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    Summary
    EudraCT Number:2015-002526-39
    Sponsor's Protocol Code Number:20010133
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2015-07-02
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2015-002526-39
    A.3Full title of the trial
    A Phase 1 Dose-escalation Study to Evaluate the Safety and Pharmacokinetics (PK) of Palifermin in Pediatric Subjects with Acute Leukemias Undergoing Myeloblative Therapy and Allogeneic Hematopoietic Stem Cell Transplant (HSCT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    To determine a safe and tolerable dose of palifermin in pediatric subjects of different age groups when administered once daily for 3 consecutive days before and 3 consecutive days after a myeloablative chemo-radiotherapy conditioning regimen with allogeneic hematopoietic stem cell transplant (HSCT).
    A.4.1Sponsor's protocol code number20010133
    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 SponsorSwedish Orphan Biovitrum AB (publ)
    B.1.3.4CountrySweden
    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 supportSwedish Orphan Biovitrum AB (publ)
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSwedish Orphan Biovitrum AB (publ)
    B.5.2Functional name of contact pointIda Gunnarsson
    B.5.3 Address:
    B.5.3.1Street AddressTomtebodavägen 23A
    B.5.3.2Town/ cityStockholm
    B.5.3.3Post code112 76
    B.5.3.4CountrySweden
    B.5.4Telephone number+46 8697 38 78
    B.5.6E-mailida.gunnarsson@sobi.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 Kepivance
    D.2.1.1.2Name of the Marketing Authorisation holderSwedish Orphan Biovitrum AB (publ)
    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.3.1Product nameKepivance
    D.3.2Product code ATC V03AF08
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous bolus use (Noncurrent)
    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
    Oral Mucositis
    E.1.1.1Medical condition in easily understood language
    Inflammation and ulceration in the mouth as complication of cancer treatment
    E.1.1.2Therapeutic area Diseases [C] - Bacterial Infections and Mycoses [C01]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine a safe and tolerable dose of palifermin in pediatric subjects of different age groups when administered once daily for 3 consecutive days before and 3 consecutive days after a myeloablative chemo-radiotherapy conditioning regimen with allogeneic HSCT.
    E.2.2Secondary objectives of the trial
    • To define the safety profile of palifermin over a range of doses in pediatric subjects with leukemia undergoing allogeneic HSCT
    • To characterize the pharmacokinetic (PK) profile of intravenous (IV) bolus injections of palifermin for multiple dose levels in pediatric subjects
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. ALL or AML requiring HSCT
    2. Age ≥ 1 and ≤ 16 years at screening
    3. Lansky performance status > 60%
    4. Candidate for allogeneic HSCT protocol:
    • Adequate kidney function: Serum creatinine: ≤ 1.5 mg/dL or creatinine clearance or radioisotope GFR ≥ 60 mL/min/1.73m2
    • Adequate liver function: Serum total bilirubin: ≤ 2.0 mg/dl; AST/ALT ≤ 4.0 x institutional upper limits of normal (IULN); Albumin ≥ 2 g/dL
    • Adequate cardiac function: shortening fraction > 29% documented by echocardiogram, or ejection fraction ≥ 50% documented by multigated acquisition scan (MUGA).
    • Adequate pulmonary function documented by corrected DLCO > 50% or oxygen saturation of
    ≥ 92% on room air if unable to perform pulmonary function tests
    • Negative for human immunodeficiency virus (HIV), hepatitis C virus (HCV), human T cell lymphotropic virus (HTLV)
    5. Identification of an HLA-compatible donor per institutional standards
    6. Assent from a minor (if the child is capable of giving assent) per Department of Health and Human Services (DHHS) guidelines listed in 21CFR 50.55 and local IRB standards.
    7. Serum amylase and lipase: ≤ 1.2 x IULN
    8. Negative serum/urine pregnancy test for females with childbearing potential within 4 days before administration of the first palifermin dose
    9. Agreement by males and females of reproductive potential to use an effective means of contraception 30 days prior to enrollment through Day +30 (end of treatment)
    E.4Principal exclusion criteria
    1. Prior treatment with palifermin or other keratinocyte growth factors
    2. Received an investigational product or device, with the exception investigational stem cell separators, in another clinical trial within 30 days before enrollment.
    3. Known to have a life threatening infection not responding well to treatment
    4. Past history of veno-occlusive disease of the liver
    5. Known sensitivity to any Escherichia coli-derived products with grade 3 to 4 allergies to
    L-asparaginase [grade 1 to 2 allergies to
    L-asparaginase will be allowed].
    6. Receiving glutamine or any other medication to reduce the incidence of OM within 30 days before enrollment
    7. Previous or concurrent malignancy other than entry diagnostic criteria and/or solid organ transplantation and/or treatment of congenital immunodeficiency
    8. History of pancreatitis
    9. Breastfeeding (giving)
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of Dose limiting toxicity
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day -10 to +16
    E.5.2Secondary end point(s)
    A Incidence and severity of AEs, change in vital signs, and incidence of laboratory abnormalities
    B PK parameters of palifermin (eg, CL, Vss, t ½z) after the 1st and 3rd IV bolus injection for multiple dose levels
    C Incidence of serum palifermin antibody formation
    E.5.2.1Timepoint(s) of evaluation of this end point
    A Day -10 to +16
    B After 1st and 3rd bolus dose
    C 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
    Single arm, open-label, dose escalation design
    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 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years10
    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: 54
    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: 9
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 9
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 9
    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
    Children under the age of 5
    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 54
    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)
    Normal treatment of that condition
    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 Pediatric Blood and Marrow Transplant Consortium
    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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