<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03023540</url>
  </required_header>
  <id_info>
    <org_study_id>CLN-PXT3003-03</org_study_id>
    <secondary_id>2015-002379-81</secondary_id>
    <nct_id>NCT03023540</nct_id>
  </id_info>
  <brief_title>Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A</brief_title>
  <acronym>PLEO-CMT-FU</acronym>
  <official_title>International, Multi-center, Open Label, Follow-up Extension Study Assessing the Long-term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Pharnext SA</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>SynteractHCR</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
    <collaborator>
      <agency>Syneos Health</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Premier Research Group plc</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Pharnext SA</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary&#xD;
      study CLN-PXT3003-02, i.e. treatment with PXT3003 or placebo, are eligible to continue in the&#xD;
      extension study CLN-PXT3003-03.&#xD;
&#xD;
      Period 1: Patients randomised to PXT3003 dose 1 or placebo in the primary study&#xD;
      (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 1 (5 mL). Patients&#xD;
      randomised to PXT3003 dose 2 (5 mL) in the primary study (CLN-PXT3003-02) continued in the&#xD;
      extension study on PXT3003 dose 2 or PXT3003 twice dose 1 (2x5 mL).&#xD;
&#xD;
      Period 2: All patients continue on twice dose 1 (2X5mL).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      PXT3003 is a rational design, fixed combination of low-dose (RS) baclofen, naltrexone&#xD;
      hydrochloride and D-sorbitol. The use of PXT3003 in a multicenter, randomised, placebo&#xD;
      controlled phase II study (CLN-PXT3003-01) was well-tolerated and safe in patients with CMT1A&#xD;
      for the three dose-levels investigated (Attarian et al., 2014). The intermediate and high&#xD;
      dose of PXT3003 demonstrated an improvement of disability in this patient population.&#xD;
&#xD;
      Subsequently a multicenter, randomised, placebo controlled phase III study (CLN-PXT3003-02)&#xD;
      to assess the efficacy and safety of PXT3003 in the treatment of patients with CMT1A was&#xD;
      initiated in December 2015. In March 2017 the first patients completed the 15-month treatment&#xD;
      with PXT3003 and rolled over into the extension study CLN-PXT3003-03.&#xD;
&#xD;
      During Period 1 (9 months), patients that were randomised to PXT3003 dose 1 or placebo in the&#xD;
      primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 1 (5 mL).&#xD;
      Patients randomised to PXT3003 dose 2 (5 mL) in the primary study (CLN-PXT3003-02) continued&#xD;
      in the extension study on on PXT3003 dose 2 or PXT3003 twice dose 1 (2x5 mL). During Period&#xD;
      2, all patients continue on twice dose 1 (2X5mL).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">March 7, 2017</start_date>
  <completion_date type="Anticipated">December 31, 2024</completion_date>
  <primary_completion_date type="Anticipated">December 31, 2024</primary_completion_date>
  <phase>Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <intervention_model_description>Patients who completed the pivotal phase III study CLN-PXT3003-02 are allowed to continue in this open-label study. In Period 1, patients who were randomly assigned to placebo or low-dose PXT3003 in pivotal phase III study were assigned to receive low-dose PXT3003 (5 mL), whereas patients assigned to high-dose PXT3003 continued on that dose (receiving the high-dose PXT3003 (5 mL) or twice the low-dose for each administration (i.e. 10 mL)). The primary objective was to assess safety and tolerability for prolonged exposure to PXT3003, and to evaluate the effect on disability in patients with mild to moderate CMT1A.&#xD;
In Period 2, all patients are allowed to continue in an open-label fashion to receive high-dose PXT3003 (receiving twice the low-dose for each administration (i.e. 10 mL). The objective is to mainly assess the safety and tolerability in the aforementioned patient population.</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Incidence of treatment-emergent adverse events (TEAEs) related to PXT3003 during the follow-up in patients with CMT1A</measure>
    <time_frame>9 or 24 months</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Incidence of all TEAEs and their evaluation of type/nature, severity/intensity, seriousness, duration, relationship to study drug, and outcome</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of adverse events leading to withdrawal of study drug</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Overall Neuropathy Limitation Scale (ONLS) score, and its arm and leg sub-items</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Charcot-Marie-Tooth Neuropathy Score - version 2 (CMTNS-V2), and its sub-items</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Nine-hole Peg Test (9-HPT)</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Quantified Muscular Testing (QMT) by hand grip and foot dorsiflexion dynamometry (mean of both sides)</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to walk 10 meters</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Compound Muscle Action Potential (CMAP) on ulnar nerve</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Sensory Nerve Action Potential (SNAP) on radial nerve</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Nerve conduction velocity (NCV)</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Quality of Life (EQ-5D)</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Visual analog scale on self-assessment of individualized main impairment in daily activities (defined at baseline with the patient)</measure>
    <time_frame>9 or 24 months</time_frame>
  </secondary_outcome>
  <other_outcome>
    <measure>Through plasma concentration of PXT3003</measure>
    <time_frame>at month 6 and 9</time_frame>
    <description>Measurement of baclofen, naltrexone and 6-beta-naltrexone plasma concentration at the through</description>
  </other_outcome>
  <other_outcome>
    <measure>Peak plasma concentration of PXT3003</measure>
    <time_frame>at month 6 and 9</time_frame>
    <description>Measurement of baclofen, naltrexone and 6-beta-naltrexone plasma concentration at the through</description>
  </other_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">187</enrollment>
  <condition>Charcot-Marie-Tooth Disease, Type IA</condition>
  <arm_group>
    <arm_group_label>PXT3003 dose 1</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Period 1, PXT3003 : Liquid oral solution (0.6 mg/mL baclofen, 0.07 mg/mL naltrexone HCl and 210 mg/mL D-sorbitol), 5 mL bid (taken morning and evening with food) for 9 consecutive months</description>
  </arm_group>
  <arm_group>
    <arm_group_label>PXT3003 dose 2</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Period 1, PXT3003: Liquid oral solution (1.2 mg/mL baclofen, 0.14 mg/mL naltrexone HCl and 420 mg/mL D-sorbitol), 5 mL bid (taken morning and evening with food) for 9 consecutive months&#xD;
Period 2, PXT3003: Liquid oral solution (0.6 mg/mL baclofen, 0.07 mg/mL naltrexone HCl and 210 mg/mL D-sorbitol), 10 mL bid (taken morning and evening with food)</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>PXT3003</intervention_name>
    <description>Liquid oral solution, 5 mL (Dose 1 or Dose 2) bid OR 10 mL (Dose 1) bid</description>
    <arm_group_label>PXT3003 dose 1</arm_group_label>
    <arm_group_label>PXT3003 dose 2</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria after September 18th 2017:&#xD;
&#xD;
          -  Patients previously randomized to study CLN-PXT3003-02 under placebo and dose 1 and&#xD;
             having completed 15 months of double-blind treatment in that study, including all&#xD;
             procedures required at the Study Termination visit (V6) or&#xD;
&#xD;
          -  Patients previously randomized to the initial study CLN-PXT3003-02 under dose 2,&#xD;
             prematurely discontinued following sponsor decision, and having performed all&#xD;
             procedures required at the Study Termination visit (V6)&#xD;
&#xD;
          -  Patients whose V6 was performed within 4 weeks before entering the extension study or&#xD;
             if not done must have a new baseline visit (VB)&#xD;
&#xD;
          -  Female patients must agree to continue using an approved method of birth control&#xD;
             throughout the extension study&#xD;
&#xD;
          -  Patients must sign a written informed consent, specific to the extension study, in&#xD;
             order to participate in this study. In case of minor children aged 16 to 18 years,&#xD;
             both parent' and children's consents should be collected&#xD;
&#xD;
        Inclusion Criteria until September 18th 2017:&#xD;
&#xD;
          -  Patients must have completed 15 months of double-blind treatment in the primary study&#xD;
             CLN-PXT3003-02, including all procedures required at the Study Termination visit (V6)&#xD;
&#xD;
          -  Female patients must agree to continue using an approved method of birth control&#xD;
             throughout the extension study&#xD;
&#xD;
          -  Patients must sign a written informed consent, specific to the extension study, in&#xD;
             order to participate in this study. In case of minor children aged 16 to 18 years,&#xD;
             both parent' and children's consents should be collected&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Any clinically significant change in health status that, in the opinion of the&#xD;
             Investigator, would prevent the subject from participating in this study or&#xD;
             successfully completing this study&#xD;
&#xD;
          -  Any unauthorized concomitant treatments, as study CLN-PXT3003-02 (e.g. including but&#xD;
             not limited to baclofen, naltrexone,sorbitol (pharmaceutical form), opioids,&#xD;
             levothyroxin, and potentially neurotoxic drugs such as amiodarone, chloroquine, cancer&#xD;
             drugs susceptible to induce peripheral neuropathy)&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>16 Years</minimum_age>
    <maximum_age>67 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Shahram Attarian, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>CHU la Timone, Marseille, France</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Teresa Sevilla, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Hospital Universitario i Politécnico La F, Valencia, Spain</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Marianne de Visser, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Academic Medical Center, Amsterdam, Netherlands</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Mark Roberts, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Selor Royal NHS Foundation Trust, Manchester, UK</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Florian Thomas, MD PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Seton Hall-Hackensack-Meridian School of Medicine, Hackensack, USA</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Department of Neurology, Cedars-Sinai Medical Center</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90048</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Department of Neurology, McKnight Brain Institute</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32610</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Kansas Medical Center</name>
      <address>
        <city>Kansas City</city>
        <state>Kansas</state>
        <zip>66160</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Brigham and Women's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Michigan Health System</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109-5322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Department of Neurology, University of Minnesota</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55455</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Department of Neurology and Psichiatry, Saint Louis University</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63104-1027</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Peripheral Neuropathy Center, Neurological Institue Building, Columbia University Medical Center</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10032</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint Luke's Rehabilitation Institute</name>
      <address>
        <city>Spokane</city>
        <state>Washington</state>
        <zip>99202-1330</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Departement of Neurology, UZ Leuven</name>
      <address>
        <city>Leuven</city>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital of Quebec</name>
      <address>
        <city>Quebec</city>
        <zip>G1J 1Z4</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cntre de Reference des Maladies Neuromusculaires, Hopital Swynghedauwl, CHU Lille</name>
      <address>
        <city>Lille</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre de Reference des Neuropathies Peripheriques Rare, Hopital Dupuytren, CHU Limoges</name>
      <address>
        <city>Limoges</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Service de Neurologie et du Sommeil, CHU Lyon Sud</name>
      <address>
        <city>Lyon</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre de Reference des Maladie Neuromusculaires, CHU la Timone</name>
      <address>
        <city>Marseille</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre de Reference des Maladie Neuromusculaires, Hotel Dieu, CHU de Nantes</name>
      <address>
        <city>Nantes</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Service de Neurologie, Hopital Kremlin Bicetre</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Departement of Neurology, Academic Medical Center</name>
      <address>
        <city>Amsterdam</city>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Department of neurology, Hospital Univesitario de Bellvitge</name>
      <address>
        <city>Barcelona</city>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Servicio de Neurologia, Hospital Universitario La Paz</name>
      <address>
        <city>Madrid</city>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centro de Diagnostico y Tratamiento, Hospital Universitario Virgen del Rocio</name>
      <address>
        <city>Sevilla</city>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Servicio de Neurologia, Hospital Universitario i Politécnic La Fe</name>
      <address>
        <city>Valencia</city>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Department of Neurology, Salford Royal NHS Foundation Trust</name>
      <address>
        <city>Salford</city>
        <state>Manchester</state>
        <zip>M6 8HD</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Belgium</country>
    <country>Canada</country>
    <country>France</country>
    <country>Netherlands</country>
    <country>Spain</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <removed_countries>
    <country>Germany</country>
  </removed_countries>
  <verification_date>March 2021</verification_date>
  <study_first_submitted>December 26, 2016</study_first_submitted>
  <study_first_submitted_qc>January 13, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 18, 2017</study_first_posted>
  <last_update_submitted>March 9, 2021</last_update_submitted>
  <last_update_submitted_qc>March 9, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">March 10, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Charcot Marie Tooth Type 1A</keyword>
  <keyword>Peripheral neuropathy</keyword>
  <keyword>PXT3003</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Tooth Diseases</mesh_term>
    <mesh_term>Charcot-Marie-Tooth Disease</mesh_term>
    <mesh_term>Nerve Compression Syndromes</mesh_term>
    <mesh_term>Hereditary Sensory and Motor Neuropathy</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03023540
org study id: CLN-PXT3003-03
secondary id: 2015-002379-81
nct id: NCT03023540
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary
      study CLN-PXT3003-02, i.e. treatment with PXT3003 or placebo, are eligible to continue in the
      extension study CLN-PXT3003-03.

      Period 1: Patients randomised to PXT3003 dose 1 or placebo in the primary study
      (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 1 (5 mL). Patients
      randomised to PXT3003 dose 2 (5 mL) in the primary study (CLN-PXT3003-02) continued in the
      extension study on PXT3003 dose 2 or PXT3003 twice dose 1 (2x5 mL).

      Period 2: All patients continue on twice dose 1 (2X5mL).
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 completed the 15-month treatment
      with PXT3003 and rolled over into the extension study CLN-PXT3003-03.

      During Period 1 (9 months), patients that were randomised to PXT3003 dose 1 or placebo in the
      primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 1 (5 mL).
      Patients randomised to PXT3003 dose 2 (5 mL) in the primary study (CLN-PXT3003-02) continued
      in the extension study on on PXT3003 dose 2 or PXT3003 twice dose 1 (2x5 mL). During Period
      2, all patients continue on twice dose 1 (2X5mL).
allocation: Non-Randomized
intervention model: Parallel Assignment
intervention model description: Patients who completed the pivotal phase III study CLN-PXT3003-02 are allowed to continue in this open-label study. In Period 1, patients who were randomly assigned to placebo or low-dose PXT3003 in pivotal phase III study were assigned to receive low-dose PXT3003 (5 mL), whereas patients assigned to high-dose PXT3003 continued on that dose (receiving the high-dose PXT3003 (5 mL) or twice the low-dose for each administration (i.e. 10 mL)). The primary objective was to assess safety and tolerability for prolonged exposure to PXT3003, and to evaluate the effect on disability in patients with mild to moderate CMT1A.
In Period 2, all patients are allowed to continue in an open-label fashion to receive high-dose PXT3003 (receiving twice the low-dose for each administration (i.e. 10 mL). The objective is to mainly assess the safety and tolerability in the aforementioned patient population.
primary purpose: Treatment
masking: None (Open Label)
measure: Incidence of treatment-emergent adverse events (TEAEs) related to PXT3003 during the follow-up in patients with CMT1A
time frame: 9 or 24 months
measure: 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
measure: Incidence of adverse events leading to withdrawal of study drug
time frame: 9 or 24 months
measure: Overall Neuropathy Limitation Scale (ONLS) score, and its arm and leg sub-items
time frame: 9 or 24 months
measure: Charcot-Marie-Tooth Neuropathy Score - version 2 (CMTNS-V2), and its sub-items
time frame: 9 or 24 months
measure: Nine-hole Peg Test (9-HPT)
time frame: 9 or 24 months
measure: Quantified Muscular Testing (QMT) by hand grip and foot dorsiflexion dynamometry (mean of both sides)
time frame: 9 or 24 months
measure: Time to walk 10 meters
time frame: 9 or 24 months
measure: Compound Muscle Action Potential (CMAP) on ulnar nerve
time frame: 9 or 24 months
measure: Sensory Nerve Action Potential (SNAP) on radial nerve
time frame: 9 or 24 months
measure: Nerve conduction velocity (NCV)
time frame: 9 or 24 months
measure: Quality of Life (EQ-5D)
time frame: 9 or 24 months
measure: 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
measure: Through plasma concentration of PXT3003
time frame: at month 6 and 9
description: Measurement of baclofen, naltrexone and 6-beta-naltrexone plasma concentration at the through
measure: Peak plasma concentration of PXT3003
time frame: at month 6 and 9
description: Measurement of baclofen, naltrexone and 6-beta-naltrexone plasma concentration at the through
arm group label: PXT3003 dose 1
arm group type: Active Comparator
description: Period 1, PXT3003 : Liquid oral solution (0.6 mg/mL baclofen, 0.07 mg/mL naltrexone HCl and 210 mg/mL D-sorbitol), 5 mL bid (taken morning and evening with food) for 9 consecutive months
arm group label: PXT3003 dose 2
arm group type: Active Comparator
description: Period 1, PXT3003: Liquid oral solution (1.2 mg/mL baclofen, 0.14 mg/mL naltrexone HCl and 420 mg/mL D-sorbitol), 5 mL bid (taken morning and evening with food) for 9 consecutive months
Period 2, PXT3003: Liquid oral solution (0.6 mg/mL baclofen, 0.07 mg/mL naltrexone HCl and 210 mg/mL D-sorbitol), 10 mL bid (taken morning and evening with food)
intervention type: Drug
intervention name: PXT3003
description: Liquid oral solution, 5 mL (Dose 1 or Dose 2) bid OR 10 mL (Dose 1) bid
arm group label: PXT3003 dose 1
arm group label: PXT3003 dose 2
criteria: 
gender: All
minimum age: 16 Years
maximum age: 67 Years
healthy volunteers: No
last name: Shahram Attarian, MD
role: Principal Investigator
affiliation: CHU la Timone, Marseille, France
last name: Teresa Sevilla, MD
role: Principal Investigator
affiliation: Hospital Universitario i Politécnico La F, Valencia, Spain
last name: Marianne de Visser, MD
role: Principal Investigator
affiliation: Academic Medical Center, Amsterdam, Netherlands
last name: Mark Roberts, MD
role: Principal Investigator
affiliation: Selor Royal NHS Foundation Trust, Manchester, UK
last name: Florian Thomas, MD PhD
role: Principal Investigator
affiliation: Seton Hall-Hackensack-Meridian School of Medicine, Hackensack, USA
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country: Belgium
country: Canada
country: France
country: Netherlands
country: Spain
country: United Kingdom
country: United States
country: Germany
responsible party type: Sponsor
mesh term: Tooth Diseases
mesh term: Charcot-Marie-Tooth Disease
mesh term: Nerve Compression Syndromes
mesh term: Hereditary Sensory and Motor Neuropathy
sharing ipd: No