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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
24
IV infusion
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
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
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
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University of Alabama at Birmingham
Birmingham, Alabama, United States
University of Alabama at Birmingham, Department of Dermatology
Birmingham, Alabama, United States
Mayo Clinic
Scottsdale, Arizona, United States
Attune Health Research Inc.
Beverly Hills, California, United States
Mayo Clinic in Florida
Jacksonville, Florida, United States
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU)
Fairway, Kansas, United States
KU Clinical and Translational Science Unit (CTSU) Rainbow
Kansas City, Kansas, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Brigham & Women's Hospital
Boston, Massachusetts, United States
Brigham and Women's Hospital - CTH
Boston, Massachusetts, United States
...and 15 more locations
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
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
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
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
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
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
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
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
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
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
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
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
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