Pivotal trial: Study design1,2

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Study design

Baseline characteristics

Phase 3, randomized, placebo-controlled, double-blind study

SCREENING

Weeks –4 to 0

All patients received stable OCS dose

TREATMENT PERIOD

52 WEEKS, N=136

NUCALA 300 mg + SOC* (n=68)

Placebo + SOC* (n=68)

  • Weeks 0 to 4, patients received stable OCS dose
  • Starting at Week 4, OCS tapering could begin

FOLLOW-UP

Weeks 52 to 60 (last dose administered at Week 48)

Study description: 52-week study in patients with EGPA that evaluated the effect of 300 mg of NUCALA administered subcutaneously once every 4 weeks plus standard of care (SOC) (n=68) vs placebo plus SOC (n=68).*

Co-primary endpoints: Total accrued duration of remission over the 52-week treatment period, defined as:

  • Birmingham Vasculitis Activity Score (BVAS)=0 (no active vasculitis) plus prednisolone or prednisone dose less than or equal to 4 mg/day
  • Proportion of patients in remission at both Week 36 and Week 48 of treatment

Select secondary endpoints—OCS reduction:

  • Difference in average daily OCS dose between NUCALA and placebo during the last 4 weeks of treatment

Other endpoints/additional analyses:

  • Annualized relapse rate (calculated rate of relapses per year)
  • Median OCS dose over time
  • Relapse subtypes

Relapse was defined as a worsening related to vasculitis, asthma, or sinonasal symptoms requiring an increase in dose of corticosteroids, increase in dose or addition of immunosuppressive therapy, or hospitalization.

Key inclusion criteria:

  • Patients with relapsing or refractory disease who were on a stable OCS dose (prednisolone or prednisone) ≥7.5 mg/day (but not >50 mg/day) for ≥4 weeks prior to baseline
  • EGPA diagnosis for at least 6 months based on the history or presence of asthma plus eosinophilia and at least 2 of the following:
    • a biopsy showing eosinophilic vasculitis or perivascular eosinophilic infiltration or eosinophil-rich granulomatous inflammation
    • neuropathy
    • pulmonary infiltrates
    • sinonasal abnormality
    • cardiomyopathy
    • glomerulonephritis
    • alveolar hemorrhage
    • palpable purpura
    • ANCA positivity

Key exclusion criteria:

  • Patients with GPA or MPA
  • Organ-threatening or life-threatening EGPA within 3 months before screening
  • Certain medications that required a predetermined washout period (eg, rituximab, cyclophosphamide)

SOC was defined as OCS +/– immunosuppressants. After Week 4, OCS (prednisolone or prednisone) dose could be tapered per physician judgment or a suggested protocol.

ANCA=antineutrophil cytoplasmic antibody; EGPA=eosinophilic granulomatosis with polyangiitis; GPA=granulomatosis with polyangiitis; MPA=microscopic polyangiitis; OCS=oral corticosteroids; SOC=standard of care.

Baseline characteristics3

  NUCALA (n=68)
 Placebo (n=68)
Age in years, mean (SD) 48.7 (12.4)  48.2 (14.3) 
Female, % 62 56
White, % 94 90
Disease duration in years, mean (SD) 5.2 (4.4) 5.9 (4.9)
History of ANCA positivity, % 19 19
Relapsing disease,* % 75 72
Refractory disease,† % 50 59
Admitted to the intensive therapy unit at least once in the past, % 12 19
Median OCS (prednisolone/prednisone) dose, mg/day 12 11
High-dose OCS (>20 mg/day), % 16 15
Immunosuppressive therapy, % 60 46

Relapsing disease was defined as at least 1 confirmed EGPA relapse (ie, requiring increase in OCS [prednisolone or prednisone] dose, initiation/increased dose of immunosuppressive therapy, or hospitalization) within the past 2 years that occurred ≥12 weeks prior to screening (Visit 1) while receiving a dose of prednisolone or prednisone of ≥7.5 mg/day.

Refractory disease was defined as EITHER failure to attain remission (BVAS=0 and OCS [prednisolone or prednisone] dose ≤7.5 mg/day) within the last 6 months following induction treatment with a standard regimen, administered for at least 3 months OR within 6 months prior to screening (Visit 1), recurrence of symptoms of EGPA (not necessarily meeting the protocol definition of relapse) while tapering OCS, occurring at any dose level of ≥7.5 mg/day prednisolone or prednisone.

 

ANCA=antineutrophil cytoplasmic antibody; BVAS=Birmingham Vasculitis Activity Score; EGPA=eosinophilic granulomatosis with polyangiitis; OCS=oral corticosteroid; SD=standard deviation.

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