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    Summary
    EudraCT Number:2018-004553-25
    Sponsor's Protocol Code Number:CCFZ533X2207
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-06-17
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2018-004553-25
    A.3Full title of the trial
    Investigator- and subject-blinded, randomized, placebo-controlled study to evaluate safety, tolerability, pharmacokinetics and efficacy of CFZ533 in pediatric and young adults with new onset type 1 diabetes mellitus (T1DM)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of safety and efficacy of CFZ533 in type 1 diabetes pediatric and young adult subjects
    A.4.1Sponsor's protocol code numberCCFZ533X2207
    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 SponsorNovartis Pharma 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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma AG
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressForum 1, Novartis Campus
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4056
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+41 61 324 1111
    B.5.5Fax number+41 61 324 8001
    B.5.6E-mailclinicaltrial.enquiries@novartis.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameIscalimab
    D.3.2Product code CFZ533
    D.3.4Pharmaceutical form Concentrate for solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNiscalimab
    D.3.9.2Current sponsor codeCFZ533
    D.3.9.3Other descriptive nameCFZ533
    D.3.9.4EV Substance CodeSUB130512
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection/infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Treatment of subjects with type 1 diabetes mellitus with residual beta cell function (RBCF), with the goal of preserving RBCF
    E.1.1.1Medical condition in easily understood language
    Type 1 diabetes mellitus
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10067584
    E.1.2Term Type 1 diabetes mellitus
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To evaluate the safety and tolerability of CFZ533 in subjects with new onset T1DM.
    • To evaluate effects of CFZ533 on pancreatic beta cell function in subjects with new-onset T1DM.
    E.2.2Secondary objectives of the trial
    • To evaluate the pharmacokinetics of CFZ533 in subjects with new onset T1DM.
    • To evaluate the treatment effect of CFZ533 on remission or partial remission in subjects with new onset T1DM.
    • To evaluate durability of effects of CFZ533 on pancreatic beta cell function in subjects with new-onset T1DM.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Willing to provide written informed consent, and if needed assent from the child on the trial.
    2. Males and females aged between 6 and 21 years (inclusive, and enrolled in stages) at screening.
    3. Body weight range from 20 to 125 kg (inclusive).
    4. Newly diagnosed auto immune type 1 diabetes confirmed by at least one positive auto antibody:glutamic acid decarboxylase (anti-GAD65), protein tyrosine phosphatase-like protein (anti-IA-2); zinc transporter 8 (anti-ZnT8); islet cell (cytoplasmic) (anti-ICA).
    5. Able to receive first dose of study drug within 56 days of diagnosis of T1DM (which may be extended to within 100 days of diagnosis in the event a screening assessment needs to be confirmed or vaccine administered.
    6. Peak stimulated C-peptide levels ≥0.2 nmol/L (0.6 ng/mL) following standard liquid mixed meal tolerance test (MMTT), to be conducted when the subject is metabolically stable, at least 2 weeks from diagnosis and within 56 days prior to randomization (or within 100 days of diagnosis in the event a screening assessment needs to be confirmed or vaccine is required).
    7. Study participants are to complete all recommended immunizations at least 4 weeks prior to first dose with study drug and in accordance with local immunization guidelines. Immunization with a live vaccine must be done at least 4 months prior to the first dose. In the event a subject has not had all vaccinations recommended according to local guidance, the screening period may be extended beyond 56 days to allow these vaccinations to be administered, but first dose of study drug must be administered within 100 days of diagnosis of T1DM. Must be willing to comply with the standard of care for diabetes management.
    8. A negative pregnancy test at screening is required for all sexually mature female subjects prior to participation in the study.
    9. Subject and/or guardian must be able to communicate well with the investigator, to understand and comply with the requirements of the study.
    E.4Principal exclusion criteria
    1. Diabetes forms other than auto immune type 1 such as maturity-onset diabetes of the young (MODY), latent autoimmune diabetes of the adult (LADA), acquired diabetes (secondary to medications or surgery), type 2 diabetes by judgement of the investigator.
    2. Diabetic ketoacidosis within 2 weeks of the baseline MMTT test.
    3. Polyglandular auto immune disease, Addison's disease, pernicious anemia, celiac sprue.
    4. Any of the following abnormal laboratory values at screening
    • total white blood cell count (WBC) outside the range 1,500-
    15,000/mm3(1.5-15.0 x 109/L)
    • neutrophil count(<1500/mm3)(<1.5 X 109 / L)
    • lymphocyte count<500/mm3(<0.5 X 109 / L)
    • hemoglobin (Hgb)<8.0 g/dL
    • platelets<100,000/mm3(<100 x 109/L)
    5. History of immunodeficiency disorders, such as HyperIgM syndrome; history of recurrent infections suggestive of immunodeficiency disorders.
    6. History of or active coagulation disorder with increased thromboembolic risk; aPTT and PT/ INR below lower limit of normal prior to inclusion.
    7. Tuberculosis infection assessed by positive QuantiFERON TB-Gold test(QFT) at screening. Subjects with a positive QFT test may participate in the study if further work up establishes conclusively that the subject has no evidence of active tuberculosis. If presence of latent tuberculosis is established, then anti tuberculosis treatment must have been initiated and maintained according to local country guidelines
    8. Chronic infection with Hepatitis B(HBV) or Hepatitis C(HCV). A positive HBV surface antigen(HBsAg) test at screening excludes a subject. Subjects with a positive HCV antibody test should have HCV RNA levels measured. Subjects with positive(detectable) HCV RNA should be excluded.
    9. Positive human immune virus test(ELISA and Western Blot) at screening.
    10. Evidence of EBV, CMV, HSV, and/or SARS-CoV-2 infection by viral load above laboratory upper limit of normal or only positive IgM serology in the absence of positive IgG at screening. Rescreening permitted in persistently asymptomatic or post-symptomatic subjects, but study drug must be able to be administered within 100 days of diagnosis of T1D.
    11. Major dental work(tooth extractions or dental surgery with access to dental pulp) within 8 days of first dose; febrile illness within 48 hrs of first dose.
    12. Use of other investigational drugs or use of immunosuppressive agents at the time of enrollment, or within 5 half-lives of enrollment, or until the expected PD effect has returned to baseline, whichever is longer; or longer if required by local regulations.
    13. History of multiple and recurring allergies or allergy to the investigational compound/compound class being used in this study.
    14. History of severe hypersensitivity reaction or anaphylaxis to biological agents, e.g. human monoclonal antibody.
    15. History of malignancy of any organ system(other than localized basal cell carcinoma of the skin), treated or untreated, within 5 years of screening, regardless of whether there is evidence of local recurrence or metastases.
    16. Active serious psychiatric disorders(diagnosed or treated by a psychiatrist), such as eating disorders and psychosis or history thereof.
    17. Any complicating medical issues or clinically abnormal laboratory results that may cause an increased safety risk to the subject as judged by the investigator.
    18. Ongoing, and up to 2 weeks prior to screening, use of medications that may affect glucose control (e.g, systemic steroids, thiazides, beta blockers). A short course of oral steroids<10 days if medically required is permissible with sponsor notification.
    19. History of drug abuse, nicotine or harmful alcohol use within 12 months prior to first dose, or evidence as determined by the investigators of such abuse at screening. For example, harmful alcohol use in adults is defined as five or more drinks per day for 5 or more days in the past 30 days. Harmful alcohol use by adolescents age 13-18 years
    is to be determined by the investigator, based on local culture and laws.
    Any alcohol use by children age 6-12 is a disqualifier.
    20. Use of any prescription drugs other than those allowed by this protocol Section 6 herbal supplements, prescribed medicinal use of cannabis/marijuana and/or over-the-counter(OTC) medication, dietary supplements(vitamins included) within two weeks prior to first dose. If needed, (i.e. an incidental and limited need) paracetamol/acetaminophen is acceptable, but must be documented in the Concomitant medications/Significant nondrug therapies page of the CRF.
    21. Taking medications prohibited by the protocol.
    22. Pregnant or nursing(lactating) women.
    23. Women of child-bearing potential, defined as all women, who are sexually active, physiologically capable of becoming pregnant(e.g. menstruating), unless they are using
    highly effective methods of contraception during dosing and for 14 weeks after stopping the investigational drug. See protocol for these methods of contraception.
    E.5 End points
    E.5.1Primary end point(s)
    1) Proportion of subjects with adverse events (AE)/serious adverse events (SAE) in treatment groups.

    2) Stimulated C-peptide AUC by mixed meal tolerance test (MMTT).
    E.5.1.1Timepoint(s) of evaluation of this end point
    1) 16 months

    2) 12 months
    E.5.2Secondary end point(s)
    1) Free CFZ533 plasma concentration.

    2) Proportion of subjects with full or partial remission.

    3) Stimulated C-peptide AUC by MMTT.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) day 1; 1 week; and 12 months

    2) 12 months

    3) 3 years
    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) 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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years6
    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: 80
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 40
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 40
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 22
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 102
    F.4.2.2In the whole clinical trial 102
    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 Decision2019-08-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-09-25
    P. End of Trial
    P.End of Trial StatusOngoing
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