Friedreich's AtaxiaPhase 2NCT01035671
Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia
A Phase 2 study testing alpha-tocopherolquinone (A0001) in people with Friedreich's Ataxia, run by Penwest Pharmaceuticals Co. at 1 site in United States.
Trial summary
This is a Phase 2a double-blind, placebo-controlled study with two dose levels of A0001 given twice daily for 28 days. Potential subjects will be screened first to determine eligibility, after which they will be randomized to receive either a high dose of A0001, a low dose of A0001 or placebo for 28 days. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- alpha-tocopherolquinone (A0001)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 42 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2009to Mar 2011
- Phase
- Phase 2
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Individuals with genetically confirmed Friedreich's Ataxia (GAA or point mutation)
- Impaired Glucose Tolerance, measured by Oral GTT
Who cannot join
- Overt Diabetes Mellitus
- Presence of clinically significant cardiovascular disease
Trial locations and contacts
1 site in 1 country.
United States1 site
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, 28 Day, Three-arm, Parallel Group Study of A0001 in the Treatment of Subjects With Friedreich's Ataxia
This is a Phase 2a double-blind, placebo-controlled study with two dose levels of A0001 given twice daily for 28 days. Potential subjects will be screened first to determine eligibility, after which they will be randomized to receive either a high dose of A0001, a low dose of A0001 or placebo for 28 days.
Eligible subjects will return within 21 days of screening for the baseline visit and randomization to one of three potential treatments. The subjects will be required to take 3 capsules of study medication in the morning with a morning meal and 3 capsules of study medication at night with an evening meal for 28 days. Additional visits to the clinic are planned for Day 14 and Day 28, at which time a number of clinical and biochemical assessments will be done.
- Conditions
- Friedreich's Ataxia
- Being tested
- alpha-tocopherolquinone (A0001), placebo
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
alpha-tocopherolquinone (A0001)Drug
28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
alpha-tocopherolquinone (A0001)Drug
28 days of high dose (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
placeboDrug
28 days of placebo oral capsules. Treatment taken twice daily with meals.
| Study group | Role | What they receive |
|---|---|---|
| Low Dose | Experimental | A0001 (0.5 g BID) Receives: Drug: alpha-tocopherolquinone (A0001) |
| High Dose | Experimental | A0001 (0.75 g BID) Receives: Drug: alpha-tocopherolquinone (A0001) |
| Placebo | Placebo comparator | Placebo Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test
Measured: Baseline, Day 14 and Day 28
Secondary outcomes (15)
Sensitivity index (SI) calculated from IVGTT
Measured: Baseline, Day 14 and Day 28
Glucose effectiveness (SG) calculated from IVGTT
Measured: Baseline, Day 14 and Day 28
AIRg for glucose and insulin during IVGTT
Measured: Baseline, Day 14 and Day 28
Fasting Glucose, Insulin and Lactate
Measured: Baseline, Day 14 and Day 28
HbA1C
Measured: Baseline, Day 14 and Day 28
Plasma 1,5-anhydroglucitol (1,5-AG) (Glycomark)
Measured: Baseline, Day 14 and Day28
Specific Activity of Complex 1 in whole blood
Measured: Baseline, Day 14 and Day 28
Timed 25 Foot Walk Test
Measured: Baseline and Day 28
FARS/neurological exam
Measured: Baseline and Day 28
9-Hole Peg Test
Measured: Baseline, Day 14 and Day 28
Vision Low Contrast Letter Acuity Test
Measured: Baseline and Day 28
Global Impression of Clinical Severity
Measured: Baseline, Day 14 and Day 28
Modified Fatigue Impact Scale
Measured: Baseline and Day 28
Activities of Daily Living
Measured: Baseline and Day 28
SF-36®
Measured: Baseline and Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2009 (estimated)
- Study startDec 2009
- Primary completionFeb 2011
- Study completionMar 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
- 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Penwest Pharmaceuticals Co.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- David Lynch, MD, PhD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01035671
- Sponsor's protocol ID
- FRD02
- Status last verified
- Apr 2011
- First submitted
- 17 Dec 2009
- Last updated
- 22 Apr 2011
- Results posted
- No
- Data monitoring committee
- No
Publications
Lynch DR, Willi SM, Wilson RB, Cotticelli MG, Brigatti KW, Deutsch EC, Kucheruk O, Shrader W, Rioux P, Miller G, Hawi A, Sciascia T. A0001 in Friedreich ataxia: biochemical characterization and effects in a clinical trial. Mov Disord. 2012 Jul;27(8):1026-33. doi: 10.1002/mds.25058. Epub 2012 Jun 28.
PubMed 22744651Friedman LS, Schadt KA, Regner SR, Mark GE, Lin KY, Sciascia T, St John Sutton M, Willi S, Lynch DR. Elevation of serum cardiac troponin I in a cross-sectional cohort of asymptomatic subjects with Friedreich ataxia. Int J Cardiol. 2013 Aug 20;167(4):1622-4. doi: 10.1016/j.ijcard.2012.04.159. Epub 2012 May 26.
PubMed 22633670
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, 28 Day, Three-arm, Parallel Group Study of A0001 in the Treatment of Subjects With Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT01035671
- Acronym / sponsor ID
- FRD02
- Conditions
- Friedreich's Ataxia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Penwest Pharmaceuticals Co.Industry (pharma, biotech, devices)
- Study start
- Dec 2009
- Primary completion
- Feb 2011 (actual)
- Study completion
- Mar 2011 (actual)
- First posted
- 21 Dec 2009 (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 is a Phase 2a double-blind, placebo-controlled study with two dose levels of A0001 given twice daily for 28 days. Potential subjects will be screened first to determine eligibility, after which they will be randomized to receive either a high dose of A0001, a low dose of A0001 or placebo for 28 days.
Eligible subjects will return within 21 days of screening for the baseline visit and randomization to one of three potential treatments. The subjects will be required to take 3 capsules of study medication in the morning with a morning meal and 3 capsules of study medication at night with an evening meal for 28 days. Additional visits to the clinic are planned for Day 14 and Day 28, at which time a number of clinical and biochemical assessments will be done.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low Dose | Experimental | Drug: alpha-tocopherolquinone (A0001)A0001 (0.5 g BID) |
| High Dose | Experimental | Drug: alpha-tocopherolquinone (A0001)A0001 (0.75 g BID) |
| Placebo | Placebo comparator | Drug: placeboPlacebo |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (2 listed)
- Individuals with genetically confirmed Friedreich's Ataxia (GAA or point mutation)
- Impaired Glucose Tolerance, measured by Oral GTT
Key exclusion criteria (2 listed)
- Overt Diabetes Mellitus
- Presence of clinically significant cardiovascular disease
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 |
|---|---|---|
| alpha-tocopherolquinone (A0001)28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals. | Drug | Low Dose |
| alpha-tocopherolquinone (A0001)28 days of high dose (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals. | Drug | High Dose |
| placebo28 days of placebo oral capsules. Treatment taken twice daily with meals. | Drug | Placebo |
Primary outcome
Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test
Time frame: Baseline, Day 14 and Day 28
15 secondary outcomes
Sensitivity index (SI) calculated from IVGTT
Time frame: Baseline, Day 14 and Day 28
Glucose effectiveness (SG) calculated from IVGTT
Time frame: Baseline, Day 14 and Day 28
AIRg for glucose and insulin during IVGTT
Time frame: Baseline, Day 14 and Day 28
Fasting Glucose, Insulin and Lactate
Time frame: Baseline, Day 14 and Day 28
HbA1C
Time frame: Baseline, Day 14 and Day 28
Plasma 1,5-anhydroglucitol (1,5-AG) (Glycomark)
Time frame: Baseline, Day 14 and Day28
Specific Activity of Complex 1 in whole blood
Time frame: Baseline, Day 14 and Day 28
Timed 25 Foot Walk Test
Time frame: Baseline and Day 28
FARS/neurological exam
Time frame: Baseline and Day 28
9-Hole Peg Test
Time frame: Baseline, Day 14 and Day 28
Vision Low Contrast Letter Acuity Test
Time frame: Baseline and Day 28
Global Impression of Clinical Severity
Time frame: Baseline, Day 14 and Day 28
Modified Fatigue Impact Scale
Time frame: Baseline and Day 28
Activities of Daily Living
Time frame: Baseline and Day 28
SF-36®
Time frame: Baseline and Day 28
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 22 Apr 2011.
- Derived from this study: PubMed 22744651Lynch DR, Willi SM, Wilson RB, Cotticelli MG, Brigatti KW, Deutsch EC, Kucheruk O, Shrader W, Rioux P, Miller G, Hawi A, Sciascia T. A0001 in Friedreich ataxia: biochemical characterization and effects in a clinical trial. Mov Disord. 2012 Jul;27(8):1026-33. doi: 10.1002/mds.25058. Epub 2012 Jun 28.
- Derived from this study: PubMed 22633670Friedman LS, Schadt KA, Regner SR, Mark GE, Lin KY, Sciascia T, St John Sutton M, Willi S, Lynch DR. Elevation of serum cardiac troponin I in a cross-sectional cohort of asymptomatic subjects with Friedreich ataxia. Int J Cardiol. 2013 Aug 20;167(4):1622-4. doi: 10.1016/j.ijcard.2012.04.159. Epub 2012 May 26.
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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 Phase 2 study testing alpha-tocopherolquinone (A0001) in people with Friedreich's Ataxia, run by Penwest Pharmaceuticals Co. 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 2 inclusion and 2 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 22 Apr 2011. 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. Some participants in this study may receive a placebo. 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test (time frame: Baseline, Day 14 and Day 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01035671, which also lists 2 linked publications.
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 22 Apr 2011.