Rosuvastatin (Crestor) in Friedreich Ataxia
|ClinicalTrials.gov Identifier: NCT02705547|
Recruitment Status : Completed
First Posted : March 10, 2016
Last Update Posted : January 10, 2018
|Condition or disease||Intervention/treatment||Phase|
|Friedreich Ataxia||Drug: Rosuvastatin||Early Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Open-label Biomarker Study of Rosuvastatin (Crestor) for the Treatment of Patients With Friedreich Ataxia|
|Study Start Date :||May 2016|
|Actual Primary Completion Date :||August 4, 2017|
|Actual Study Completion Date :||August 4, 2017|
Experimental: Rosuvastatin (Crestor)
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 oral administration of Rosuvastatin (10 mg) for 3 months
Other Name: Crestor
- Change in ApoA-1 serum protein levels from baseline to Week 12 visit [ Time Frame: 12 weeks ]Serum ApoA-1 protein levels will be collected at baseline and again at the Week 12 visit.
- Change in frataxin levels from baseline to Week 12 visit [ Time Frame: 12 weeks ]Frataxin levels in whole blood and buccal cells will be collected at baseline and again at the Week 12 visit.
- Change in platelet metabolism from baseline to Week 12 visit [ Time Frame: 12 weeks ]Platelet metabolism will be assessed by performing liquid chromatography-mass spectrometry analysis on whole blood samples collected at baseline and again at the Week 12 visit.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02705547
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19103|
|Principal Investigator:||David Lynch, MD PhD||Children's Hospital of Philadelphia|