High Dose Ascorbic Acid Treatment of CMT1A

NCT00484510 | Completed Phase 2 DMC

• 2 other identifiers: HIC074406MP2FMDA4193
• Study Type: interventional
• Target: 110
• Locations: 1 country
• Sites: 3

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.

Conditions

Charcot-Marie-Tooth Disease, Type Ia

Keywords

Ascorbic Acid; Vitamin C; Charcot Marie Tooth; CMT; CMT1a

Outcome Measures

Primary Outcomes (1)

• Mean change in the CMT Neuropathy Scale following high dose ascorbic acid ingestion, assessed at baseline and every 6 months throughout the trial.

  25 months per subject from baseline to completion.

Secondary Outcomes (1)

• Evaluation of PMP22 mRNA levels of myelinated peripheral nerve fibers.

  Baseline and Month 24.

Study Arms (2)

Ascorbic Acid

EXPERIMENTAL

Drug: Ascorbic acid (Vitamin C)

Placebo

PLACEBO COMPARATOR

Drug: placebo

Interventions

Ascorbic acid (Vitamin C) DRUG

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).

Ascorbic Acid

placebo DRUG

Eight 500 mg capsules/day of placebo. Subjects will take four (4)capsules each morning and four (4) capsules each evening for 24 months.

Placebo

Eligibility Criteria

• Age: 13 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Wayne State University: lead
• Muscular Dystrophy Association: collaborator
• Charcot-Marie-Tooth Association: collaborator

Study Sites (3)

Johns Hopkins University, Dept of Neurology

Baltimore, Maryland, 21287, United States

Location

Wayne State University, Dept of Neurology

Detroit, Michigan, 48201, United States

Location

University of Rochester Medical Center, Dept of Neurology

Rochester, New York, 14642, United States

Location

Related Publications (1)

• 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.

  PMID: 23797954 DERIVED

MeSH Terms

Conditions

Charcot-Marie-Tooth Disease

Interventions

Ascorbic Acid

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Sugar Acids; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Hydroxy Acids; Carbohydrates

Study Officials

• Richard A Lewis, MD

  Wayne State University, Dept. of Neurology

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: June 8, 2007
• First Posted: June 11, 2007
• Study Start: April 1, 2007
• Primary Completion: December 1, 2012
• Study Completion: December 1, 2012
• Last Updated: March 6, 2013

Record last verified: 2013-03

Locations

US (3)