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    Summary
    EudraCT Number:2019-002121-30
    Sponsor's Protocol Code Number:1704R2133
    National Competent Authority:Lithuania - SMCA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-11-27
    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 ConcernedLithuania - SMCA
    A.2EudraCT number2019-002121-30
    A.3Full title of the trial
    An Open-label Study with a Nonrandomized Single-dose Phase in Subjects with Suspected or Confirmed Aerobic Gram-negative Bacterial Infections followed by a Randomized, Multiple-dose, Active-controlled Phase in Subjects with Suspected or Confirmed Complicated Urinary Tract Infection (cUTI), Hospital-acquired Bacterial Pneumonia (HABP) or Ventilator-associated Bacterial Pneumonia (VABP) to Assess the Safety, Tolerability, and Pharmacokinetics of Cefiderocol in Hospitalized Pediatric Subjects 3 Months to < 18 Years of Age
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of cefiderocol or standard of care for paediatric patients with confirmed or suspected Gram-negative bacterial infections receiving a single dose and paediatric patents with confirmed or suspected complicated urinary tract infections, hospital-acquired bacterial pneumonia (HABP) or ventilator-associated bacterial pneumonia (VABP) receiving multiple doses to assess the safety, tolerability and exposure of cefiderocol.
    A.3.2Name or abbreviated title of the trial where available
    APEKS-PEDI
    A.4.1Sponsor's protocol code number1704R2133
    A.5.4Other Identifiers
    Name:INDNumber:116,787
    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 SponsorShionogi B.V.
    B.1.3.4CountryNetherlands
    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 supportShionogi & Co., Ltd
    B.4.2CountryJapan
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShionogi & Co., Ltd
    B.5.2Functional name of contact pointCorporate Communications Department
    B.5.3 Address:
    B.5.3.1Street Address1-8, Doshomachi 3-chome
    B.5.3.2Town/ cityChuo-Ku, Osaka
    B.5.3.3Post code541-0045
    B.5.3.4CountryJapan
    B.5.4Telephone number+81662097885
    B.5.6E-mailshionogiclintrials-admin@shionogi.co.jp
    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.3.2Product code S-649266
    D.3.4Pharmaceutical form Powder 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 INNCefiderocol
    D.3.9.3Other descriptive nameS-649266
    D.3.9.4EV Substance CodeSUB131099
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Single dose: Pneumonia, cUTI, complicated intra-abdominal infections [cIAI], hospital acquired bacterial pneumonia [HABP]/ventilator-associated bacterial pneumonia [VABP], and sepsis or bloodstream infections [BSI]).
    Multiple dose: cUTI, HABP/VABP
    E.1.1.1Medical condition in easily understood language
    Pneumonia, urinary tract infections, intra-abdominal infections and sepsi caused by Gram-negative bacterial infections
    E.1.1.2Therapeutic area Diseases [C] - Bacterial Infections and Mycoses [C01]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level SOC
    E.1.2Classification code 10021881
    E.1.2Term Infections and infestations
    E.1.2System Organ Class 10021881 - Infections and infestations
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10076918
    E.1.2Term Hospital acquired pneumonia
    E.1.2System Organ Class 10021881 - Infections and infestations
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10065153
    E.1.2Term Ventilator associated pneumonia
    E.1.2System Organ Class 10021881 - Infections and infestations
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLT
    E.1.2Classification code 10046577
    E.1.2Term Urinary tract infections
    E.1.2System Organ Class 10021881 - Infections and infestations
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10040047
    E.1.2Term Sepsis
    E.1.2System Organ Class 10021881 - Infections and infestations
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 24.1
    E.1.2Level PT
    E.1.2Classification code 10003997
    E.1.2Term Bacteraemia
    E.1.2System Organ Class 10021881 - Infections and infestations
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To assess the safety and tolerability of cefiderocol after single-dose
    administration in hospitalized pediatric subjects 3 months to < 12 years
    of age with suspected or confirmed aerobic Gram-negative bacterial
    infections
    - To assess the pharmacokinetics (PK) of cefiderocol after single-dose
    administration in hospitalized pediatric subjects 3 months to < 12 years
    of age with suspected or confirmed aerobic Gram-negative bacterial
    infections
    - To assess the safety and tolerability of cefiderocol after multiple-dose
    administration in hospitalized pediatric subjects 3 months to < 18 years
    of age with suspected or confirmed cUTI, HABP, or VABP
    - To assess the PK of cefiderocol after multiple-dose administration in
    hospitalized pediatric subjects 3 months to < 18 years of age with
    suspected or confirmed cUTI, HABP, or VABP
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject's parent(s) or legally authorized representative(s) (LAR)[s])
    provides written informed consent in accordance with regional- and
    country-specific laws and regulations.

    2. Subject provides written informed assent, when feasible (age of
    assent to be determined by institutional review board/independent
    ethics committee [IRBs/IECs] or be consistent with local legal
    requirements).

    3. Hospitalized subject is 3 months to < 18 years of age at the time
    written informed consent/assent is obtained for the multiple-dose
    phase. Hospitalized subject is 3 months to < 12 years of age at the time
    written informed consent/assent is obtained for the single-dose phase.
    Premature babies will not be restricted, but the subject must have an
    adjusted or postnatal age of 3 months.

    4. Single-dose phase: Subject has a suspected or confirmed infection (including but not limited to cUTI, cIAI, pneumonia, HABP/VABP, and sepsis or BSI) that requires hospitalization for treatment with IV antibiotics.

    Multiple-dose phase: Subject has a suspected or confirmed cUTI (with a positive urine dipstick test for pyuria and, when possible, microscopy
    demonstrating = 105 colony forming units (CFU)/mL in a midstream urine specimen or = 5 x 104 CFU/mL in a clean catheter catch urine
    specimen) or HABP, or VABP that requires hospitalization for treatment with IV antibiotics.

    5. If subject is a sexually active female of childbearing potential and has
    reached menarche or Tanner stage 3, subject agrees to use barrier
    contraception (including condom, diaphragm, or cervical cap) with
    spermicide or agrees to use a highly effective method of contraception
    (including contraceptive implant, injectable contraceptive, combination
    oral contraceptive, or an intrauterine [IUD] contraceptive device) from
    Screening up to 28 days after administration of the last dose of
    cefiderocol.
    E.4Principal exclusion criteria
    1. Subject has a documented history of any hypersensitivity or allergic
    reaction to any β-lactam antibiotic (Note: for β-lactams, a history of a
    mild rash followed by uneventful re-exposure is not a contraindication to
    enrollment).
    2. Multiple-dose only: Subject has an infection caused only by a
    confirmed Gram-positive pathogen.
    3. Subject has a suspected or confirmed central nervous system (CNS)
    infection (eg, meningitis, brain abscess, shunt infection) or osteomyelitis
    (which would require prolonged antibiotic therapy).
    4. Subject has cystic fibrosis.
    5. Single-dose phase: Subject has moderate or severe renal impairment
    based on estimated glomerular filtration rate (eGFR) (based on the
    Schwartz equation if ≥ 3 months to < 1 year of age and modified Bedside
    Schwartz equation if ≥ 1 to < 18 years of age) of < 60 mL/min/1.73 m2
    at Screening.
    Multiple-dose phase: Subject has an eGFR (based on the Schwartz
    equation if ≥ 3 months to < 1 year of age and modified Bedside Schwartz
    equation if ≥ 1 to < 18 years of age) of < 15 mL/min/1.73 m2 at
    Screening. See Protocol Section 7.5.3.1.2 for eGFR calculations and
    ranges.
    6. Subject has end-stage renal disease (ESRD), is on hemodialysis (HD),
    or receiving continuous venovenous hemofiltration (CVVH).
    7. Subject has experienced shock in the prior month or is in shock at the
    time of Screening.
    8. Subject has severe neutropenia or is severely immunocompromised.
    9. Subject has multiorgan failure.
    10. Subjects with a life expectancy of < 30 days due to severity of a concurrent illness.
    11. Subject is a female who has a positive pregnancy test at Screening.
    12. Subject is a female who is breastfeeding.
    13. Subject has received any other investigational medicinal product
    (IMP) within 30 days.
    14. Subject has any condition or circumstance that, in the opinion of the
    investigator, would compromise the safety of the subject or the quality
    of the study data, including acute trauma to the pelvis or urinary tract.
    15. Subject is receiving vasopressor therapy at screening.
    E.5 End points
    E.5.1Primary end point(s)
    Pharmacokinetics (PK) and
    safety of cefiderocol in subjects 3 months to < 18 years of age.
    E.5.1.1Timepoint(s) of evaluation of this end point
    PK for single dose (cohort 2) - hours 1, 3, 3.5, 5 and 8
    PK for single dose (cohorts 3 and 4) - hours 3, 5 and 8
    PK for multi-dose (cohorts 1 and 2) - hours 1, 3, 3.5, 5 and 8
    PK for multi-dose (cohorts 3 and 4) - hours 3, 5 and 8
    E.5.2Secondary end point(s)
    NA
    E.5.2.1Timepoint(s) of evaluation of this end point
    NA
    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) 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Standard of care
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    Mexico
    Panama
    Philippines
    United States
    Lithuania
    Spain
    Czechia
    Greece
    Ukraine
    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 years3
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months9
    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: 85
    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: 44
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 25
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects may not be able to understand or personally sign ICF at the time of screening due to their young age and/or medical condition.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 85
    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)
    Patents withdrawn or discontinued from study treatment should receive additional standard of care antibiotic therapy if n the judgement of the investigator such treatment is clinically indicated.
    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 PENTA ID Innovation
    G.4.3.4Network Country Italy
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-04-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-01
    P. End of Trial
    P.End of Trial StatusOngoing
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