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

Descriptive Information
Brief Title † Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A
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 Disease Type 1A
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 Description 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.
Study Phase Phase 3
Study Type † Interventional
Study Design †
Primary Outcome Measure † Incidence of treatment-emergent adverse events (TEAEs) related to PXT3003 during the follow-up in patients with CMT1A
Secondary Outcome Measure † Incidence of all TEAEs and their evaluation of type/nature, severity/intensity, seriousness, duration, relationship to study drug, and outcome
Condition † Charcot-Marie-Tooth Disease, Type IA
Intervention † DrugPXT3003
Study Arms / Comparison Groups PXT3003 dose 1 PXT3003: Liquid oral solution, 5 mL bid (taken morning and evening with food) for 9 consecutive months PXT3003 dose 2 PXT3003: Liquid oral solution, 5 mL bid (taken morning and evening with food) for 9 consecutive 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 † 323
Start Date † March 7, 2017
Completion Date January 2019
Primary Completion Date January 2019
Eligibility Criteria † Inclusion Criteria: - Patients must have completed 15 months of double-blind treatment in the primary study CLN-PXT3003-02, including all procedures required at the Study Termination visit (V6) - 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 Exclusion Criteria: - Any clinically significant change in health status that, in the opinion of the Investigator, would prevent the subject from participating in this study or successfully completing this study - Any unauthorized concomitant treatments, as study CLN-PXT3003-02 (e.g. including but not limited to baclofen, naltrexone,sorbitol (pharmaceutical form), opioids, levothyroxin, and potentially neurotoxic drugs such as amiodarone, chloroquine, cancer drugs susceptible to induce peripheral neuropathy)
Gender All
Ages 16 Years - 67 Years
Accepts Healthy Volunteers No
Contacts †† Rene Goedkoop, MD, +33 1 41 09 22 30, rgoedkoop@pharnext.com
Location Countries † Belgium
Administrative Information
NCT ID † NCT03023540
Organization ID CLN-PXT3003-03
Secondary IDs †† 2015-002379-81
Responsible Party Sponsor
Study Sponsor † Pharnext SA
Collaborators †† SynteractHCR
Investigators † Principal Investigator: Shahram Attarian, MD, CHU la Timone, Marseille, France
Information Provided By
Verification Date April 2017
First Received Date † December 26, 2016
Last Updated Date April 13, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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