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    Summary
    EudraCT Number:2021-006223-18
    Sponsor's Protocol Code Number:PXL-770-011
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2022-01-17
    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 ConcernedGermany - BfArM
    A.2EudraCT number2021-006223-18
    A.3Full title of the trial
    A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL770 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Trial on patients with adrenomyeloneuropathy to assess PXL770's behaviour, a new potential medicine, in the body
    A.3.2Name or abbreviated title of the trial where available
    START770
    A.4.1Sponsor's protocol code numberPXL-770-011
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05146284
    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 SponsorPoxel S.A.
    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 supportPoxel S.A.
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPoxel S.A.
    B.5.2Functional name of contact pointCarole THANG
    B.5.3 Address:
    B.5.3.1Street Address259-261 Avenue Jean Jaurès
    B.5.3.2Town/ cityLyon
    B.5.3.3Post code69007
    B.5.3.4CountryFrance
    B.5.4Telephone number+33437372010
    B.5.6E-mailcarole.thang@poxelpharma.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePXL770
    D.3.2Product code PXL770
    D.3.4Pharmaceutical form Film-coated tablet
    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.3.9.1CAS number 1523493-78-8
    D.3.9.2Current sponsor codePXL770
    D.3.9.3Other descriptive namePXL770
    D.3.9.4EV Substance CodeSUB179447
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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
    Adrenomyeloneuropathy (AMN)
    E.1.1.1Medical condition in easily understood language
    Neurodegenerative disease or Congenital, hereditary and neonatal diseases and abnormalitites
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Efficacy : To assess the PK of PXL770 in AMN subjects at the daily dose of 500mg OD and 250mg BID after 4 weeks of treatment
    E.2.2Secondary objectives of the trial
    1. To assess the safety and tolerability of PXL770 in AMN subjects at the daily dose of 500mg OD and 250mg BID after 12 weeks of treatment
    2.To assess the effect of PXL770 at the daily dose of 500mg OD and 250mg BID on PD biomarkers:
    - VLCFA in plasma
    - NfL in plasma
    - Lipid and glycemic parameters
    in AMN subjects up to 12 weeks of treatment
    3.To compare the 2 doses regimens of PXL770 (BID versus OD) on PK, safety and PD parameters in AMN subjects up to 12 weeks of treatment
    Exploratory Objectives : To assess the effect of PXL770 at the daily dose of 500mg OD and 250mg BID on other parameters:
    - VLCFA in PBMC
    - Biomarkers of inflammation
    - Biomarkers of PXL770 mechanism of action
    in AMN subjects up to 12 weeks of treatmente enter information in English and add any other language that is applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male subjects with either a confirmed diagnosis of AMN by genetic testing or a family history of ALD together with an elevation in VLCFA obtained from overnight fasting plasma sample at V1 (Screening Visit).
    2. Age: ≥ 18 to ≤ 65 years at informed consent signature.
    3. Normal brain MRI or brain MRI showing non-specific abnormalities that can be observed in AMN subjects without signs of C-ALD
    E.4Principal exclusion criteria
    Target disease exclusions
    1. Any progressive neurological disease other than AMN.
    2. Arrested or progressing C-ALD as defined by cerebral lesions.
    3. Prior receipt of an allogeneic hematopoietic stem cell transplant or gene therapy.
    E.5 End points
    E.5.1Primary end point(s)
    Primary plasma PK parameters of PXL770: Cmax and AUC0-24 at V3 for 500mg OD treatment group and Cmax and AUC0-8 at Week 4 for 250mg BID treatment group.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 4
    E.5.2Secondary end point(s)
    Secondary plasma PK parameters of PXL770:
    • Ctrough at V3, V4 and V5-EoT,
    • Cavg, CLss/F, AUC0-8 (for 500mg OD treatment group) tmax and AUClast at V3.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 4, week 8 and week 12
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Same IMP but different dosing regimen
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA2
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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 years
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days15
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 22
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 24
    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 Decision2022-05-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-29
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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