Trial record 9 of 294 for:
"Charcot-Marie-Tooth disease"
High Dose Ascorbic Acid Treatment of CMT1A
This study has been completed.
Sponsor:
Wayne State University
Collaborators:
Muscular Dystrophy Association
Charcot-Marie-Tooth Association
Information provided by (Responsible Party):
Michael E. Shy, MD, Wayne State University
ClinicalTrials.gov Identifier:
NCT00484510
First received: June 8, 2007
Last updated: March 4, 2013
Last verified: March 2013
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Purpose
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 |
|---|---|---|
|
Charcot-Marie-Tooth Disease, Type Ia |
Drug: Ascorbic acid (Vitamin C) Drug: placebo |
Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator) Primary Purpose: Treatment |
| 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. |
Resource links provided by NLM:
Genetics Home Reference related topics:
Charcot-Marie-Tooth disease
hereditary neuropathy with liability to pressure palsies
U.S. FDA Resources
Further study details as provided by Wayne State University:
Primary Outcome Measures:
- 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. ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Evaluation of PMP22 mRNA levels of myelinated peripheral nerve fibers. [ Time Frame: Baseline and Month 24. ] [ Designated as safety issue: No ]
| Enrollment: | 110 |
| Study Start Date: | April 2007 |
| Study Completion Date: | December 2012 |
| Primary Completion Date: | December 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| 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 |
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.
|
Detailed Description:
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.
Eligibility| Ages Eligible for Study: | 13 Years to 70 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00484510
Locations
| 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 | |
Sponsors and Collaborators
Wayne State University
Muscular Dystrophy Association
Charcot-Marie-Tooth Association
Investigators
| Principal Investigator: | Richard A Lewis, MD | Wayne State University, Dept. of Neurology |
More Information
Additional Information:
No publications provided
| Responsible Party: | Michael E. Shy, MD, Professor, Wayne State University |
| ClinicalTrials.gov Identifier: | NCT00484510 History of Changes |
| Other Study ID Numbers: | HIC074406MP2F, MDA4193 |
| Study First Received: | June 8, 2007 |
| Last Updated: | March 4, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Wayne State University:
|
Ascorbic Acid Vitamin C Charcot Marie Tooth CMT CMT1a |
Additional relevant MeSH terms:
|
Charcot-Marie-Tooth Disease Nerve Compression Syndromes Hereditary Sensory and Motor Neuropathy Tooth Diseases Nervous System Malformations Nervous System Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Polyneuropathies Peripheral Nervous System Diseases Neuromuscular Diseases Congenital Abnormalities |
Genetic Diseases, Inborn Stomatognathic Diseases Ascorbic Acid Vitamins Antioxidants Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Protective Agents Physiological Effects of Drugs Micronutrients Growth Substances |
ClinicalTrials.gov processed this record on May 23, 2013