Dermatomyositis
Conditions
Brief summary
The purpose of this research study is to evaluate the long-term safety, and tolerability of PF-06823859 study drug in adult participants with Dermatomyositis (DM) from a qualifying study.
Interventions
DRUGAnti-Beta Interferon (PF-06823859)
IV infusion
Sponsors
Pfizer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Eligible participants will have completed the treatment period of a qualifying Dermatomyositis parent study. All participants will receive active study drug.
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Participants aged ≥18 and ≤80 with moderate to severe dermatomyositis (DM), that have completed the treatment period of a qualifying study. * Capable of giving signed informed consent. * Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures.
Exclusion criteria
* Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study * Participants who met discontinuation criteria at any point during the participating qualifying studies. * Participants with an ongoing safety event in the qualifying studies which, in the opinion of the investigator or sponsor, is an ongoing safety concern OR the participant has met safety monitoring criteria in the qualifying study that has not resolved.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | From Day 1 of dosing maximum up to Week 68 | An Adverse Event (AE) was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE was considered treatment emergent relative to a given treatment if the event occurred for the first time during the effective duration of treatment and was not seen prior to the start of treatment, or the event was seen prior to the start of treatment but increased in severity during treatment. AEs included both serious adverse events (SAEs) and all non-SAEs. |
| Number of Participants With Laboratory Abnormalities | From Day 1 of dosing maximum up to Week 68 | Hematology laboratory parameters: hemoglobin (grams per deciliter \[g/dL\]); hematocrit (percentage \[%\]); lymphocytes (10\^3 per (/) millimeter\[mm\]\^3); lymphocytes/leukocytes (%); neutrophils (10\^3/mm\^3) less than (\<)0.8\*lower limit of normal (LLN), leukocytes (10\^3/mm\^3) \<0.6\*LLN, neutrophils (10\^3/mm\^3); basophils (10\^3/mm\^3); basophils/leukocytes (%); monocytes/leukocytes (%); activated partial thromboplastin time (seconds \[sec\]); prothrombin time (sec) more than (\>)1.2\*upper limit of normal (ULN). Clinical chemistry: potassium (milliequivalents per liter \[mEq/L\]); bicarbonate (mEq/L) \<0.9\*LLN, creatine kinase (units per liter \[U/L\]) \>2.0\*ULN, glucose (milligram per deciliter \[mg/dl\]); glucose-fasting (mg/dl) \>1.5\*ULN. Urinalysis: Urine glucose; ketones; urine protein; urine hemoglobin; nitrite; leukocyte esterase; hyaline casts (1/per leukocytosis promoting factor (more than or equal to \[\>=\] 1, urine erythrocytes (scalar); urine leukocytes (scalar) \>=20. |
| Number of Participants According to Categorization of Changes in Vital Signs | From Day 1 of dosing maximum up to Week 68 | Vital signs included the following parameters: sitting diastolic blood pressure (millimetres of mercury \[mmHg\]) change \>=20 mmHg increase; sitting systolic blood pressure (mmHg) change \>=30 mmHg increase, sitting diastolic blood pressure (mmHg) change \>=20 mmHg decrease and sitting systolic blood pressure (mmHg) change \>=30 mmHg decrease. |
| Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | From Day 1 of dosing maximum up to Week 68 | ECG parameters evaluated were: PR interval value \>=300 milliseconds (msec); QRS duration value \>=200 msec; QT interval value \>=500 msec; corrected QT Interval using Fridericia's formula (QTCF) 450 less than or equal to (\<=) value \<480 msec, 480 \<=value\<500 msec and value\>=500 msec. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Weeks 12, 24, 36, 48 and 52 | CDASI is a validated DM-specific instrument designed to systematically quantify the extent of cutaneous disease. Disease involvement in 15 different anatomical locations was rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia were also captured. Total CDASI damage score was based on the physician's evaluation of two damage (poikiloderma, calcinosis) measures, and presence and severity of Gottron's papules. Total CDASI damage score ranged from 0 to 32, where higher scores indicated higher level of skin damage. |
| Total Improvement Score (TIS) at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Weeks 12, 24, 36, 48 and 52 | There are 6 core set measure that comprised of TIS: 1) Physician Global Assessment Score \[PhGA\] (from Myositis Disease Activity Assessment Tool \[MDAAT\], 0-100 mm or 0-10 centimeter (cm) on visual analogue scale \[VAS\], higher scores= worse health status); 2) Patient Global Assessment Score \[PtGA\] (0-100 mm or 0-10 cm on VAS, higher scores= worse status); 3) Manual Muscle Testing-8 (MMT-8) designated muscle groups (0-80, lower scores= higher level of disability); 4) Health Assessment Questionnaire Disability Index \[HAQ-DI\] (0-3, higher scores= worse status); 5) Global Extramuscular Disease Activity (from MDAAT, 0-10 cm on a VAS, higher scores= higher level of disability); 6) Participant's most elevated muscle enzymes. TIS was sum of all 6 improvement scores associated with the change in each core set measure. TIS ranged from 0 to 100; where TIS\>=20 shows minimal improvement, TIS \>=40 shows moderate improvement and TIS \>= 60 shows major improvement. |
| Change From Baseline in Physician Global Assessment (PhGA) Score at Week 12, 24, 36, 48 and 52: Muscle Cohort | Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52 | PhGA: Investigator was asked to evaluate the participant's overall disease activity on a VAS of 0 cm (very good) to 10 cm (very poor), higher scores indicated worse health status. |
| Change From Baseline in Patient Global Assessment (PtGA) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52 | PtGA was the assessment of the severity of disease by the participant/participant's guardian, using a VAS from 0 mm (no evidence of disease activity) to 100 mm (extremely active or severe disease activity). Higher score indicated worse status. |
| Change From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 52 | Baseline (before dose 1), Week 52 | CDASI is a validated DM-specific instrument designed to systematically quantify the extent of cutaneous disease. Disease involvement in 15 different anatomical locations was rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia were also captured. Total CDASI activity score was based on the physician's evaluation of three activities (erythema, scale, erosion/ulceration), presence and severity of Gottron's papules, periungual changes and alopecia. Total CDASI activity score ranged from 0 to 100, where higher scores indicated higher levels of disability. |
| Change From Baseline in Health Assessment Questionnaire and Disease Index (HAQ-DI) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52 | HAQ-DI consisted of 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. |
| Change From Baseline in Creatine Kinase at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52 | Creatine kinase is a muscle enzyme measured in units per liter (U/L). |
| Change From Baseline in Extramuscular Global Assessment From the Myositis Disease Activity Assessment Tool (MDAAT) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52 | MDAAT tool measures the degree of disease activity of extramuscular organ systems and muscle on a VAS of 0 to 10 cm, higher scores indicated higher level of disability. |
| Change From Baseline in Manual Muscle Testing-8 Designated Muscle Groups (MMT-8) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52 | MMT-8 is a tool that assesses muscle strength using manual muscle testing. Eight designated muscles are tested unilaterally with a total potential summed score of 0-80. Lower scores indicated a higher level of disability. |
| Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48 | Baseline (before dose 1), Weeks 12, 24, 36 and 48 | CDASI is a validated DM-specific instrument designed to systematically quantify the extent of cutaneous disease. Disease involvement in 15 different anatomical locations was rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia were also captured. Total CDASI activity score was based on the physician's evaluation of three activities (erythema, scale, erosion/ulceration), presence and severity of Gottron's papules, periungual changes and alopecia. Total CDASI activity score ranged from 0 to 100, where higher scores indicated higher levels of disability. |
| Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Weeks 12, 24, 36, 48 and 52 | CDASI is a validated DM-specific instrument designed to systematically quantify the extent of cutaneous disease. Disease involvement in 15 different anatomical locations was rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia were also captured. Total CDASI activity score was based on the physician's evaluation of three activities (erythema, scale, erosion/ulceration), presence and severity of Gottron's papules, periungual changes and alopecia. Total CDASI activity score ranged from 0 to 100, where higher scores indicated higher levels of disability. |
| Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Baseline (before dose on Day 1), Weeks 12, 24, 36, 48 and 52 | CDASI is a validated DM-specific instrument designed to systematically quantify the extent of cutaneous disease. Disease involvement in 15 different anatomical locations was rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia were also captured. Total CDASI damage score was based on the physician's evaluation of two damage (poikiloderma, calcinosis) measures, and presence and severity of Gottron's papules. Total CDASI damage score ranged from 0 to 32, where higher scores indicated higher level of skin damage. |
Countries
Hungary, Poland, Spain, United States
Participant flow
Recruitment details
Eligible participants with moderate to severe dermatomyositis (DM) who completed the treatment period of qualifying study C0251002 \[NCT03181893\] and had agreement from their study doctor to continue active treatment were enrolled in this study. A total of 24 participants (9 participants from the skin predominant cohort \[Amended stage 2\] and 15 participants from the muscle predominant cohort \[Stage 3\]) of the study C0251002 were enrolled in this open label, long-term extension (OLE) study.
Pre-assignment details
As planned, in participant flow discontinuations were reported by treatment.
Participants by arm
| Arm | Count |
|---|---|
| PF-06823859 600mg: All Participants Participants with DM received PF-06823859 600 mg IV once every 4 weeks. The treatment duration was up to 48 weeks (treatment period was up through and including Week 52). There was a follow-up period of 16 weeks post treatment period, however participants were assessed for safety from Day 1 of treatment up to end of study (Week 68). | 24 |
| Total | 24 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Follow-up | Withdrawal by Subject | 1 |
| Treatment Period | Lack of Efficacy | 1 |
| Treatment Period | Other | 1 |
| Treatment Period | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | PF-06823859 600mg: All Participants |
|---|---|
| Age, Continuous All Participants | 49.79 Years STANDARD_DEVIATION 12.73 |
| Age, Continuous Muscle Cohort | 48.60 Years STANDARD_DEVIATION 14.19 |
| Age, Continuous Skin Cohort | 51.78 Years STANDARD_DEVIATION 10.32 |
| Ethnicity (NIH/OMB) All Participants Hispanic or Latino | 5 Participants |
| Ethnicity (NIH/OMB) All Participants Not Hispanic or Latino | 19 Participants |
| Ethnicity (NIH/OMB) All Participants Unknown or Not Reported | 0 Participants |
| Ethnicity (NIH/OMB) Muscle Cohort Hispanic or Latino | 4 Participants |
| Ethnicity (NIH/OMB) Muscle Cohort Not Hispanic or Latino | 11 Participants |
| Ethnicity (NIH/OMB) Muscle Cohort Unknown or Not Reported | 0 Participants |
| Ethnicity (NIH/OMB) Skin Cohort Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Skin Cohort Not Hispanic or Latino | 8 Participants |
| Ethnicity (NIH/OMB) Skin Cohort Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) All Participants American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) All Participants Asian | 0 Participants |
| Race (NIH/OMB) All Participants Black or African American | 0 Participants |
| Race (NIH/OMB) All Participants More than one race | 0 Participants |
| Race (NIH/OMB) All Participants Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) All Participants Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) All Participants White | 24 Participants |
| Race (NIH/OMB) Muscle Cohort American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Muscle Cohort Asian | 0 Participants |
| Race (NIH/OMB) Muscle Cohort Black or African American | 0 Participants |
| Race (NIH/OMB) Muscle Cohort More than one race | 0 Participants |
| Race (NIH/OMB) Muscle Cohort Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Muscle Cohort Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) Muscle Cohort White | 15 Participants |
| Race (NIH/OMB) Skin Cohort American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Skin Cohort Asian | 0 Participants |
| Race (NIH/OMB) Skin Cohort Black or African American | 0 Participants |
| Race (NIH/OMB) Skin Cohort More than one race | 0 Participants |
| Race (NIH/OMB) Skin Cohort Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Skin Cohort Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) Skin Cohort White | 9 Participants |
| Sex: Female, Male All Participants Female | 20 Participants |
| Sex: Female, Male All Participants Male | 4 Participants |
| Sex: Female, Male Muscle Cohort Female | 11 Participants |
| Sex: Female, Male Muscle Cohort Male | 4 Participants |
| Sex: Female, Male Skin Cohort Female | 9 Participants |
| Sex: Female, Male Skin Cohort Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 9 | 0 / 15 | 0 / 24 |
| other Total, other adverse events | 7 / 9 | 13 / 15 | 20 / 24 |
| serious Total, serious adverse events | 1 / 9 | 2 / 15 | 3 / 24 |
Outcome results
Primary
Number of Participants According to Categorization of Changes in Vital Signs
Vital signs included the following parameters: sitting diastolic blood pressure (millimetres of mercury \[mmHg\]) change \>=20 mmHg increase; sitting systolic blood pressure (mmHg) change \>=30 mmHg increase, sitting diastolic blood pressure (mmHg) change \>=20 mmHg decrease and sitting systolic blood pressure (mmHg) change \>=30 mmHg decrease.
Time frame: From Day 1 of dosing maximum up to Week 68
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for skin cohort/analysis set, muscle cohort/analysis set and all participants/safety analysis set.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Number of Participants According to Categorization of Changes in Vital Signs | Increase: sitting diastolic blood pressure (mmHg) | 4 Participants |
| PF-06823859 600mg: Skin Cohort | Number of Participants According to Categorization of Changes in Vital Signs | Increase: sitting systolic blood pressure (mmHg) | 4 Participants |
| PF-06823859 600mg: Skin Cohort | Number of Participants According to Categorization of Changes in Vital Signs | Decrease: sitting diastolic blood pressure (mmHg) | 1 Participants |
| PF-06823859 600mg: Skin Cohort | Number of Participants According to Categorization of Changes in Vital Signs | Decrease: sitting systolic blood pressure (mmHg) | 1 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants According to Categorization of Changes in Vital Signs | Decrease: sitting systolic blood pressure (mmHg) | 3 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants According to Categorization of Changes in Vital Signs | Increase: sitting diastolic blood pressure (mmHg) | 4 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants According to Categorization of Changes in Vital Signs | Decrease: sitting diastolic blood pressure (mmHg) | 4 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants According to Categorization of Changes in Vital Signs | Increase: sitting systolic blood pressure (mmHg) | 2 Participants |
| PF-06823859 600mg: All Participants | Number of Participants According to Categorization of Changes in Vital Signs | Decrease: sitting systolic blood pressure (mmHg) | 4 Participants |
| PF-06823859 600mg: All Participants | Number of Participants According to Categorization of Changes in Vital Signs | Increase: sitting systolic blood pressure (mmHg) | 6 Participants |
| PF-06823859 600mg: All Participants | Number of Participants According to Categorization of Changes in Vital Signs | Decrease: sitting diastolic blood pressure (mmHg) | 5 Participants |
| PF-06823859 600mg: All Participants | Number of Participants According to Categorization of Changes in Vital Signs | Increase: sitting diastolic blood pressure (mmHg) | 8 Participants |
Primary
Number of Participants According to Categorization of Electrocardiogram (ECG) Findings
ECG parameters evaluated were: PR interval value \>=300 milliseconds (msec); QRS duration value \>=200 msec; QT interval value \>=500 msec; corrected QT Interval using Fridericia's formula (QTCF) 450 less than or equal to (\<=) value \<480 msec, 480 \<=value\<500 msec and value\>=500 msec.
Time frame: From Day 1 of dosing maximum up to Week 68
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for skin cohort/analysis set, muscle cohort/analysis set and all participants/safety analysis set.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | PR interval: value >=300 msec | 0 Participants |
| PF-06823859 600mg: Skin Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QRS duration: value >=200 msec | 0 Participants |
| PF-06823859 600mg: Skin Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QT interval: >=500 msec | 0 Participants |
| PF-06823859 600mg: Skin Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QTCF: 450<=value<480 msec | 2 Participants |
| PF-06823859 600mg: Skin Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QTCF: 480<=value<500 msec | 0 Participants |
| PF-06823859 600mg: Skin Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QTCF: value>=500 msec | 0 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QTCF: value>=500 msec | 0 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | PR interval: value >=300 msec | 0 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QTCF: 450<=value<480 msec | 1 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QTCF: 480<=value<500 msec | 0 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QRS duration: value >=200 msec | 0 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QT interval: >=500 msec | 0 Participants |
| PF-06823859 600mg: All Participants | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QRS duration: value >=200 msec | 0 Participants |
| PF-06823859 600mg: All Participants | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QT interval: >=500 msec | 0 Participants |
| PF-06823859 600mg: All Participants | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QTCF: value>=500 msec | 0 Participants |
| PF-06823859 600mg: All Participants | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QTCF: 450<=value<480 msec | 3 Participants |
| PF-06823859 600mg: All Participants | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | PR interval: value >=300 msec | 0 Participants |
| PF-06823859 600mg: All Participants | Number of Participants According to Categorization of Electrocardiogram (ECG) Findings | QTCF: 480<=value<500 msec | 0 Participants |
Primary
Number of Participants With Laboratory Abnormalities
Hematology laboratory parameters: hemoglobin (grams per deciliter \[g/dL\]); hematocrit (percentage \[%\]); lymphocytes (10\^3 per (/) millimeter\[mm\]\^3); lymphocytes/leukocytes (%); neutrophils (10\^3/mm\^3) less than (\<)0.8\*lower limit of normal (LLN), leukocytes (10\^3/mm\^3) \<0.6\*LLN, neutrophils (10\^3/mm\^3); basophils (10\^3/mm\^3); basophils/leukocytes (%); monocytes/leukocytes (%); activated partial thromboplastin time (seconds \[sec\]); prothrombin time (sec) more than (\>)1.2\*upper limit of normal (ULN). Clinical chemistry: potassium (milliequivalents per liter \[mEq/L\]); bicarbonate (mEq/L) \<0.9\*LLN, creatine kinase (units per liter \[U/L\]) \>2.0\*ULN, glucose (milligram per deciliter \[mg/dl\]); glucose-fasting (mg/dl) \>1.5\*ULN. Urinalysis: Urine glucose; ketones; urine protein; urine hemoglobin; nitrite; leukocyte esterase; hyaline casts (1/per leukocytosis promoting factor (more than or equal to \[\>=\] 1, urine erythrocytes (scalar); urine leukocytes (scalar) \>=20.
Time frame: From Day 1 of dosing maximum up to Week 68
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for skin cohort/analysis set, muscle cohort/analysis set and all participants/safety analysis set.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| PF-06823859 600mg: Skin Cohort | Number of Participants With Laboratory Abnormalities | 8 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants With Laboratory Abnormalities | 14 Participants |
| PF-06823859 600mg: All Participants | Number of Participants With Laboratory Abnormalities | 22 Participants |
Primary
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An Adverse Event (AE) was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE was considered treatment emergent relative to a given treatment if the event occurred for the first time during the effective duration of treatment and was not seen prior to the start of treatment, or the event was seen prior to the start of treatment but increased in severity during treatment. AEs included both serious adverse events (SAEs) and all non-SAEs.
Time frame: From Day 1 of dosing maximum up to Week 68
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for skin cohort/analysis set, muscle cohort/analysis set and all participants/safety analysis set.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| PF-06823859 600mg: Skin Cohort | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 7 Participants |
| PF-06823859 600mg: Muscle Cohort | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 13 Participants |
| PF-06823859 600mg: All Participants | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 20 Participants |
Secondary
Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52
CDASI is a validated DM-specific instrument designed to systematically quantify the extent of cutaneous disease. Disease involvement in 15 different anatomical locations was rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia were also captured. Total CDASI activity score was based on the physician's evaluation of three activities (erythema, scale, erosion/ulceration), presence and severity of Gottron's papules, periungual changes and alopecia. Total CDASI activity score ranged from 0 to 100, where higher scores indicated higher levels of disability.
Time frame: Weeks 12, 24, 36, 48 and 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for skin cohort/analysis set and muscle cohort/analysis set. Here, Number Analyzed signifies number evaluable for specified rows.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Week 24 | 5.0 Units on a scale | Standard Deviation 3.71 |
| PF-06823859 600mg: Skin Cohort | Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Week 48 | 4.6 Units on a scale | Standard Deviation 4.45 |
| PF-06823859 600mg: Skin Cohort | Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Week 36 | 5.5 Units on a scale | Standard Deviation 5.13 |
| PF-06823859 600mg: Skin Cohort | Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Week 52 | 4.4 Units on a scale | Standard Deviation 4.85 |
| PF-06823859 600mg: Skin Cohort | Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Week 12 | 4.4 Units on a scale | Standard Deviation 2.45 |
| PF-06823859 600mg: Muscle Cohort | Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Week 52 | 2.2 Units on a scale | Standard Deviation 2.52 |
| PF-06823859 600mg: Muscle Cohort | Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Week 12 | 4.4 Units on a scale | Standard Deviation 3.44 |
| PF-06823859 600mg: Muscle Cohort | Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Week 24 | 4.6 Units on a scale | Standard Deviation 3.81 |
| PF-06823859 600mg: Muscle Cohort | Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Week 36 | 3.9 Units on a scale | Standard Deviation 3.27 |
| PF-06823859 600mg: Muscle Cohort | Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52 | Week 48 | 3.1 Units on a scale | Standard Deviation 3.03 |
Secondary
Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52
CDASI is a validated DM-specific instrument designed to systematically quantify the extent of cutaneous disease. Disease involvement in 15 different anatomical locations was rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia were also captured. Total CDASI damage score was based on the physician's evaluation of two damage (poikiloderma, calcinosis) measures, and presence and severity of Gottron's papules. Total CDASI damage score ranged from 0 to 32, where higher scores indicated higher level of skin damage.
Time frame: Weeks 12, 24, 36, 48 and 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for skin cohort/analysis set and muscle cohort/analysis set. Here Number Analyzed signifies number evaluable for specified rows.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Week 24 | 1.9 Units on a scale | Standard Deviation 2.26 |
| PF-06823859 600mg: Skin Cohort | Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Week 48 | 1.7 Units on a scale | Standard Deviation 2.12 |
| PF-06823859 600mg: Skin Cohort | Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Week 36 | 1.4 Units on a scale | Standard Deviation 2.33 |
| PF-06823859 600mg: Skin Cohort | Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Week 52 | 1.6 Units on a scale | Standard Deviation 1.59 |
| PF-06823859 600mg: Skin Cohort | Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Week 12 | 0.9 Units on a scale | Standard Deviation 1.25 |
| PF-06823859 600mg: Muscle Cohort | Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Week 52 | 1.3 Units on a scale | Standard Deviation 3.19 |
| PF-06823859 600mg: Muscle Cohort | Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Week 12 | 1.6 Units on a scale | Standard Deviation 3.07 |
| PF-06823859 600mg: Muscle Cohort | Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Week 24 | 1.9 Units on a scale | Standard Deviation 3.41 |
| PF-06823859 600mg: Muscle Cohort | Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Week 36 | 1.9 Units on a scale | Standard Deviation 3.43 |
| PF-06823859 600mg: Muscle Cohort | Absolute Values of CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Week 48 | 1.3 Units on a scale | Standard Deviation 2.83 |
Secondary
Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48
CDASI is a validated DM-specific instrument designed to systematically quantify the extent of cutaneous disease. Disease involvement in 15 different anatomical locations was rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia were also captured. Total CDASI activity score was based on the physician's evaluation of three activities (erythema, scale, erosion/ulceration), presence and severity of Gottron's papules, periungual changes and alopecia. Total CDASI activity score ranged from 0 to 100, where higher scores indicated higher levels of disability.
Time frame: Baseline (before dose 1), Weeks 12, 24, 36 and 48
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for skin cohort/analysis set and muscle cohort/analysis set. Here Number Analyzed signifies number of participants evaluable for specified rows.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) |
|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48 | Change at Week 12 | -5.28 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48 | Change at Week 24 | -4.11 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48 | Change at Week 36 | -4.51 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48 | Change at Week 48 | -4.56 Units on a scale |
| PF-06823859 600mg: Muscle Cohort | Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48 | Change at Week 48 | -1.70 Units on a scale |
| PF-06823859 600mg: Muscle Cohort | Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48 | Change at Week 12 | -0.87 Units on a scale |
| PF-06823859 600mg: Muscle Cohort | Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48 | Change at Week 36 | -1.40 Units on a scale |
| PF-06823859 600mg: Muscle Cohort | Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48 | Change at Week 24 | -0.67 Units on a scale |
Secondary
Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52
CDASI is a validated DM-specific instrument designed to systematically quantify the extent of cutaneous disease. Disease involvement in 15 different anatomical locations was rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia were also captured. Total CDASI damage score was based on the physician's evaluation of two damage (poikiloderma, calcinosis) measures, and presence and severity of Gottron's papules. Total CDASI damage score ranged from 0 to 32, where higher scores indicated higher level of skin damage.
Time frame: Baseline (before dose on Day 1), Weeks 12, 24, 36, 48 and 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for skin cohort/analysis set and muscle cohort/analysis set. Here Number Analyzed signifies number evaluable for specified rows.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Change at Week 24 | -1.8 Units on a scale | Standard Deviation 2.33 |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Change at Week 48 | -2.0 Units on a scale | Standard Deviation 2 |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Change at Week 36 | -2.3 Units on a scale | Standard Deviation 2.25 |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Change at Week 52 | -2.1 Units on a scale | Standard Deviation 2.09 |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Change at Week 12 | -2.4 Units on a scale | Standard Deviation 2.77 |
| PF-06823859 600mg: Muscle Cohort | Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Change at Week 52 | -0.9 Units on a scale | Standard Deviation 1.08 |
| PF-06823859 600mg: Muscle Cohort | Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Change at Week 12 | -0.8 Units on a scale | Standard Deviation 1.15 |
| PF-06823859 600mg: Muscle Cohort | Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Change at Week 24 | -0.5 Units on a scale | Standard Deviation 1.19 |
| PF-06823859 600mg: Muscle Cohort | Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Change at Week 36 | -0.5 Units on a scale | Standard Deviation 1.06 |
| PF-06823859 600mg: Muscle Cohort | Change From Baseline in CDASI Damage Score at Weeks 12, 24, 36, 48, and 52 | Change at Week 48 | -0.9 Units on a scale | Standard Deviation 1.24 |
Secondary
Change From Baseline in Creatine Kinase at Weeks 12, 24, 36, 48 and 52: Muscle Cohort
Creatine kinase is a muscle enzyme measured in units per liter (U/L).
Time frame: Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for muscle cohort/analysis set. Here Number Analyzed signifies number evaluable for specified rows.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) |
|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Creatine Kinase at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 12 | -98.93 Units per liter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Creatine Kinase at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 24 | -58.53 Units per liter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Creatine Kinase at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 36 | -97.62 Units per liter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Creatine Kinase at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 48 | -72.04 Units per liter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Creatine Kinase at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 52 | -94.04 Units per liter |
Secondary
Change From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 52
CDASI is a validated DM-specific instrument designed to systematically quantify the extent of cutaneous disease. Disease involvement in 15 different anatomical locations was rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia were also captured. Total CDASI activity score was based on the physician's evaluation of three activities (erythema, scale, erosion/ulceration), presence and severity of Gottron's papules, periungual changes and alopecia. Total CDASI activity score ranged from 0 to 100, where higher scores indicated higher levels of disability.
Time frame: Baseline (before dose 1), Week 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for skin cohort/analysis set and muscle cohort/analysis set. Here, Number of Participants Analyzed signifies number evaluable for this outcome measure.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 52 | -4.67 Units on a scale |
| PF-06823859 600mg: Muscle Cohort | Change From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 52 | -2.51 Units on a scale |
Secondary
Change From Baseline in Extramuscular Global Assessment From the Myositis Disease Activity Assessment Tool (MDAAT) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort
MDAAT tool measures the degree of disease activity of extramuscular organ systems and muscle on a VAS of 0 to 10 cm, higher scores indicated higher level of disability.
Time frame: Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for muscle cohort/analysis set. Here Number Analyzed signifies number evaluable for specified rows.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) |
|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Extramuscular Global Assessment From the Myositis Disease Activity Assessment Tool (MDAAT) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 12 | 0.05 centimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Extramuscular Global Assessment From the Myositis Disease Activity Assessment Tool (MDAAT) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 24 | 0.23 centimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Extramuscular Global Assessment From the Myositis Disease Activity Assessment Tool (MDAAT) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 36 | 0.01 centimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Extramuscular Global Assessment From the Myositis Disease Activity Assessment Tool (MDAAT) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 48 | -0.16 centimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Extramuscular Global Assessment From the Myositis Disease Activity Assessment Tool (MDAAT) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 52 | -0.56 centimeter |
Secondary
Change From Baseline in Health Assessment Questionnaire and Disease Index (HAQ-DI) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort
HAQ-DI consisted of 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.
Time frame: Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for muscle cohort/analysis set. Here Number Analyzed signifies number evaluable for specified rows.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) |
|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Health Assessment Questionnaire and Disease Index (HAQ-DI) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 12 | 0.00 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Health Assessment Questionnaire and Disease Index (HAQ-DI) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 24 | -0.10 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Health Assessment Questionnaire and Disease Index (HAQ-DI) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 36 | -0.07 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Health Assessment Questionnaire and Disease Index (HAQ-DI) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 48 | -0.12 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Health Assessment Questionnaire and Disease Index (HAQ-DI) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 52 | -0.18 Units on a scale |
Secondary
Change From Baseline in Manual Muscle Testing-8 Designated Muscle Groups (MMT-8) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort
MMT-8 is a tool that assesses muscle strength using manual muscle testing. Eight designated muscles are tested unilaterally with a total potential summed score of 0-80. Lower scores indicated a higher level of disability.
Time frame: Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for muscle cohort/analysis set. Here Number Analyzed signifies number evaluable for specified rows.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) |
|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Manual Muscle Testing-8 Designated Muscle Groups (MMT-8) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 12 | -0.06 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Manual Muscle Testing-8 Designated Muscle Groups (MMT-8) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 24 | 1.85 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Manual Muscle Testing-8 Designated Muscle Groups (MMT-8) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 36 | 4.92 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Manual Muscle Testing-8 Designated Muscle Groups (MMT-8) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 48 | 8.00 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Manual Muscle Testing-8 Designated Muscle Groups (MMT-8) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 52 | 9.84 Units on a scale |
Secondary
Change From Baseline in Patient Global Assessment (PtGA) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort
PtGA was the assessment of the severity of disease by the participant/participant's guardian, using a VAS from 0 mm (no evidence of disease activity) to 100 mm (extremely active or severe disease activity). Higher score indicated worse status.
Time frame: Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for muscle cohort/analysis set. Here Number Analyzed signifies number evaluable for specified rows.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) |
|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Patient Global Assessment (PtGA) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 12 | 3.70 millimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Patient Global Assessment (PtGA) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 24 | -9.63 millimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Patient Global Assessment (PtGA) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 36 | -9.10 millimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Patient Global Assessment (PtGA) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 48 | -8.36 millimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Patient Global Assessment (PtGA) Score at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 52 | -6.28 millimeter |
Secondary
Change From Baseline in Physician Global Assessment (PhGA) Score at Week 12, 24, 36, 48 and 52: Muscle Cohort
PhGA: Investigator was asked to evaluate the participant's overall disease activity on a VAS of 0 cm (very good) to 10 cm (very poor), higher scores indicated worse health status.
Time frame: Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for muscle cohort/analysis set. Here Number Analyzed signifies number evaluable for specified rows.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) |
|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Physician Global Assessment (PhGA) Score at Week 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 12 | 0.17 centimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Physician Global Assessment (PhGA) Score at Week 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 24 | 0.29 centimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Physician Global Assessment (PhGA) Score at Week 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 36 | -0.18 centimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Physician Global Assessment (PhGA) Score at Week 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 48 | -0.48 centimeter |
| PF-06823859 600mg: Skin Cohort | Change From Baseline in Physician Global Assessment (PhGA) Score at Week 12, 24, 36, 48 and 52: Muscle Cohort | Change at Week 52 | -0.60 centimeter |
Secondary
Total Improvement Score (TIS) at Weeks 12, 24, 36, 48 and 52: Muscle Cohort
There are 6 core set measure that comprised of TIS: 1) Physician Global Assessment Score \[PhGA\] (from Myositis Disease Activity Assessment Tool \[MDAAT\], 0-100 mm or 0-10 centimeter (cm) on visual analogue scale \[VAS\], higher scores= worse health status); 2) Patient Global Assessment Score \[PtGA\] (0-100 mm or 0-10 cm on VAS, higher scores= worse status); 3) Manual Muscle Testing-8 (MMT-8) designated muscle groups (0-80, lower scores= higher level of disability); 4) Health Assessment Questionnaire Disability Index \[HAQ-DI\] (0-3, higher scores= worse status); 5) Global Extramuscular Disease Activity (from MDAAT, 0-10 cm on a VAS, higher scores= higher level of disability); 6) Participant's most elevated muscle enzymes. TIS was sum of all 6 improvement scores associated with the change in each core set measure. TIS ranged from 0 to 100; where TIS\>=20 shows minimal improvement, TIS \>=40 shows moderate improvement and TIS \>= 60 shows major improvement.
Time frame: Weeks 12, 24, 36, 48 and 52
Population: Safety Analysis Set included all participants enrolled who took at least 1 dose of study intervention, regardless of which stage the participant entered from. In this outcome measure data was planned to be reported for muscle cohort/analysis set. Here Number Analyzed signifies number evaluable for specified rows.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) |
|---|---|---|---|
| PF-06823859 600mg: Skin Cohort | Total Improvement Score (TIS) at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Week 12 | 15.03 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Total Improvement Score (TIS) at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Week 24 | 15.67 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Total Improvement Score (TIS) at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Week 36 | 18.17 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Total Improvement Score (TIS) at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Week 48 | 19.61 Units on a scale |
| PF-06823859 600mg: Skin Cohort | Total Improvement Score (TIS) at Weeks 12, 24, 36, 48 and 52: Muscle Cohort | Week 52 | 23.66 Units on a scale |