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    Summary
    EudraCT Number:2014-001032-11
    Sponsor's Protocol Code Number:OMS721-TMA-001
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-06-25
    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 number2014-001032-11
    A.3Full title of the trial
    A Phase 2, uncontrolled, three-stage, dose-escalation cohort study to evaluate the safety, pharmacokinetics, pharmacodynamics, immunogenicity, and clinical activity of OMS721 in adults with thrombotic microangiopathies.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the safety, pharmacokinetics, pharmacodynamics, immunogenicity, and clinical activity of study drug OMS721 in adults with thrombotic microangiopathies.
    A.4.1Sponsor's protocol code numberOMS721-TMA-001
    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 SponsorOmeros Corporation
    B.1.3.4CountryUnited States
    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 supportOmeros Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPSI CRO AG
    B.5.2Functional name of contact pointProject Manager
    B.5.3 Address:
    B.5.3.1Street AddressBaarestrasse 113a
    B.5.3.2Town/ cityZug
    B.5.3.3Post code6300
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+482221002004818
    B.5.5Fax number+48222100220
    B.5.6E-mailMalgorzata.Kozak-Regwelska@psi-cro.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.3.2Product code OMS721
    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 INNnot yet available
    D.3.9.2Current sponsor codeOMS721
    D.3.9.3Other descriptive nameOMS721 100 mg/ml Injection solution, OMS00620646, OMS620646, MASP-2 Antibody
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 Yes
    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
    Three forms of thrombotic microangiopathies (TMA):
    - atypical hemolytic uremic syndrome (aHUS)
    - hematopoietic stem cell transplant (HSCT)-associated TMA
    - thrombotic thrombocytopenic purpura (TTP)
    E.1.1.1Medical condition in easily understood language
    thrombosis in capillaries and arterioles
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level PT
    E.1.2Classification code 10043645
    E.1.2Term Thrombotic microangiopathy
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The co-primary objectives of this study are to:
    • Assess the safety and tolerability of multiple-dose administration of OMS721 in subjects with TMA
    • Evaluate the clinical activity of multiple-dose administration of OMS721 in subjects with TMA
    E.2.2Secondary objectives of the trial
    • Determine the pharmacokinetics (PK) of multiple-dose administration of OMS721 in subjects with TMA
    • Determine the pharmacodynamics (PD) of multiple-dose administration of OMS721 in subjects with TMA
    • Determine the immunogenicity of multiple-dose administration of OMS721 in subjects with TMA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Competent to provide informed consent.
    2. Voluntarily provide informed consent in accordance with regulations and governing ethics committee requirements prior to any procedures or evaluations performed specifically for the sole purpose of the study.
    3. Are age ≥18 at screening (Visit 1).
    4. Have a diagnosis of TMA in accordance with one of the following three categories:
    • Primary aHUS, diagnosed clinically and having ADAMTS13 activity >10% in plasma. Patients are eligible with or without a documented complement mutation or anti-CFH antibody. Patients are categorized according to their response to plasma therapy (plasma exchange or plasma infusion):
    o Plasma therapy-resistant aHUS patients must have all of the following: 1) screening platelet count < 150,000/μL despite at least four plasma therapy treatments prior to screening; 2) evidence of microangiopathic hemolysis (presence of schistocytes, serum lactate dehydrogenase (LDH) > upper limit of normal (ULN), or haptoglobin < LLN); and 3) serum creatinine > ULN.
    o Chronic plasma therapy-responsive aHUS patients (plasma therapy-sensitive) must require at least once-per-week plasma therapy for four weeks before first dose of OMS721 with serum creatinine >ULN.
    • TTP defined as having all of the following:
    o Platelet count < 150,000/μL
    o Evidence of microangiopathic hemolysis (presence of schistocytes, serum LDH > ULN, or haptoglobin < LLN)
    o ADAMTS13 activity ≤ 10% during the current episode of TTP or historically
    • Persistent HSCT-associated TMA defined as having all of the following at least two weeks following modification or discontinuation of calcineurin inhibitor treatment or at least 30 days after the transplant:
    o Platelet count < 150,000/μL
    o Evidence of microangiopathic hemolysis (presence of schistocytes, serum LDH > ULN, or haptoglobin < LLN)
    o Renal dysfunction (doubling of serum creatinine from pre-transplant).
    5. No clinically apparent alternative explanation for thrombocytopenia and anemia.
    6. If sexually active and of childbearing potential, must agree to practice a highly effective method of birth control until the end of the study, defined as one that results in a low failure rate (i.e., less than 1% per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices, sexual abstinence or vasectomized partner.
    E.4Principal exclusion criteria
    Subjects will be excluded from the study for any of the following reasons:
    1. Had eculizumab therapy within three months prior to screening.
    2. Have STEC-HUS.
    3. Have a positive direct Coombs test.
    4. Have an active systemic bacterial or fungal infection requiring antimicrobial therapy (prophylactic antimicrobial therapy administered as standard of care is allowed).
    5. Baseline resting heart rate < 45 beats per minute or > 115 beats per minute.
    6. Baseline QTcF > 470 milliseconds.
    7. Have malignant hypertension (diastolic blood pressure [BP] > 120 mm Hg with bilateral hemorrhages or “cotton-wool” exudates on funduscopic examination).
    8. Have a poor prognosis with a life expectancy of less than three months in the opinion of the investigator.
    9. Are pregnant or lactating.
    10. Have received treatment with an investigational drug or device within four weeks prior to screening.
    11. Have abnormal liver function tests defined as ALT or AST > five times ULN.
    12. Have a positive test for human immunodeficiency virus (HIV) antibodies.
    13. Are an employee of Omeros, an investigator, a study staff member, or their immediate family member.
    14. Have a known hypersensitivity to any constituent of the product.
    15. Presence of any condition that the Investigator believes would put the subject at risk.
    E.5 End points
    E.5.1Primary end point(s)
    • Safety as assessed by AEs, vital signs, ECG and clinical laboratory tests
    • Clinical activity as assessed by platelet count
    E.5.1.1Timepoint(s) of evaluation of this end point
    from baseline across all evaluations/visits
    E.5.2Secondary end point(s)
    • TMA clinical activity
    o Serum LDH
    o Serum haptoglobin
    o Hemoglobin
    o Serum creatinine
    o TMA-related symptoms
    o Need for plasma therapy (plasma exchange or plasma infusion)
    o Need for dialysis
    • PK parameters including maximum concentration (Cmax), time to maximum concentration (Tmax), elimination half-life (t½), area under time-concentration curve (AUC), clearance (CL), and volume of distribution (Vz)
    • PD measure of inhibition of ex vivo lectin pathway activation
    • Presence of ADA response
    E.5.2.1Timepoint(s) of evaluation of this end point
    pre-dose, 5 min, 2 hours, 24 hours, 48 hours, 72 hours
    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 Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity
    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 Yes
    E.8.1.7.1Other trial design description
    ascending-dose-escalation study
    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 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 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
    Belarus
    Belgium
    Bulgaria
    Germany
    Hong Kong
    Italy
    Lithuania
    Malaysia
    New Zealand
    Poland
    Switzerland
    Taiwan
    Thailand
    Turkey
    United Kingdom
    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.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 70
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 19
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 49
    F.4.2.2In the whole clinical trial 89
    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 Decision2014-07-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-10-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-10-16
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