FRDA is an inherited disease that causes progressive damage to the nervous system
UCLA Researchers are seeking patients 8-to-17 years old who have been diagnosed with Friedreich's Ataxia for a Phase III clinical research trial. The purpose of the trial is to examine the possible therapeutic effects of the drug idebenone (SNT-MC17) on the nervous system and heart.
Friedreich's Ataxia (FRDA) is an extremely rare disease, with no approved medication, that typically begins in childhood or early adolescence with symptoms that are slow and progressive. These include muscle weakness in the arms and legs; loss of coordination, vision impairment, curvature of the spine (scoliosis), and various heart disorders including atrial fibrillation, and enlargement of the heart.
A Phase III trial is a clinical trial to show the effectiveness and safety of a drug. The Food & Drug Administration requests such trials before a drug can be submitted for regulatory review and market approval. Earlier studies have shown idebenone to be effective with minimal side effects. The purpose of this study is to further show idebenone's clinical benefit on the nervous system and the hearts of patients with FRDA. The trial is being led by Dr. Susan Perlman, UCLA clinical professor of neurology.
Ambulatory Patients between the ages of 8-to-17 years-old are sought who have been diagnosed with FRDA. Participation in the study will require a nine month commitment. For six of those months participants will be treated with the medication or placebo (an inactive drug with no drug substance in it). Study volunteers will be assigned randomly to receive either one of the two drug doses or placebo. Santhera Pharmaceuticals, the developer of SNT-MC17/idebenone, will offer all patients that complete the study the opportunity to participate in a follow up study. In that study, all participants will receive the high-dose of SNT-MC17/idebenone for up to one year free of charge.
Patients participating in this current trial will be asked to visit UCLA five times for screening and monitoring. All costs associated with the travel will be fully reimbursed.
This study is funded by Santhera Pharmaceuticals