Rosuvastatin (Crestor) in Friedreich Ataxia

Study Overview
=================
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 Description
-----------------
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.

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

Conditions
-----------------
Friedreich Ataxia

Intervention / Treatment
-----------------
* Drug: Rosuvastatin


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Subjects with Friedreich Ataxia confirmed by genetic testing Adults between the ages of 18 and 65 Stable quinone dose (at least 1000 mg of Idebenone or 200 mg Coenzyme Q10) for 14 days prior to study entry and for the duration of the study Females who are not pregnant or breast feeding, and who do not intend to become pregnant. Subject has voluntarily signed consent form Willingness and ability to comply with all study procedures Exclusion Criteria: Treatment with statins during the six previous months before study inclusion Currently active or unresolved liver or kidney disease Known history of renal insufficiency or creatine kinase >2 x ULN Use of red rice yeast during the previous six months before inclusion Current use of niacin and/or fibric acid derivatives Current use of cyclosporine Use of any investigational product within 30 days of baseline visit

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Rosuvastatin (Crestor)<br>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. | Drug: Rosuvastatin<br>* Daily oral administration of Rosuvastatin (10 mg) for 3 months<br>* Other names: Crestor;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in ApoA-1 serum protein levels from baseline to Week 12 visit | Serum ApoA-1 protein levels will be collected at baseline and again at the Week 12 visit. | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in frataxin levels from baseline to Week 12 visit | Frataxin levels in whole blood and buccal cells will be collected at baseline and again at the Week 12 visit. | 12 weeks | 
| Change in platelet metabolism from baseline to Week 12 visit | 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. | 12 weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Friedreich Ataxia