Clinical Trial: Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: International, Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study Assessing in Parallel Groups the Efficacy and Safety of 2 Doses of PXT3003 in Patients With Analysis of Covariance on the summary mean of ONLS at 12 and 15 months adjusted for baseline ONLS values.
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Responder rate to PXT3003 therapy defined as a patients improving on ONLS at end of treatment [ Time Frame: 15 months of treatment ]
- Arm and leg sub-items of ONLS [ Time Frame: 12 and 15 months of treatment ]
- Charcot-Marie-Tooth Neuropathy Score version 2 (CMTNS-v2), including its sub-items [ Time Frame: 12 and 15 months of treatment ]
- Nine-hole Peg Test (9-HPT) performed on non-dominant hand [ Time Frame: 12 and 15 months of treatment ]
- Quantified Muscular Testing (QMT) by Hand grip and Foot dorsiflexion dynamometry (mean of both sides) [ Time Frame: 12 and 15 months of treatment ]
- Time to walk 10 meters [ Time Frame: 12 and 15 months of treatment ]
- Compound Muscle Amplitude Potential (CMAP) in ulnar or median nerves (non-dominant side) [ Time Frame: 12 and 15 months of treatment ]mV
- Sensory Nerve Potential Amplitude (SNAP) in radial nerve (non-dominant side) [ Time Frame: 12 and 15 months of treatment ]µV
- Nerve Conduction Velocity (non-dominant side) [ Time Frame: 12 and 15 months of treatment ]m/sec
- Quality of life measured by the EuroQol 5-Dimensional Health-related Quality of Life scale (EQ-5D-L) [ Time Frame: 12 and 15 months of treatment ]
- visual analog scale on self-assessment of the individualized main impairment in daily activities defined at baseline with the patient [ Time Frame: 12 and 15 months of treatment ]
- Safety and tolerability of PXT3003: assessment of some AE incidence and of the change in physical examination, vital signs, clinical laboratory variables, and ECGs. [ Time Frame: 15 months of treatment ]Incidence of Treatment Emergent Adverse Events (TEAE), of related TEAE with moderate or severe intensity, AE leading to withdrawal of study drug, and Serious Adverse Events,
Original Secondary Outcome: Same as current
Information By: Pharnext SA
Dates:
Date Received: September 28, 2015
Date Started: December 2015
Date Completion: March 2018
Last Updated: January 4, 2017
Last Verified: January 2017