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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-004228-41 | EudraCT Number |
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A Study looking at Investigational drug and Placebo administered to adult Patients with moderate to severe Dermatomyositis
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo ARM | Placebo Comparator |
| |
| PF-06823859 ARM high | Experimental |
| |
| PF-06823859 ARM low | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PF-06823859 low | Drug | A humanized immunoglobulin neutralizing antibody |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 12 (Stage 1, Stage 2 and Amended Stage 2) | The treatment effect was defined as the difference (mean chg from baseline at Week12 in the active treatment group minus that in the placebo group) in the mean change of CDASI activity score from baseline at Week 12. The score (range: 0-100) consists of the extent score (ES), Gottorn hands score (GHS), peringual score (PS) and allopecia score (AS). ES (range: 0-90) was obtained by summing up scores for the total erythema (ER [0-45], redness of the skin or mucous membranes), scaling (SC [0-30], peeling of the skin) and erosion/ulceration (EU [0-15], presence of the deeper wound). Total ER, SC and EU scores were calculated as a sum of the contributions from 15 individual areas of the body. GHS characterizes papules (swellings) on hand and is a sum of the papule's characterization score (0-6) and ulceration score (0-1). PS (0-2) characterizes abnormalities around nails. The AS (0-1) characterizes hair loss. Higher scores indicate greater disease severity. | Baseline and Week 12 |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAE) (Stage 3) | Adverse event (AE) was any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE was any untoward medical occurrence that at any dose resulted in any of following outcomes/deemed significant for any other reason: death; initial /prolonged inpatient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly/birth defect and suspected transmission via a Pfizer product of an infectious agent, pathogenic or non-pathogenic. AEs included both serious (if occurred) and all non-serious adverse events. TEAEs are events between first dose of study drug and up to Week 40 that were absent before treatment or that worsened relative to pretreatment state. | Up to Week 40 |
| Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 3) | Hemoglobin(HGB),hematocrit,erythrocytes(ery.),HDL cholesterol(chl.)<0.8*lower limit of normal(LLN);reticulocytes (ret.), ret./ery.(%)<0.5*LLN,>1.5*upper limit of normal (ULN);ery. mean corpuscular(EMC) volume,EMC HGB concentration,potassium,chloride,calcium,bicarbonate<0.9*LLN,>1.1*ULN;platelets<0.5*LLN,>1.75*ULN; leukocytes(leu.),glucose<0.6*LLN,>1.5*ULN;lymphocytes(lym.), lym./leu.(%),neutrophils (neu.), neu./leu.(%), protein,albumin<0.8*LLN,>1.2*ULN;basophils(bas.), bas./leu.(%), eosinophils(eos.), eos./leu., monocytes(mon.), mon./leu.(%), urate>1.2*ULN;bilirubin (total, direct,indirect)>1.5*ULN;aspartate/alanine aminotransferase,gamma glutamyl transferase,lactate dehydrogenase,alkaline phosphatase>3.0*ULN;urea nitrogen,creatinine,triglycerides, chl.>1.3*ULN; sodium <0.95*LLN,>1.05*ULN; creatine kinase >2.0*ULN;Urine: pH<4.5,>8;glucose, ketones,protein, HGB,urobilinogen,bilirubin,nitrite,leukocyte esterase>=1;ery., leu.>= 20;hyaline casts>1;bacteria>20. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With TEAEs and SAEs (Stage 1 and Stage 2) | AE was any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE was any untoward medical occurrence that at any dose resulted in any of following outcomes/deemed significant for any other reason: death; initial /prolonged inpatient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly/birth defect and suspected transmission via a Pfizer product of an infectious agent, pathogenic or non-pathogenic. AEs included both serious (if occurred) and all non-serious adverse events. TEAEs are events between first dose of study drug and up to Week 28 that were absent before treatment or that worsened relative to pretreatment state. |
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Inclusion Criteria for Patients with Skin Predominant Activity:
Must have CDASI Activity score of greater than or equal to 14, and have failed at least 1 standard of care systemic treatment, (eg, corticosteroids).
Confirmation of DM by the investigator and two of the following:
Post DM diagnosis; standard of care workup for DM must have been completed prior to entry into this research study.
Willing to provide 8 biopsies during the course of the research study
Inclusion Criteria for Patients with Muscle Predominant Activity:
MMT-8 ≤136/150 and PhGA, VAS ≥3 cm (0-10 cm) by visual analog scale (VAS)
Sum of PhGA, VAS, PtGA, and extramuscular global assessment VAS scores is ≥10 cm (0-10 cm) VAS for each.
Exclusion Criteria for Patients with Skin Predominant Activity:
Exclusion Criteria for Patients with Muscle Predominant Activity:
Similar to patients with skin predominant activity; Intake of >20 mg oral prednisone/day, or equivalent
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | United States | ||
| The University of Alabama at Birmingham |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42273013 | Derived | Aggarwal R, Peeva E, Fiorentino DF, Vleugels RA, Mangold AR, Werth VP, Oemar BS, Rath JN, Sloan A, Chu M. Rapid Onset of Response in Adults with Dermatomyositis Receiving Dazukibart: A Phase 2, Double-Blind, Randomized, Placebo-Controlled Study. Clin Cosmet Investig Dermatol. 2026 Jun 4;19:581733. doi: 10.2147/CCID.S581733. eCollection 2026. | |
| 41874840 |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
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A total of 75 participants were randomized at 19 centers in 5 countries: 32, 9, 16 and 18 participants were treated in Stage 1, Stage 2, Amended Stage 2 and Stage 3, respectively.
A fixed sequence design with crossover at Week 12 was employed in Amended Stage 2 and Stage 3 to provide all participants with the opportunity to receive active drug during the treatment period.
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| ID | Title | Description |
|---|---|---|
| FG000 | (Stage 1) Placebo | Participants in this group were randomized to receive placebo on Day 1, Week 4, and Week 8 in Stage 1 After week 12, participants went into a follow up period. |
| FG001 | (Stage 1) PF-06823859 600 mg Intravenous (IV) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Baseline to Week 12 (All Stages) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 20, 2021 | May 26, 2023 |
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| Placebo Arm |
| Drug |
Placebo contains histidine, sucrose, PS80, ethylene diamine, and triacetic acid |
|
| PF-06823859 high | Drug | A humanized immunoglobulin neutralizing antibody |
|
| Up to Week 40 |
| Number of Participants With Vital Sign Abnormalities (Stage 3) | Abnormality in vital signs: Sitting pulse rate <40 beats per minute (bpm) to >120 bpm, sitting diastolic blood pressure (DBP) < 50 millimeter of mercury (mmHg), sitting systolic blood pressure (SBP) <90 mmHg. | Baseline up to Week 40 |
| Number of Participants With Electrocardiogram (ECG) Abnormalities (Stage 3) | ECG abnormalities criteria included: 1) QTc interval adjusted according to Fridericia formula (QTcF) (msec): >450, >480, >500, increase from baseline >=30, increase from baseline >=60; 2) Pulse rate (PR) (msec): >=300, change from baseline (Chg) >=25% or 50%; 3) QT (msec): >=500; 4) QRS (msec): >=200, Chg >=25% or 50%. Categories, with at least 1 participant having ECG abnormality in any of the reporting arms, were reported in this outcome measure. | Baseline up to Week 40 |
| Up to Week 28 |
| Number of Participants With TEAEs and SAEs (Amended Stage 2) | AE was any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE was any untoward medical occurrence that at any dose resulted in any of following outcomes/deemed significant for any other reason: death; initial /prolonged inpatient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly/birth defect and suspected transmission via a Pfizer product of an infectious agent, pathogenic or non-pathogenic. AEs included both serious (if occurred) and all non-serious adverse events. TEAEs are events between first dose of study drug and up to Week 40 that were absent before treatment or that worsened relative to pretreatment state. | Up to Week 40 |
| Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 1 and Stage 2) | HGB,hematocrit,ery.,HDL chl.<0.8*LLN;ret., ret./ery. (%)<0.5*LLN,>1.5*ULN;EMC volume,EMC HGB,EMC HGB concentration,potassium,chloride,calcium,bicarbonate<0.9*LLN,>1.1*ULN;platelets<0.5*LLN,>1.75*ULN;leu.,glucose<0.6*LLN,>1.5*ULN;lym., lym./leu.(%), neu., neu./leu. (%), protein,albumin <0.8*LLN,>1.2*ULN;bas., bas./leu.(%), eos., eos./leu., mon., mon./leu.(%), urate >1.2*ULN;bilirubin (total, direct, indirect)>1.5*ULN;aspartate/alanine aminotransferase, gamma glutamyl transferase, lactate dehydrogenase, alkaline phosphatase>3.0*ULN;urea nitrogen, creatinine, triglycerides, chl.>1.3*ULN; sodium <0.95*LLN,>1.05*ULN; creatine kinase >2.0*ULN;Urine: pH<4.5,>8;glucose, ketones, protein, HGB, urobilinogen,bilirubin,nitrite,leukocyte esterase>=1;ery., leu.>= 20;hyaline casts>1;bacteria>20. Clinical significance of laboratory parameters was determined at the investigator's discretion. | Up to Week 28 |
| Number of Participants With Clinically Significant Laboratory Abnormalities (Amended Stage 2) | HGB,hematocrit,ery.,HDL chl.<0.8*LLN;ret., ret./ery. (%)<0.5*LLN,>1.5*ULN;EMC volume,EMC HGB,EMC HGB concentration,potassium,chloride,calcium,bicarbonate<0.9*LLN,>1.1*ULN;platelets<0.5*LLN,>1.75*ULN;leu.,glucose<0.6*LLN,>1.5*ULN;lym., lym./leu.(%), neu., neu./leu. (%), protein,albumin <0.8*LLN,>1.2*ULN;bas., bas./leu.(%), eos., eos./leu., mon., mon./leu.(%), urate >1.2*ULN;bilirubin (total, direct, indirect)>1.5*ULN;aspartate/alanine aminotransferase, gamma glutamyl transferase, lactate dehydrogenase, alkaline phosphatase>3.0*ULN;urea nitrogen, creatinine, triglycerides, chl.>1.3*ULN; sodium <0.95*LLN,>1.05*ULN; creatine kinase >2.0*ULN;Urine: pH<4.5,>8;glucose, ketones, protein, HGB, urobilinogen,bilirubin,nitrite,leukocyte esterase>=1;ery., leu.>= 20;hyaline casts>1;bacteria>20. Clinical significance of laboratory parameters was determined at the investigator's discretion. | Up to Week 40 |
| Number of Participants With Vital Sign Abnormalities (Stage 1 and Stage 2) | Abnormality in vital signs: Sitting pulse rate <40 bpm to >120 bpm, sitting DBP < 50 mmHg, sitting SBP <90 mmHg. | Up to Week 28 |
| Number of Participants With Vital Sign Abnormalities (Amended Stage 2) | Abnormality in vital signs: Sitting pulse rate <40 bpm to >120 bpm, sitting DBP < 50 mmHg, sitting SBP <90 mmHg. | Up to Week 40 |
| Number of Participants With ECG Abnormalities (Stage 1 and Stage 2) | ECG abnormalities criteria included: 1) QTc interval adjusted according to Fridericia formula (QTcF) (msec): >450, >480, >500, increase from baseline >=30, increase from baseline >=60; 2) Pulse rate (PR) (msec): >=300, change from baseline (Chg) >=25% or 50%; 3) QT (msec): >=500; 4) QRS (msec): >=200, Chg >=25% or 50%. Categories, with at least 1 participant having ECG abnormality in any of the reporting arms, were reported in this outcome measure. | Up to Week 28 |
| Number of Participants With ECG Abnormalities (Amended Stage 2) | ECG abnormalities criteria included: 1) QTc interval adjusted according to Fridericia formula (QTcF) (msec): >450, >480, >500, increase from baseline >=30, increase from baseline >=60; 2) Pulse rate (PR) (msec): >=300, change from baseline (Chg) >=25% or 50%; 3) QT (msec): >=500; 4) QRS (msec): >=200, Chg >=25% or 50%. Categories, with at least 1 participant having ECG abnormality in any of the reporting arms, were reported in this outcome measure. | Up to Week 40 |
| Change From Baseline in CDASI Activity Score at at All Scheduled Timepoints Through Week 12 (Stage 1, Stage 2 and Amended Stage 2) | The treatment effect was defined as the difference (mean change from baseline at Weeks 1, 4, 8 in the active treatment group minus the mean change from baseline at Weeks 1, 4, 8 in the placebo group) in the mean change of CDASI activity score from baseline at scheduled timepoints. The score (range: 0-100) consists of the ES, GHS, PS and AS. ES (range: 0-90) was obtained by summing up scores for the total erythema (ER [0-45], redness of the skin or mucous membranes), scaling (SC [0-30], peeling of the skin) and erosion/ulceration (EU [0-15], presence of the deeper wound). Total ER, SC and EU scores were calculated as a sum of the contributions from 15 individual areas of the body. GHS characterizes papules (swellings) on hand and is a sum of the papule's characterization score (0-6) and ulceration score (0-1). PS (0-2) characterizes abnormalities around nails. The AS (0-1) characterizes hair loss. Higher scores indicate greater disease severity. | Baseline, Week 1, Week 4, and Week 8 (except for Week 12 which is a primary outcome measure) |
| Change From Baseline in CDASI Activity Score at All Scheduled Timepoints Through Week 12 (Stage 3) | The treatment effect was defined as the difference (mean change from baseline at Weeks 1, 4, 8,12 in the active treatment group minus the mean change from baseline at Weeks 1, 4, 8, 12 in the placebo group) in the mean change of CDASI activity score from baseline at scheduled timepoints. The score (range: 0-100) consists of the ES, GHS, PS and AS. ES (range: 0-90) was obtained by summing up scores for the total erythema (ER [0-45], redness of the skin or mucous membranes), scaling (SC [0-30], peeling of the skin) and erosion/ulceration (EU [0-15], presence of the deeper wound). Total ER, SC and EU scores were calculated as a sum of the contributions from 15 individual areas of the body. GHS characterizes papules (swellings) on hand and is a sum of the papule's characterization score (0-6) and ulceration score (0-1). PS (0-2) characterizes abnormalities around nails. The AS (0-1) characterizes hair loss. Higher scores indicate greater disease severity. | Baseline, Week 1, Week 4, Week 8 and Week 12 |
| Absolute Values of CDASI Activity Score at All Scheduled Timepoints Through Week 12 (All Stages) | The CDASI activity score (range: 0-100) consists of the ES, GHS, PS and AS. ES (range: 0-90) was obtained by summing up scores for the total erythema (ER [0-45], redness of the skin or mucous membranes), scaling (SC [0-30], peeling of the skin) and erosion/ulceration (EU [0-15], presence of the deeper wound). Total ER, SC and EU scores were calculated as a sum of the contributions from 15 individual areas of the body. GHS characterizes papules (swellings) on hand and is a sum of the papule's characterization score (0-6) and ulceration score (0-1). PS (0-2) characterizes abnormalities around nails. The AS (0-1) characterizes hair loss. Higher scores indicate greater disease severity. | Baseline, Week 1, Week 4, Week 8 and Week 12 |
| Absolute Values of CDASI Damage Score at All Scheduled Timepoints Through Week 12 (All Stages) | The Damage Score (DS) was calculated as a sum of the total poilkiloderma score (POLS), total calcinosis score (CALS) and Gotorn's hands damage score (GHDS). The POLS characterizes specific dispigmentation in the particulal area and calcinosis score characterizes calcification of the skin in the particular area. The POLS and the CALS are summed up over 15 individual areas in the body and each of them has range 0-15. The GHDS has the range 0-2 so that the DS has the range 0-32. Higher scores indicate greater disease severity. | Baseline, Week 1, Week 4, Week 8 and Week 12 |
| Absolute Values for Total Improvement Score (TIS) at Week 12 and Intermediate Scheduled Time Points (Stage 3) | The TIS was the sum of all 6 improvement scores where higher score indicates worse status (PhGA [from the MDAAT, 0-20 scale], PtGA [0-10 scale], MMT [0-35 scale], HAQ-DI [0-10 scale], muscle enzymes [0-7.5 scale], and extramuscular global assessment [0-20 scale]) associated with the change in each core set measure. A total improvement score between 0 and 100 corresponded to the degree of improvement, with higher scores corresponding to a greater degree of improvement: of ≥20 represented minimal improvement, a score of ≥40 represented moderate improvement, and a score of ≥60 represented major improvement. | Week 4, Week 8 and Week 12 |
| Change From Baseline in the Core Set Measures (CSM) of the TIS (Global Disease Activity [PhGA] and Extramuscular Global Assessment [EmGA]) (Stage 3) | PhGA: assessment of the severity of disease by the physician. The physician used the visual analog scale and put a mark on 0 cm (best) -10 cm (worst) scale where higher score indicated worse status. EmGA: overall evaluation of disease activity in all extramuscular systems using visual analog scale 0 cm (best) -10 cm (worst) scale where higher score indicated worse status. | Baseline, Week 4, Week 8 and Week 12 |
| Change From Baseline in the CSM of the TIS (PtGA) (Stage 3) | PtGA: assessment of the severity of disease by the participant/participant's guardian, using a visual analog scale from 0 mm (no evidence of disease activity) to 100 mm (extremely active or severe disease activity). Higher score indicated worse status. | Baseline, Week 4, Week 8 and Week 12 |
| Change From Baseline in the CSM of the TIS (MMT8 and HAQ01-HAQ-DI) (Stage 3) | Manual Muscle Testing-8 designated muscle groups (MMT-8): was a set of 8 designated muscles tested unilaterally generally on right side (left side used unless right side cannot be used). Potential score range was from 0 to 80, where higher scores denoted better health status. HAQ-DI: contained eight sections (including dressing & grooming, arising, eating, walking, hygiene, grip, reach, and activities). Each section had multiple questions that the participant used to rank their functionality and ranged from 0 to 3 where 0 = without any difficulty and 3 = unable to do. For each participant, the average ranking was calculated for each of the eight sections. HAQ-DI had a score range of 0 to 3, where higher score reflected worse status. | Week 4, Week 8 and Week 12 |
| Change From Baseline in the CSM of the TIS (Aldolase and Creatine Kinase) (Stage 3) | The LS mean (with 90% CI) of CSM of the TIS (aldolase and creatine kinase) at weeks 4, 8, 12 were presented. | Baseline, Week 4, Week 8 and Week 12 |
| Birmingham |
| Alabama |
| 35249 |
| United States |
| The University of Alabama at Birmingham | Birmingham | Alabama | 35294 | United States |
| Mayo Clinic Arizona Research Pharmacy | Phoenix | Arizona | 85054 | United States |
| Mayo Clinic | Scottsdale | Arizona | 85259 | United States |
| Attune Health Research Inc. | Beverly Hills | California | 90211 | United States |
| Freidenrich Center for Translational Research at Stanford University | Palo Alto | California | 94304 | United States |
| Mayo Clinic Florida | Jacksonville | Florida | 32224 | United States |
| University Of Miami Hospital | Miami | Florida | 33125 | United States |
| University of Miami Hospital Clinical Translational Research Site (Infusion site) | Miami | Florida | 33136 | United States |
| KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) | Fairway | Kansas | 66205 | United States |
| The University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Johns Hopkins Bayview Medical Center | Baltimore | Maryland | 21224-6821 | United States |
| Brigham and Women's Hospital - ACC | Boston | Massachusetts | 02115 | United States |
| Brigham and Women's Hospital - CTC | Boston | Massachusetts | 02115 | United States |
| Brigham and Women's Hospital - CTH | Boston | Massachusetts | 02115 | United States |
| Brigham and Women's Hospital | Boston | Massachusetts | 02115 | United States |
| Clinical Research Unit (CRU) | Minneapolis | Minnesota | 55455 | United States |
| Department of Medicine Division of Rheumatic and Autoimmune Disease | Minneapolis | Minnesota | 55455 | United States |
| Lillehei Clinical Research Unit (LCRU) | Minneapolis | Minnesota | 55455 | United States |
| University of Minnesota Health Rheumatology Clinic | Minneapolis | Minnesota | 55455 | United States |
| University of Minnesota, Department of Dermatology | Minneapolis | Minnesota | 55455 | United States |
| Center for Outpatient Health | St Louis | Missouri | 63108 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| New York University School of Medicine | New York | New York | 10016 | United States |
| NYU Langone Health Clinical Research Center | New York | New York | 10016 | United States |
| Mount Sinai Doctors Dermatology | New York | New York | 10028 | United States |
| Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| OHSU, Center for Health and Healing CHH2 | Portland | Oregon | 97239 | United States |
| Oregon Clinical & Translational Research Institute | Portland | Oregon | 97239 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | United States |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75390-9191 | United States |
| University of Utah MidValley Dermatology | Murray | Utah | 84107 | United States |
| Center for Clinical & Translational Science | Salt Lake City | Utah | 84108 | United States |
| Universitaetsklinikum Tuebingen | Tübingen | 72076 | Germany |
| University of Debrecen | Debrecen | Hajdú-Bihar | H-4032 | Hungary |
| Nova Reuma spolka partnerska | Bialystok | 15-707 | Poland |
| Centrum Medyczne Plejady | Krakow | 30-363 | Poland |
| Hospital Universitario 12 de Octubre | Madrid | 28041 | Spain |
| Hospital Quiron Infanta Luisa | Seville | 41010 | Spain |
| Prybylski JP, Zhu J', Banfield C, Mukherjee A, Purohit V. Early exposure-response modeling of an interferon-beta monoclonal antibody (dazukibart) in adults with dermatomyositis. J Pharmacokinet Pharmacodyn. 2026 Mar 24;53(3):17. doi: 10.1007/s10928-026-10022-1. |
| 39798982 | Derived | Fiorentino D, Mangold AR, Werth VP, Christopher-Stine L, Femia A, Chu M, Musiek ACM, Sluzevich JC, Graham LV, Fernandez AP, Aggarwal R, Rieger K, Page KM, Li X, Hyde C, Rath N, Sloan A, Oemar B, Banerjee A, Salganik M, Banfield C, Neelakantan S, Beebe JS, Vincent MS, Peeva E, Vleugels RA. Efficacy, safety, and target engagement of dazukibart, an IFNbeta specific monoclonal antibody, in adults with dermatomyositis: a multicentre, double-blind, randomised, placebo-controlled, phase 2 trial. Lancet. 2025 Jan 11;405(10473):137-146. doi: 10.1016/S0140-6736(24)02071-3. |
Participants in this group were randomized to receive PF-06823859 600 mg on Day 1, Week 4, and Week 8 in Stage 1. After week 12, participants went into a follow up period.
| FG002 | (Stage 2) Placebo | Participants in this group were randomized to receive placebo on Day 1, Week 4, and Week 8 in Stage 2. After week 12, participants went into a follow up period. |
| FG003 | (Stage 2) PF-06823859 150 mg IV | Participants in this group were randomized to receive PF-06823859 150 mg on Day 1, Week 4, and Week 8. After week 12, participants went into a follow up period. |
| FG004 | (Stage 2) PF-06823859 600 mg IV | Dosing occurred Day 1, Week 4, and Week 8. After week 12, participants went into a follow-up period. |
| FG005 | (Amended Stage 2) Placebo Then Switched to PF-06823859 150 mg IV at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| FG006 | (Amended Stage 2) Placebo Then Switched to PF-06823859 600 mg IV at Week 12 | Dosing occurred on Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| FG007 | (Amended Stage 2) PF-06823859 150 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16 and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| FG008 | (Amended Stage 2) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| FG009 | (Stage 3) Placebo Then Switched to PF-06823859 600 mg IV at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants entered a 4-month follow-up period or rolled over to the long-term extension study. |
| FG010 | (Stage 3) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants entered a 4-month follow-up period or rolled over to the long-term extension study. |
| COMPLETED |
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| NOT COMPLETED |
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|
| Weeks 12-24 (Amended Stage 2, Stage 3) |
|
|
The safety analysis population included all participants who had received at least 1 dose of study drug or placebo.
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| ID | Title | Description |
|---|---|---|
| BG000 | (Stage 1) Placebo | Participants in this group were randomized to receive placebo on Day 1, Week 4, and Week 8. After week 12, participants went into a follow up period. |
| BG001 | (Stage 1) PF-06823859 600 mg Intravenous (IV) | Participants in this group were randomized to receive PF-06823859 600 mg on Day 1, Week 4, and Week 8. After week 12, participants went into a follow up period. |
| BG002 | (Stage 2) Placebo | Participants in this group were randomized to receive placebo on Day 1, Week 4, and Week 8. After week 12, participants went into a follow up period. |
| BG003 | (Stage 2) PF-06823859 150 mg IV | Participants in this group were randomized to receive PF-06823859 150 mg on Day 1, Week 4, and Week 8 After week 12, participants went into a follow up period. |
| BG004 | (Stage 2) PF-06823859 600 mg IV | Dosing occurred Day 1, Week 4, and Week 8. After week 12, participants went into a follow-up period. |
| BG005 | (Amended Stage 2) Placebo Then Switched to PF-06823859 150 mg IV at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| BG006 | (Amended Stage 2) Placebo Then Switched to PF-06823859 600 mg IV at Week 12 | Dosing occurred on Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| BG007 | (Amended Stage 2) PF-06823859 150 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16 and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| BG008 | (Amended Stage 2) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| BG009 | (Stage 3) Placebo Then Switched to PF-06823859 600 mg IV at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants entered a 4-month follow-up period or rolled over to the long-term extension study. |
| BG010 | (Stage 3) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants entered a 4-month follow-up period or rolled over to the long-term extension study. |
| BG011 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 12 (Stage 1, Stage 2 and Amended Stage 2) | The treatment effect was defined as the difference (mean chg from baseline at Week12 in the active treatment group minus that in the placebo group) in the mean change of CDASI activity score from baseline at Week 12. The score (range: 0-100) consists of the extent score (ES), Gottorn hands score (GHS), peringual score (PS) and allopecia score (AS). ES (range: 0-90) was obtained by summing up scores for the total erythema (ER [0-45], redness of the skin or mucous membranes), scaling (SC [0-30], peeling of the skin) and erosion/ulceration (EU [0-15], presence of the deeper wound). Total ER, SC and EU scores were calculated as a sum of the contributions from 15 individual areas of the body. GHS characterizes papules (swellings) on hand and is a sum of the papule's characterization score (0-6) and ulceration score (0-1). PS (0-2) characterizes abnormalities around nails. The AS (0-1) characterizes hair loss. Higher scores indicate greater disease severity. | The Full Analysis Set (FAS) in Stage 1, Stage 2, and Amended Stage 2 included all participants who received at least 1 dose of randomized treatment in in Stage 1, Stage 2, or Amended Stage 2. | Posted | Mean | Standard Deviation | Units on a scale | Baseline and Week 12 |
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| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAE) (Stage 3) | Adverse event (AE) was any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE was any untoward medical occurrence that at any dose resulted in any of following outcomes/deemed significant for any other reason: death; initial /prolonged inpatient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly/birth defect and suspected transmission via a Pfizer product of an infectious agent, pathogenic or non-pathogenic. AEs included both serious (if occurred) and all non-serious adverse events. TEAEs are events between first dose of study drug and up to Week 40 that were absent before treatment or that worsened relative to pretreatment state. | The safety analysis set in Stage 3 (SAS3) included all participants who received at least 1 dose of randomized treatment in Stage 3. | Posted | Count of Participants | Participants | Up to Week 40 |
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| Primary | Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 3) | Hemoglobin(HGB),hematocrit,erythrocytes(ery.),HDL cholesterol(chl.)<0.8*lower limit of normal(LLN);reticulocytes (ret.), ret./ery.(%)<0.5*LLN,>1.5*upper limit of normal (ULN);ery. mean corpuscular(EMC) volume,EMC HGB concentration,potassium,chloride,calcium,bicarbonate<0.9*LLN,>1.1*ULN;platelets<0.5*LLN,>1.75*ULN; leukocytes(leu.),glucose<0.6*LLN,>1.5*ULN;lymphocytes(lym.), lym./leu.(%),neutrophils (neu.), neu./leu.(%), protein,albumin<0.8*LLN,>1.2*ULN;basophils(bas.), bas./leu.(%), eosinophils(eos.), eos./leu., monocytes(mon.), mon./leu.(%), urate>1.2*ULN;bilirubin (total, direct,indirect)>1.5*ULN;aspartate/alanine aminotransferase,gamma glutamyl transferase,lactate dehydrogenase,alkaline phosphatase>3.0*ULN;urea nitrogen,creatinine,triglycerides, chl.>1.3*ULN; sodium <0.95*LLN,>1.05*ULN; creatine kinase >2.0*ULN;Urine: pH<4.5,>8;glucose, ketones,protein, HGB,urobilinogen,bilirubin,nitrite,leukocyte esterase>=1;ery., leu.>= 20;hyaline casts>1;bacteria>20. | The safety analysis set in Stage 3 (SAS3) included all participants who received at least 1 dose of randomized treatment in Stage 3. | Posted | Count of Participants | Participants | Up to Week 40 |
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| Primary | Number of Participants With Vital Sign Abnormalities (Stage 3) | Abnormality in vital signs: Sitting pulse rate <40 beats per minute (bpm) to >120 bpm, sitting diastolic blood pressure (DBP) < 50 millimeter of mercury (mmHg), sitting systolic blood pressure (SBP) <90 mmHg. | The SAS3 included all participants who received at least 1 dose of randomized treatment in Stage 3. | Posted | Count of Participants | Participants | Baseline up to Week 40 |
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| Primary | Number of Participants With Electrocardiogram (ECG) Abnormalities (Stage 3) | ECG abnormalities criteria included: 1) QTc interval adjusted according to Fridericia formula (QTcF) (msec): >450, >480, >500, increase from baseline >=30, increase from baseline >=60; 2) Pulse rate (PR) (msec): >=300, change from baseline (Chg) >=25% or 50%; 3) QT (msec): >=500; 4) QRS (msec): >=200, Chg >=25% or 50%. Categories, with at least 1 participant having ECG abnormality in any of the reporting arms, were reported in this outcome measure. | The SAS3 included all participants who received at least 1 dose of randomized treatment in Stage 3. | Posted | Count of Participants | Participants | Baseline up to Week 40 |
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| Secondary | Number of Participants With TEAEs and SAEs (Stage 1 and Stage 2) | AE was any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE was any untoward medical occurrence that at any dose resulted in any of following outcomes/deemed significant for any other reason: death; initial /prolonged inpatient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly/birth defect and suspected transmission via a Pfizer product of an infectious agent, pathogenic or non-pathogenic. AEs included both serious (if occurred) and all non-serious adverse events. TEAEs are events between first dose of study drug and up to Week 28 that were absent before treatment or that worsened relative to pretreatment state. | The safety analysis set in Stage 1 (SAS1) and safety analysis set in Stage 2 (SAS2) included all participants who received at least 1 dose of randomized treatment in Stage 1 and Stage 2, respectively. | Posted | Count of Participants | Participants | Up to Week 28 |
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| Secondary | Number of Participants With TEAEs and SAEs (Amended Stage 2) | AE was any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE was any untoward medical occurrence that at any dose resulted in any of following outcomes/deemed significant for any other reason: death; initial /prolonged inpatient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly/birth defect and suspected transmission via a Pfizer product of an infectious agent, pathogenic or non-pathogenic. AEs included both serious (if occurred) and all non-serious adverse events. TEAEs are events between first dose of study drug and up to Week 40 that were absent before treatment or that worsened relative to pretreatment state. | The safety analysis set in Amended Stage 2 (SASA2) includes all participants who received at least one dose of randomized treatment in Amended Stage 2. | Posted | Count of Participants | Participants | Up to Week 40 |
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| Secondary | Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 1 and Stage 2) | HGB,hematocrit,ery.,HDL chl.<0.8*LLN;ret., ret./ery. (%)<0.5*LLN,>1.5*ULN;EMC volume,EMC HGB,EMC HGB concentration,potassium,chloride,calcium,bicarbonate<0.9*LLN,>1.1*ULN;platelets<0.5*LLN,>1.75*ULN;leu.,glucose<0.6*LLN,>1.5*ULN;lym., lym./leu.(%), neu., neu./leu. (%), protein,albumin <0.8*LLN,>1.2*ULN;bas., bas./leu.(%), eos., eos./leu., mon., mon./leu.(%), urate >1.2*ULN;bilirubin (total, direct, indirect)>1.5*ULN;aspartate/alanine aminotransferase, gamma glutamyl transferase, lactate dehydrogenase, alkaline phosphatase>3.0*ULN;urea nitrogen, creatinine, triglycerides, chl.>1.3*ULN; sodium <0.95*LLN,>1.05*ULN; creatine kinase >2.0*ULN;Urine: pH<4.5,>8;glucose, ketones, protein, HGB, urobilinogen,bilirubin,nitrite,leukocyte esterase>=1;ery., leu.>= 20;hyaline casts>1;bacteria>20. Clinical significance of laboratory parameters was determined at the investigator's discretion. | The SAS1 and SAS2 included all participants who received at least 1 dose of randomized treatment in Stage 1 and Stage 2, respectively. | Posted | Count of Participants | Participants | Up to Week 28 |
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| Secondary | Number of Participants With Clinically Significant Laboratory Abnormalities (Amended Stage 2) | HGB,hematocrit,ery.,HDL chl.<0.8*LLN;ret., ret./ery. (%)<0.5*LLN,>1.5*ULN;EMC volume,EMC HGB,EMC HGB concentration,potassium,chloride,calcium,bicarbonate<0.9*LLN,>1.1*ULN;platelets<0.5*LLN,>1.75*ULN;leu.,glucose<0.6*LLN,>1.5*ULN;lym., lym./leu.(%), neu., neu./leu. (%), protein,albumin <0.8*LLN,>1.2*ULN;bas., bas./leu.(%), eos., eos./leu., mon., mon./leu.(%), urate >1.2*ULN;bilirubin (total, direct, indirect)>1.5*ULN;aspartate/alanine aminotransferase, gamma glutamyl transferase, lactate dehydrogenase, alkaline phosphatase>3.0*ULN;urea nitrogen, creatinine, triglycerides, chl.>1.3*ULN; sodium <0.95*LLN,>1.05*ULN; creatine kinase >2.0*ULN;Urine: pH<4.5,>8;glucose, ketones, protein, HGB, urobilinogen,bilirubin,nitrite,leukocyte esterase>=1;ery., leu.>= 20;hyaline casts>1;bacteria>20. Clinical significance of laboratory parameters was determined at the investigator's discretion. | The SASA2 includes all participants who received at least one dose of randomized treatment in Amended Stage 2. | Posted | Count of Participants | Participants | Up to Week 40 |
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| Secondary | Number of Participants With Vital Sign Abnormalities (Stage 1 and Stage 2) | Abnormality in vital signs: Sitting pulse rate <40 bpm to >120 bpm, sitting DBP < 50 mmHg, sitting SBP <90 mmHg. | The SAS1 and SAS2 included all participants who received at least 1 dose of randomized treatment in Stage 1 and Stage 2, respectively. | Posted | Count of Participants | Participants | Up to Week 28 |
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| Secondary | Number of Participants With Vital Sign Abnormalities (Amended Stage 2) | Abnormality in vital signs: Sitting pulse rate <40 bpm to >120 bpm, sitting DBP < 50 mmHg, sitting SBP <90 mmHg. | The SASA2 includes all participants who received at least one dose of randomized treatment in Amended Stage 2. | Posted | Count of Participants | Participants | Up to Week 40 |
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| Secondary | Number of Participants With ECG Abnormalities (Stage 1 and Stage 2) | ECG abnormalities criteria included: 1) QTc interval adjusted according to Fridericia formula (QTcF) (msec): >450, >480, >500, increase from baseline >=30, increase from baseline >=60; 2) Pulse rate (PR) (msec): >=300, change from baseline (Chg) >=25% or 50%; 3) QT (msec): >=500; 4) QRS (msec): >=200, Chg >=25% or 50%. Categories, with at least 1 participant having ECG abnormality in any of the reporting arms, were reported in this outcome measure. | The SAS1 and SAS2 included all participants who received at least 1 dose of randomized treatment in Stage 1 and Stage 2, respectively. | Posted | Count of Participants | Participants | Up to Week 28 |
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| Secondary | Number of Participants With ECG Abnormalities (Amended Stage 2) | ECG abnormalities criteria included: 1) QTc interval adjusted according to Fridericia formula (QTcF) (msec): >450, >480, >500, increase from baseline >=30, increase from baseline >=60; 2) Pulse rate (PR) (msec): >=300, change from baseline (Chg) >=25% or 50%; 3) QT (msec): >=500; 4) QRS (msec): >=200, Chg >=25% or 50%. Categories, with at least 1 participant having ECG abnormality in any of the reporting arms, were reported in this outcome measure. | The SASA2 includes all participants who received at least one dose of randomized treatment in Amended Stage 2. | Posted | Count of Participants | Participants | Up to Week 40 |
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| Secondary | Change From Baseline in CDASI Activity Score at at All Scheduled Timepoints Through Week 12 (Stage 1, Stage 2 and Amended Stage 2) | The treatment effect was defined as the difference (mean change from baseline at Weeks 1, 4, 8 in the active treatment group minus the mean change from baseline at Weeks 1, 4, 8 in the placebo group) in the mean change of CDASI activity score from baseline at scheduled timepoints. The score (range: 0-100) consists of the ES, GHS, PS and AS. ES (range: 0-90) was obtained by summing up scores for the total erythema (ER [0-45], redness of the skin or mucous membranes), scaling (SC [0-30], peeling of the skin) and erosion/ulceration (EU [0-15], presence of the deeper wound). Total ER, SC and EU scores were calculated as a sum of the contributions from 15 individual areas of the body. GHS characterizes papules (swellings) on hand and is a sum of the papule's characterization score (0-6) and ulceration score (0-1). PS (0-2) characterizes abnormalities around nails. The AS (0-1) characterizes hair loss. Higher scores indicate greater disease severity. | The FAS in Stage 1, Stage 2, and Amended Stage 2 included all participants who received at least 1 dose of randomized treatment in in Stage 1, Stage 2, or Amended Stage 2. | Posted | Mean | Standard Deviation | Units on a scale | Baseline, Week 1, Week 4, and Week 8 (except for Week 12 which is a primary outcome measure) |
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| Secondary | Change From Baseline in CDASI Activity Score at All Scheduled Timepoints Through Week 12 (Stage 3) | The treatment effect was defined as the difference (mean change from baseline at Weeks 1, 4, 8,12 in the active treatment group minus the mean change from baseline at Weeks 1, 4, 8, 12 in the placebo group) in the mean change of CDASI activity score from baseline at scheduled timepoints. The score (range: 0-100) consists of the ES, GHS, PS and AS. ES (range: 0-90) was obtained by summing up scores for the total erythema (ER [0-45], redness of the skin or mucous membranes), scaling (SC [0-30], peeling of the skin) and erosion/ulceration (EU [0-15], presence of the deeper wound). Total ER, SC and EU scores were calculated as a sum of the contributions from 15 individual areas of the body. GHS characterizes papules (swellings) on hand and is a sum of the papule's characterization score (0-6) and ulceration score (0-1). PS (0-2) characterizes abnormalities around nails. The AS (0-1) characterizes hair loss. Higher scores indicate greater disease severity. | The Full Analysis Set in Stage 3 (FAS3) included all participants who received at least 1 dose of randomized treatment in Stage 3. | Posted | Mean | Standard Deviation | Units on a scale | Baseline, Week 1, Week 4, Week 8 and Week 12 |
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| Secondary | Absolute Values of CDASI Activity Score at All Scheduled Timepoints Through Week 12 (All Stages) | The CDASI activity score (range: 0-100) consists of the ES, GHS, PS and AS. ES (range: 0-90) was obtained by summing up scores for the total erythema (ER [0-45], redness of the skin or mucous membranes), scaling (SC [0-30], peeling of the skin) and erosion/ulceration (EU [0-15], presence of the deeper wound). Total ER, SC and EU scores were calculated as a sum of the contributions from 15 individual areas of the body. GHS characterizes papules (swellings) on hand and is a sum of the papule's characterization score (0-6) and ulceration score (0-1). PS (0-2) characterizes abnormalities around nails. The AS (0-1) characterizes hair loss. Higher scores indicate greater disease severity. | The FAS included all participants who received at least 1 dose of randomized treatment in any study stage. | Posted | Mean | Standard Deviation | Units on a scale | Baseline, Week 1, Week 4, Week 8 and Week 12 |
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| Secondary | Absolute Values of CDASI Damage Score at All Scheduled Timepoints Through Week 12 (All Stages) | The Damage Score (DS) was calculated as a sum of the total poilkiloderma score (POLS), total calcinosis score (CALS) and Gotorn's hands damage score (GHDS). The POLS characterizes specific dispigmentation in the particulal area and calcinosis score characterizes calcification of the skin in the particular area. The POLS and the CALS are summed up over 15 individual areas in the body and each of them has range 0-15. The GHDS has the range 0-2 so that the DS has the range 0-32. Higher scores indicate greater disease severity. | The FAS included all participants who received at least 1 dose of randomized treatment in any study stage. | Posted | Mean | Standard Deviation | Units on a scale | Baseline, Week 1, Week 4, Week 8 and Week 12 |
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| Secondary | Absolute Values for Total Improvement Score (TIS) at Week 12 and Intermediate Scheduled Time Points (Stage 3) | The TIS was the sum of all 6 improvement scores where higher score indicates worse status (PhGA [from the MDAAT, 0-20 scale], PtGA [0-10 scale], MMT [0-35 scale], HAQ-DI [0-10 scale], muscle enzymes [0-7.5 scale], and extramuscular global assessment [0-20 scale]) associated with the change in each core set measure. A total improvement score between 0 and 100 corresponded to the degree of improvement, with higher scores corresponding to a greater degree of improvement: of ≥20 represented minimal improvement, a score of ≥40 represented moderate improvement, and a score of ≥60 represented major improvement. | The FAS3 included all participants who received at least 1 dose of randomized treatment in Stage 3. | Posted | Least Squares Mean | 90% Confidence Interval | Units on a scale | Week 4, Week 8 and Week 12 |
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| Secondary | Change From Baseline in the Core Set Measures (CSM) of the TIS (Global Disease Activity [PhGA] and Extramuscular Global Assessment [EmGA]) (Stage 3) | PhGA: assessment of the severity of disease by the physician. The physician used the visual analog scale and put a mark on 0 cm (best) -10 cm (worst) scale where higher score indicated worse status. EmGA: overall evaluation of disease activity in all extramuscular systems using visual analog scale 0 cm (best) -10 cm (worst) scale where higher score indicated worse status. | The Full Analysis Set in Stage 3 (FAS3) included all participants who received at least 1 dose of randomized treatment in Stage 3. | Posted | Least Squares Mean | 90% Confidence Interval | Centimeter (cm) | Baseline, Week 4, Week 8 and Week 12 |
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| Secondary | Change From Baseline in the CSM of the TIS (PtGA) (Stage 3) | PtGA: assessment of the severity of disease by the participant/participant's guardian, using a visual analog scale from 0 mm (no evidence of disease activity) to 100 mm (extremely active or severe disease activity). Higher score indicated worse status. | The FAS3 included all participants who received at least 1 dose of randomized treatment in Stage 3. | Posted | Least Squares Mean | 90% Confidence Interval | Millimeter (mm) | Baseline, Week 4, Week 8 and Week 12 |
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| Secondary | Change From Baseline in the CSM of the TIS (MMT8 and HAQ01-HAQ-DI) (Stage 3) | Manual Muscle Testing-8 designated muscle groups (MMT-8): was a set of 8 designated muscles tested unilaterally generally on right side (left side used unless right side cannot be used). Potential score range was from 0 to 80, where higher scores denoted better health status. HAQ-DI: contained eight sections (including dressing & grooming, arising, eating, walking, hygiene, grip, reach, and activities). Each section had multiple questions that the participant used to rank their functionality and ranged from 0 to 3 where 0 = without any difficulty and 3 = unable to do. For each participant, the average ranking was calculated for each of the eight sections. HAQ-DI had a score range of 0 to 3, where higher score reflected worse status. | The FAS3 included all participants who received at least 1 dose of randomized treatment in Stage 3. | Posted | Least Squares Mean | 90% Confidence Interval | Units on a scale | Week 4, Week 8 and Week 12 |
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| Secondary | Change From Baseline in the CSM of the TIS (Aldolase and Creatine Kinase) (Stage 3) | The LS mean (with 90% CI) of CSM of the TIS (aldolase and creatine kinase) at weeks 4, 8, 12 were presented. | The FAS3 included all participants who received at least 1 dose of randomized treatment in Stage 3. | Posted | Least Squares Mean | 90% Confidence Interval | Units per litre (U/L) | Baseline, Week 4, Week 8 and Week 12 |
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Up to Week 40
The safety analysis set includes all participants who received at least one dose of randomized treatment in any stage. The AEs reported were treatment emergent.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Participants who received placebo in any stage. | 1 | 45 | 3 | 45 | 22 | 45 |
| EG001 | PF-06823859 150 mg IV | Participants who received PF-06823859 150 mg IV in any stage. | 0 | 17 | 0 | 17 | 13 | 17 |
| EG002 | PF-06823859 600 mg IV | Participants who received PF-06823859 600 mg IV in any stage. | 1 | 47 | 4 | 47 | 18 | 47 |
| EG003 | Total | This arm included all participants (32, 9, 16 and 18 participants who were randomized and treated in Stage 1, Stage 2, Amended Stage 2, and Stage 3, respectively) in the safety analysis set | 1 | 75 | 5 | 75 | 45 | 75 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cytopenia | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Leukopenia | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Colitis microscopic | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Ocular icterus | Hepatobiliary disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Haemophagocytic lymphohistiocytosis | Immune system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Central nervous system lesion | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Blood potassium decreased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Weight increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Parathyroid tumour benign | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v25.1 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Mental impairment | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Vaginal haemorrhage | Reproductive system and breast disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Dermatomyositis | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Rosacea | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 9, 2022 | May 26, 2023 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D003882 | Dermatomyositis |
| ID | Term |
|---|---|
| D017285 | Polymyositis |
| D009220 | Myositis |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
Not provided
Not provided
| Withdrawal by Subject |
|
| Adverse Event |
|
| 65-84 Years |
|
| >=85 Years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Black or African American |
|
| Asian |
|
| American Indian or Alaska Native |
|
| Native Hawaiian or Other Pacific Islander |
|
| Other |
|
| Multiracial |
|
| Not reported |
|
| OG007 | (Amended Stage 2) PF-06823859 150 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16 and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG008 | (Amended Stage 2) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
|
|
Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants entered a 4-month follow-up period or rolled over to the long-term extension study. |
|
|
|
| Counts |
|---|
| Participants |
|
|
| OG002 | (Stage 2) Placebo | Participants in this group were randomized to receive placebo on Day 1, Week 4, and Week 8. After week 12, participants went into a follow up period. |
| OG003 | (Stage 2) PF-06823859 150 mg IV | Participants in this group were randomized to receive PF-06823859 150 mg on Day 1, Week 4, and Week 8 After week 12, participants went into a follow up period. |
| OG004 | (Stage 2) PF-06823859 600 mg IV | Dosing occurred Day 1, Week 4, and Week 8. After week 12, participants went into a follow-up period. |
|
|
| OG002 | (Amended Stage 2) PF-06823859 150 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16 and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG003 | (Amended Stage 2) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
|
|
Participants were randomized to receive PF-06823859 600 mg on Day 1, Week 4, and Week 8 in Stage 1.
| OG002 | (Stage 2) Placebo | Participants in this group were randomized to receive placebo on Day 1, Week 4, and Week 8. After week 12, participants went into a follow up period. |
| OG003 | (Stage 2) PF-06823859 150 mg IV | Participants in this group were randomized to receive PF-06823859 150 mg on Day 1, Week 4, and Week 8 After week 12, participants went into a follow up period. |
| OG004 | (Stage 2) PF-06823859 600 mg IV | Dosing occurred Day 1, Week 4, and Week 8. After week 12, participants went into a follow-up period. |
|
|
Dosing occurred on Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG002 | (Amended Stage 2) PF-06823859 150 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16 and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG003 | (Amended Stage 2) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
|
|
Participants in this group were randomized to receive PF-06823859 150 mg on Day 1, Week 4, and Week 8 After week 12, participants went into a follow up period.
| OG004 | (Stage 2) PF-06823859 600 mg IV | Dosing occurred Day 1, Week 4, and Week 8. After week 12, participants went into a follow-up period. |
|
|
| OG003 |
| (Amended Stage 2) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 |
Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
|
|
| OG003 | (Stage 2) PF-06823859 150 mg IV | Participants in this group were randomized to receive PF-06823859 150 mg on Day 1, Week 4, and Week 8 After week 12, participants went into a follow up period. |
| OG004 | (Stage 2) PF-06823859 600 mg IV | Dosing occurred Day 1, Week 4, and Week 8. After week 12, participants went into a follow-up period. |
|
|
Dosing occurred Day 1, Weeks 4, 8, 12, 16 and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG003 | (Amended Stage 2) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
|
|
| PF-06823859 600 mg IV (Stage 1) |
Participants were randomized to receive PF-06823859 600 mg on Day 1, Week 4, and Week 8 in Stage 1. |
| OG002 | (Stage 2) Placebo | Participants in this group were randomized to receive placebo on Day 1, Week 4, and Week 8. After week 12, participants went into a follow up period. |
| OG003 | (Stage 2) PF-06823859 150 mg IV | Participants in this group were randomized to receive PF-06823859 150 mg on Day 1, Week 4, and Week 8 After week 12, participants went into a follow up period. |
| OG004 | (Stage 2) PF-06823859 600 mg IV | Dosing occurred Day 1, Week 4, and Week 8. After week 12, participants went into a follow-up period. |
| OG005 | (Amended Stage 2) Placebo Then Switched to PF-06823859 150 mg IV at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG006 | (Amended Stage 2) Placebo Then Switched to PF-06823859 600 mg IV at Week 12 | Dosing occurred on Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG007 | (Amended Stage 2) PF-06823859 150 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16 and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG008 | (Amended Stage 2) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
|
|
Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants entered a 4-month follow-up period or rolled over to the long-term extension study. |
|
|
| (Stage 2) Placebo |
Participants in this group were randomized to receive placebo on Day 1, Week 4, and Week 8. After week 12, participants went into a follow up period. |
| OG003 | (Stage 2) PF-06823859 150 mg IV | Participants in this group were randomized to receive PF-06823859 150 mg on Day 1, Week 4, and Week 8 After week 12, participants went into a follow up period. |
| OG004 | (Stage 2) PF-06823859 600 mg IV | Dosing occurred Day 1, Week 4, and Week 8. After week 12, participants went into a follow-up period. |
| OG005 | (Amended Stage 2) Placebo Then Switched to PF-06823859 150 mg IV at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG006 | (Amended Stage 2) Placebo Then Switched to PF-06823859 600 mg IV at Week 12 | Dosing occurred on Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG007 | (Amended Stage 2) PF-06823859 150 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16 and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG008 | (Amended Stage 2) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG009 | (Stage 3) Placebo Then Switched to PF-06823859 600 mg IV at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG010 | (Stage 3) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants entered a 4-month follow-up period or rolled over to the long-term extension study. |
|
|
| OG003 | (Stage 2) PF-06823859 150 mg IV | Participants in this group were randomized to receive PF-06823859 150 mg on Day 1, Week 4, and Week 8 After week 12, participants went into a follow up period. |
| OG004 | (Stage 2) PF-06823859 600 mg IV | Dosing occurred Day 1, Week 4, and Week 8. After week 12, participants went into a follow-up period. |
| OG005 | (Amended Stage 2) Placebo Then Switched to PF-06823859 150 mg IV at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG006 | (Amended Stage 2) Placebo Then Switched to PF-06823859 600 mg IV at Week 12 | Dosing occurred on Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG007 | (Amended Stage 2) PF-06823859 150 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16 and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG008 | (Amended Stage 2) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants then entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG009 | (Stage 3) Placebo Then Switched to PF-06823859 600 mg IV at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, 20. At Week 24, participants entered a 4-month follow-up period or rolled over to the long-term extension study. |
| OG010 | (Stage 3) PF-06823859 600 mg IV Then Switched to Placebo at Week 12 | Dosing occurred Day 1, Weeks 4, 8, 12, 16, and 20. At Week 24, participants entered a 4-month follow-up period or rolled over to the long-term extension study. |
|
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| Participants |
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