Clinical Trial: Rosuvastatin (Crestor) in Friedreich Ataxia

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Open-label Biomarker Study of Rosuvastatin (Crestor) for the Treatment of Patients With Friedreich Ataxia

Brief Summary: This study is an exploratory open-label clinical trial of Rosuvastatin in patients with Friedreich ataxia (FRDA). This is an outpatient trial with the goal of enrolling 10 evaluable adults with genetically confirmed FRDA who are between the ages of 18-65. Subjects will receive 10mg of oral Rosuvastatin daily for three months.

Detailed Summary: Friedreich ataxia (FRDA) is a progressive neurodegenerative disease of children and adults for which there is presently no therapy. Much of the current work in FRDA is aimed at finding new targets for drug therapies. Recent work at the University of Pennsylvania has discovered that serum ApoA-1 protein levels are lower in people with FRDA when compared with control levels. ApoA-1 is the main protein found in high-density lipoprotein (HDL) cholesterol and individuals with FRDA frequently have low HDL levels; the current study proposes to assess if administration of HMG-CoA reductase inhibitors for 3 months alters ApoA-1 protein levels in FRDA. Although the significance of ApoA-1 levels among FRDA patients is currently unknown, this study is proposed as an exploratory study to further examine this protein. If ApoA-1 protein levels increase over the course of treatment, future studies may additionally focus on examining this as a potential therapeutic treatment.
Sponsor: Children's Hospital of Philadelphia

Current Primary Outcome: 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.


Original Primary Outcome: Change in ApoA-1 serum protein levels [ Time Frame: Baseline visit compared to Day 84 visit ]

ApoA-1 serum levels have been found to be lower in FRDA patients than in control subjects. We will evaluate the effect of treatment with Rosuvastatin on ApoA-1 serum levels to see if this may be a useful biomarker of disease.


Current Secondary Outcome:

  • 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.


Original Secondary Outcome:

  • Change in frataxin levels [ Time Frame: Baseline visit compared to Day 84 visit ]
    Patients with FRDA do not make enough of the protein frataxin. We will evaluate if administration of Rosuvastatin has any effect on frataxin levels in whole blood and buccal cell samples.
  • Change in platelet metabolism [ Time Frame: Baseline visit compared to Day 84 visit ]
    Platelet metabolism studies will be conducted at baseline and Day 84 visits to see if there is any effect of administration of Rosuvastatin on this biomarker of disease.


Information By: Children's Hospital of Philadelphia

Dates:
Date Received: March 5, 2016
Date Started: May 2016
Date Completion: August 2018
Last Updated: March 15, 2017
Last Verified: March 2017