High Dose Ascorbic Acid Treatment of CMT1A
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|ClinicalTrials.gov Identifier: NCT00484510|
Recruitment Status : Completed
First Posted : June 11, 2007
Last Update Posted : March 6, 2013
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|Condition or disease||Intervention/treatment||Phase|
|Charcot-Marie-Tooth Disease, Type Ia||Drug: Ascorbic acid (Vitamin C) Drug: placebo||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||110 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|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.|
|Study Start Date :||April 2007|
|Actual Primary Completion Date :||December 2012|
|Actual Study Completion Date :||December 2012|
|Experimental: Ascorbic Acid||
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).
|Placebo Comparator: 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.
- Mean change in the CMT Neuropathy Scale following high dose ascorbic acid ingestion, assessed at baseline and every 6 months throughout the trial. [ Time Frame: 25 months per subject from baseline to completion. ]
- Evaluation of PMP22 mRNA levels of myelinated peripheral nerve fibers. [ Time Frame: Baseline and Month 24. ]
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|Ages Eligible for Study:||13 Years to 70 Years (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- The subject has CMT1A, defined by the duplication on chromosome 17p11.2 performed by either Pulse Field Gel Electrophoresis or Fluorescence In Situ Hybridization (FISH) by a CLIA certified laboratory, OR the subject has a first or second degree relative with a documented duplication performed by the above methods AND the subject has uniform motor conduction slowing of the median or ulnar nerve between 16 and 30 m/s.
- The subject is between 13 and 70 years of age.
- The subject, if 18 years or older, has signed the Informed Consent Form and agrees to follow the stipulations of the protocol.
- If the subject is less than 18, his or her parents or guardians have signed the Informed Consent Form and agree to follow the stipulations of the protocol. The subject has also signed a written assent form.
- A known neuropathy from another source (For example, diabetes, drug induced, alcohol, etc.)
- The subject has ever received Vincristine.
- The subject has a known allergy to ascorbic acid.
- The subject has ever had kidney stones.
- The subject has a known history of G6PD deficit.
- The subject has a history of hemochromatosis.
- The subject suffers from a serious illness or medical condition that is not stabilized or that could require hospitalization.
- The subject has a high ascorbic acid level at screening.
- The subject is pregnant or nursing.
- The subject, in the opinion of the investigator, is unlikely to comply with the study protocol or is unsuitable for any other reason.
- The subject participates to another clinical trial or is still within a washout period of a previous clinical trial.
- The subject is taking neurotoxic medications.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00484510
|United States, Maryland|
|Johns Hopkins University, Dept of Neurology|
|Baltimore, Maryland, United States, 21287|
|United States, Michigan|
|Wayne State University, Dept of Neurology|
|Detroit, Michigan, United States, 48201|
|United States, New York|
|University of Rochester Medical Center, Dept of Neurology|
|Rochester, New York, United States, 14642|
|Principal Investigator:||Richard A Lewis, MD||Wayne State University, Dept. of Neurology|
|Responsible Party:||Michael E. Shy, MD, Professor, Wayne State University|
|Other Study ID Numbers:||
|First Posted:||June 11, 2007 Key Record Dates|
|Last Update Posted:||March 6, 2013|
|Last Verified:||March 2013|
Charcot Marie Tooth
Nerve Compression Syndromes
Hereditary Sensory and Motor Neuropathy
Nervous System Malformations
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Peripheral Nervous System Diseases
Genetic Diseases, Inborn
Physiological Effects of Drugs
Molecular Mechanisms of Pharmacological Action