Rosuvastatin (Crestor) in Friedreich Ataxia

• ClinicalTrials.gov Identifier: NCT02705547
• Recruitment Status: Completed
• First Posted: March 10, 2016
• Last Update Posted: January 10, 2018
• Sponsor: Children's Hospital of Philadelphia
• Collaborator: Friedreich's Ataxia Research Alliance
• Information provided by (Responsible Party): Children's Hospital of Philadelphia

Tracking Information

• First Submitted Date: March 5, 2016
• First Posted Date: March 10, 2016
• Last Update Posted Date: January 10, 2018
• Study Start Date: May 2016
• Actual Primary Completion Date: August 4, 2017 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 20, 2016)

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.

Original Primary Outcome Measures (submitted: March 5, 2016)

Change in ApoA-1 serum protein levels [ Time Frame: Baseline visit compared to Day 84 visit ]

ApoA-1 serum levels have been found to be lower in FRDA patients than in control subjects. We will evaluate the effect of treatment with Rosuvastatin on ApoA-1 serum levels to see if this may be a useful biomarker of disease.

Current Secondary Outcome Measures (submitted: April 20, 2016)

• 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.
• 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.

Original Secondary Outcome Measures (submitted: March 5, 2016)

• Change in frataxin levels [ Time Frame: Baseline visit compared to Day 84 visit ]
  Patients with FRDA do not make enough of the protein frataxin. We will evaluate if administration of Rosuvastatin has any effect on frataxin levels in whole blood and buccal cell samples.
• Change in platelet metabolism [ Time Frame: Baseline visit compared to Day 84 visit ]
  Platelet metabolism studies will be conducted at baseline and Day 84 visits to see if there is any effect of administration of Rosuvastatin on this biomarker of disease.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Rosuvastatin (Crestor) in Friedreich Ataxia
• Official Title: Open-label Biomarker Study of Rosuvastatin (Crestor) for the Treatment of Patients With Friedreich Ataxia
• 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.
• Study Type: Interventional
• Study Phase: Early Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Friedreich Ataxia

Intervention

Drug: Rosuvastatin

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

Other Name: Crestor

Study Arms

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.

Intervention: Drug: Rosuvastatin

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: April 20, 2016): 12
• Original Estimated Enrollment (submitted: March 5, 2016): 10
• Actual Study Completion Date: August 4, 2017
• Actual Primary Completion Date: August 4, 2017 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 65 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02705547
• Other Study ID Numbers: 16-012659
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Children's Hospital of Philadelphia
• Study Sponsor: Children's Hospital of Philadelphia
• Collaborators: Friedreich's Ataxia Research Alliance

Investigators

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

• PRS Account: Children's Hospital of Philadelphia
• Verification Date: January 2018