A Study to Evaluate PF-06823859 in People With Active Idiopathic Inflammatory Myopathies
Research type
Research Study
Full title
C0251006 - A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study To Evaluate The Efficacy And Safety Of Pf-06823859 In Participants With Active Idiopathic Inflammatory Myopathies (Including Participants With Active Dermatomyositis Or Polymyositis)
IRAS ID
1008220
Contact name
Keith Kanik
Contact email
Sponsor organisation
Pfizer Inc.
Research summary
This study will investigate the efficacy and safety of PF-06823859, in adult participants with idiopathic inflammatory myopathies (IIM), including participants with dermatomyositis (DM) or polymyositis (PM). IIM are a group of chronic autoimmune disorders which are rare, serious, and often debilitating to patients due to muscle weakness. Eligible participants are required to have active disease at the time of enrollment and are required to enter the study on protocol-permitted standard of care background medication. The primary objective of the study is to evaluate the efficacy of PF-06823859 in reducing muscle symptoms in adult participants with active DM and adult participants with active PM. The study will be conducted in 2 parallel cohorts: Cohort 1 (DM, N=111) and Cohort 2 (PM, N=159). The study includes a Screening Period and a Treatment Period for all participants, and an Observation Period for participants who discontinue study intervention early. Eligible participants will be randomly assigned (2:1) to receive the study drug (PF-06823859) or a placebo during the Treatment Period. The study drug or placebo will be given as an intravenous infusion every 4 weeks from Day 1 to Week 48 of the study. Participants permanently discontinuing from study drug before Week 52 will be asked to continue with the study visits until Week 52 as part of an Observation Period. At the end of the study (Week 52) visit, eligible participants will have the option to participate in an open-label extension study in which all participants receive PF-06823859. This study will involve visits with the study doctor every 4 weeks. Study procedures include blood tests, urine collection, clinical questionnaires/assessments, physical examination, vital signs, height, weight, lung function tests, electrocardiogram, skin photography, and use of digital tools for physical activity monitoring and video recording of a single assessment.
REC name
West of Scotland REC 1
REC reference
23/WS/0159
Date of REC Opinion
21 Dec 2023
REC opinion
Further Information Favourable Opinion