Rosuvastatin (Crestor) in Friedreich Ataxia

NCT ID: NCT02705547

Last Updated: 2021-03-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-05-31
• Study Completion Date: 2017-08-04

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.

Conditions

Friedreich Ataxia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Rosuvastatin (Crestor)

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.

Group Type: EXPERIMENTAL

Rosuvastatin

Intervention Type: DRUG

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

Interventions

Rosuvastatin

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

Intervention Type: DRUG

Other Intervention Names

Crestor

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Friedreich's Ataxia Research Alliance

OTHER

Sponsor Role: collaborator

Children's Hospital of Philadelphia

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

David Lynch, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

Locations

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Countries

United States

Other Identifiers

16-012659

Identifier Type: -

Identifier Source: org_study_id