NCT02705547

Rosuvastatin (Crestor) in Friedreich Ataxia

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

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Children's Hospital of Philadelphia · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Friedreich Ataxia

Interventions

Type	Name	Description
DRUG	Rosuvastatin	Daily oral administration of Rosuvastatin (10 mg) for 3 months

Timeline

• Start date: 2016-05-01
• Primary completion: 2017-08-04
• Completion: 2017-08-04
• First posted: 2016-03-10
• Last updated: 2021-03-25

Locations

1 site across 1 country: United States