NCT01401257

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

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.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 80 (actual)
• Sponsor: Pharnext S.C.A. · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• Charcot-Marie-Tooth Disease
• Hereditary Neuropathy With Liability to Pressure Palsies
• Genetic Disorders

Interventions

Type	Name	Description
DRUG	PXT3003 Low dose	Liquid,5 ml, twice a day, 12-month treatment
DRUG	PXT3003 Intermediate Dose	Liquid,5 ml, twice a day, 12-month treatment
DRUG	PXT3003 High Dose	Liquid,5 ml, twice a day, 12-month treatment
OTHER	Placebo	Liquid,5 ml, twice a day, 12-month treatment

Timeline

• Start date: 2010-12-01
• Primary completion: 2011-10-01
• Completion: 2012-12-01
• First posted: 2011-07-25
• Last updated: 2017-11-22
• Results posted: 2017-02-16

Locations

6 sites across 1 country: France