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    Summary
    EudraCT Number:2021-001577-24
    Sponsor's Protocol Code Number:NI-0501-14
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-10-14
    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 ConcernedGermany - PEI
    A.2EudraCT number2021-001577-24
    A.3Full title of the trial
    A two-cohort, open-label, single arm, multicenter study to evaluate efficacy, safety and tolerability, pharmacokinetics and pharmacodynamics, of emapalumab in children and adults with macrophage activation syndrome (MAS) in Still’s disease (including systemic juvenile idiopathic arthritis and Adult onset Still’s disease) or with MAS in Systemic lupus erythematous
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate efficacy, safety and tolerability, pharmacokinetics and pharmacodynamics, of emapalumab in children and adults with macrophage activation syndrome (MAS)
    A.3.2Name or abbreviated title of the trial where available
    NI-0501-14
    A.4.1Sponsor's protocol code numberNI-0501-14
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/109/2020
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSwedish Orphan Biovitrum AG (Sobi AG)
    B.1.3.4CountrySwitzerland
    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 AG (Sobi AG)
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSwedish Orphan Biovitrum AG (Sobi AG)
    B.5.2Functional name of contact pointClinical Development Swedish Orphan
    B.5.3 Address:
    B.5.3.1Street AddressChemin des Aulx 12
    B.5.3.2Town/ cityPlan-les-Ouates
    B.5.3.3Post code1228
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number0041225519163
    B.5.6E-mailRAclinical@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 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/3/10/749
    D.3 Description of the IMP
    D.3.1Product nameEmapalumab
    D.3.2Product code NI-0501
    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 INNEmapalumab
    D.3.9.2Current sponsor codeNI-0501
    D.3.9.4EV Substance CodeSUB188645
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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.IMP: 2
    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/3/10/749
    D.3 Description of the IMP
    D.3.1Product nameEmapalumab
    D.3.2Product code NI-0501
    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 INNEmapalumab
    D.3.9.2Current sponsor codeNI-0501
    D.3.9.4EV Substance CodeSUB188645
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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
    Macrophage activation syndrome (MAS) in the context of Systemic juvenile inflammatory arthritis (sJIA) and Adult onset Still’s disease (AOSD).
    MAS in the context of pediatric and adult Systemic lupus erythematous (SLE).
    E.1.1.1Medical condition in easily understood language
    Children and adults with macrophage activation syndrome (MAS)
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10071583
    E.1.2Term Haemophagocytic lymphohistiocytosis
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate efficacy of emapalumab in the treatment of patients in:
    - Cohort 1: Macrophage Activation Syndrome (MAS) in the context of systemic juvenile idiopathic arthritis and adult onset Still's disease
    (AOSD).
    - Cohort 2: MAS in the context of pediatric and adult systemic lupus
    erythematosus (SLE).
    E.2.2Secondary objectives of the trial
    - To demonstrate efficacy of emapalumab with respect to tapering of glucocorticoids (GCs).
    - To evaluate the time to onset of response to emapalumab treatment.
    - To evaluate efficacy of emapalumab with respect to overall response
    (OR).
    - To evaluate the sustained efficacy of emapalumab treatment.
    - To evaluate the patient's survival after treatment with emapalumab.
    - To evaluate the safety and tolerability of emapalumab.
    - To evaluate patient-reported outcome of MAS in patients treated with emapalumab.
    - To determine the pharmacokinetic (PK) profile of emapalumab.
    - To determine the pharmacodynamic (PD) profile of emapalumab.
    - To determine the immunogenicity of emapalumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Run-in phase in all cohorts
    1. Informed consent provided by the patient or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as required by local law.
    2. Male and female patients aged between 6 months and 80 years of age at the time of diagnosis of active MAS.
    3. MAS defined as per the criteria defined below for each cohort and
    requiring treatment with GCs as per standard of care.
    Interventional phase in all cohorts
    1. Informed consent provided by the patient or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as as required by local law.
    2. Male and female patients aged between 6 months and 80 years of age at the time of diagnosis of active MAS.
    3. Patients who have shown an inadequate response to high dose intravenous (i.v.) GCs administered for at least 3 days according to local standard clinical practice, including but not limited to pulses of 30 mg/kg methylprednisolone (mPDN) on 3 consecutive days. High i.v. GCs dose is recommended not to be lower than 2 mg/kg/ day PDN equivalent (or at least 60 mg/day in pediatric patients of 30 kg or more, and at least 1 g/day in adult MAS patients). In case of rapid worsening of the patient's condition and/or laboratory parameters, as per Investigator judgment, inclusion may occur within less than 3 days from starting high dose GCs.
    4. Diagnosis of active MAS confirmed by the treating rheumatologist,
    having ascertained the followings:
    a. Febrile patients presenting with ferritin > 684 ng/mL.
    b. and any 2 of:
    i. Platelet count ≤ 181 x109/L
    ii. Aspartate aminotransferase (AST)-level > 48 U/L
    iii. Triglycerides > 156 mg/dL
    iv. Fibrinogen level ≤ 360 mg/dL
    5. Female patients of child-bearing potential (sexually or non-sexually
    active). Female patients who are sexually active must be willing to use highly effective methods of contraception from study drug initiation to 6 months after the last dose of study drug.
    Specific inclusion criteria for Cohort 1 and Cohort 2
    6. Cohort 1:
    a. Confirmed sJIA diagnosis. For patients presenting with MAS in the context of the onset of sJIA, high presumption of sJIA will suffice for eligibility.
    b. Confirmed diagnosis of AOSD as per Yamaguchi criteria (Yamaguchi et al. 1992)
    7. Cohort 2:
    a. Confirmed diagnosis of SLE as per Systemic Lupus International Collaborating Clinics (SLICC) 2012 criteria.
    E.4Principal exclusion criteria
    1. Primary hemophagocytic lymphohistiocytosis (p-HLH) documented by either the presence of a known causative genetic mutation or abnormal perforin expression or CD107a degranulation assay as described with p-HLH or by the presence of family history.
    2. Confirmed malignancy. Note: patients with a suspected malignancy should have mononuclear cells typed by flow cytometry and/or tissue biopsy, as applicable, to rule out malignancy.
    3. Treatment with canakinumab, Janus kinase (JAK) inhibitors, Tumour necrosis factor (TNF) inhibitors and tocilizumab at the time of emapalumab initiation.
    4. Ongoing treatment with anakinra at a dose above 4 mg/kg/ day at time of emapalumab initiation.
    5. Patients treated with etoposide for MAS in the last 1 month.
    6. Presence of any medical or psychological condition or laboratory result that in the opinion of the Investigator can interfere with the patient's ability to comply with the protocol requirements or makes the patient not appropriate for inclusion to the study and treatment with emapalumab.
    7. Foreseeable inability to cooperate with given instructions or study
    procedures.
    8. Clinically active mycobacteria (typical and atypical), Histoplasma Capsulatum, or Salmonella infections.
    9. Evidence of leishmania infections.
    10. Evidence of latent tuberculosis.
    11. History of hypersensitivity or allergy to any component of the study
    drug.
    12. Receipt of a Bacillus Calmette-Guerin (BCG) vaccine within 12 weeks prior to screening.
    13. Receipt of a live or attenuated live (other than BCG) vaccine within 4 weeks prior to screening.
    14. Pregnancy or lactating female patients.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients with complete response (CR) at Week 8 after first administration of emapalumab.
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 8
    E.5.2Secondary end point(s)
    - GCs tapering to a dose < 50 % of prednisolone (PDN) equivalent at the time of emapalumab start or to the same (or lower) dose being administered before the occurrence of MAS (in patients already treated for the underlying condition) whichever occurs first at any time during the study.
    - GCs tapering to ≤ 1 mg/kg/day of PDN equivalent at any time during
    the study.
    - Time to achieve GCs tapering as defined in the 2 bullets above.
    - Time to first CR.
    - Proportion of patients with overall response (OR) as defined by CR or PR (partial response).
    - Time to first overall response (OR) as defined by CR or PR.
    - MAS recurrence at any time after achievement of CR.
    - Withdrawal from the study due to lack of response as per Investigator decision.
    - Survival time.
    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 No
    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) 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.8.2.4Number of treatment arms in the trial1
    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 EEA19
    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
    Canada
    China
    Japan
    United States
    Russian Federation
    Belgium
    Czechia
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    Sweden
    United Kingdom
    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 days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months4
    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: 22
    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: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 26
    F.4.2.2In the whole clinical trial 41
    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)
    return to 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 Decision2022-08-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-10-27
    P. End of Trial
    P.End of Trial StatusOngoing
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