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    Summary
    EudraCT Number:2015-002379-81
    Sponsor's Protocol Code Number:CLN-PXT3003-03
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2017-06-12
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2015-002379-81
    A.3Full title of the trial
    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.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An International, multi-center, FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.
    A.4.1Sponsor's protocol code numberCLN-PXT3003-03
    A.5.4Other Identifiers
    Name:US IND Number:122505
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPharnext SA
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPharnext SA
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPharnext SA
    B.5.2Functional name of contact pointAdrian Hepner
    B.5.3 Address:
    B.5.3.1Street Address46 rue Saint Lazare
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75009
    B.5.3.4CountryFrance
    B.5.4Telephone number+33(0)141092230
    B.5.6E-mailahepner@pharnext.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1260
    D.3 Description of the IMP
    D.3.2Product code PXT3003 Dose 1
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBACLOFEN
    D.3.9.1CAS number 1134-47-0
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameNA
    D.3.9.4EV Substance CodeSUB05667MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.6
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNALTREXONE
    D.3.9.1CAS number 16676-29-2
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameNALTREXONE HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB14629MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.07
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSorbitol
    D.3.9.1CAS number 50-70-4
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameD-SORBITOL
    D.3.9.4EV Substance CodeSUB20837
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number21
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1260
    D.3 Description of the IMP
    D.3.2Product code PXT3003 Dose 2 (equivalent to twice Dose 1)
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBACLOFEN
    D.3.9.1CAS number 1134-47-0
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameNA
    D.3.9.4EV Substance CodeSUB05667MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.2
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNALTREXONE
    D.3.9.1CAS number 16676-29-2
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameNALTREXONE HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB14629MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.14
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSorbitol
    D.3.9.1CAS number 50-70-4
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameD-SORBITOL
    D.3.9.4EV Substance CodeSUB20837
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number42
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Charcot-Marie-Tooth Disease type 1A
    E.1.1.1Medical condition in easily understood language
    Charcot-Marie-Tooth Disease type 1A
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10008414
    E.1.2Term Charcot-Marie-Tooth disease
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Period 1: The main objective of the study is to assess the long-term safety and tolerability of PXT3003 up to 2 years, and to increase the exposed population receiving PXT3003 for at least 9 months.

    Period 2: For patients continuing after V9, the main objective will be to offer patients the opportunity to access to twice Dose 1 equivalent to Dose 2 of PXT3003, with a regular safety follow-up.
    E.2.2Secondary objectives of the trial
    Period 1:
    Secondary objectives:
    - To gather up-to-2-year data to estimate the long-term effect of PXT3003 on clinical, functional and electrophysiological endpoints;
    - To compare the effect of PXT3003 in patients receiving PXT3003 active dose from the start of the primary study to patients assigned to a delayed start (i.e., who received placebo during the primary study) in order to determine if PXT3003 slows the progression of the disease
    - To assess the evolution of potential blood biomarkers and molecular changes in skin biopsy on the longer term.
    Period 2: For patients continuing after V9, sefty follow-up and ONLS score follow-up on a 6-month frequency.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    •Skin Biopsy (ancillary sub-study) for molecular biology will be done at the end of the study (9-month visit)
    •MRI measure (ancillary sub-study) of muscle/fat index in the leg (by MRI quantification of lower limb muscles and fatty muscle infiltration) will be performed at the end of the study (9-month visit).
    Period 2
    After V9, no sub-study
    E.3Principal inclusion criteria
    Period 1
    • Patients under P and D1 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) or patients under D2 prematurely discontinued from CLN-PXT3003-02 (due to sponsor decision on September 18th, 2017) must have performed an early V6
    • Patients who completed the V6 assessments within 4 weeks prior to extension study or patients who have completed a new baseline visit (VB) if the V6 assessments were not completed in the 4 weeks prior to study entry.
    • 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. For minor patients, both patient’s and parents’ consent shall be collected.

    Period 2
    • Patients must sign a written informed consent to continue after V9 to access to study treatment.
    E.4Principal exclusion criteria
    Patients with any of the following criteria will be excluded from entry:
    1. Presenting 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.
    2. Any unauthorized concomitant treatments, in the 4 weeks preceding study entry, as study CLN-PXT3003-02.
    E.5 End points
    E.5.1Primary end point(s)
    Period 1
    To assess the safety and tolerability of PXT3003 in CMT1A patients during a long-term period, the primary endpoint will be the incidence of Treatment-Emergent Adverse Events (TEAEs) Related to the Investigational Product during the follow-up, in patients exposed to PXT3003 for up to 24 months or 9 months.
    The incidence of TEAEs in the two groups P / D1-9m and P/ D2-9m will be compared to the first 9-month placebo period in the primary study.
    The incidence of TEAEs in the two groups D1-24m, D2-24m will be compared to the first 9-month period in the primary study for their corresponding group.
    Period 2
    After V9, the incidence of TEAEs related to investigational product will be described.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Period 1: 9 month after the inclusion in the open-label study (either Dose 1 or Dose 2 equivalent to twice Dose 1)

    Period2: every 6 months until the end of the study, when 1st marketing authorization for PXT3003 is obtained (estimated in 2024)
    E.5.2Secondary end point(s)
    PERIOD 1:

    • Safety Endpoints:

    Other safety endpoints will be assessed:
    - Incidence of all TEAEs and their evaluation of type/nature, severity/intensity, seriousness, duration, relationship to study drug, and outcome;
    - Incidence of AE leading to withdrawal of study drug;
    - Changes in laboratory parameters, ECGs, vital signs and physical examination

    • Efficacy Endpoints:

    The clinical response will be assessed by the change over time of the following efficacy endpoints:
    - the Overall Neuropathy Limitation Scale (ONLS) score, and its arm and leg sub-items;
    - the Charcot-Marie-Tooth Neuropathy Score - version 2 (CMTNS-V2), and its sub-items;
    - the functional assessments measured by Nine-hole Peg Test (9-HPT), Quantified Muscular Testing (QMT) by hand grip and foot dorsiflexion dynamometry (mean of both sides), and Time to walk 10 meters;
    - electrophysiological parameters assessing sensory and motor responses of ulnar and radial nerves (non-dominant side) including Compound Muscle Action Potential (CMAP) on ulnar nerve; Sensory Nerve Action Potential (SNAP) on radial nerve; and Nerve conduction velocity (NCV);
    - the EuroQol 5-Dimensional Health-related Quality of Life scale (EQ-5D);
    - the individualized main impairment in daily activities (defined at baseline with the patient) by self-assessment on visual analog scale.

    For the patients receiving up to 24 months of active dose along the two successive studies (from groups D1-24m and D2-24m) the clinical response will be assessed by the change from V1/baseline of each endpoint.

    For the patients who received placebo during the 15-month primary study and active drug during the 9-month extension study (from groups P/D1-9m and P/D2-9m) the clinical response will be assessed after 9 months of PXT3003 treatment by the change from V6/baseline.

    The Percentage of responders to PXT3003 defined as patients improving on ONLS at end of treatment (V9) will be assessed in the patients receiving the active dose of PXT3003 up to 24 months (from groups D1-24m, D2-24m).

    PERIOD 2:

    • Safety Endpoints:

    After V9,
    - the incidence of all TEAEs and their evaluation of type/nature, severity/intensity, seriousness, duration, relationship to study drug, and outcome;
    - the incidence of related TEAEs
    - the incidence of TEAE leading to withdrawal of study drug
    - the incidence of Serious TEAE, Related Serious TEAEs and Serious TEAE leading to withdrawal of the study drug
    - changes in laboratory parameters, ECGs, vital signs and physical examination

    will be described.

    • Efficacy Endpoints:

    After V9, the Overall Neuropathy Limitation Scale (ONLS) score, and its arm and leg sub-items shall be assessed every 6 months and the clinical response will be assessed by the change over time of ONLS
    E.5.2.1Timepoint(s) of evaluation of this end point
    Period 1: 9 month after the inclusion in the open-label study (either Dose 1 or Dose 2 equivalent to twice Dose 1)

    Period2: every 6 months until the end of the study, when 1st marketing authorization for PXT3003 is obtained (estimated in 2024)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Open Labelled, Parallel Group
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Belgium
    Canada
    France
    Netherlands
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years8
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 4
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 285
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 137
    F.4.2.2In the whole clinical trial 187
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    Aucun
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-06-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-07-28
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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