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Rosuvastatin (Crestor) in Friedreich Ataxia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02705547
Recruitment Status : Completed
First Posted : March 10, 2016
Last Update Posted : March 25, 2021
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Friedreich Ataxia Drug: Rosuvastatin Early Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label Biomarker Study of Rosuvastatin (Crestor) for the Treatment of Patients With Friedreich Ataxia
Study Start Date : May 2016
Actual Primary Completion Date : August 4, 2017
Actual Study Completion Date : August 4, 2017

Arm Intervention/treatment
Experimental: 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.
Drug: Rosuvastatin
Daily oral administration of Rosuvastatin (10 mg) for 3 months
Other Name: Crestor

Primary Outcome Measures :
  1. Change in ApoA-1 serum protein levels from baseline to Week 12 visit [ Time Frame: 12 weeks ]
    Serum ApoA-1 protein levels will be collected at baseline and again at the Week 12 visit.

Secondary Outcome Measures :
  1. Change in frataxin levels from baseline to Week 12 visit [ Time Frame: 12 weeks ]
    Frataxin levels in whole blood and buccal cells will be collected at baseline and again at the Week 12 visit.

  2. Change in platelet metabolism from baseline to Week 12 visit [ Time Frame: 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02705547

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United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19103
Sponsors and Collaborators
Children's Hospital of Philadelphia
Friedreich's Ataxia Research Alliance
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Principal Investigator: David Lynch, MD PhD Children's Hospital of Philadelphia
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Responsible Party: Children's Hospital of Philadelphia Identifier: NCT02705547    
Other Study ID Numbers: 16-012659
First Posted: March 10, 2016    Key Record Dates
Last Update Posted: March 25, 2021
Last Verified: March 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Children's Hospital of Philadelphia:
Friedreich Ataxia
Additional relevant MeSH terms:
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Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Rosuvastatin Calcium
Anticholesteremic Agents
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors