Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

High Dose Ascorbic Acid Treatment of CMT1A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00484510
Recruitment Status : Completed
First Posted : June 11, 2007
Last Update Posted : March 6, 2013
Sponsor:
Collaborators:
Muscular Dystrophy Association
Charcot-Marie-Tooth Association
Information provided by (Responsible Party):
Michael E. Shy, MD, Wayne State University

Tracking Information
First Submitted Date  ICMJE June 8, 2007
First Posted Date  ICMJE June 11, 2007
Last Update Posted Date March 6, 2013
Study Start Date  ICMJE April 2007
Actual Primary Completion Date December 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 8, 2007)
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. ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 8, 2007)
Evaluation of PMP22 mRNA levels of myelinated peripheral nerve fibers. [ Time Frame: Baseline and Month 24. ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE High Dose Ascorbic Acid Treatment of CMT1A
Official Title  ICMJE A Randomized, Placebo-controlled, Double Masked 120 Subject "Futility Design" Clinical Trial of Ascorbic Acid Treatment of Charcot Marie Tooth Disease Type 1A.
Brief Summary 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.
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Charcot-Marie-Tooth Disease, Type Ia
Intervention  ICMJE
  • 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).
  • 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.
Study Arms  ICMJE
  • Experimental: Ascorbic Acid
    Intervention: Drug: Ascorbic acid (Vitamin C)
  • Placebo Comparator: Placebo
    Intervention: Drug: placebo
Publications * Lewis RA, McDermott MP, Herrmann DN, Hoke A, Clawson LL, Siskind C, Feely SM, Miller LJ, Barohn RJ, Smith P, Luebbe E, Wu X, Shy ME; Muscle Study Group. High-dosage ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A: results of a randomized, double-masked, controlled trial. JAMA Neurol. 2013 Aug;70(8):981-7. doi: 10.1001/jamaneurol.2013.3178.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 4, 2013)
110
Original Estimated Enrollment  ICMJE
 (submitted: June 8, 2007)
120
Actual Study Completion Date  ICMJE December 2012
Actual Primary Completion Date December 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 13 Years to 70 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00484510
Other Study ID Numbers  ICMJE HIC074406MP2F
MDA4193
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Michael E. Shy, MD, Wayne State University
Study Sponsor  ICMJE Wayne State University
Collaborators  ICMJE
  • Muscular Dystrophy Association
  • Charcot-Marie-Tooth Association
Investigators  ICMJE
Principal Investigator: Richard A Lewis, MD Wayne State University, Dept. of Neurology
PRS Account Wayne State University
Verification Date March 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP