Rosuvastatin (Crestor) in Friedreich Ataxia

Early Phase 1

Completed

• Conditions: Friedreich Ataxia
• Interventions: Drug: Rosuvastatin

• Registration Number: NCT02705547
• Lead Sponsor: Children's Hospital of Philadelphia

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.

Study Timeline

• Study First Submitted: 2016-03-05
• Study First Submitted QC: 2016-03-05
• Study First Posted: 2016-03-10
• Study Start: 2016-05
• Primary Completion: 2017-08-04
• Study Completion: 2017-08-04
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-23
• Last Update Posted: 2021-03-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Rosuvastatin (Crestor)	Rosuvastatin	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.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.
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.

Trial Locations

Locations (1)

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States