ClinicalTrials.gov Identifier: NCT02705547 First received: March 5, 2016
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19103
Principal Investigator: David Lynch, MD PhD
This is an open-label study of Rosuvastatin (Crestor) in patients with FRDA. Study subjects will receive 10 mg of Rosuvastatin daily for 3 months.Daily administration of oral tablets of Rosuvastatin, 10 mg.
Context: Friedreich ataxia (FRDA) is a progressive neurodegenerative disease of children and adults for which there is presently no therapy. It has previously been reported that patients with FRDA have lower than expected HDL levels, and recent work by Dr. Blair at the University of Pennsylvania has found that serum ApoA-1 levels in FRDA patients are lower than those in controls. ApoA-1 levels are controlled by HMG-CoA activity, which is elevated in FRDA, and we hope to examine whether HMG-CoA levels can be inhibited by giving statins to patients with FRDA. Inhibition of this pathway has been proposed as a treatment for a variety of neurodegenerative disorders, and we will examine if FRDA may be one of them by looking at various biomarkers in the disease.
Thursday, March 10, 2016
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