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    Summary
    EudraCT Number:2020-004805-30
    Sponsor's Protocol Code Number:CLN-PXT3003-06
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-08
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2020-004805-30
    A.3Full title of the trial
    A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 3 trial of PXT3003 in Charcot-Marie-Tooth (CMT) Type 1A patients
    A.4.1Sponsor's protocol code numberCLN-PXT3003-06
    A.5.4Other Identifiers
    Name:IND numberNumber: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 pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address46 rue Saint Lazare
    B.5.3.2Town/ cityParis
    B.5.3.3Post code7009
    B.5.3.4CountryFrance
    B.5.4Telephone number+19178853598
    B.5.5Fax number+33141092231
    B.5.6E-mailmisrael@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.3.1Product nameNA
    D.3.2Product code PXT3003
    D.3.4Pharmaceutical form Oral solution in sachet
    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.3.9.3Other descriptive name(RS)-baclofen
    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 16590-41-3
    D.3.9.3Other descriptive namenaltrexone hydrochloride
    D.3.9.4EV Substance CodeSUB09143MIG
    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.3.9.3Other 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.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral solution in sachet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Charcot Marie Tooth Type 1A
    E.1.1.1Medical condition in easily understood language
    Hereditary Motor and Sensory Neuropathy
    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
    To evaluate the efficacy of treatment with PXT3003 (a fixed-dose combination of (RS)-baclofen, naltrexone hydrochloride, and D-sorbitol) compared to placebo in subjects with CMT1A
    E.2.2Secondary objectives of the trial
    To evaluate the safety and tolerability of PXT3003 treatment in subjects with CMT1A
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Male and non-pregnant female subjects, aged 16 to 65 years with a genetically proven diagnosis of CMT1A. Notes: a) A report of a genetic test confirming PMP22 duplication and therefore a diagnosis of CMT1A must be available in teh subject's reecord at the clinical site. b) In the absence of a report of a genetic test confirming PMP22 duplication in teh subject's medical record, a confirmatory genetic test must be conducted via the central laboratory as part of Screening. c) In the exceptional case wherein subject was randomized into the study without meeting (a) or (b), an unscheduled confirmatory genetic test will be performed. In the event of a negative genetic test result, the subject will be withdrawn from the study.
    2) Able to provide written informed consent/assent and comply with study procedures
    3) Mild-to-moderate severity assessed by CMTNS-v2 score >2 and ≤18
    4) Muscle weakness in at least foot dorsiflexion on clinical assessment
    5) Ulnar nerve motor conduction time of at least 15m/s
    6) If taking prescribed psychoactive drug(s) (e.g. antidepressants, stimulants, tranquilizers, anti-epileptics) for CMT1A, should be on a stable dose for at least 4 weeks prior to randomization, which is not planned to be changed
    7) If taking prescribed or ‘over-the-counter’ (OTC) analgesic medication(s) (e.g. paracetamol/acetaminophen, nonsteroidal anti-inflammatory drugs [NSAIDs]) for CMT1A, should be on a stable dose for at least 2 weeks prior to randomization, which is not planned to be changed
    8) If female, subject must be: (a) Surgically sterilized via hysterectomy, bilateral oophorectomy, or bilateral tubal ligation; or (b) Of childbearing potential and using a birth control method such as:
    • Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
    o Oral
    o Intravaginal
    o Transdermal
    • Progestogen-only hormonal contraception associated with inhibition of ovulation:
    o Oral
    o Injectable
    o Implantable
    • IUD
    • IUS
    • Bilateral tubal occlusion
    • Vasectomized partner
    • Sexual abstinence;
    or (c) Of non-childbearing potential (i.e., no menses for ≥ 12 consecutive months without any other underlying medical cause)
    9) If male, the subject must have had a vasectomy or must use a reliable method of birth control with their partner or total abstinence from sexual intercourse. The subject must agree to continue using their selected method of birth control with their sexual partner during the study and for 120 days after study completion.

    For subjects consenting to enter the OLE Period, the following inclusion criteria will be confirmed/reassessed on Screening Day (SV6) of the OLE Period:
    1. Able to provide written informed consent/assent and comply with study procedures.
    2. If female, subject must be (a) surgically sterilized via hysterectomy, bilateral oophorectomy, or bilateral tubal ligation; or (b) of childbearing potential and using a birth control method such as:
    • Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
    o Oral
    o Intravaginal
    o Transdermal
    • Progestogen-only hormonal contraception associated with inhibition of ovulation:
    o Oral
    o Injectable
    o Implantable
    • IUD
    • IUS
    • Bilateral tubal occlusion
    • Vasectomized partner
    • Sexual abstinence;
    or (c) of non-childbearing potential (ie, no menses for ≥ 12 consecutive months without any other underlying medical cause)
    9) If male, the subject must have had a vasectomy or must use a reliable method of birth control with their partner or total abstinence from sexual intercourse. The subject must agree to continue using their selected method of birth control with their sexual partner during the study and for 120 days after study completion.
    E.4Principal exclusion criteria
    1) Subjects previously enrolled in any PXT3003 study
    2) Subjects living in the same household and enrolled in a PXT3003 study (due to potential lack of adequate storage for study material, risk of mixing treatments and potential unblinding)
    3) CMT of any subtype other than 1A
    4) ONLS score of 0
    5) Known clinically significant motor or sensory abnormalities secondary to a different neurological cause. Note: subjects with diagnosis of unilateral carpal tunnel syndrome at least 1 year prior to Screening Visit that is asymptomatic at the time of Screening Visit will not be excluded from participating in this study
    6) Subjects who have had any surgery or have a concomitant disorder that reduces the mobility of the ankle or wrist making it, in the opinion of the investigator, difficult to assess the efficacy of the treatment. Note: subjects with surgical repair of unilateral carpal tunnel syndrome will not be excluded from participating in this study
    7) Known peripheral neuropathy, myopathy, or neuromuscular disorder of any other kind. Note: subjects with diagnosis of unilateral carpal tunnel syndrome at least 1 year prior to Screening Visit that is asymptomatic at the time of Screening Visit will not be excluded from participating in this study
    8) Any other clinically significant and/or uncontrolled medical condition that in the opinion of the investigator, could be a confound, may increase subject´s risk, or may preclude successful participation or completion of the study
    9) Known hypersensitivity or intolerance to PXT3003 (or matching placebo), including any of its active ingredients, and/or any of its excipients.
    10) Concomitant treatments including but not limited to baclofen, naltrexone, sorbitol (pharmaceutical form), opioids, potent central nervous system depressants, and potentially neurotoxic drugs such as amiodarone, chloroquine, and chemotherapeutics capable of inducing peripheral neuropathy. Subjects able to stop these medications at least 2 weeks before randomization and for the study duration may be included. Subjects with positive urine drug screen at Baseline Visit will be excluded, except for permitted use of codeine and benzodiazepines.
    11) History of porphyria
    12) Diagnosis or history of substance use disorder by DSM-V criteria within the past 12 months
    13) Medical or recreational use of marijuana in the 3 months prior to the Screening Visit
    14) Active suicidality
    15) Currently active major depression, as determined by a BDI-II score ≥ 20
    16) Currently lactating, pregnant, or planning on becoming pregnant during the study
    17) ALT or AST levels greater than 2 times the ULN
    18) Significant renal impairment as determined by GFR of less than 50 mL/min
    19) Subject has participated in an investigational drug or device study within 30 days prior to the Screening Visit or plans to participate in an investigational drug or device study during the course of this study
    20) Subject is a dependent and/or relative of the Sponsor or investigator
    For subjects consenting to enter the OLE Period, the following criteria will be confirmed/reassessed on SV6 of the OLE Period:
    1. Any clinically significant and/or uncontrolled medical condition that, in the opinion of the investigator, could be a confound factor, may increase subject's risk or may preclude successful participation or completion of the study
    2. Concomitant treatments including but not limited to baclofen, naltrexone, sorbitol (pharmaceutical form) other than PXT3003 taken in the Double-blind Treatment Period of this study, opioids, potent CNS depressants, and potentially neurotoxic drugs such as amiodarone, chloroquine, and chemotherapeutics capable of inducing peripheral neuropathy. Subjects able to stop these medications at least 2 weeks before randomization and for the study duration may be included
    3. Diagnosis or history of substance use disorder by Diagnostic and Statistical Manual of Mental Disorders-5th Edition Criteria within the past 12 months
    4. Active suicidiality
    5. Currently active major depression, as determined by a BDI-II score ≥ 20
    6. Currently lactating, pregnant or planning on becoming pregnant during the study
    7. ALT or AST levels greater than 2 x ULN relative to Baseline (1)
    8. Estimated GFR of less than 50 mL/min (1)
    (1) Note: If the laboratory values are not normal (out of range) the subjects will be contacted and therapy will be stopped
    Note: PXT3003 will be dispensed to all subjects before laboratory results are available and if the laboratory results are out of range, ie, subject meeting the exclusion criteria the subject will be called immediately to stop taking PXT3003. One retest within 4 weeks may be performed in consultation with the Medical Monitor if any of the above laboratory abnormalities are found. In case of eligibility determination after the retest, subjects will restart taking 2 weeks of half dose of PXT3003
    E.5 End points
    E.5.1Primary end point(s)
    The change in the modified Overall Neuropathy Limitation Scale (mONLS) between baseline and the Month 15 visit.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 15 visit
    E.5.2Secondary end point(s)
    The change from baseline to month 15 in the following outcome measures in hierarchical order:
    1) 10-Meter Walk Test (10mWT)
    2) Quantified Muscular Testing (bilateral foot dorsiflexion dynamometry)
    3) Patient Global Impression of Severity (PGI-S)
    4) Patient Global Impression of Change (PGI-C)*
    5) Charcot-Marie-Tooth Neuropathy Score, version 2 (CMTNS-v2)
    6) Quantified Muscular Testing (hand grip)
    * Because the PGI-C is already a change assessment, the change from Baseline is not needed for this endpoint.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Month 15 visit
    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 Yes
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    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
    Denmark
    France
    Germany
    Israel
    Italy
    Spain
    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
    For Double-blind Treatment Period - LVLS - completion of the Month 15 Visit by the last subject.
    For OLE Period - The duration of OLE Period will be up to two years and will be based on Sponsor discretion. Justification of a potential extension beyond the two-year period will be evaluated and agreed upon with the competent authorities of Denmark (DKMA).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days5
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months5
    E.8.9.2In all countries concerned by the trial days23
    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: 2
    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: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 381
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 183
    F.4.2.2In the whole clinical trial 387
    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
    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 Decision2021-07-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-01-31
    P. End of Trial
    P.End of Trial StatusOngoing
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