pre-screening questionnaire to review eligibility criteria
- Receive a study drug (Elamipretide) as a daily shot (injection)
- Perform various vision testing
- Undergo heart function evaluations
- Have research blood tests at each study visit
- Complete questionnaires and speech testing
- Keep a subject dosing diary
Contact one of the CHOP study coordinators to learn more about what study procedures are involved and if you may be eligible to participate.
Courtney Cheek Park: Phone: (267) 426-9667; Email: firstname.lastname@example.org
McKenzie Wells: Phone: (267) 426-9608; Email: email@example.com
Medina Keita: Phone: (267) 426-7584; Email: firstname.lastname@example.org
Kellie McIntyre: Phone: (215) 590-2975; Email: email@example.com
View the Study flyer