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    Summary
    EudraCT Number:2021-002864-36
    Sponsor's Protocol Code Number:PKRPC001
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-06-28
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2021-002864-36
    A.3Full title of the trial
    A prospective randomized, double-blind, placebo-controlled, multi-center phase IIb study to evaluate the efficacy and safety of mocravimod as an adjunctive and maintenance treatment in adult in acute myeloid leukemia (AML) patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT)
    Studio di fase IIb prospettico randomizzato, in doppio cieco, controllato con placebo, multicentrico, volto a valutare l'efficacia e la sicurezza di mocravimod come trattamento aggiuntivo e di mantenimento in pazienti adulti affetti da leucemia mieloide acuta (LMA) in fase di trapianto allogenico di cellule staminali ematopoietiche (HSCT).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase IIb placebo-controlled efficacy and safety study of mocravimod as an adjunctive and maintenance treatment in AML patients undergoing allo-HSCT (MO-TRANS Study)
    Studio di efficacia e sicurezza di fase IIb, controllato con placebo, di mocravimod come trattamento aggiuntivo e di mantenimento in pazienti affetti da leucemia mieloide acuta sottoposti a allo-HSCT (studio MO-TRANS)
    A.3.2Name or abbreviated title of the trial where available
    MO-TRANS Study
    MO-TRANS Study
    A.4.1Sponsor's protocol code numberPKRPC001
    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 SponsorPriothera SAS
    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 supportPriothera S.A.S.
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPriothera S.A.S.
    B.5.2Functional name of contact pointClinical Sciences & Operations
    B.5.3 Address:
    B.5.3.1Street Address57 avenue Général de Gaulle
    B.5.3.2Town/ citySaint-Louis
    B.5.3.3Post code68300
    B.5.3.4CountryFrance
    B.5.4Telephone number0685535546
    B.5.6E-mailmalika.souquieres@priothera.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/3/21/2504
    D.3 Description of the IMP
    D.3.1Product nameMocravimod
    D.3.2Product code [KRP203, 1.0 mg]
    D.3.4Pharmaceutical form Capsule, hard
    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 INNMocravimod
    D.3.9.1CAS number 509088-69-1
    D.3.9.2Current sponsor codeKRP203
    D.3.9.4EV Substance CodeSUB195626
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 placeboCapsule, hard
    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
    Adjunctive and maintenance treatment to HSCT
    Trattamento aggiuntivo e di mantenimento al trapianto
    E.1.1.1Medical condition in easily understood language
    Adjunctive and maintenance treatment to HSCT
    Trattamento aggiuntivo e di mantenimento al trapianto
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10000886
    E.1.2Term Acute myeloid leukemia
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare the efficacy of mocravimod to that of placebo
    Confrontare l'efficacia del mocravimod con quella del placebo
    E.2.2Secondary objectives of the trial
    To compare mocravimod's effect on overall survival (OS) to that of placebo
    Per confrontare l'effetto di mocravimod sulla sopravvivenza globale (OS) con quello del placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Subjects with a diagnosis of AML (excluding acute promyelocytic leukemia) according to the World Health Organization (WHO) 2016 classification of AML and related precursor neoplasms, including secondary AML after an antecedent hematological disease (e.g. myelodysplastic syndrome) and therapy-related AML.
    2.Subjects with European LeukemiaNet (ELN) high risk AML in CR1, intermediate risk AML in CR1 if MRDpos, or AML of any risk in CR2.
    - Complete remission is defined as: < 5% marrow blasts by morphologic examination and no circulating peripheral blasts and blasts with Auer rods; absence of extramedullary disease; absolute neutrophil count = 1.0x109/L (1000/µL); platelet count = 100x109/L (100 000/µL)
    3.Subjects planned to undergo allogeneic HSCT
    4.Life expectancy = 6 months at screening.
    5.Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
    6.Male or female, age = 18 years and = 75 years.
    1.Soggetti con diagnosi di AML (esclusa leucemia promielocitica acuta) secondo la classificazione 2016 dell'Organizzazione Mondiale della Sanità (OMS) di AML e relative neoplasie precursori, inclusa AML secondaria dopo una malattia ematologica antecedente (es. sindrome mielodisplastica) e AML correlata alla terapia.
    2.Soggetti con LMA ad alto rischio European LeukemiaNet (ELN) in CR1, LMA a rischio intermedio in CR1 se MRDpos, o LMA di qualsiasi rischio in CR2.
    - La remissione completa è definita come: < 5% di blasti midollari all'esame morfologico e assenza di blasti periferici circolanti e blasti con bastoncelli di Auer; assenza di malattia extramidollare; conta assoluta dei neutrofili = 1,0x109/L (1000/µL); conta piastrinica = 100x109/L (100 000/µL)
    3.Soggetti previsti per il trapianto allogenico
    4. Aspettativa di vita = 6 mesi allo screening.
    5. Performance status ECOG (Eastern Cooperative Oncology Group) pari a 0 o 1
    6.Maschio o femmina, età = 18 anni e = 75 anni.
    E.4Principal exclusion criteria
    1.Planned use of anti-thymocyte globulin (ATG), post-transplantation cyclophosphamide, sirolimus, mycophenolate mofetil, abatacept, or any approved or non-approved medication other than MTX plus CsA or MTX plus TAC for GVHD prophylaxis.
    2.Planned use of serotherapy during conditioning, including ATG and alemtuzumab.
    3.Planned ex vivo major graft manipulation, including T-cell depletion or CD34+ selection.
    4.Subjects having received prior allogeneic HSCT or recipients of a solid organ transplant.
    5.Immunosuppressive drugs for concomitant disease. Subjects must be able to be off prednisone (> 10 mg/day) or other immunosuppressive medications for at least 3 days prior to the start of treatment of the study. Physiologic replacement dosing of hydrocortisone is permissible.
    6.Require treatments for cardiac dysfunction
    7.Subjects with acute promyelocytic leukemia.
    8.Blast crisis of chronic myeloid leukemia
    9.Cardiac dysfunction
    10.Pulmonary dysfunction.
    11.Significant liver disease or liver injury or known history of alcohol abuse, chronic liver or biliary disease. Hepatic dysfunction as defined by aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 2.5 x upper limit of normal (ULN); or total bilirubin > 1.5 x ULN
    12.Renal dysfunction with creatinine clearance < 60 mL/min by the Cockcroft-Gault formula.
    13.History of stroke or intracranial hemorrhage within 1 year prior to screening.
    14.Active clinically significant infection (viral, bacterial, or fungal) that requires ongoing antimicrobial therapy and in the judgment of the investigator represents a risk to proceeding with HSCT.


    1. Uso pianificato di globulina antitimocitaria (ATG), ciclofosfamide post-trapianto, sirolimus, micofenolato mofetile, abatacept o qualsiasi farmaco approvato o non approvato diverso da MTX più CsA o MTX più TAC per la profilassi della GVHD.
    2. Uso pianificato della sieroterapia durante il condizionamento, inclusi ATG e alemtuzumab.
    3. Manipolazione pianificata dell'innesto ex vivo, inclusa la deplezione dei linfociti T o la selezione di CD34+.
    4.Soggetti che hanno ricevuto un precedente HSCT allogenico o destinatari di un trapianto di organo solido.
    5. Farmaci immunosoppressori per malattie concomitanti. I soggetti devono poter essere assenti dal prednisone (> 10 mg/die) o da altri farmaci immunosoppressori per almeno 3 giorni prima dell'inizio del trattamento dello studio. È consentito il dosaggio sostitutivo fisiologico dell'idrocortisone.
    6.Richiedere trattamenti per la disfunzione cardiaca
    7.Soggetti con leucemia promielocitica acuta.
    8. Crisi esplosiva di leucemia mieloide cronica
    9. Disfunzione cardiaca
    10. Disfunzione polmonare.
    11. Malattia epatica significativa o danno epatico o storia nota di abuso di alcol, malattie croniche del fegato o delle vie biliari. Disfunzione epatica definita dall'aspartato aminotransferasi (AST) e/o dall'alanina aminotransferasi (ALT) > 2,5 x limite superiore della norma (ULN); o bilirubina totale > 1,5 x ULN
    12. Disfunzione renale con clearance della creatinina < 60 ml/min secondo la formula di Cockcroft-Gault
    13.Anamnesi di ictus o emorragia intracranica entro 1 anno prima dello screening.
    14. Un'infezione attiva clinicamente significativa (virale, batterica o fungina) che richiede una terapia antimicrobica in corso e, a giudizio dello sperimentatore, rappresenta un rischio per procedere con l'HSCT.
    E.5 End points
    E.5.1Primary end point(s)
    Relapse-free survival (RFS)
    Sopravvivenza libera da recidive (RFS)
    E.5.1.1Timepoint(s) of evaluation of this end point
    following 46 events occurred (relapse or death of any cause)
    a seguito di 46 eventi verificatisi (recidiva o morte per qualsiasi causa)
    E.5.2Secondary end point(s)
    - Overall survival (OS)
    -Time to relapse
    -Non-relapse mortality



    - Sopravvivenza globale (OS)
    - Tempo di ricaduta
    - Mortalità senza recidiva
    E.5.2.1Timepoint(s) of evaluation of this end point
    Event-driven trial
    Prova guidata dagli eventi
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Quality of Life (QoL)
    Qualità della vita (QoL)
    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 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 trial3
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA54
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Israel
    Japan
    United States
    France
    Spain
    Switzerland
    Germany
    Italy
    United Kingdom
    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 (last subject last visit – LSLV)
    LVLS (ultimo soggetto ultima visita – LSLV)
    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 months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    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 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: 200
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 49
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 249
    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)
    After participation, subjects will be managed in accordance with clinical practice, i.e. as per local guidelines and standard of care.
    Dopo la partecipazione, i soggetti saranno gestiti secondo la normale pratica clinica, ovvero secondo le linee guida locali e gli standard di cura.
    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 Decision2022-09-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-09-12
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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