Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A (NCT01401257) | Clinical Trial Compass
CompletedPhase 2
Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
France80 participantsStarted 2010-12
Plain-language summary
The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.
Who can participate
Age range18 Years – 65 Years
SexALL
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Inclusion Criteria:
* DNA proven CMT1A
* Muscle weakness in at least foot dorsiflexion (clinical assessment)
* Age between 18 and 65 years
* Male or non pregnant, non breastfeeding female
* CMT neuropathy score at screening ≤ 20
* Agrees to perform electrorophysiological studies and two cutaneous biopsies for determination of PMP22 expression and histology
* Providing signed written informed consent to participate in the study and willing and able to comply with all study procedures and scheduled visits
Exclusion Criteria:
* Patients with another neurological disease
* Patients using unauthorized concomitant treatments, ascorbic acid, opioids, levothyroxine and potentially neurotoxic drugs. Patients who can/agree to stop these medications 4 weeks before randomization can be included
* Patients who have participated in another trial of investigational drug within the past 30 days
* Concomitant major systemic disease
* Clinically significant history of unstable medical illness over the last 30 days (unstable angina…)
* History of significant hematologic, kidney, liver disease, or insulin-dependent diabetes
* Clinically significant abnormalities on the prestudy laboratory evaluation, physical evaluation, electrocardiogram (ECG)
* ASAT/ALAT levels above the upper limit of normal (ULN). However, patients with an isolated elevation of either ASAT or ALAT (\<1.5 ULN) can be included at investigators" discretion if the remaining liver function tests are normal and if ASAT or ALAT …