Clinical trial NCT04551989: Mepolizumab Long-term Study to Assess Real World Safety and Effectiveness of Eosinophilic Granulomatosis With Polyangiitis (EGPA) in Japan
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Churg-Strauss Syndrome
Eosinophilic Granulomatosis With Polyangiitis
Keywords: NUCALA
Eosinophilic granulomatosis with polyangiitis
Real world setting
Long term safety and efficacy
Lead sponsor: GlaxoSmithKline
Sponsor class: INDUSTRY
First posted: 2020-09-16
Enrollment: 118
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Any Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESI)
timeFrame: Up to 96 weeks
measure: Number of Participants With Adverse Drug Reactions (ADRs)
timeFrame: Up to 96 weeks
Secondary outcomes:
measure: Percentage of Participants With Clinical Symptoms
timeFrame: At 96 weeks
measure: Percentage of Participants With Eosinophilic Granulomatosis With Polyangiitis (EGPA) Relapse
timeFrame: Up to 96 weeks
measure: Annualized Rate of Hospitalization for EGPA-related Events
timeFrame: Up to 96 weeks
measure: Annualized Rate of Emergency Room/Unscheduled Visit for EGPA-related Events
timeFrame: Up to 96 weeks
measure: Average Daily Dose (Prednisolone-equivalent) of Oral Corticosteroid (OCS)
timeFrame: Week 0, Weeks 9-12, Weeks 21-24, Weeks 33-36, Weeks 45-48, Weeks 57-60, Weeks 69-72, Weeks 81-84, Weeks 93-96
measure: Number of Participants by Dosing Categories Relative to Average Daily OCS (Prednisolone-equivalent)
timeFrame: Week 0, Weeks 9-12, Weeks 21-24, Weeks 33-36, Weeks 45-48, Weeks 57-60, Weeks 69-72, Weeks 81-84, Weeks 93-96
Other outcomes:
Locations:
city: Aichi
country: Japan
facility: GSK Investigational Site
city: Gunma
country: Japan
facility: GSK Investigational Site
city: Hyogo
country: Japan
facility: GSK Investigational Site
city: Hyogo
country: Japan
facility: GSK Investigational Site
city: Hyogo
country: Japan
facility: GSK Investigational Site
city: Hyogo
country: Japan
facility: GSK Investigational Site
city: Ishikawa
country: Japan
facility: GSK Investigational Site
city: Ishikawa
country: Japan
facility: GSK Investigational Site
city: Kanagawa
country: Japan
facility: GSK Investigational Site
city: Kanagawa
country: Japan
facility: GSK Investigational Site
city: Kanagawa
country: Japan
facility: GSK Investigational Site
city: Kanagawa
country: Japan
facility: GSK Investigational Site
city: Kochi
country: Japan
facility: GSK Investigational Site
city: Mie
country: Japan
facility: GSK Investigational Site
city: Mie
country: Japan
facility: GSK Investigational Site
city: Miyagi
country: Japan
facility: GSK Investigational Site
city: Osaka
country: Japan
facility: GSK Investigational Site
city: Osaka
country: Japan
facility: GSK Investigational Site
city: Saitama
country: Japan
facility: GSK Investigational Site
city: Shiga
country: Japan
facility: GSK Investigational Site
city: Shizuoka
country: Japan
facility: GSK Investigational Site
city: Shizuoka
country: Japan
facility: GSK Investigational Site
city: Tokyo
country: Japan
facility: GSK Investigational Site
city: Tokyo
country: Japan
facility: GSK Investigational Site
city: Tokyo
country: Japan
facility: GSK Investigational Site
city: Tokyo
country: Japan
facility: GSK Investigational Site
city: Tokyo
country: Japan
facility: GSK Investigational Site
city: Tokyo
country: Japan
facility: GSK Investigational Site
city: Tokyo
country: Japan
facility: GSK Investigational Site
city: Tokyo
country: Japan
facility: GSK Investigational Site
city: Tokyo
country: Japan
facility: GSK Investigational Site
city: Tottori
country: Japan
facility: GSK Investigational Site
city: Wakayama
country: Japan
facility: GSK Investigational Site
city: Yamaguchi
country: Japan
facility: GSK Investigational Site
Study officials:
affiliation: GlaxoSmithKline
name: GSK Clinical Trials
role: STUDY_DIRECTOR
References: