Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia
NCT ID: NCT01035671
Last Updated: 2011-04-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
42 participants
INTERVENTIONAL
2009-12-31
2011-03-31
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Low Dose
A0001 (0.5 g BID)
alpha-tocopherolquinone (A0001)
28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
High Dose
A0001 (0.75 g BID)
alpha-tocopherolquinone (A0001)
28 days of high dose (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
Placebo
Placebo
placebo
28 days of placebo oral capsules. Treatment taken twice daily with meals.
Interventions
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alpha-tocopherolquinone (A0001)
28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
alpha-tocopherolquinone (A0001)
28 days of high dose (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
placebo
28 days of placebo oral capsules. Treatment taken twice daily with meals.
Eligibility Criteria
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Inclusion Criteria
* Impaired Glucose Tolerance, measured by Oral GTT
Exclusion Criteria
* Presence of clinically significant cardiovascular disease
18 Years
60 Years
ALL
No
Sponsors
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Penwest Pharmaceuticals Co.
INDUSTRY
Responsible Party
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Penwest Pharmaceuticals Co.
Principal Investigators
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David Lynch, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital of Philadelphia
Locations
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Countries
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References
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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.
Friedman 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.
Other Identifiers
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FRD02
Identifier Type: -
Identifier Source: org_study_id
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