Clinical Trial: Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: International, Multi-center, Double Blind 9-month Follow-up Extension Study Assessing the Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth

Brief Summary:

All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary study CLN-PXT3003-02, i.e. 15-month double-blind treatment with PXT3003 or placebo, will be eligible to continue in the extension study CLN-PXT3003-03.

Patients randomised to PXT3003 dose 1 or 2 in the primary study (CLN-PXT3003-02) will continue in the extension study at the same dose, while the patients who received placebo will be assigned to one of the two active doses by a second randomization at the entry in the extension study.


Detailed Summary:

PXT3003 is a rational design, fixed combination of low-dose (RS) baclofen, naltrexone hydrochloride and D-sorbitol. The use of PXT3003 in a multicenter, randomised, placebo controlled phase II study (CLN-PXT3003-01) was well-tolerated and safe in patients with CMT1A for the three dose-levels investigated (Attarian et al., 2014). The intermediate and high dose of PXT3003 demonstrated an improvement of disability in this patient population.

Subsequently a a multicenter, randomised, placebo controlled phase III study (CLN-PXT3003-02) to assess the efficacy and safety of PXT3003 in the treatment of patients with CMT1A was initiated in December 2015. In March 2017 the first patients will have completed the 15-month treatment with PXT3003. Thereafter, patients will be allowed for entry in this extension study (CLN-PXT3003-03) for a 9-month treatment with PXT3003. Thus patients initially randomised to active treatment will have used PXT3003 for 24 months, whereas patients initially randomized to inactive treatment will have used PXT3003 for 9 months.


Sponsor: Pharnext SA

Current Primary Outcome: Incidence of treatment-emergent adverse events (TEAEs) related to PXT3003 during the follow-up in patients with CMT1A [ Time Frame: 9 or 24 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Incidence of all TEAEs and their evaluation of type/nature, severity/intensity, seriousness, duration, relationship to study drug, and outcome [ Time Frame: 9 or 24 months ]
  • Incidence of adverse events leading to withdrawal of study drug [ Time Frame: 9 or 24 months ]
  • Overall Neuropathy Limitation Scale (ONLS) score, and its arm and leg sub-items [ Time Frame: 9 or 24 months ]
  • Charcot-Marie-Tooth Neuropathy Score - version 2 (CMTNS-V2), and its sub-items [ Time Frame: 9 or 24 months ]
  • Nine-hole Peg Test (9-HPT) [ Time Frame: 9 or 24 months ]
  • Quantified Muscular Testing (QMT) by hand grip and foot dorsiflexion dynamometry (mean of both sides) [ Time Frame: 9 or 24 months ]
  • Time to walk 10 meters [ Time Frame: 9 or 24 months ]
  • Compound Muscle Action Potential (CMAP) on ulnar nerve [ Time Frame: 9 or 24 months ]
  • Sensory Nerve Action Potential (SNAP) on radial nerve [ Time Frame: 9 or 24 months ]
  • Nerve conduction velocity (NCV) [ Time Frame: 9 or 24 months ]
  • Quality of Life [ Time Frame: 9 or 24 months ]
    EuroQol 5-Dimensional Health-related Quality of Life scale (EQ-5D
  • Visual analog scale on self-assessment of individualized main impairment in daily activities (defined at baseline with the patient) [ Time Frame: 9 or 24 months ]


Original Secondary Outcome: Same as current

Information By: Pharnext SA

Dates:
Date Received: December 26, 2016
Date Started: March 7, 2017
Date Completion: January 2019
Last Updated: April 13, 2017
Last Verified: April 2017