An Extension Study for Participants Who Have Completed the Treatment Period of a Qualifying Parent Study

AN OPEN LABEL, LONG-TERM EXTENSION STUDY TO INVESTIGATE THE SAFETY OF PF-06823859 ADMINISTERED TO ADULT PARTICIPANTS ≥18 AND ≤80 WITH ACTIVE DERMATOMYOSITIS.

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 Overview

• Status: Completed
• Conditions: Dermatomyositis
• Intervention / Treatment: Drug: Anti-Beta Interferon (PF-06823859)

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 2

Contacts and Locations

Study Locations

• Hungary
  • Debrecen, Hungary, 4032
    • Debreceni Egyetem Klinikai Kozpont
• Poland
  • Krakow, Poland, 30-363
    • Centrum Medyczne Plejady
  • Podlaskie
    • Białystok, Podlaskie, Poland, 15-707
      • Nova Reuma Społka Partnerska
• Spain
  • Madrid, Spain, 28041
    • Hospital Universitario 12 de Octubre
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • University of Alabama at Birmingham
    • Birmingham, Alabama, United States, 35294
      • University of Alabama at Birmingham, Department of Dermatology
  • Arizona
    • Scottsdale, Arizona, United States, 85259
      • Mayo Clinic
  • California
    • Beverly Hills, California, United States, 90211
      • Attune Health Research Inc.
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic in Florida
  • Kansas
    • Fairway, Kansas, United States, 66205
      • KU Clinical Research Center - Clinical and Translational Science Unit (CTSU)
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center
    • Kansas City, Kansas, United States, 66160
      • KU Clinical and Translational Science Unit (CTSU) Rainbow
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Brigham & Women's Hospital
    • Boston, Massachusetts, United States, 02115
      • Brigham and Women's Hospital - CTH
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine
    • Saint Louis, Missouri, United States, 63108
      • Washington University School of Medicine
    • Saint Louis, Missouri, United States, 63108
      • Center for Outpatient Health
  • New York
    • New York, New York, United States, 10029
      • Icahn School of Medicine at Mount Sinai
    • New York, New York, United States, 10016
      • NYU Langone Health Clinical Research Center
    • New York, New York, United States, 10028
      • Mount Sinai Doctors Dermatology
    • New York, New York, United States, 10017
      • NYU Langone Radiology - Ambulatory Care Center East 41st Street
    • New York, New York, United States, 10016
      • NYU Grossman School of Medicine, The Ronald O. Perelman Department of Dermatology
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic Foundation
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Center for Human Phenomic Science
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania, Perelman Center for Advanced Medicine (PCAM)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Anti-Beta Interferon drug (PF-06823859); IV infusion	Drug: Anti-Beta Interferon (PF-06823859); IV infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	From Day 1 of dosing maximum up to Week 68
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.	From Day 1 of dosing maximum up to Week 68

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	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.	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.	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.	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.	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.	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.	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.	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).	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.	Baseline (before dose on day 1), Weeks 12, 24, 36, 48 and 52

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2021
• Primary Completion (Actual): November 20, 2023
• Study Completion (Actual): November 20, 2023

Study Registration Dates

• First Submitted: December 1, 2021
• First Submitted That Met QC Criteria: January 10, 2022
• First Posted (Actual): January 14, 2022

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 5, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Connective Tissue Diseases; Skin Diseases; Myositis; Polymyositis; Dermatomyositis; Anti-Infective Agents; Antineoplastic Agents; Immunologic Factors; Physiological Effects of Drugs; Antiviral Agents; Interferons; Interferon-beta
• Other Study ID Numbers: C0251008; 2021-004787-10 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No