High Dose Ascorbic Acid Treatment of CMT1A

• Status: Completed
• Trial end date: 2012-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Wayne State University
• Collaborators: Charcot-Marie-Tooth Association; Muscular Dystrophy Association
• Treatments: Ascorbic Acid; Vitamins

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.