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Rosuvastatin (Crestor) in Friedreich Ataxia

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

Tracking Information
First Submitted Date  ICMJE March 5, 2016
First Posted Date  ICMJE March 10, 2016
Last Update Posted Date January 10, 2018
Study Start Date  ICMJE May 2016
Actual Primary Completion Date August 4, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (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  ICMJE
 (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.
Change History
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE
 (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  ICMJE Rosuvastatin (Crestor) in Friedreich Ataxia
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Early Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Friedreich Ataxia
Intervention  ICMJE Drug: Rosuvastatin
Daily oral administration of Rosuvastatin (10 mg) for 3 months
Other Name: Crestor
Study Arms  ICMJE 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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 20, 2016)
12
Original Estimated Enrollment  ICMJE
 (submitted: March 5, 2016)
10
Actual Study Completion Date  ICMJE August 4, 2017
Actual Primary Completion Date August 4, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02705547
Other Study ID Numbers  ICMJE 16-012659
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Children's Hospital of Philadelphia
Study Sponsor  ICMJE Children's Hospital of Philadelphia
Collaborators  ICMJE Friedreich's Ataxia Research Alliance
Investigators  ICMJE
Principal Investigator: David Lynch, MD PhD Children's Hospital of Philadelphia
PRS Account Children's Hospital of Philadelphia
Verification Date January 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP