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Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A

Charcot-Marie-Tooth Disease Clinical Trial

Official title:
A Phase II, Randomized, Placebo-controlled Trial of the Safety, Efficacy, Pharmacodynamics and Pharmacokinetics of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A.

Clinical Trial Summary

The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.

Clinical Trial Description

In addition to the safety and tolerability of the treatment, clinical, electrophysiological and biological endpoints (PMP22 mRNA, skin biopsy histology and plasma biomarkers) will be assessed. Standard laboratory tests and drug plasma concentrations will also be measured. Because of the slow progression of the disease and the nature of the observed symptoms, a minimum duration of 12 months of treatment is required in order to observe a potential improvement in any of the efficacy parameters. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01401257
Study type Interventional
Source Pharnext SA
Contact
Status Completed
Phase Phase 2
Start date December 2010
Completion date December 2012
View Details
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