Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT) (NCT02579759) | Clinical Trial Compass
CompletedPhase 3
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)
United States323 participantsStarted 2015-12
Plain-language summary
The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This double-blind study will assess in parallel groups 2 doses of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.
Who can participate
Age range16 Years – 65 Years
SexALL
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Inclusion Criteria:
* Male or female, aged from 16 to 65 years;
* Patient with a proven genetic diagnosis of CMT1A;
* Mild-to-moderate severity assessed by Charcot-Marie-Tooth Neuropathy Score (version 2) with a score \>2 and ≤18;
* Muscle weakness in at least foot dorsiflexion;
* Motor nerve conduction of the ulnar nerve of at least 15 m/sec;
* 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:
* Any other associated cause of peripheral neuropathy such as diabetes;
* Patient with another significant neurological disease or a concomitant major systemic disease;
* Clinically significant history of unstable medical illness since the last 30 days (unstable angina, cancer…) that may jeopardize the participation in the study;
* Significant hematologic disease, hepatitis or liver failure, renal failure;
* Limb surgery within six months before randomization or planned before trial completion;
* Clinically significant abnormalities on the pre-study laboratory evaluation, physical evaluation, electrocardiogram (ECG);
* Elevated ASAT/ALAT (\> 3 x ULN) and elevated serum creatinine levels (\> 1.25 x ULN);
* History of recent alcohol or drug abuse or non-adherence with treatment or other experimental protocols;
* Patient using unauthorized concomitant treatments including but not limited to baclofen, naltrexone, sorbitol (pharmaceutical form), opioids, levothyroxin and…
What they're measuring
1
Overall Neuropathy Limitation Scale (ONLS) Total Score