search
Back to results

Rosuvastatin (Crestor) in Friedreich Ataxia

Primary Purpose

Friedreich Ataxia

Status
Completed
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
Rosuvastatin
Sponsored by
Children's Hospital of Philadelphia
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Friedreich Ataxia focused on measuring Friedreich Ataxia

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's Hospital of Philadelphia

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Rosuvastatin (Crestor)

Arm Description

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.

Outcomes

Primary Outcome Measures

Change in ApoA-1 serum protein levels from baseline to Week 12 visit
Serum ApoA-1 protein levels will be collected at baseline and again at the Week 12 visit.

Secondary Outcome Measures

Change in frataxin levels from baseline to Week 12 visit
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
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.

Full Information

First Posted
March 5, 2016
Last Updated
March 23, 2021
Sponsor
Children's Hospital of Philadelphia
Collaborators
Friedreich's Ataxia Research Alliance
search

1. Study Identification

Unique Protocol Identification Number
NCT02705547
Brief Title
Rosuvastatin (Crestor) in Friedreich Ataxia
Official Title
Open-label Biomarker Study of Rosuvastatin (Crestor) for the Treatment of Patients With Friedreich Ataxia
Study Type
Interventional

2. Study Status

Record Verification Date
March 2021
Overall Recruitment Status
Completed
Study Start Date
May 2016 (undefined)
Primary Completion Date
August 4, 2017 (Actual)
Study Completion Date
August 4, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital of Philadelphia
Collaborators
Friedreich's Ataxia Research Alliance

4. Oversight

Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Friedreich Ataxia
Keywords
Friedreich Ataxia

7. Study Design

Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Rosuvastatin (Crestor)
Arm Type
Experimental
Arm Description
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 Type
Drug
Intervention Name(s)
Rosuvastatin
Other Intervention Name(s)
Crestor
Intervention Description
Daily oral administration of Rosuvastatin (10 mg) for 3 months
Primary Outcome Measure Information:
Title
Change in ApoA-1 serum protein levels from baseline to Week 12 visit
Description
Serum ApoA-1 protein levels will be collected at baseline and again at the Week 12 visit.
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Change in frataxin levels from baseline to Week 12 visit
Description
Frataxin levels in whole blood and buccal cells will be collected at baseline and again at the Week 12 visit.
Time Frame
12 weeks
Title
Change in platelet metabolism from baseline to Week 12 visit
Description
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.
Time Frame
12 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David Lynch, MD PhD
Organizational Affiliation
Children's Hospital of Philadelphia
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19103
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Rosuvastatin (Crestor) in Friedreich Ataxia

We'll reach out to this number within 24 hrs