Rosuvastatin (Crestor) in Friedreich Ataxia
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|ClinicalTrials.gov Identifier: NCT02705547|
Recruitment Status : Completed
First Posted : March 10, 2016
Last Update Posted : March 25, 2021
|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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|