Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A (PLEO-CMT-FU)


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 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. September 18th 2017, the PXT3003 dose 2 was prematurely discontinued by the Sponsor. 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 up to 24 months, whereas patients initially randomized to inactive treatment will have used PXT3003 for up to 9 months. Visit for more details.


  • Drug: PXT3003

For study details, please contact Jennifer Steshyn:


  • 16 to 67 years old
  • Patients previously randomized to study CLN-PXT3003-02 under placebo and dose 1 and having completed 15 months of double-blind treatment in that study, including all procedures required at the Study Termination visit (V6) or
  • Patients previously randomized to the initial study CLN-PXT3003-02 under dose 2, prematurely discontinued following sponsor decision, and having performed all procedures required at the Study Termination visit (V6)
  • Patients whose V6 was performed within 4 weeks before entering the extension study or if not done must have a new baseline visit (VB)
  • Female patients must agree to continue using an approved method of birth control throughout the extension study
  • Patients must sign a written informed consent, specific to the extension study, in order to participate in this study. In case of minor children aged 16 to 18 years, both parent' and children's consents should be collected

Additional criteria apply, please contact Jennifer Steshyn:


18 and under
18 to 65



Can be done from home



Charcot-Marie Tooth, Charcot-Marie-Tooth disease, Neurology

Principal Investigator

S.H. Subramony, MD



Contact Information


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