Friedreich AtaxiaEarly Phase 1NCT02705547
Rosuvastatin (Crestor) in Friedreich Ataxia
A Early Phase 1 study testing Rosuvastatin in people with Friedreich Ataxia, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- Rosuvastatin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- May 2016to 4 Aug 2017
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19103
About this study
Official title: Open-label Biomarker Study of Rosuvastatin (Crestor) for the Treatment of Patients With Friedreich Ataxia
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.
Read the 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.
- Conditions
- Friedreich Ataxia
- Being tested
- Rosuvastatin
- Keywords
- Friedreich Ataxia
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RosuvastatinDrug
Also called Crestor
Daily oral administration of Rosuvastatin (10 mg) for 3 months
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Rosuvastatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 12 weeks
Secondary outcomes (2)
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.
Measured: 12 weeks
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.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2016 (estimated)
- Study startMay 2016
- Primary completion4 Aug 2017
- Study completion4 Aug 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Collaborators
- Friedreich's Ataxia Research Alliance
- Responsible party
- Sponsor
- Investigators
- David Lynch, MD PhD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02705547
- Sponsor's protocol ID
- 16-012659
- Status last verified
- Mar 2021
- First submitted
- 5 Mar 2016
- Last updated
- 25 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label Biomarker Study of Rosuvastatin (Crestor) for the Treatment of Patients With Friedreich Ataxia
- ClinicalTrials.gov ID
- NCT02705547
- Acronym / sponsor ID
- 16-012659
- Conditions
- Friedreich Ataxia
- Keywords
- Friedreich Ataxia
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Collaborators
- Friedreich's Ataxia Research Alliance
- Study start
- May 2016
- Primary completion
- 4 Aug 2017 (actual)
- Study completion
- 4 Aug 2017 (actual)
- First posted
- 10 Mar 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rosuvastatin (Crestor) | Experimental | Drug: RosuvastatinThis is an open-label study of Rosuvastatin (Crestor) in patients with FRDA. Study subjects will receive 10 mg of Rosuvastatin daily for 3 months. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RosuvastatinAlso: CrestorDaily oral administration of Rosuvastatin (10 mg) for 3 months | Drug | Rosuvastatin (Crestor) |
Primary outcome
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.
2 secondary outcomes
Change in frataxin levels from baseline to Week 12 visit
Time frame: 12 weeks
Change in platelet metabolism from baseline to Week 12 visit
Time frame: 12 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2021.
- Individual participant dataSharing planned: No
Related trials for Friedreich Ataxia
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Lynch, MD PhD principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Rosuvastatin in people with Friedreich Ataxia, run by Children's Hospital of Philadelphia at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in ApoA-1 serum protein levels from baseline to Week 12 visit (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2021.