Rosuvastatin (Crestor) in Friedreich Ataxia

• ClinicalTrials.gov Identifier: NCT02705547
• Recruitment Status: Completed
• First Posted: March 10, 2016
• Last Update Posted: March 25, 2021
• Sponsor: Children's Hospital of Philadelphia
• Collaborator: Friedreich's Ataxia Research Alliance
• Information provided by (Responsible Party): Children's Hospital of Philadelphia

Study Description

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.

Study Design

• 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

Arms and Interventions

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

Outcome Measures

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.

Eligibility Criteria

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

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

Contacts and Locations

Locations

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

Investigators

• Principal Investigator: David Lynch, MD PhD; Children's Hospital of Philadelphia

More Information

• Responsible Party: Children's Hospital of Philadelphia
• ClinicalTrials.gov Identifier: NCT02705547
• Other Study ID Numbers: 16-012659
• First Posted: March 10, 2016
• 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:

Ataxia; Cerebellar Ataxia; Friedreich Ataxia; Dyskinesias; 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; Antimetabolites; Molecular Mechanisms of Pharmacological Action; Lipid Regulating Agents; Hydroxymethylglutaryl-CoA Reductase Inhibitors; Enzyme Inhibitors