Rosuvastatin (Crestor) in Friedreich Ataxia | NCT02705547

Studies
Rosuvastatin (Crestor) in Friedreich Ataxia

NCT02705547

Sponsor

Children's Hospital of Philadelphia

Status

Completed

Last Update Posted

Mar 25, 2021Actual

Enrollment

12Actual

Phase

Early Phase 1

Conditions

Friedreich Ataxia

Interventions

Rosuvastatin

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2021

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

May 2016

Primary Completion Date

Aug 4, 2017Actual

Completion Date

Aug 4, 2017Actual

First Submitted Date

Mar 5, 2016

First Submission Date that Met QC Criteria

Mar 5, 2016

First Posted Date

Mar 10, 2016Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 23, 2021

Last Update Posted Date

Mar 25, 2021Actual

Name	Class
Friedreich's Ataxia Research Alliance	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Early Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

12Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Rosuvastatin (Crestor)	Experimental	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

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Rosuvastatin	Drug	Daily oral administration of Rosuvastatin (10 mg) for 3 months	Rosuvastatin (Crestor)

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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.	12 weeks

Secondary Outcomes

Measure	Description	Time Frame
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.	12 weeks
Change in platelet metabolism from baseline to Week 12 visit

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

65 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
David Lynch, MD PhD	Children's Hospital of Philadelphia	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital of Philadelphia		Philadelphia	Pennsylvania	19103	United States

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D005621	Friedreich Ataxia

Ancestor Terms

ID	Term
D013132	Spinocerebellar Degenerations
D002526	Cerebellar Diseases
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D000068718	Rosuvastatin Calcium

Ancestor Terms

ID	Term
D013449	Sulfonamides
D000577	Amides
D009930	Organic Chemicals
D005464	Fluorobenzenes

Browse Leaves

Not provided

Browse Branches

Not provided