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    Summary
    EudraCT Number:2017-003434-87
    Sponsor's Protocol Code Number:CPX-MA-1201
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2017-08-24
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2017-003434-87
    A.3Full title of the trial
    A Phase 1/Pilot Study of CPX-351 for Children, Adolescents and Young Adults with Recurrent or Refractory
    Hematologic Malignancies.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety Study of CPX-351 in Children With Relapsed Leukemia or Lymphoma.
    A.4.1Sponsor's protocol code numberCPX-MA-1201
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/200/2017
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCincinnati Children's Hospital Medical Center
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportJazz Pharmaceuticals
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJazz Pharmaceuticals
    B.5.2Functional name of contact pointClinical Trial Disclosure
    B.5.3 Address:
    B.5.3.1Street Address1818 Market Street
    B.5.3.2Town/ cityPhiladelphia
    B.5.3.3Post code19103
    B.5.3.4CountryUnited States
    B.5.4Telephone number+12158323750
    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 Yes
    D.2.1.1.1Trade name Vyxeos
    D.2.1.1.2Name of the Marketing Authorisation holderJazz Pharmaceuticals
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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/3/11/942
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Lyophilisate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraventricular use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCytarabine
    D.3.9.1CAS number 147-94-4
    D.3.9.4EV Substance CodeSUB06880MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDaunorubicin Hydrochloride
    D.3.9.1CAS number 23431-50-6
    D.3.9.4EV Substance CodeSUB01556MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.44
    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) Yes
    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
    Acute Myeloid Leukemia
    E.1.1.1Medical condition in easily understood language
    Acute Myeloid Leukemia
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10060558
    E.1.2Term Acute myeloid leukemia recurrent
    E.1.2System Organ Class 100000012987
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine a safe and tolerable dose of CPX-351 in children and adolescents with relapsed or refractory hematopoietic malignancies and to recommend a dose for future studies.
    E.2.2Secondary objectives of the trial
    To estimate the overall response rate to a single course of CPX-351 to young patients with recurrent or
    refractory hematologic malignancies.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Age 1-21 yrs at the time of study enrollment
    - Diagnosis of a hematologic malignancy [acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or aggressive lymphoma]
    - Karnofsky ≥ 50% for patients > 16 years of age and Lansky ≥ 50 for patients ≤16 years of age
    - Patients must have fully recovered from acute toxicities of prior therapy
    - Adequate Organ Function requirements, defined as:
    o Platelet count ≥ 20 X 109/L (20,000/mcgL) (may receive platelet transfusion)
    o Hemoglobin ≥ 8.0 g/dL (may receive RBC transfusion)
    o Adequate serum creatinine based on age/gender or a 24 hour creatinine clearance/ radioisotope determined GFR ≥ 70mL/min/1.73 m2
    o Direct bilirubin ≤ 1.5 X upper limit of normal (ULN) for age
    o SGPT (ALT) , 5.0 X ULN for age and institution (unless elevation is related to leukemia involvement)
    o Shortening fraction of ≥27% by echocardiogram, or Ejection fraction of ≥ 50% by gated radionuclide study or echocardiogram
    E.4Principal exclusion criteria
    - Patients with Acute Promyelocytic Leukemia (APML), Down Syndrome, Fanconi Anemia, ALL and CNS leukemia (CNS status 3).
    - Pregnant or breast-feeding women and males or females of reproductive potential unwilling to use an effective contraceptive method.
    - Growth factors that support platelet or white cell number or function must not have been administered within the 7 days prior to enrollment.
    - Patients who are currently receiving another investigational drug or anti-cancer agents (with the exception of intrathecal cytarabine and oral hydroxyurea. Hydroxyurea must be discontinued 24 hours prior to initiation of protocol therapy).
    - Patients who have an uncontrolled infection, history of Wilson’s disease or other copper metabolism, had major surgery within 4 weeks of enrollment or received prior radiation to the mediastinum.
    - Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study.
    E.5 End points
    E.5.1Primary end point(s)
    ● Determine rate of dose limiting toxicities
    ● Number of participants with dose limiting toxicities to determine maximum tolerated dose
    ● Pharmacokinetics: Serum concentration of CPX-351 components and metabolites
    E.5.1.1Timepoint(s) of evaluation of this end point
    ● Day 56
    ● Day 56
    ● Day 10
    E.5.2Secondary end point(s)
    ● Overall response rate after a single course of CPX-351
    ● Electrocardigram, echocardiogram, peripheral blood cardiac troponin-T (cTn-T) and brain natriuretic peptide (BnP)
    E.5.2.1Timepoint(s) of evaluation of this end point
    ● Day 28
    ● Day 30
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    PK study
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    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 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 No
    E.8.3 Will this trial be conducted at a single site globally? Yes
    E.8.4 Will this trial be conducted at multiple sites globally? No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    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
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years5
    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: 27
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    This clinical trial will be conducted in children in different age groups, including children younger than 12 years of age.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 27
    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)
    Standard of care and/or supportive care therapy.
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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