Friedreich Ataxia
Conditions
Keywords
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 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.
Interventions
DRUGRosuvastatin
Daily oral administration of Rosuvastatin (10 mg) for 3 months
Sponsors
Friedreich's Ataxia Research Alliance
Children's Hospital of Philadelphia
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in ApoA-1 serum protein levels from baseline to Week 12 visit | 12 weeks | Serum ApoA-1 protein levels will be collected at baseline and again at the Week 12 visit. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in frataxin levels from baseline to Week 12 visit | 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 | 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. |
Countries
United States
Outcome results
None listed