Mepolizumab Long-term Study to Assess Real World Safety and Effectiveness of Eosinophilic Granulomatosis With Polyangiitis (EGPA) in Japan (MARS)

A Single Arm, Multi-center Study to Assess the Long-term Real-world Safety and Effectiveness of Nucala in EGPA Patients Who Have Already Used Nucala for at Least 96 Weeks in Japan

Eosinophilic granulomatosis with polyangiitis (EGPA), formerly known as the Churg-Strauss syndrome, is a systemic necrotizing vasculitis that affects small and medium sized blood vessels. NUCALA® (mepolizumab 300 milligrams [mg], subcutaneous administration) was approved in Japan in 2018 for the treatment of EGPA in adult participants. This is a single-arm, multi-center, prospective, non-interventional study that aims to assess long-term (2 to 4 years) real-world safety and effectiveness of NUCALA. Approximately 120 participants who completed the NUCALA Post Marketing Surveillance (PMS) study (National Clinical Trial [NCT]03557060) will be enrolled in the study.

NUCALA is a registered trademark of GlaxoSmithKline (GSK) group of companies.

Study Overview

• Status: Active, not recruiting
• Conditions: Churg-Strauss Syndrome

• Study Type: Observational
• Enrollment (Anticipated): 120

Contacts and Locations

Study Locations

• Japan
  • Aichi, Japan, 489-8642
    • GSK Investigational Site
  • Gunma, Japan, 371-8511
    • GSK Investigational Site
  • Hyogo, Japan, 653-0013
    • GSK Investigational Site
  • Hyogo, Japan, 670-8520
    • GSK Investigational Site
  • Hyogo, Japan, 674-0063
    • GSK Investigational Site
  • Hyogo, Japan, 675-8611
    • GSK Investigational Site
  • Ishikawa, Japan, 920-8530
    • GSK Investigational Site
  • Ishikawa, Japan, 923-8560
    • GSK Investigational Site
  • Kanagawa, Japan, 252-0392
    • GSK Investigational Site
  • Kanagawa, Japan, 216-8511
    • GSK Investigational Site
  • Kanagawa, Japan, 241-0801
    • GSK Investigational Site
  • Kanagawa, Japan, 241-0811
    • GSK Investigational Site
  • Kochi, Japan, 780-8522
    • GSK Investigational Site
  • Mie, Japan, 510-8567
    • GSK Investigational Site
  • Mie, Japan, 511-0061
    • GSK Investigational Site
  • Miyagi, Japan, 980-8574
    • GSK Investigational Site
  • Osaka, Japan, 534-0021
    • GSK Investigational Site
  • Osaka, Japan, 533-0024
    • GSK Investigational Site
  • Saitama, Japan, 350-8550
    • GSK Investigational Site
  • Shiga, Japan, 520-2192
    • GSK Investigational Site
  • Shizuoka, Japan, 430-8525
    • GSK Investigational Site
  • Shizuoka, Japan, 436-8555
    • GSK Investigational Site
  • Tokyo, Japan, 113-8655
    • GSK Investigational Site
  • Tokyo, Japan, 113-8603
    • GSK Investigational Site
  • Tokyo, Japan, 104-8560
    • GSK Investigational Site
  • Tokyo, Japan, 113-8431
    • GSK Investigational Site
  • Tokyo, Japan, 204-8585
    • GSK Investigational Site
  • Tokyo, Japan, 173-8606
    • GSK Investigational Site
  • Tokyo, Japan, 183-8524
    • GSK Investigational Site
  • Tokyo, Japan, 190-0014
    • GSK Investigational Site
  • Tokyo, Japan, 194-0023
    • GSK Investigational Site
  • Tottori, Japan, 680-0833
    • GSK Investigational Site
  • Wakayama, Japan, 640-8558
    • GSK Investigational Site
  • Yamaguchi, Japan, 755-8505
    • GSK Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Adult participants with EGPA who have already received NUCALA treatment for 96 weeks after its market launch in Japan.

Description

Inclusion Criteria:

• Adult participants with EGPA of >=20 years of age inclusive, at the time of signing the informed consent.
• Participants must have a current clinical diagnosis of EGPA by physician.

• Participants have continuously used NUCALA for at least 96 weeks for the treatment of EGPA as mentioned in the current label in Japan.

  • Participants thus were registered and completed the NUCALA PMS study (special drug use investigation; Protocol Number 208505, NCT03557060) prior to be enrolled in this study.

• Physician's decision to continue treatment with NUCALA for the treatment of EGPA as mentioned in the current label in Japan.
• Prior to commencing any study related activities, participants must be able and willing to provide written informed consent.

Exclusion Criteria:

• Participants who have previously discontinued NUCALA treatment for EGPA for more than 12 weeks.
• Participating in another clinical trial within the past 12 months, in which the participant has been exposed to an investigational or non-investigational pharmaceutical product.
• Participants with any reasons that in physician's opinion would place the participants at risk.
• Participants who are pregnant or breastfeeding.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Participants with EGPA who have received NUCALA treatment; Data will be collected of participants who have already received NUCALA for 96 weeks in routine clinical practice.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with Adverse events (AEs), Serious adverse events (SAEs) and Adverse events of special interest (AESI)	AEs, SAEs, and AESIs will be collected.	Up to 96 weeks
Number of participants with Adverse drug reactions (ADRs)	ADRs related to NUCALA will be collected.	Up to 96 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of participants with clinical symptoms	Clinical symptoms relevant to EGPA in systemic vasculitis will be assessed.	Up to 96 weeks
Percentage of participants with EGPA relapse	EGPA relapse is defined as any of the following with worsening EGPA: increased dose of oral corticosteroids (OCS), initiation/increased dose of immuno-suppressive agents or EGPA treatment with hospitalization.	Up to 96 weeks
Number of participants with hospitalization for EGPA-related events	Participants with hospitalization for EGPA-related events will be assessed.	Up to 96 weeks
Number of participants with emergency room/unscheduled visits for EGPA-related events	Participants with emergency room/unscheduled visits for EGPA-related events will be assessed.	Up to 96 weeks
Number of participants receiving prednisolone-equivalent of OCS over 96 weeks	Number of participants receiving prednisolone-equivalent of OCS over 96 weeks will be assessed.	Up to 96 weeks
Percentage of participants in each category of average daily prednisolone-equivalent of OCS over 96 weeks	Percentage of participants in each category of average daily prednisolone-equivalent of OCS over 96 weeks will be assessed. The dosing categories will include: zero, greater than (>)0 to less than or equal to (<=)4.0 mg/day, >4.0 to <=7.5 mg/day, >7.5 mg/day.	Up to 96 weeks

Collaborators and Investigators

• Sponsor: GlaxoSmithKline

Study record dates

Study Major Dates

• Study Start (Actual): December 11, 2020
• Primary Completion (Anticipated): April 3, 2023
• Study Completion (Anticipated): April 3, 2023

Study Registration Dates

• First Submitted: September 11, 2020
• First Submitted That Met QC Criteria: September 11, 2020
• First Posted (Actual): September 16, 2020

Study Record Updates

• Last Update Posted (Actual): September 21, 2022
• Last Update Submitted That Met QC Criteria: September 20, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Keywords: NUCALA; Eosinophilic granulomatosis with polyangiitis; Real world setting; Long term safety and efficacy
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Respiratory Tract Diseases; Immune System Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Autoimmune Diseases; Lung Diseases; Vasculitis; Lung Diseases, Interstitial; Systemic Vasculitis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Granuloma; Granulomatosis with Polyangiitis; Churg-Strauss Syndrome
• Other Study ID Numbers: 213684

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

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