Mepolizumab efficacy and pathogenesis studies in patients with early-onset EGPA.

JRCT ID: jRCTs031230675

Registered date:05/03/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: eosinophilic granulomatosis with polyangiitis, EGPA
• Date of first enrollment: 05/03/2024
• Target sample size: 20
• Study type: Interventional

Intervention(s)

For acute onset EGPA with inadequate response to steroid pulse therapy:
Mepo group: Mepolizumab 300 mg subcutaneously once every 4 weeks in addition to standard therapy (covered by insurance)
Placebo group: Standard therapy plus once-every-four-weeks subcutaneous injection of a placebo (normalsaline)

Outcome(s)

• Primary Outcome: Steroid dose reduction: Patients whose PSL dose at week 8 was less than 20 mg/day were defined as successful steroid dose reduction cases, and the proportion of successful cases was compared between the two groups.

Secondary Outcome

Number of days in hospital
Steroid dose reduction, change from baseline in PSL dose (%) (8W, 24W)
Daily oral steroid dose (daily dose at 8W and 24W)
Total (cumulative) steroid dosage (total up to 8W and 24W)
VAS assessment of 8 subjective symptoms
BVAS
ADL
ACT
Grip strength
Peripheral blood eosinophil count, serum total IgE, serum IgG4, urinary EDN, serum Gal-10, MBP, periostin, urinary leukotriene E4, expiratory NO
ANCA value
Serum BNP, pulse rate, CTR on chest Xp (cardiac hypertrophy)
Serum CK, D-dimer
Adverse events (AEs)
Serum and urinary biomarker changes (serum cytokine multiplex, cell surface indicators, single cell analysis)
Urinary lipid mediators

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1) Patients with EGPA who meet both the Lanham and ACR/eular criteria 2022
2) Adult patients 18 years of age or older with untreated, acute-onset EGPA.
(Untreated refers to patients who have not started standard therapy for their vasculitis symptoms.)
(Acute onset refers to the period within 2 months of the onset of vasculitis symptoms.)
3) Patients who have not received anti-IL-5 antibodies in the six months prior to EGPA testing.
4) Patients with inadequate symptom improvement after initiation of steroid pulse therapy at the time of enrollment allocation
5) Patients whose consent has been obtained by the patient

Exclude criteria

(1) Cases with difficulty in determining efficacy due to complications of other diseases with vasculitis symptoms (MPA, GPA, PAN, etc.)
(2) Critical cases of EGPA (cerebrovascular disease with life-threatening prognosis, severe heart failure, gastrointestinal ischemia with risk of ileus or perforation, or fatal organ failure such as renal failure)
(3) Pregnant or planning to become pregnant. Also, those who are breastfeeding.
(4) Other cases deemed inappropriate by the principal/assistant investigator.
(5) When written consent cannot be obtained

Related Information

• Primary Sponsor: Taniguchi Masami
• Secondary Sponsor: GlaxoSmithKline K.K. UK Headquarters