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    Summary
    EudraCT Number:2021-003528-33
    Sponsor's Protocol Code Number:VIB0551.P2.S2.NMO
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-03-22
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2021-003528-33
    A.3Full title of the trial
    An Open-label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects with Neuromyelitis Optica Spectrum Disorder
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Neuromyelitis Optica Spectrum Disorder Inebilizumab Study in Children and Adolescents
    A.4.1Sponsor's protocol code numberVIB0551.P2.S2.NMO
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/213/2022
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorHorizon Therapeutics Ireland DAC
    B.1.3.4CountryIreland
    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 supportHorizon Therapeutics Ireland DAC
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHorizon Therapeutics Ireland DAC
    B.5.2Functional name of contact pointAlan Mac Neice
    B.5.3 Address:
    B.5.3.1Street Address70 St. Stephen’s Green
    B.5.3.2Town/ cityDublin 2
    B.5.3.3Post codeD02 E2X4
    B.5.3.4CountryIreland
    B.5.4Telephone number+35317722228
    B.5.6E-mailamacneice@horizontherapeutics.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 nameInebilizumab
    D.3.2Product code VIB0551
    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 INNInebilizumab
    D.3.9.1CAS number 1299440-37-1
    D.3.9.2Current sponsor codeVIB0551 (formerly known as MEDI-551)
    D.3.9.4EV Substance CodeSUB189141
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 Yes
    D.3.11.13.1Other medicinal product typeInebilizumab (formerly known as MEDI-551) is a humanized, affinity-optimized, afucosylated IgG1 kappa monoclonal antibody (mAb), known as 16C4-aFuc.
    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
    Neuromyelitis optica spectrum disorder (NMOSD; also known as Devic's syndrome and previously known as neuromyelitis optica [NMO])
    E.1.1.1Medical condition in easily understood language
    Disorder of the central nervous system, which is a rare condition that damages the optic nerve (optic neuritis) and spinal cord (myelitis).
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10077875
    E.1.2Term Neuromyelitis optica spectrum disorder
    E.1.2System Organ Class 10029205 - Nervous 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
    1. To characterize the PK of inebilizumab administered in pediatric subjects with NMOSD
    2. To characterize the PD of inebilizumab administered in pediatric subjects with NMOSD
    3. To assess the safety and tolerability of inebilizumab administered in pediatric subjects with NMOSD
    E.2.2Secondary objectives of the trial
    1. To assess disease activity when inebilizumab is administered in pediatric subjects with NMOSD
    2. To assess health-related quality of life (HRQoL) when inebilizumab is administered in pediatric subjects with NMOSD
    3. To assess visual acuity when inebilizumab is administered in pediatric subjects with NMOSD
    4. To assess disability when inebilizumab is administered in pediatric subjects with NMOSD
    5. To characterize the immunogenicity of inebilizumab administered in pediatric subjects with NMOSD
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent and any locally required data privacy authorization obtained from the subject’s legally authorized representative in accordance with regional laws or regulations and the subject’s assent, when applicable, prior to performing any protocol-related procedures.
    2. Male or female subjects, minimum body weight of 15 kg, age 2 to < 18 years at the time of screening.
    3. Positive serum anti-AQP4-IgG result at screening (verified by the central laboratory) and diagnosed with NMOSD
    4. Documented history of one or more NMOSD acute relapses within the last year, or 2 or more NMOSD acute relapses within 2 years prior to screening.
    5. Female subjects of childbearing potential who are sexually active with a nonsterilized male partner must agree to use a highly effective method of contraception from screening until 6 months after the final dose of investigational product (IP)
    6. Nonsterilized male subjects who are sexually active with a female partner of childbearing potential must agree to use a male condom from Day 1 until 3 months after the final dose of IP.
    E.4Principal exclusion criteria
    1. Any condition that, in the opinion of the Investigator, would interfere with the evaluation or administration of the IP or interpretation of subject safety or study results
    2. Concurrent/previous enrollment in another clinical study involving an investigational treatment within 4 weeks or 5 published half-lives of the investigational treatment, whichever is the longer, prior to Day 1
    3. Females who are breastfeeding, pregnant, or who intend to become pregnant at any time from screening until 6 months after the final dose of IP
    4. Known history of allergy or reaction to any component of the IP formulation or history of anaphylaxis following any biologic therapy
    5. Evidence of alcohol, drug, or chemical abuse, or a recent history of such abuse < 1 year prior to Day 1
    6. Major surgery within 8 weeks prior to screening
    7. Spontaneous or induced abortion, still or live birth, or pregnancy ≤ 4 weeks prior to screening
    8. Evidence of significant hepatic, renal, or metabolic dysfunction or significant hematological abnormality
    9. Receipt of rituximab or any experimental B-cell depleting agent within 6 months prior to screening unless B-cell counts have returned to ≥ one-half the LLN
    10. Receipt of intravenous immunoglobulin (IVIG) within one month prior to Day 1
    11. Receipt of particular immunosuppressive therapy within 2 months prior to Day 1
    12. Receipt of natalizumab (Tysabri®) within 6 months prior to Day 1
    13. Severe drug allergic history or anaphylaxis to 2 or more food products or medicine
    14. Diagnosed with a concurrent autoimmune disease that is uncontrolled (unless approved by the medical monitor)
    15. Receipt of any of the following:
    a. Any live or attenuated vaccine within 4 weeks prior to Day 1
    b. Bacillus Calmette-Guérin vaccine within one year of screening
    c. Blood transfusion within 4 weeks prior to screening or during screening
    16. Clinically significant serious active or chronic viral, bacterial, or fungal infection that requires treatment with anti-infectives, within 2 months prior to Day 1
    17. Known history of congenital or acquired immunodeficiency that predisposes the subject to infection
    18. Positive test for chronic hepatitis B infection at screening
    19. Positive test for hepatitis C virus antibody
    20. Negative test for varicella zoster virus (VZV)-IgG
    21. History of cancer, apart from squamous cell or basal cell carcinoma of the skin treated with documented success of curative therapy > 3 months prior to Day 1
    22. History of active or latent tuberculosis
    23. For subjects who may undergo MRI scans: Unable to undergo an MRI scan (eg, hypersensitivity to Gd containing MRI contrast agents, implanted pacemakers, defibrillators, or other metallic objects on or inside the body that limit performing MRI scans), or unable to tolerate or comply with the MRI procedure.
    E.5 End points
    E.5.1Primary end point(s)
    1. PK parameters, including maximum observed concentration (Cmax), area under the concentration-time curve from time 0 to 14 days post dose (AUC0-14d) and from time 0 extrapolated to infinity (AUC0-inf), systemic clearance, terminal elimination half-life (t½), and volume of distribution at steady state (Vss)
    2. CD20-positive B-cell counts on Days 1, 8, 15, 29, 57, 85, 113, 155, and 197
    3. Safety and tolerability assessments, including incidence of adverse events (AEs)/serious adverse events (SAEs)/adverse events of special interest (AESIs) and changes in laboratory parameters and vital signs
    E.5.1.1Timepoint(s) of evaluation of this end point
    From randomization until end of the trial
    E.5.2Secondary end point(s)
    1. Disease activity endpoints include:
    − Time to first relapse
    − Proportion of relapse-free subjects
    − Annualized relapse rate
    2. HRQoL endpoints include:
    − Change in Euro Quality of Life-5 Dimension Youth (EQ-5D-Y) score
    − Change in Pediatric Quality of Life Inventory (PedsQL)
    3. Change in visual acuity
    4. Change in EDSS
    5. Presence of anti-drug antibody (ADA)
    E.5.2.1Timepoint(s) of evaluation of this end point
    From randomization until end of the trial
    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 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.8.2.4Number 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    Argentina
    Brazil
    Canada
    Serbia
    United Kingdom
    United States
    France
    Netherlands
    Poland
    Spain
    Sweden
    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
    The study will be complete when the last active subject completes the Week 80/EDV visit (Week 52 visit for those subjects who wish to continue receiving inebilizumab through a Sponsor-supported managed access program or equivalent or discontinues prematurely from the study).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months10
    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: 15
    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: 8
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 7
    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 No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Pediatric subjects 2 to < 18 years of age
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 15
    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)
    If Investigator believes the subject would benefit from continued treatment with IP, the subject can continue receiving IP after their participation in the trial through a Sponsor-supported named-patient program. The subjects must complete the W52 visit to be eligible to continue treatment and will exit the study following the W52 visit. The next dosing for these subjects will be administered in the named-patient program 6 months after the last dose of IP in this study.
    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 Decision2022-04-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-24
    P. End of Trial
    P.End of Trial StatusOngoing
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