Main
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
|
EUCTR |
Last refreshed on:
|
26 September 2023 |
Main ID: |
EUCTR2020-004228-41-ES |
Date of registration:
|
01/09/2021 |
Prospective Registration:
|
No |
Primary sponsor: |
|
Public title:
|
A placebo-controlled study to evaluate the efficacy, safety and tolerability of PF-06823859 in adult subjects with Dermatomyositis
|
Scientific title:
|
A PHASE 2 DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF PF-06823859 IN ADULT SUBJECTS WITH DERMATOMYOSITIS |
Date of first enrolment:
|
28/05/2021 |
Target sample size:
|
76 |
Recruitment status: |
Not Recruiting |
URL:
|
https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-004228-41 |
Study type:
|
Interventional clinical trial of medicinal product |
Study design:
|
Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: yes Other trial design description: Fixed sequence design If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 3
|
Phase:
|
Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): no
|
|
Countries of recruitment
|
Germany
|
Hungary
|
Italy
|
Poland
|
Spain
|
United States
| | |
Contacts
|
Name:
|
Clinical Trials.gov Call Centre
|
Address:
|
235 East 42nd Street
NY 10017
New York
United States |
Telephone:
|
+18007181021 |
Email:
|
ClinicalTrials.gov_Inquiries@pfizer.com |
Affiliation:
|
Pfizer Inc. |
|
Name:
|
Clinical Trials.gov Call Centre
|
Address:
|
235 East 42nd Street
NY 10017
New York
United States |
Telephone:
|
+18007181021 |
Email:
|
ClinicalTrials.gov_Inquiries@pfizer.com |
Affiliation:
|
Pfizer Inc. |
| |
Key inclusion & exclusion criteria
|
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-childbear
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 immunos
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
|
Health Condition(s) or Problem(s) studied
|
Dermatomyositis MedDRA version: 20.0
Level: PT
Classification code 10012503
Term: Dermatomyositis
System Organ Class: 10040785 - Skin and subcutaneous tissue disorders
|
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
|
Intervention(s)
|
Product Name: PF-06823859 Product Code: PF-06823859 Pharmaceutical Form: Solution for injection INN or Proposed INN: PF-06823859 Current Sponsor code: PF-06823859 Other descriptive name: Humanised IgG1K monoclonal antibody against interferon beta Concentration unit: mg/g milligram(s)/gram Concentration type: equal Concentration number: 100- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Intravenous use
|
Primary Outcome(s)
|
Primary 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.
|
Main Objective: 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
|
Secondary Objective: 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
|
Timepoint(s) of evaluation of this end point: 12 weeks
|
Secondary Outcome(s)
|
Timepoint(s) of evaluation of this end point: Various timepoints
|
Secondary 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.
|
Secondary ID(s)
|
2020-004228-41-DE
|
NCT03181893
|
C0251002
|
Source(s) of Monetary Support
|
Pfizer Inc.
|
Ethics review
|
Status: Approved
Approval date: 29/04/2021
Contact:
|
|