An Extension Study for Participants Who Have Completed the Treatment Period of a Qualifying Parent Study
- Conditions
- Dermatomyositis
- Interventions
- Drug: Anti-Beta Interferon (PF-06823859)
- Registration Number
- NCT05192200
- Lead Sponsor
- Pfizer
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 24
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Anti-Beta Interferon drug (PF-06823859) Anti-Beta Interferon (PF-06823859) IV infusion
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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 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 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 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.
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.
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.
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 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 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).
Trial Locations
- Locations (24)
Mayo Clinic
🇺🇸Scottsdale, Arizona, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Debreceni Egyetem Klinikai Kozpont
🇭🇺Debrecen, Hungary
Nova Reuma Społka Partnerska
🇵🇱Białystok, Podlaskie, Poland
Centrum Medyczne Plejady
🇵🇱Krakow, Poland
KU Clinical and Translational Science Unit (CTSU) Rainbow
🇺🇸Kansas City, Kansas, United States
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
University of Alabama at Birmingham, Department of Dermatology
🇺🇸Birmingham, Alabama, United States
Brigham & Women's Hospital
🇺🇸Boston, Massachusetts, 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
Brigham and Women's Hospital - CTH
🇺🇸Boston, Massachusetts, United States
Center for Outpatient Health
🇺🇸Saint Louis, Missouri, United States
NYU Grossman School of Medicine, The Ronald O. Perelman Department of Dermatology
🇺🇸New York, New York, United States
NYU Langone Health Clinical Research Center
🇺🇸New York, New York, United States
NYU Langone Radiology - Ambulatory Care Center East 41st Street
🇺🇸New York, New York, United States
Mount Sinai Doctors Dermatology
🇺🇸New York, New York, United States
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
Center for Human Phenomic Science
🇺🇸Philadelphia, Pennsylvania, United States
University of Pennsylvania, Perelman Center for Advanced Medicine (PCAM)
🇺🇸Philadelphia, Pennsylvania, United States
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States