Rosuvastatin (Crestor) in Friedreich Ataxia

NCT02705547 | Completed Early Phase 1

• 1 other identifier: 16-012659
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Friedreich Ataxia

Keywords

Friedreich Ataxia

Outcome Measures

Primary Outcomes (1)

• 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 (2)

• Change in frataxin levels from baseline to Week 12 visit

  12 weeks

• Change in platelet metabolism from baseline to Week 12 visit

  12 weeks

Study Arms (1)

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

Rosuvastatin DRUG

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

Also known as: Crestor

Rosuvastatin (Crestor)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Currently active or unresolved liver or kidney disease
• Known history of renal insufficiency or creatine kinase \>2 x ULN
• Current use of niacin and/or fibric acid derivatives
• Current use of cyclosporine
• Use of any investigational product within 30 days of baseline visit

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• Friedreich's Ataxia Research Alliance: collaborator

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19103, United States

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

Rosuvastatin Calcium

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Sulfonamides; Amides; Organic Chemicals; Fluorobenzenes; Hydrocarbons, Fluorinated; Hydrocarbons, Halogenated; Hydrocarbons; Sulfones; Sulfur Compounds; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• David Lynch, MD PhD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 5, 2016
• First Posted: March 10, 2016
• Study Start: May 1, 2016
• Primary Completion: August 4, 2017
• Study Completion: August 4, 2017
• Last Updated: March 25, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)