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    The EU Clinical Trials Register currently displays   44335   clinical trials with a EudraCT protocol, of which   7366   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2021-002864-36
    Sponsor's Protocol Code Number:PKRPC001
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-09-03
    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 ConcernedFrance - ANSM
    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 in acute myeloid leukemia (AML) patients undergoing allogeneic hematopoietic stem cell transplant (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 in AML patients undergoing HSCT
    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 S.A.S.
    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 du Général de Gaulle
    B.5.3.2Town/ citySaint-Louis
    B.5.3.3Post code68300
    B.5.3.4CountryFrance
    B.5.4Telephone number+33 (0) 6 85 53 55 46
    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 No
    D.2.5.1Orphan drug designation number
    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 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 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
    Patients undergoing allogeneic hematopoietic stem cell transplant (HSCT) for acute myeloid leukemia (AML)
    E.1.1.1Medical condition in easily understood language
    Patients undergoing allogeneic hematopoietic stem cell transplant (HSCT) for acute myeloid leukemia (AML)
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10018651
    E.1.2Term Graft versus host disease
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess mocravimod’s effect as an adjunctive therapy to HSCT on GVHD and GVL after a 12-month treatment period.
    E.2.2Secondary objectives of the trial
    To assess mocravimod’s effect on overall survival (OS) at 24 months, following a 12-month treatment and a 12-month follow-up period.
    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 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 ELN high risk AML in CR1 or any other AML in CR2. (Complete remission with incomplete count recovery [CRi] is also allowable).
    o Complete remission is defined as leukemia clearance (< 5% marrow blasts and no circulating peripheral blasts) in conjunction with normal values for absolute neutrophil count and platelet count, no extramedullary manifestation of leukemia and no need for repeat blood transfusions.
    o CRi is defined as meeting all complete remission criteria except for an absolute neutrophil count < 1,000/μL or platelet count < 100,000/μL.
    3. Subjects planned to undergo allogeneic HSCT, with all of the following parameters met:
    o Use of fully matched related or unrelated donor (10/10 HLA-matched), and
    o Use of granulocyte colony-stimulating factor (G-CSF) mobilized peripheral blood stem cells, and
    o Use of protocol-approved (myeloablative) conditioning regimen, and
    o Use of CsA and MTX as GVHD prophylaxis
    4. Life expectancy ≥ 6 months at screening.
    5. Karnofsky Performance Status (KPS) ≥ 70%.
    6. Male or female, age ≥ 18 years and ≤ 75 years.
    Subjects ≥ 65 years must have a Sorror (hematopoietic cell transplantation-specific comorbidity index [HCT-CI]) Score ≤ 3.
    7. Able and willing to provide written informed consent and comply with the trial protocol and procedures.
    8. For females of childbearing potential who are sexually active and males who have sexual contact with a female of childbearing potential: willingness to use reliable methods of contraception (oral contraceptives, contraceptive injection, male vasectomy, condom with spermicidal agent, or sexual abstinence).
    Contraception is to be used from the Screening visit until the EOT visit, and in any case for at least 6 months after the last dose of IMP.
    A female is considered of childbearing potential following menarche and until becoming post-menopausal unless permanently sterile. Women are considered post-menopausal and not of childbearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least 6 weeks before IMP treatment. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow-up hormone level assessment, she is considered not of childbearing potential.
    9. Sexually active males must use a condom during intercourse from the Screening visit until at least 6 months after the last dose of IMP and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the IMP via seminal fluid.
    10. Affiliation to a national health insurance scheme (according to applicable local requirements).
    E.4Principal exclusion criteria
    1.Planned use of tacrolimus, anti-thymocyte globulin (ATG), post-transplantation cyclophosphamide, or mycophenolate mofetil for GVHD prophylaxis.
    2.Planned use of serotherapy during conditioning
    3.Planned ex vivo major graft manipulation
    4.Subjects having received prior allogeneic HSCT or recipients of a solid organ transplant
    5.Vaccination within 4 weeks prior to screening
    6.Immunosuppressive drugs for concomitant disease. Subjects off prednisone or other immunosuppressive medications <3 days prior study treatment
    7.Major surgery within 4weeks prior screening or a major wound, not fully healed
    8.Require any of the following treatments for cardiac dysfunction:
    -treatment with medication that impairs cardiac conduction
    -Concomitant use of agents known to prolong the QT interval unless they can be permanently discontinued for the duration of the study
    -Treatment with quinidine
    9.Subjects with acute promyelocytic leukemia
    10.Diagnosis of any previous or concomitant malignancy, except subjects diagnosed with localized basal cell carcinoma of the skin or in situ cervical cancer, or subjects who have completed treatment with curative intent for the malignancy at least 6 months prior to enrollment
    11.Blast crisis of chronic myeloid leukemia
    12.Concurrent severe and/or uncontrolled medical condition including:
    -Clinically significant pulmonary fibrosis
    -Tuberculosis, except for history of successfully treated tuberculosis or history of prophylactic treatment after positive PPD skin reaction
    -Subjects receiving daily therapies for asthma
    -Subjects with any other types of clinically significant obstructive pulmonary disease
    -Uncontrolled diabetes mellitus as assessed by the investigator or diabetes complicated with organ involvement such as diabetic nephropathy or retinopathy
    -Uncontrolled seizure disorder
    -Uncontrolled depression or history of suicide attempts/ideation.
    13.Cardiac dysfunction as defined by:
    -Myocardial infarction within the last 3 months of trial entry
    -Reduced left ventricular function with an ejection fraction <40% as measured by MUGA scan or echocardiogram within 6weeks before signing ICF
    -History or presence of stable or unstable IHD, myocarditis or cardiomyopathy
    -NYHA Class II-IV congestive heart failure
    -Unstable cardiac arrhythmias including history of or presence of symptomatic bradycardia
    -Resting heart rate ECG <60bpm
    -History or current diagnosis of ECG abnormalities indicating significant risk of safety such as: Concomitant clinically significant cardiac arrhythmias, or sino-atrial heart block, clinically significant atrioventricular (AV) block, bundle branch block or resting QTc >450 msec for males and >470 msec for females at Screening or Baseline ECG
    -History or presence of symptomatic arrhythmia or arrhythmia requiring treatment or of clinical significance
    -Uncontrolled arterial hypertension; if controlled, the medication must be stable for 3 months prior to baseline
    -Requiring treatment with prohibited medication
    -History of syncope of suspected cardiac origin
    -History of familial long QT syndrome or known family history of Torsades de Pointes
    14.Pulmonary dysfunction as defined by oxygen saturation <90% on room air. PFT is required only in the case of symptomatic or prior known impairments within 6weeks before signing ICF -with pulmonary function <50% corrected diffusing capacity of the lung for carbon monoxide (DLCO) and <50% predicted forced expiratory volume in 1 second (FEV1).
    15.Significant liver disease or liver injury or known history of alcohol abuse, chronic liver or biliary disease
    16.Hepatic dysfunction as defined by AST and/or ALT >5 x upper limit of normal; or total bilirubin <2.5 mg/dL, unless attributable to Gilbert’s syndrome
    17.Renal dysfunction with serum creatinine >2.0 mg/dL
    18.History of stroke or intracranial hemorrhage within 6months prior screening
    19.Active clinically significant infection requiring ongoing antimicrobial therapy and in the judgment of the investigator represents a risk to proceeding with HSCT
    20.History of HIV or active infection with hepatitis B or hepatitis C virus defined as a positive HIV antibody, hepatitis B surface antigen or hepatitis C antigen
    21.Positive pregnancy test or breastfeeding of subject (women of childbearing potential only)
    22.Known allergy to any of the components of mocravimod
    23.Diagnosis of macular edema at screening. Subjects with a history of macular edema will enter the study provided they do not have macular edema at screening
    24.Participation in another interventional or concomitant clinical trial within 4weeks prior screening
    25.Any other condition that, in the opinion of the investigator, makes the subject ineligible for the study
    26.Subjects under legal protection measure and/or inability or unwillingness to comply with the requirements and procedures of the trial
    E.5 End points
    E.5.1Primary end point(s)
    Refractory GVHD-free, relapse-free survival (rGRFS) at Month 12 after HSCT
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 12 after HSCT
    E.5.2Secondary end point(s)
    - Overall survival (OS) at Month 24 after HSCT;
    - Relapse-free survival (RFS) at Month 24 after HSCT;
    - Survival free from refractory acute GVHD (aGVHD) at Month 12 after HSCT;
    - Survival free from moderate/severe chronic GVHD (cGVHD) at Month 12 and at Month 24 after HSCT;
    - Non-relapse related mortality at Month 12 and at Month 24 after HSCT;
    - Cumulative incidence of relapse at Month 12 and at Month 24 after HSCT;
    - rGRFS at Month 24 after HSCT;
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Month 24 after HSCT;
    - Month 12 and at Month 24 after HSCT.
    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 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 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 concerned8
    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 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
    Australia
    Canada
    Israel
    United States
    Belgium
    France
    Germany
    Italy
    Netherlands
    Spain
    Switzerland
    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
    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: 140
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 110
    F.4.2.2In the whole clinical trial 160
    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.
    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 Decision2021-11-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-19
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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