Rosuvastatin (Crestor) in Friedreich Ataxia

Early Phase 1CompletedNCT02705547

Children's Hospital of Philadelphia12 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Friedreich Ataxia

Interventions

RosuvastatinDRUG

Daily oral administration of Rosuvastatin (10 mg) for 3 months

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Outcomes

Primary Outcomes

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.

Time frame: 12 weeks

Secondary Outcomes

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.

Time frame: 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.

Time frame: 12 weeks

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.