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    Summary
    EudraCT Number:2014-003233-26
    Sponsor's Protocol Code Number:CD-ON-CAT-8015-1053
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-05-26
    Trial results View 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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2014-003233-26
    A.3Full title of the trial
    A Pivotal Multicenter Trial of Moxetumomab Pasudotox in Relapsed/Refractory Hairy Cell Leukemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical Trial of Investigational Agent (Moxetumomab Pasudotox) in
    Patients with Relapsed/ Refractory Hairy Cell Leukemia
    A.4.1Sponsor's protocol code numberCD-ON-CAT-8015-1053
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01829711
    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 SponsorMedImmune, LLC
    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 supportMedImmune, LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedImmune, LLC
    B.5.2Functional name of contact pointInformation Center
    B.5.3 Address:
    B.5.3.1Street Addressn/a
    B.5.3.2Town/ cityn/a
    B.5.3.3Post coden/a
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrialenquiries@medimmune.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/3/08/592
    D.3 Description of the IMP
    D.3.1Product nameMoxetumomab Pasudotox
    D.3.2Product code CAT-8015
    D.3.4Pharmaceutical form Concentrate 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 INNMoxetumomab Pasudotox
    D.3.9.1CAS number 1020748-57-5
    D.3.9.2Current sponsor codeCAT-8015
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number0.90 to 1.10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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 Yes
    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
    Hairy Cell Leukemia
    E.1.1.1Medical condition in easily understood language
    A specific type of cancer of the blood
    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 10019055
    E.1.2Term Hairy cell leukemia
    E.1.2System Organ Class 100000012994
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10019056
    E.1.2Term Hairy cell leukemia variant
    E.1.2System Organ Class 100000012994
    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 the rate of durable complete response (CR) in multiply relapsed HCL with moxetumomab pasudotox.
    E.2.2Secondary objectives of the trial
    -Determine the overall response rate (ORR), progression-free survival (PFS), time to treatment failure (TTF), and duration of responses (CR and partial response [PR]).
    -Confirm the tolerability and safety of moxetumomab pasudotox in patients with HCL.
    -Evaluate immunogenicity and pharmacokinetics (PK) of moxetumomab pasudotox.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients must have histologically confirmed hairy cell leukemia or hairy cell leukemia variant with a need for therapy based on at least one of the following criteria:
    a. neutrophils < 1.0x 10(9)/L
    b. platelets < 100 x 10(9)/L
    c. hemoglobin < 10 g/dL
    d. symptomatic splenomegaly
    2. Patients must be Pseudomonas-immunotoxin naïve
    3. Patients must have received at least 2 prior systemic therapies, including 2 courses of courses of a PNA, or 1 course of either rituximab or BRAF inhibitor following a single prior course of PNA.
    4. Men or women age ≥ 18 years. Because this disease does not generally occur in children, children are excluded from this study.
    5. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
    6. Patients must have adequate organ function as defined below:
    a. total bilirubin ≤ 1.5 mg/dL, unless consistent with Gilbert’s (ratio between total and direct bilirubin > 5)
    b. AST and ALT ≤ 3x upper limit of normal (ULN)
    c. alkaline phosphatase ≤ 2.5 ULN
    d. serum creatinine ≤ 1.5 mg/dL or creatinine clearance ≥ 60 mL/min as estimated by the Cockcroft-Gault equation
    7. Prothrombin time (PT)/international normalized ratio (INR) or partial thromboplastin time (PTT) < 2.5 ULN, fibrinogen ≥ 0.5 lower limit of normal; if on warfarin, INR < 3.5, if on any other anticoagulation, PT < 2.5 x baseline
    8. Ability to understand and the willingness to sign a written informed consent document.
    9. Life expectancy ≥ 6 months.
    10. Females of childbearing potential who are sexually active with a nonsterilized male partner must use a highly effective method of contraception prior to study entry and or the duration of study participation, and must agree to continue using such precautions for 4 months after completion of moxetumomab pasudotox administration; cessation of contraception after this point should be discussed with a responsible physician. Periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of contraception.
    ◦ Females of childbearing potential are defined as those who are not surgically sterile (ie, bilateral tubal ligation, bilateral oophorectomy, or complete hysterectomy) or those who are premenarchal or postmenopausal (defined as 12 months with no menses without an alternative medical cause).
    ◦ A highly effective method of contraception is defined as one that results in a low failure rate (ie, less than 1% per year) when used consistently and correctly.
    ◦ Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
    11. Nonsterilized males who are sexually active with a female partner of childbearing potential must use a highly effective method of contraception from Day 1 for 60 days after receipt of the final dose of investigational product.
    E.4Principal exclusion criteria
    1. Patients who have had chemotherapy, immunotherapy or radiotherapy within 4 weeks prior to initiation of treatment.
    2. Patients who are receiving any other investigational agents.
    3. Patients with known brain metastases should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events (AEs).
    4. Patients with retinal or choroidal detachment identified during the screening ophthalmologic evaluation.
    5. Pregnant or breastfeeding females. The effects of moxetumomab pasudotox on the developing fetus are unknown. Because there is an unknown but potential risk for AEs in nursing infants secondary to treatment of the mother with moxetumomab pasudotox breastfeeding should be discontinued if the mother is treated with moxetumomab pasudotox.
    6. Positive for Hepatitis B core antibody or surface antigen unless the patient is on Lamivudine or Entecavir and Hepatitis B Viral DNA load is < 2000 IU/mL.
    7. Active second malignancy requiring treatment other than minor resection of indolent cancers like basal cell and squamous skin cancers.
    8. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, uncontrolled hypertension, cardiac arrhythmia, malaria infection, or psychiatric illness/social situations that would limit compliance with study requirements.
    9. Known human immunodeficiency virus (HIV)-positive patients unless taking appropriate anti-HIV medications with a CD4 count of > 200. Otherwise, there may be increased risk of lethal infections when temporarily suppressing normal B-cells.
    10. History of allogeneic bone marrow transplant.
    11. Patients with history of both thromboembolism and known congenital hypercoagulable conditions.
    12. Uncontrolled pulmonary infection, pulmonary edema
    13. Oxygen saturation at rest < 88% measured by pulse oximetry or PaO2 ≤ 55 mm Hg
    14. Serum albumin < 2 g/dL
    15. Radioimmunotherapy within 2 years prior to enrollment in the study.
    16. Absolute neutrophil count (ANC) < 1.0 x 10(9)/L, or platelet count < 50 x 10(9)/L, unless judged by the investigator to be due to underlying disease (ie, potentially reversible with anti-neoplastic therapy). A patient will not be excluded because of pancytopenia ≥ Grade 3, or erythropoietin dependence, if due to disease, based on the results of bone marrow studies.
    17. Patients with < 50% of predicted forced expiratory volume (FEV1) or < 50% of predicted diffusing capacity for carbon monoxide, corrected for hemoglobin concentration and alveolar volume (DLCO). Note: Patients with no prior history of pulmonary illness are not required to have pulmonary function testing (PFT). Forced expiratory volume will be assessed after bronchodilator therapy.
    18. Patients with history of thrombotic microangiopathy or thrombotic microangiopathy / HUS.
    19. Patients with corrected QT interval (Frederica) elevation > 500 msec (manually over-read by medically qualified person) based on at least two separate 12-lead ECGs.
    20. Patients on high dose estrogen (defined as > 0.625 mg/day of an estrogen compound).
    21. Patients with clinical evidence of disseminated intravascular coagulation (Grade 3-4)
    E.5 End points
    E.5.1Primary end point(s)
    Durable Complete response
    E.5.1.1Timepoint(s) of evaluation of this end point
    Approximately 12 months from the start of treatment
    E.5.2Secondary end point(s)
    Overall Response Rate (ORR), progression free survival (PFS), time to treatment failute, duration fo response (CR and PR), Safety, Immunogenicty, and Pharmokokinetics
    E.5.2.1Timepoint(s) of evaluation of this end point
    Duration of study
    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 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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA50
    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
    Argentina
    Belgium
    Brazil
    Canada
    Czech Republic
    Denmark
    Finland
    France
    Germany
    Greece
    Ireland
    Israel
    Italy
    Mexico
    Netherlands
    Norway
    Poland
    Portugal
    Spain
    Sweden
    United Kingdom
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    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: 70
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 7
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Elderly
    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 55
    F.4.2.2In the whole clinical trial 77
    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
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-07-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-05-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-04-29
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