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    Summary
    EudraCT Number:2017-003652-22
    Sponsor's Protocol Code Number:INCB50465-206
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-03-29
    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 ConcernedAustria - BASG
    A.2EudraCT number2017-003652-22
    A.3Full title of the trial
    A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia (AIHA)
    A.4.1Sponsor's protocol code numberINCB50465-206
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03538041
    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 SponsorIncyte Coorporation
    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 supportIncyte Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIncyte Corporation
    B.5.2Functional name of contact pointMedical Information
    B.5.3 Address:
    B.5.3.1Street Address1801 Augustine Cut-Off
    B.5.3.2Town/ cityWilmington
    B.5.3.3Post codeDE 19803
    B.5.3.4CountryUnited States
    B.5.6E-mailglobalmedinfo@incyte.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 INCB050465 1 mg
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot yet assigned
    D.3.9.2Current sponsor codeINCB050465
    D.3.9.3Other descriptive nameINCB050465 HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB183790
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code INCB050465 2.5 mg
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot yet assigned
    D.3.9.2Current sponsor codeINCB050465
    D.3.9.3Other descriptive nameINCB050465 HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB183790
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2.5
    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
    Autoimmune Hemolytic Anemia (AIHA), immunohemolytic anemia, autoimmune hemolytic anemia, immune complex hemolytic anemia.
    Warm AIHA, Cold AIHA
    E.1.1.1Medical condition in easily understood language
    Autoimmune Hemolytic Anemia (AIHA)
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10003825
    E.1.2Term Autoimmune hemolytic anemia
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of INCB050465 in the treatment of participants with AIHA.
    • Proportion of participants attaining a CR (defined as hemoglobin > 12 g/dL not attributed to transfusion effect and the normalization of hemolytic markers) at any visit from Week 6 to Week 12.
    • Proportion of participants attaining a PR (defined as hemoglobin 10-12 g/dL or ≥ 2 g/dL increase from baseline not attributed to transfusion effect and the normalization of hemolytic markers) at any visit from Week 6 to Week 12.

    To evaluate the safety of INCB050465 administered as repeat doses in participants with AIHA.
    • Safety and tolerability will be assessed by monitoring AEs, measuring vital signs and ECGs, and conducting clinical laboratory blood and urine sample assessments.
    E.2.2Secondary objectives of the trial
    To further evaluate the efficacy of INCB050465 in the treatment of participants with AIHA.
    • Proportion of participants attaining a CR during postbaseline visits.
    • Proportion of participants attaining a PR during postbaseline visits.
    • Proportion of participants attaining a ≥ 2 g/dL increase in hemoglobin from baseline.
    • Mean, change, and percentage change from baseline of hemoglobin.
    • Proportion of participants requiring transfusions.
    • Proportion of participants who achieve normalization of hemolytic markers. (Hemolysis markers: hemoglobin, haptoglobin, LDH, reticulocyte count, total bilirubin, and direct/indirect bilirubin.)
    • Change of daily usage of prednisone.
    • FACIT-F sub-scale assessment.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A participant who meets all of the following criteria may be included in the study:
    1. Men or women, aged 18 years or older.
    2. Diagnosis of AIHA based on the presence of hemolytic anemia and serological evidence of anti-erythrocyte antibodies, detectable by the DAT as follows:
    a. Warm: DAT positive for IgG only or IgG plus C3d
    b. Cold (CAD): DAT positive for C3d only, with cold agglutinins of I specificity
    c. Mixed: DAT positive for IgG and C3d, with coexistence of warm autoantibodies and high titer cold agglutinins
    3. Participants who have disease progression after treatment with standard therapies that are known to confer clinical benefit, or who are intolerant to treatment, or who refuse standard treatment. There is no limit to the number of prior treatment regimens.
    4. Hemoglobin 7 to 10 g/dL. (as determined by local laboratory).
    5. No evidence of a lymphoproliferative malignancy or other autoimmune-related underlying conditions (eg, systemic lupus erythematosus, Castleman's disease, Sjögren's syndrome, or other autoimmune diseases).
    6. ECOG performance status score of 0 to 2.
    7. Willingness to avoid pregnancy or fathering children based on the criteria below:
    a. Woman of nonchildbearing potential (ie, surgically sterile with a hysterectomy and/or bilateral oophorectomy OR postmenopausal, defined by last menstrual period > 12 months before screening and confirmed by FSH).
    b. Woman of childbearing potential who has a negative serum pregnancy test at screening and who agrees to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through at least 93 days after the last dose of study drug. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the participant and her understanding confirmed.
    c. Man who agrees to take appropriate precautions to avoid fathering children (with at least 99% certainty) from screening through at least 93 days after the last dose of study drug. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the participant and his understanding confirmed.
    8. Able to comprehend and willing to sign an ICF.
    9. Willingness to receive PJP prophylaxis during the study period.

    E.4Principal exclusion criteria
    A participant who meets any of the following criteria will be excluded from the study:
    1. Pregnant or breastfeeding women.
    2. Concurrent conditions and history of other diseases:
    a. History or clinical manifestations of significant unstable metabolic, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, urological, neurological, or psychiatric disorders.
    b. Current or previous malignancy within 5 years of study entry, except basal or squamous cell skin cancer with removal considered to be curative, superficial bladder cancer, prostate intraepithelial neoplasm, carcinoma in situ of the cervix, or other noninvasive or indolent malignancy without sponsor approval.
    c. Clinically significant cardiac disease, including unstable angina, acute myocardial infarction, and or cardiac conduction issues within 6 months of the date of study drug administration.
    d. Current New York Heart Association Class II to IV congestive heart failure or uncontrolled arrhythmia.
    3. Inadequate hematologic function defined as follows (as determined by local laboratory):
    a. ANC < 1.5× 109/L.
    b. Platelet count < 100 × 109/L.
    4. Severely impaired liver function (Child-Pugh Class C) or ALT or AST ≥ 2 × ULN on repeated assessment.
    5. Impaired renal function with estimated creatinine clearance less than 45 mL/min.
    6. Anti-phospholipid antibodies positive or elevated anti-streptolysin antibodies.
    7. Hepatitis B (HBV) or hepatitis C (HCV) infection: Participants who are positive for the hepatitis B surface antibody or hepatitis B core antibody, will be eligible if they are negative for HBV-DNA; these participants should be considered for prophylactic antiviral therapy. Participants who are positive for anti-HCV antibody will be eligible if they are negative for HCV-RNA.
    8. Known HIV infection or positivity on immunoassay. Note: HIV screening test is optional for participants enrolled in the United States, but participants with known HIV infection enrolled in the United States will be excluded.
    9. History or presence of an abnormal ECG, that in the investigator’s opinion is clinically meaningful. Participants with screening QTc interval> 470 milliseconds for males and > 480 milliseconds for females (corrected by Fridericia) are excluded. In the event that a single QTcF is >470 milliseconds for males or >480 milliseconds for females, the participant may enroll if the average QTcF for 3 ECGs is <470 milliseconds for males and <480 milliseconds for females.
    10. Use of the following medications:
    a. Treatment with rituximab within 3 months of the baseline visit.
    b. Use of immunosuppressive therapy within 28 days of the baseline visit. Immunosuppressive therapy includes, but is not limited to, cyclosporine A, tacrolimus, or high-dose corticosteroids. Participants receiving corticosteroids must be at a dose level ≤ 20 mg/day (prednisone or equivalent corticosteroid dose) within 14 days of the baseline visit.
    c. Chronic or current active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment or exposure to a live vaccine within 28 days of the baseline visit.
    d. Use or expected use during the study of any prohibited medications, including potent CYP3A4 inhibitors or inducers within 14 days or 5 half-lives (whichever is longer) before the baseline visit.
    e. Current treatment or treatment within 30 days or 5 half-lives (whichever is longer) before the baseline visit with another investigational medication, or current enrollment in another investigational drug protocol.
    f. Use of any prohibited medications within 14 days or 5 half-lives (whichever is longer) before the baseline visit.
    11. Known hypersensitivity or severe reaction to INCB050465 or its excipients.
    12. Unable to swallow oral medication, malabsorption syndrome, disease significantly affecting gastrointestinal function, total resection of the stomach or small bowel, ulcerative colitis, symptomatic inflammatory bowel disease, or partial or complete bowel obstruction.
    13. Current alcohol or drug use that, in the opinion of the investigator, will interfere with the participant's ability to comply with the dose regimen and study evaluations.
    14. Participants who, in the opinion of the investigator, are unable or unlikely to comply with the dose regimen and study evaluations.
    15. Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants attaining a CR (defined as hemoglobin >12 g/dL not attributed to transfusion effect and the normalization of hemolytic markers) at any visit from Week 6 to Week 12.
    • Proportion of participants attaining a PR (defined as hemoglobin 10-12 g/dL or at least ≥ 2 g/dL increase from baseline not attributed to transfusion effect and the normalization of hemolytic markers) at any visit from Week 6 to Week 12.

    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy: From Week 6 to Week 12
    Safety: Throughout the study
    E.5.2Secondary end point(s)
    • Proportion of participants attaining a CR during postbaseline visits.
    • Proportion of participants attaining a PR during postbaseline visits.
    • Proportion of participants attaining a ≥ 2 g/dL increase in hemoglobin from baseline.
    • Mean, change, and percentage change from baseline of hemoglobin.
    • Proportion of participants requiring transfusions.
    • Proportion of participants who achieve normalization of hemolytic markers. (Hemolysis markers: hemoglobin, haptoglobin, LDH, reticulocyte count, total bilirubin, and direct/indirect bilirubin.)
    • Change of daily usage of prednisone.
    • FACIT-F sub-scale assessment.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy: Throughout the study
    PK: Week 1: predose; week 2 and week 8: predose, at 1h +- 10min, at 2h +- 10min and at 4h +- 30 min; week 12: predose
    PD: DAT for IgG and C3b and cold agglutinin levels: at screening and week 12; reticulocyte count, haptoglobin, total bilirubin, direct/indirect bilirubin, and LDH: at screening, day 1, weeks 1, 2, 4, 6, 8 and 10; complement assessment (CH50, C3, and C4): at day 1 and week 12
    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 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 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    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
    Austria
    France
    Italy
    United States
    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
    The end of the study will occur when all participants have completed all applicable follow-up assessments.
    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 months3
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    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: 17
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 25
    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 for treatment of AIHA
    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 Decision2018-07-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-18
    P. End of Trial
    P.End of Trial StatusOngoing
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