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    Summary
    EudraCT Number:2024-000321-41
    Sponsor's Protocol Code Number:P-Monofer-PED-01
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2024-10-15
    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 number2024-000321-41
    A.3Full title of the trial
    A phase III, prospective, open-label, multi-center trial of ferric derisomaltose in children 0 to <18 years of age with iron deficiency anemia due to non-dialysis dependent chronic kidney disease or with iron deficiency anemia who are intolerant or unresponsive to oral iron.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase III, prospective, open-label, multi-center trial of ferric derisomaltose in children 0 to <18 years of age with iron deficiency anemia due to non-dialysis dependent chronic kidney disease or with iron deficiency anemia who are intolerant or unresponsive to oral iron.
    A.4.1Sponsor's protocol code numberP-Monofer-PED-01
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1266-9073
    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 SponsorPharmacosmos A/S
    B.1.3.4CountryDenmark
    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 supportPharmacosmos A/S
    B.4.2CountryDenmark
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPharmacosmos A/S
    B.5.2Functional name of contact pointClinical R&D
    B.5.3 Address:
    B.5.3.1Street AddressRørvangsvej 30
    B.5.3.2Town/ cityHolbæk
    B.5.3.3Post code4300
    B.5.3.4CountryDenmark
    B.5.6E-mailmen@pharmacosmos.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 Monoferric
    D.2.1.1.2Name of the Marketing Authorisation holderPharmacosmos A/S
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameMonoferric
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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
    Iron deficiency anemia due to non-dialysis dependent chronic kidney disease or iron deficiency anemia who are intolerant or unresponsive to oral iron.
    E.1.1.1Medical condition in easily understood language
    Iron deficiency anemia due to non-dialysis dependent chronic kidney disease or iron deficiency anemia who are intolerant or unresponsive to oral iron.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    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
    Ability of IV ferric derisomaltose to increase Hb.
    E.2.2Secondary objectives of the trial
    Effect of ferric derisomaltose on relevant iron related biochemical parameters.
    PK parameters (only obtained from the first 8 subjects in each age cohort).
    Safety of IV ferric derisomaltose.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A subject will be eligible for inclusion in the trial if he/she fulfils the following criteria:
    1. Subjects <18 years
    2. IDA caused by different etiologies such as gastrointestinal disease, NDD-CKD, or other conditions leading to IDA*
    3. Hb concentration less than the 5th percentile for age and sex-specific reference range (Appendix B)
    4. Subjects with NDD-CKD (a) or who are intolerant or unresponsive to oral iron (b):
    a) NDD-CKD:
    TSAT ≤35 % or s-ferritin <100 ng/mL
    eGFR <60 mL/min/1.73m2
    If on ESA, receiving stable ESA regimen defined as dose adjustments no more than ± 20 % for ≥8 weeks prior to screening
    b) Documented history of intolerance or unresponsiveness to oral iron therapy** for at least one month*** prior to trial enrolment. The subject would not be a candidate for oral iron again:
    TSAT ≤20 % or s-ferritin <100 ng/mL
    5) Informed consent and child assent, as age-appropriate, obtained before any trial-related activities and willingness to participate. LAR of the subject must sign and date the ICF (according to local requirements). The child must sign and date the CAF or provide oral assent, if required according to local requirements
    *The etiology (also if unknown) for IDA should be documented in the medical history and verified in the source document.
    **The intolerance and non-response to oral iron treatment should be documented with sign and symptoms in the medical history and verified in the source document.
    ***There should be a documentation of intolerance or unresponsiveness to at least one month of prescribed oral iron therapy per Investigator’s judgment within the last 9 months and the subject would not be candidate for oral iron again. The therapy should have been intended for at least one month, but not necessarily administered for one month.
    E.4Principal exclusion criteria
    A subject will not be eligible for inclusion in this trial if he/she fulfils any of the following criteria:
    1. Anemia caused by factors other than IDA according to Investigator's judgment
    2. S-ferritin >600 ng/mL
    3. Hb ≤5.0 g/dL
    4. Iron overload or disturbances in utilization of iron (e.g. hemochromatosis and hemosiderosis)
    5. ALAT and/or ASAT >2 times upper limit of normal (e.g. decompensated liver cirrhosis or active hepatitis)
    6. Pregnant or nursing female subjects. In order to avoid pregnancy, female subjects of childbearing potential have to use adequate contraception (e.g. intrauterine devices, hormonal contraceptives, or double barrier method) or be abstinent during the whole trial period and 7 days after the last dosing. Childbearing potential refers to all female subjects ≥12 years old or <12 years old who have started menstruating
    7. Previous serious hypersensitivity reactions to any IV iron compounds including ferric derisomaltose
    8. Received an investigational drug within 30 days prior to screening
    9. Treatment with IV iron within 10 days prior to screening
    10. Treatment with blood transfusion, radiotherapy, chemotherapy or other drugs that suppress the bone marrow, and drugs which have anemia as side effect within 30 days prior to screening
    11. Planned elective surgery (or planned surgery during the trial period) where significant blood loss is expected within the last 30 days prior to screening
    12. Any non-viral infection (non-viral infection that has been fully treated before the baseline visit is accepted)
    13. Any other laboratory abnormality, medical condition, or psychiatric disorders which, in the opinion of the Investigator, will put the subject’s disease management at risk or may result in the subject being unable to comply with the trial requirements
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of subjects with a Hb increase of ≥1 g/dL (NDD-CKD) or 2 g/dL (intolerant or unresponsive to oral iron) from baseline at any time from week 1 to week 8 in the absence of transfusions or ESA dose change or other iron supplementation.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline at any time from week 1 to week 8.
    E.5.2Secondary end point(s)
    • Time to increase Hb ≥1 g/dL (NDD-CKD) or 2 g/dL (intolerant or unresponsive to oral iron)
    • Incidence of subjects who achieve a serum (s-) ferritin of ≥100 ng/mL at weeks 1, 2, 4, and 8
    • Incidence of subjects who achieve a transferrin saturation (TSAT) of ≥35 % (NDD-CKD) or ≥20 % (intolerant or unresponsive to oral iron) at weeks 1, 2, 4, and 8
    • Change in Hb, s-ferritin, TSAT, and s-iron from baseline to weeks 1, 2, 4, and 8
    E.5.2.1Timepoint(s) of evaluation of this end point
    At weeks 1, 2, 4, and 8
    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
    Pharmacokinetic
    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) 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    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 Yes
    F.1.1Number of subjects for this age range: 200
    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.1.1.3.1Number of subjects for this age range: 2
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 18
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 80
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 100
    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 200
    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)
    None
    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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