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    Summary
    EudraCT Number:2016-004554-14
    Sponsor's Protocol Code Number:PIX111
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2016-11-24
    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 number2016-004554-14
    A.3Full title of the trial
    A Phase 1, Dose-Escalation Study of Pixantrone Monotherapy in Pediatric Patients with Relapsed or Refractory Cancer
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to evaluate the safety, pharmacokinetics and anti-tumor activity of pixantrone in pediatric patients with cancer
    A.4.1Sponsor's protocol code numberPIX111
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02800889
    A.5.4Other Identifiers
    Name:INDNumber:62,678
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/310/2016
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCTI BioPharma Corp.
    B.1.3.4CountryUnited States
    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 supportCTI BioPharma Corp.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCTI BioPharma Corp.
    B.5.2Functional name of contact pointSarah Telzrow
    B.5.3 Address:
    B.5.3.1Street Address3101 Western Avenue, Suite #600
    B.5.3.2Town/ citySeattle
    B.5.3.3Post codeWA 98121
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1206-272-4426
    B.5.5Fax number+1206-284-6206
    B.5.6E-mailstelzrow@ctibiopharma.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 Yes
    D.2.1.1.1Trade name Pixuvri
    D.2.1.1.2Name of the Marketing Authorisation holderCTI Life Sciences Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.4Pharmaceutical form Powder for concentrate and solution for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNpixantrone
    D.3.9.1CAS number 144510-96-3
    D.3.9.2Current sponsor codepixantrone
    D.3.9.3Other descriptive namePIXANTRONE
    D.3.9.4EV Substance CodeSUB21304
    D.3.10 Strength
    D.3.10.1Concentration unit mg/m2 milligram(s)/square meter
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number40 to 70
    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
    Relapsed or refractory solid tumors (excluding those with CNS tumors) or lymphoma
    E.1.1.1Medical condition in easily understood language
    Relapsed or refractory cancer
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10007050
    E.1.2Term Cancer
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Dose Escalation :
    The primary objectives in the dose-escalation phase are to define the dose-limiting toxicities (DLT) of pixantrone and identify the maximum tolerated dose (MTD) of pixantrone given as monotherapy to pediatric
    patients.

    Expansion cohort:
    The primary objective in the expansion cohort phase is to evaluate anti-tumor activity of pixantrone in expansion cohort patients treated at the MTD as assessed by consensus criteria for each malignancy
    under treatment, including:
    - overall response rate
    - complete response rate
    - partial response rate
    - duration of overall response
    E.2.2Secondary objectives of the trial
    Overall study:
    - To explore and characterize the safety and tolerability of pixantrone given as monotherapy to pediatric patients
    - To evaluate the anti-tumor activity of pixantrone given as monotherapy to pediatric patients across all dose cohorts at or below the MTD
    - To characterize the pharmacokinetics of pixantrone given as monotherapy to pediatric patients (including Cmax, AUC0-24, t1/2, Ctrough, and, Csteadystate)
    - To evaluate treatment effects on cardiac function as assessed by electrocardiogram, echocardiogram (LVEF, LVSF, EA ratio, IVRT, and LV myocardial strain), high sensitivity troponin T, galectin-3, and NTproBNP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient and/or guardian have signed an Informed Consent Form and Assent approved by the Institutional Review Board or Institutional Ethics Committee, as appropriate and necessary, on a per-age basis
    2. Age 6 months to 21 years old (initial qualifying diagnosis must have been made at or before the age
    of 18 and the patient must be under the care of a pediatric hematologist/oncologist)
    3. Patient received a diagnosis of lymphoma or any non-hematologic malignancy (except central nervous system [CNS] tumors) for which the patient is considered relapsed or refractory. (NOTE: CNS metastases are allowable in patients who are deemed not at risk for progression during the first 30 days, who are neurologically stable, and, if on corticosteroids, have been on a stable corticosteroid dose for at least 2 weeks.) Patients who have >1 malignancy ongoing during screening are not eligible
    4. Patient must have one or more of the following treatment statuses:
    • Has failed at least 2 prior lines of chemotherapy
    • Has no curative chemotherapy treatment option available
    • Is not considered a candidate for available chemotherapy treatment options
    5. In dose-escalation accrual, patients may have un-measurable disease (such as bone marrow/bone involvement or diffuse tumors)
    6. In dose-escalation accrual, patients may have un-measurable disease in cases where the standard of care would indicate the need for adjuvant chemotherapy after definitive surgery or radiation, but for whom no standard chemotherapy options are available
    7. In expansion cohort accrual, patients must have disease that is evaluable or measurable for response and progression per standard criteria for their diagnosis (Refer to the Appendices: RECIST 1.1 Criteria for Evaluation of Solid Tumors, Including Neuroblastoma, Appendix 18.4], Evaluation of Neuroblastoma [Appendix 18.6], and the Lymphoma Staging and Disease Response Criteria [Appendix 18.5])
    8. Karnofsky-Lansky performance status (as per age of patient) ≥50 (Appendix 18.1)
    9. Patient must have one or more of the following cardiac function measurements by echocardiogram:
    • Left ventricular ejection fraction (LVEF) ≥55%
    • Left ventricular shortening fraction (LVSF) ≥27%
    10. Hemoglobin ≥8 g/dL (can be post-transfusion)
    11. Platelet count ≥75 × 109/L
    12. Absolute neutrophil count (ANC) ≥0.75 × 109/L
    13. Serum direct/conjugated bilirubin ≤1.5 × upper limit of normal (ULN); patients with clinically diagnosed Gilbert's syndrome and total bilirubin ≤5 × ULN may be enrolled
    14. Aspartate aminotransferase (AST; also called serum glutamicoxaloacetic transaminase [SGOT]) and alanine aminotransferase (ALT; also called serum glutamic-pyruvic transaminase [SGPT]) ≤2.5 × ULN, or ≤5 × ULN if elevation is due to hepatic involvement by tumor
    15. Serum creatinine ≤2 × ULN
    16. Patients must meet the following criteria with respect to prior cancer therapy, radiotherapy, and
    stem cell transplants:
    a.Myelosuppressive chemotherapy: At least 3 weeks must have elapsed since the completion of myelosuppressive chemotherapy (4 weeks if prior nitrosourea) and resolution of all nonhematologic toxicities to ≤ grade 1.
    b.Biologic (anti-neoplastic agent):
    • Oral tyrosine kinase inhibitors or other similar agents. At least 7 days must have elapsed since the completion of therapy with a biologic agent and all non-hematologic toxicities must have resolved to ≤grade 1 prior to enrollment
    • Anti-IGFR-1R and other monoclonal antibodies: the shorter of 3 halflives or 6 weeks must have elapsed since previous monoclonal antibody therapy and resolution of all non-hematologic toxicities to ≤grade 1
    prior to enrollment
    c. Myeloid Growth Factor: Must not have received within 1 week prior to entry into this study (2 weeks in the case of PEG-filgrastim)
    d.Radiotherapy: At least 4 weeks must have elapsed and all nonhematologic toxicities must have resolved to ≤grade 1 prior to enrollment. Previously radiated lesions cannot be used to assess response unless those sites are the sites of disease progression
    e.Stem cell transplant (SCT): For autologous SCT, ≥3 months must have elapsed. For allogeneic SCT, ≥6 months must have elapsed and there must be no evidence of active graft versus host disease
    17. All acute toxicities related to prior treatment recovered to grade ≤1, except alopecia and hematologic parameters specified in the inclusion criteria
    18. Willingness and ability to comply with the visit schedule and assessments required by the study protocol, including effective birth control (Section 5.4) for sexually active patients
    E.4Principal exclusion criteria
    1. Investigator-predicted life expectancy of less than two months
    2. Investigator-predicted inability to tolerate pixantrone monotherapy
    treatment adverse effects for less than two months
    3. Prior anthracycline treatment with a cumulative dose exceeding 450
    mg/m2 (calculated based on doxorubicin equivalents; Appendix 18.3)
    4. Active National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) ≥grade 3 infection, or a lower grade infection deemed resistant or refractory to available antimicrobial agents, or infection requiring ongoing antibiotic treatment
    5. Major surgery ≤7 days and/or with incomplete/inadequate wound healing prior to start of study
    treatment
    6. Known acute or chronic hepatitis B or hepatitis C virus infection
    7. Known seropositivity for human immunodeficiency virus (HIV)
    8. Any experimental/investigational therapy ≤28 days prior to start of
    study treatment
    9. Myocardial infarction within the past 6 months
    10. New York Heart Association class II, III or IV heart failure
    11. Any contraindication, known allergy, or hypersensitivity to any
    investigational drug(s)
    12. Pregnant or lactating
    13. Planned radiotherapy or surgical procedures for the qualifying malignancy
    14. Any psychological, familial, sociological, or geographical condition potentially hampering compliance with the study procedures or followup schedules
    15. Other severe and/or uncontrolled medical disease that could compromise participation in the study, or any medical or psychiatric condition that, in the opinion of the investigator, would make pixantrone administration hazardous or obscure the interpretation of data
    E.5 End points
    E.5.1Primary end point(s)
    Dose Escalation phase:
    - Occurence of suspected DLT

    Expansion Cohort phase:
    - Disease response
    E.5.1.1Timepoint(s) of evaluation of this end point
    Dose Escalation phase:
    Cycle 1 (day 1, 8, 15, 16, 22)

    Expansion Cohort phase:
    - baseline, day 22, end of trial
    E.5.2Secondary end point(s)
    Safety :
    - Number of AEs and SAEs
    - Pharmacokinetics assessments
    E.5.2.1Timepoint(s) of evaluation of this end point
    - baseline, day 1, 8, 15, 16, 22 of each site, End of trial
    - Cycle 1, Day 1
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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
    Dose Escalation 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? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    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 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
    Last Visit last subject
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months6
    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 Yes
    F.1.1Number of subjects for this age range: 35
    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: 16
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 13
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    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 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.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 35
    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
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Children's Hospital Los Angeles
    G.4.3.4Network Country United States
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation ICON Central Laboratories
    G.4.3.4Network Country United States
    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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