Rosuvastatin (Crestor) in Friedreich Ataxia

• Status: Completed
• Trial end date: 2017-08-04
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital of Philadelphia
• Collaborator: Friedreich's Ataxia Research Alliance
• Treatments: Rosuvastatin Calcium

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