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    Summary
    EudraCT Number:2020-004228-41
    Sponsor's Protocol Code Number:C0251002
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-03-26
    Trial results View 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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2020-004228-41
    A.3Full title of the trial
    A PHASE 2 DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF PF-06823859 IN ADULT SUBJECTS WITH DERMATOMYOSITIS
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A placebo-controlled study to evaluate the efficacy, safety and tolerability of PF-06823859 in adult subjects with Dermatomyositis
    A.4.1Sponsor's protocol code numberC0251002
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03181893
    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 SponsorPfizer Inc.
    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 supportPfizer Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer Inc.
    B.5.2Functional name of contact pointClinical Trials.gov Call Centre
    B.5.3 Address:
    B.5.3.1Street Address235 East 42nd Street
    B.5.3.2Town/ cityNew York
    B.5.3.3Post codeNY 10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number+18007181021
    B.5.6E-mailClinicalTrials.gov_Inquiries@pfizer.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 namePF-06823859
    D.3.2Product code PF-06823859
    D.3.4Pharmaceutical form Solution for injection
    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 INNPF-06823859
    D.3.9.2Current sponsor codePF-06823859
    D.3.9.3Other descriptive nameHumanised IgG1K monoclonal antibody against interferon beta
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    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
    Dermatomyositis
    E.1.1.1Medical condition in easily understood language
    Dermatomyositis is a rare inflammatory disease marked by muscle weakness and a distinctive severe skin rash
    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 PT
    E.1.2Classification code 10012503
    E.1.2Term Dermatomyositis
    E.1.2System Organ Class 10040785 - Skin and subcutaneous tissue disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    STAGE 1 (complete):
    •To evaluate the efficacy of PF-06823859 in adult participants with moderate to severe DM.

    STAGE 2:
    •To estimate the efficacy of PF-06823859 in adult participants with moderate to severe DM across two stages.

    AMENDED STAGE 2:
    •To estimate the efficacy of PF-06823859 in adult participants with moderate to severe DM across Stage 1 and Stage 2.

    STAGE 3:
    •To evaluate the safety and tolerability of PF-06823859 in adult DM participants with moderate to severe active muscle disease
    E.2.2Secondary objectives of the trial
    STAGE 1 (complete):
    •To evaluate the efficacy of PF-06823859 over time.
    •To determine the safety, and tolerability, of PF-06823859.

    STAGE 2:
    •To estimate the efficacy of PF-06823859 overtime across two Stages.
    •To determine the safety, and tolerability, of PF-06823859 across two Stages.

    AMENDED STAGE 2:
    •To estimate the efficacy of PF-06823859 over time across Stage 1 and Stage 2.
    •To determine the safety, and tolerability, of PF-06823859 across Stage 1 and Stage 2.

    STAGE 3:
    •To evaluate the efficacy of PF-06823859 over time in adult DM participants with moderate active muscle disease
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    STAGE 1 and 2:
    3. Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score, v2-a (See Appendix 2) (Activity ≥14, and have failed at least 1 standard of care systemic treatment, (eg, corticosteroids).
    5. Participant has had a standard work up for dermatomyositis (prior to) baseline.
    a. Stable interstitial lung disease related to DM that is not severe in the opinion of the investigator is allowed, ie, no supplemental oxygen permitted.
    b. If their DM diagnosis is within 2 years of the screening visit, then they must have completed either:
    • Age appropriate malignancy screening eg, computerized tomography, (CT) of the chest/abdomen/pelvis if indicated; or
    • PET CT of chest/abdomen/pelvis at least once by the baseline visit.
    6. Willing to provide 6 skin punch biopsies; (4) skin punch biopsies at pre-dose Day 1, Visit 2, and (2) skin punch biopsies at Week 12.
    7. Male participants able to father children and female participants of childbearing potential must agree to use 2 highly effective methods of contraception throughout the duration of the study, including the follow-up period until the end of study.
    8. Female participants of non-childbearing potential must meet at least 1 of the following criteria:
    a. Achieved postmenopausal status, defined as follows: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; status may be confirmed with a serum follicle-stimulating hormone (FSH) level confirming the postmenopausal state;
    b. Have undergone a documented hysterectomy and/or bilateral oophorectomy;
    c. Have medically confirmed ovarian failure.
    All other female participants (including female participants with tubal ligations) are considered to be of childbearing potential.

    Amended STAGE 2:
    If a participant meets eligibility for both the Stage 2, (skin focused cohort) and the Stage 3, (muscle disease cohort) the patient should be placed in the Stage 3 muscle disease cohort.
    All inclusion criteria is the same as Stage 1 and Stage 2 with the exception to Inclusion Criteria #6.
    6. Willing to provide at least (8) vs. 6 skin punch biopsies; (4) skin punch biopsies at pre-dose Day 1 Visit 2, (2) skin punch biopsies at Week 12, Visit 6 and (2) skin punch biopsies at Week 24 Visit 9.

    STAGE 3:
    3. Meets one of the following two criteria:
    • MMT-8 ≤ 136/150 and PhGA, VAS ≥ 3 cm (0-10 cm) by visual analog scale (VAS) (Appendix 21)
    • Sum of PhGA, VAS, PtGA, and extramuscular global assessment VAS scores (Appendix 20) is ≥ 10 cm (0-10 cm) VAS for each)
    4. Participant has failed at least two or more adequate courses of an immunosuppressive agent or immunomodulatory agent, including IVIG, at a dose known to be effective for rheumatologic diseases.
    5. Participant has had a standard work up for dermatomyositis (prior to) baseline.
    a. Stable interstitial lung disease related to DM that is not severe in the opinion of the investigator is allowed, ie, no supplemental oxygen permitted. completed either:
    • Age appropriate malignancy screening eg, computerized tomography, (CT) of the chest/abdomen/pelvis if indicated; or
    • PET CT of chest/abdomen/pelvis at least once by the baseline visit.
    6. Male participants able to father children and female participants of childbearing potential must agree to use 2 highly effective methods of contraception throughout the duration of the study, including the follow-up period until the end of study.
    7. Female participants of non-childbearing potential must meet at least 1 of the following criteria:
    a. Achieved postmenopausal status, defined as follows: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; status may be confirmed with a serum follicle-stimulating hormone (FSH) level confirming the postmenopausal state;
    b. Have undergone a documented hysterectomy and/or bilateral oophorectomy;
    c. Have medically confirmed ovarian failure.
    d. All other female participants (including female participants with tubal
    ligations) are considered to be of childbearing potential.

    [See section 4 of the Protocol for the full list of Inclusion Criteria]
    E.4Principal exclusion criteria
    STAGE 1 AND STAGE 2:
    2. Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
    7. Abnormal labs:
    • Hemoglobin <10 g/dL;
    • Neutrophils <1.0 x 109/L;
    • Lymphocytes <500 cells/uL;
    • Platelets <75 x 109/L;
    • Creatinine clearance <60 ml/min according to modified Cockcroft-Gault equation;
    • Alkaline phosphatase >2.5 x upper normal limit;
    • Total bilirubin ≥1.5 x upper limit of normal.
    Note: Elevation of aspartate aminotransferase (AST), alanine aminotransferase(ALT), lactate dehydrogenase (LDH) , creatinine kinase (CK) or aldolasedue to muscle involvement (in the opinion of the investigator) are allowed if gamma glutamyl transferase, GGT <1.5 upper limit normal.
    12. Have received the following within 60 days of Day 1:
    • Any intra muscular (IM) or IV steroid injection.
    • Tofacitinib or any other Janus kinase (JAK) inhibitors.
    • Any change in dose of an immunosuppressive/immunomodulatory or antimalarial agent. Dose must be stable for 60 days prior to Day 1 and remain stable through Week 12.
    • Inhaled immunosuppressive agents can be used during the study however must be on a stable dose 60 days prior to Day 1 and remain stable through Week 12. (Immunosuppressive ophthalmic drops are allowed without any restrictions).
    • Disease -modifying antirheumatic drugs (DMARD)s, (methotrexate, sulfasalazine, hydroxychloroquine, and leflunomide). Less frequently used medications include gold salts, azathioprine, and cyclosporine. Dose must be stable for 60 days prior to Day 1 and remain stable through Week 12.
    • Participants may be on one of the following cytotoxic agents: methotrexate, azathioprine, leflunomide, mycophenolate, or 6 MP, but not on any combination of these cytotoxic agents.
    Use of IV or IM antibacterials, antivirals, antifungals, or anti-parasitic agents within 60 days of Day 1. Substitution of IM agents for oral agents because of gastro intestinal, (GI) intolerance may be acceptable, as long as it does not otherwise meet the criteria for a serious infection (requires hospitalization or use of other IV antibiotics) (See exclusion #24).
    25. Have acute coronary syndrome (eg, myocardial infarction, unstable angina pectoris) or any history of significant cerebrovascular disease within 24 weeks of screening. A screening 12-lead electrocardiogram (ECG) that demonstrates clinically significant abnormalities requiring treatment (eg, acute myocardial infarction, serious tachy- or brady- arrhythmias) or that is indicative of serious underlying heart disease Wolff-Parkinson-White syndrome).
    26. Have cancer or a history of cancer within 5 years of screening (other than adequately treated cutaneous basal cell, squamous cell carcinoma, or carcinoma in situ of the uterine cervix with no evidence of recurrence within the previous 5 years).

    Amended STAGE 2:
    All exclusion criteria is the same as Stage 1 and Stage 2 with the exception of Exclusion Criterion #7 Abnormal labs and Exclusion Criterion #12
    Exclusion Criterion #7:
    • Hemoglobin <9 g/dL;
    • Creatinine clearance <50 mL/min according to modified Cockcroft-Gault equation.
    Exclusion Criterion #12:
    • Any change in dose of an immunosuppressive/immunomodulatory or antimalarial agent. Dose must be stable for 60 days prior to Day 1 and remain stable through Week 24.

    STAGE 3:
    5. Drug induced myopathy, metabolic myopathy, muscular dystrophy, cancer associated DM, mixed connective tissue disease-associated DM, (eg, overlap syndrome) and polymyositis.
    6. Significant concurrent disease or conditions other than DM that may influence response to the study drug or safety.
    18. Active bacterial, viral, fungal, mycobacterial or other infections (including but not limited to tuberculosis [TB] and atypical mycobacterial disease, granulomatous disease on chest x-ray). History of recurrent bacterial, viral, fungal, mycobacterial or other infections (including but not limited to TB and atypical mycobacterial disease, granulomatous disease on chest x-ray) that would substantially increase the risk to the participant if he or she participates in the study.
    33. Participant has severe muscle damage defined as a global muscle damage score >5 on a 10cm VAS scale which is a component on the Myositis Damage Index (MDI) Appendix 23

    [See section 4 of the Protocol for the full list of Exclusion Criteria]
    E.5 End points
    E.5.1Primary end point(s)
    STAGE 1, STAGE 2 AND AMENDED STAGE 2
    •Change from baseline in CDASI activity score at Week 12.

    STAGE 3
    •Incidence of adverse events (AEs) laboratory abnormalities, changes in vital signs, and electrocardiogram (ECG) findings.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 weeks
    E.5.2Secondary end point(s)
    STAGE 1, STAGE 2 AND AMENDED STAGE 2
    •Absolute values and change from baseline of CDASI activity and CDASI damage scores at all scheduled time points. (Exception: The change from baseline in CDASI activity score at Week 12 is a primary endpoint).
    •Incidence of adverse events (AEs) laboratory abnormalities, changes in vital signs, and electrocardiogram (ECG) findings.

    STAGE 3
    •Total Improvement Score (TIS) at Week 12 and intermediate scheduled time points
    •Change from baseline in the core Set Measures (CSM) of the TIS including PGA, PtGA, MMT, HAQ-DI, muscle enzymes, and extra-muscular activity, (MDAAT)
    •Absolute values and change from baseline of CDASI activity and CDASI damage scores at all scheduled time points.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Various timepoints
    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 Yes
    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 No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Fixed sequence design
    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 trial3
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    United States
    Germany
    Hungary
    Italy
    Poland
    Spain
    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
    Last participant last visit (LSLV) is defined as the date the investigator reviews the last participant’s final safety data and determines that no further evaluation is required for the participant to complete the trial.
    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 months0
    E.8.9.1In the Member State concerned days11
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days9
    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: 27
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 7
    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 76
    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)
    Subjects can continue on standard of care treatments for dermatomyositis after the end of the trial
    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 Decision2021-07-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-07-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-11-18
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