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

Descriptive Information
Brief Title † Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
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.
Brief 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.
Study Phase Phase 2
Study Type † Interventional
Study Design † Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary Outcome Measure † Safety and tolerability of PXT3003
Secondary Outcome Measure † To obtain preliminary data on the efficacy of PXT3003 on clinical scores and functional tests
Condition † Charcot-Marie-Tooth Disease Hereditary Neuropathy With Liability to Pressure Palsies Genetic Disorders Tooth Disorders
Intervention † DrugPXT3003
Study Arms / Comparison Groups PXT3003 Low dose Oral Liquid formulation, 1/100, bid, 12 months PXT3003 Intermediate dose Oral Liquid formulation, 1/50, bid, 12 months PXT3003 High dose Oral Liquid formulation, 1/10, bid, 12 months Placebo Oral Liquid formulation, bid, 12 months
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 80
Start Date † December 2010
Completion Date December 2012
Primary Completion Date October 2011
Eligibility Criteria † 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 (
Gender Both
Ages 18 Years - 65 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † France
Administrative Information
NCT ID † NCT01401257
Organization ID CLN-PXT3003-01
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Pharnext, SAS
Collaborators ††
Investigators † Principal Investigator: Shahram ATTARIAN, MD, Hôpital La Timone
Information Provided By
Verification Date July 2011
First Received Date † July 20, 2011
Last Updated Date September 11, 2013
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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