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Sponsors and Collaborators: |
Wayne State University Muscular Dystrophy Association Charcot-Marie-Tooth Association |
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Information provided by: | Wayne State University |
ClinicalTrials.gov Identifier: | NCT00484510 |
This study will look at the impact of ascorbic acid (Vitamin C) on the progression of disease in people with CMT1A as compared to volunteers receiving a placebo. This study will assess whether is it futile to proceed with a larger, longer-term, placebo-controlled study.
Condition | Intervention | Phase |
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Charcot-Marie-Tooth Disease, Type Ia |
Drug: Ascorbic acid (Vitamin C) Drug: placebo |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | A Randomized, Placebo-Controlled, Double Masked 120 Subject "Futility Design" Clinical Trial of Ascorbic Acid Treatment of Charcot Marie Tooth Disease Type 1A. |
Estimated Enrollment: | 120 |
Study Start Date: | April 2007 |
Estimated Study Completion Date: | December 2010 |
Estimated Primary Completion Date: | December 2010 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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Ascorbic Acid: Experimental |
Drug: Ascorbic acid (Vitamin C)
Eight 500 mg capsules/day of ascorbic acid. Subjects will take four (4)capsules each morning and four (4) capsules each evening for 24 months. (Total 4 gr/day).
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Placebo: Placebo Comparator |
Drug: placebo
Eight 500 mg capsules/day of placebo. Subjects will take four (4)capsules each morning and four (4) capsules each evening for 24 months.
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Charcot Marie Tooth disease (CMT), or inherited peripheral neuropathies, are among the most frequent heritable disorders, affecting approximately 1 in 2500 people. The most frequent genetic form of CMT is CMT1A. CMT1A is caused by a 1.4 Mb duplication within chromosome 17p11.2 in the region containing the PMP22 gene. Most subjects with CMT1A have a "typical" phenotype characterized by onset in childhood or early adulthood, distal weakness, sensory loss, foot deformities and absent reflexes. How increased expression of PMP22 causes these disabilities is unknown but is currently being investigated in both animal and tissue culture systems. In this study, researchers will evaluate whether ascorbic acid (Vitamin C), administered orally, slows clinical progression of CMT1A and affects the PMP22 mRNA levels of myelinated peripheral nerve fibers obtained from biopsies of glabrous skin.
Ages Eligible for Study: | 13 Years to 70 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Maryland | |
Johns Hopkins University, Dept of Neurology | Recruiting |
Baltimore, Maryland, United States, 21287 | |
Contact: Lora Clawson 410-624-4346 lclawson@jhmi.edu | |
Principal Investigator: Ahmet Hoke, MD | |
United States, Michigan | |
Wayne State University, Dept of Neurology | Recruiting |
Detroit, Michigan, United States, 48201 | |
Contact: Lisa Rowe 313-577-1689 lrowe@med.wayne.edu | |
Principal Investigator: Michael Shy, MD | |
United States, New York | |
University of Rochester Medical Center, Dept of Neurology | Recruiting |
Rochester, New York, United States, 14642 | |
Contact: Patty Smith 585-275-0581 Patty_Smith@urmc.rochester.edu | |
Principal Investigator: David Herrmann, MD |
Principal Investigator: | Richard A Lewis, MD | Wayne State University, Dept. of Neurology |
Responsible Party: | Wayne State University School of Medicine ( Richard A. Lewis, MD ) |
Study ID Numbers: | HIC074406MP2F, MDA4193 |
Study First Received: | June 8, 2007 |
Last Updated: | January 11, 2008 |
ClinicalTrials.gov Identifier: | NCT00484510 |
Health Authority: | United States: Food and Drug Administration |
Ascorbic Acid Vitamin C Charcot Marie Tooth CMT CMT1a |
Tooth Diseases Nervous System Malformations Roussy Levy hereditary areflexic dystasia Charcot-Marie-Tooth Disease Polyneuropathies Neurodegenerative Diseases Nerve Compression Syndromes Tomaculous neuropathy Heredodegenerative Disorders, Nervous System |
Neuromuscular Diseases Genetic Diseases, Inborn Peripheral Nervous System Diseases Hereditary Motor and Sensory Neuropathies Stomatognathic Diseases Congenital Abnormalities Ascorbic Acid Charcot Marie Tooth disease Charcot-Marie-Tooth disease, Type 1A |
Antioxidants Molecular Mechanisms of Pharmacological Action Growth Substances Vitamins Physiological Effects of Drugs |
Nervous System Diseases Micronutrients Protective Agents Pharmacologic Actions |