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

Active, not recruiting

• Conditions: Churg-Strauss Syndrome; Eosinophilic Granulomatosis With Polyangiitis

• Registration Number: NCT04551989
• Lead Sponsor: GlaxoSmithKline

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-09-11
• Study First Submitted QC: 2020-09-11
• Study First Posted: 2020-09-16
• Study Start: 2020-12-11
• Status Verified: 2022-09
• Last Update Submitted: 2022-09-20
• Last Update Posted: 2022-09-21
• Primary Completion: 2023-04-03
• Study Completion: 2023-04-03

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 120

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of Participants With Any Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESI)	Up to 96 weeks	An AE is any untoward medical occurrence in a clinical study participant, temporally associated with study participation, whether or not considered related to study participation. An SAE is defined as any untoward medical occurrence that, at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect, and other situations which involve medical or scientific judgment. An AESI may be of scientific and medical concern related to the treatment, monitored, and rapidly communicated by investigator to sponsor. AESIs included Hypersensitivity (including anaphylaxis), Infections and Malignant tumors.
Number of Participants With Adverse Drug Reactions (ADRs)	Up to 96 weeks	An ADR is defined as an AE for which the investigator classifies the possible relationship to study intervention as "Yes". ADRs related to NUCALA were collected.

Secondary Outcome Measures

Name	Time	Method
Percentage of Participants With Clinical Symptoms	At 96 weeks	Clinical symptoms as assessed by 9 organ-systems (i.e. systemic, skin, mucous membranes/eyes, ears/nose/throat, chest, cardiovascular, abdominal, renal, nervous system \[motor and sensory\]) relevant to EGPA in systemic vasculitis were assessed. Data were summarized for following categories; none, active, worsening, active + worsening. "None" is defined as absence of clinical symptoms. "Active disease" is defined as follows: The participant has clinical symptoms, or worsening of symptoms is noted as compared to those in the preceding observation period and the status in the preceding observation period was assessed as "None". "Worsening" is defined as follows: The participant has worsening clinical symptoms, or worsening of symptoms is noted as compared to those in the preceding observation period and the status in the preceding observation period was assessed as "Active disease" or "Worsening". Percentage values are rounded off.
Percentage of Participants With Eosinophilic Granulomatosis With Polyangiitis (EGPA) Relapse	Up to 96 weeks	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. Percentage of participants with EGPA relapse were reported. Percentage values are rounded off.
Annualized Rate of Hospitalization for EGPA-related Events	Up to 96 weeks	Annualized rate of hospitalization = (Number of corresponding hospital visits \* 365)/ (number of days in the observation period). The annualized rate and associated 95 percent (%) confidence intervals (CIs) were calculated using a negative binomial generalized linear model with logarithm of time as an offset variable, without covariate. Number (estimated event per person year) and 95% CI were reported.
Annualized Rate of Emergency Room/Unscheduled Visit for EGPA-related Events	Up to 96 weeks	Annualized rate of Emergency Room/Unscheduled Visit = (Number of corresponding Emergency Room/Unscheduled Visits \* 365)/(number of days in the observation period). The annualized rate and associated 95% CIs were calculated using a negative binomial generalized linear model with logarithm of time as an offset variable, without covariate. Number (estimated event per person year) and 95% CI were reported.
Average Daily Dose (Prednisolone-equivalent) of Oral Corticosteroid (OCS)	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	Average daily dose of OCS for each participant was calculated by 12-weekly periods as: Total dosage of OCS (mg) / Total duration of administration of OCS (day). Total duration of administration is defined as: Last date of period minus later date of (first date of each period or start date of OCS) +1.
Number of Participants by Dosing Categories Relative to Average Daily OCS (Prednisolone-equivalent)	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	Number of participants by each category of average daily prednisolone-equivalent of OCS were assessed. The dosing categories included: zero, greater than (\>)0 to less than or equal to (\<=) 4.0 milligrams per day (mg/day), \>4.0 to \<=7.5 mg/day, \>7.5 mg/day.

Trial Locations

Locations (1)

GSK Investigational Site; 🇯🇵 Yamaguchi, Japan