Criteria about the Vitamin C Trials in the USA

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(Last Updated 7/18/2007)

Neuromuscular Trial/Study

DISEASE CLASSIFICATION(S):

Charcot-Marie-Tooth Disease(CMT)

NAME OF CLINICAL TRIAL/STUDY:

High-Dose Ascorbic Acid Treatment of CMT1A

http://www.mda.org/research/view_ctrial.aspx?id=186

PURPOSE AND RATIONALE:

Charcot-Marie-Tooth disease (CMT) is among the most frequent of

genetic disorders, affecting approximately one in 2,500 people. The

most frequent genetic form of CMT is CMT1A. CMT1A is caused by a

duplication of chromosome 17 in the region containing the PMP22

gene.

Most people with CMT1A experience symptom onset in childhood or

early adulthood, have distal (furthest from the center of the body)

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 levels in nerve fibers in skin (obtained

from skin biopsies).

STUDY DETAILS:

This is a randomized, placebo-controlled, double-blind study,

meaning that participants are randomly assigned to receive ascorbic

acid or a placebo (inert substance), and that neither participants

nor investigators will know who is getting which substance until the

trial is over. Each participant will be in the trial for about two

years (25 months).

Participants will undergo testing using the CMT Neuropathy Scale at

the start of the trial and every six months thereafter. The CMT

Neuropathy Scale involves measurements of strength and movement in

the arms and legs, as well as assessments of sensitivity to pin

prick and vibration, and nerve conduction studies.

Participants will undergo skin biopsies for nerve fiber examination

at study entry and at month 24.

The study is funded in part by MDA.

OPENING DATES:

April 2007

CLOSING DATES:

December 2010

TARGET NUMBER OF PARTICIPANTS:

120

RECRUITMENT STATUS:

Open

ELIGIBILITY REQUIREMENTS:

Inclusion Criteria

Participants must

have a diagnosis of CMT1A, defined by the duplicaton on chromosome

17; or, must have a first- or second-degree relative with a

documented duplication and slowing of conduction in the median or

ulnar nerve to between 16 and 30 meters per second

be between 13 and 70 years old

if 18 or older, sign the informed consent document and agree to

follow the stipulations of the trial procedures (protocol)

if younger than 18, have his or her parents or guardians sign the

informed consent document and agree to follow the stipulations of

the trial procedures (protocol); and sign a written " assent " form

himself or herself

Exclusion Criteria

Participants must not

have a known neuropathy (nerve disorder) in addition to CMT1A (for

example, a neuropathy from diabetes or drugs or alcohol)

have ever received vincristine (a cancer medication)

have a known allergy to ascorbic acid

have had kidney stones

have a disorder called G6PD deficit

have a disorder called hemochromatosis

have a serious illness or medical condition that is not stabilized

or that could require hospitalization

have a high ascorbic acid level at screening

be pregnant or nursing

be in another clinical trial or be within a " washout " period of a

previous clinical trial

be taking neurotoxic (nerve-damaging) medications

be unlikely to comply with the study procedures (protocol) or be

unsuitable for any other reason, in the opinion of the investigator

CONTACT INFORMATION:

US LOCATIONS

land

Lora Clawson

Study Coordinator

s Hopkins University

Neurology

Baltimore, MD

United States

Phone: (410) 624-4346

lclawson@...

Michigan

Rowe

Study Coordinator

Wayne State University

Neurology

Detroit, MI

United States

Phone: (313) 577-1689

lrowe@...

New York

Patty

Study Coordinator

University of Rochester Medical Center

Neurology

Rochester, NY

United States

Phone: (585) 275-0581

Patty_@...