Up to 4 weeks
with stable regimen of hypereosinophilic
syndrome (HES) therapy
32 weeks; N=108
NUCALA 300 mg SC + stable HES therapy (n=54)
Placebo + stable HES therapy (n=54)
Administered every 4 weeks
32-week, multicenter, randomized, double blind, placebo-controlled, parallel-group study comparing NUCALA 300 mg SC to placebo, each added to stable HES therapy in 108 patients aged ≥12 years with HES.
Key inclusion criteria
- Diagnosed with HES* ≥ 6 months prior to baseline
- Blood eosinophil counts ≥ 1000 cells/µL during screening
- ≥2 HES flares† in the past 12 months
- At least one of these HES flares must not have been related to a decrease in therapy during the 4 weeks prior to the flare
Key exclusion criteria
- Patients with non-hematologic secondary HES (eg, drug hypersensitivity, parasitic helminth infection, HIV infection, non-hematologic malignancy)
- Patients with FIP1L1-PDGFRA kinase-positive HES
Stable HES therapy
- Existing HES therapy, which included but was not limited to chronic or episodic corticosteroids, immunosuppressants, or cytotoxic therapy
Primary endpoint results:
Proportion of patients who experienced HES flares(s)‡ during the 32-week study or withdrew. NUCALA 28% vs placebo 56%, P=0.002
Select secondary endpoints:
Time to first flare, change from baseline in Brief Fatigue Inventory Item 3 at Week 32
*HES diagnosis was based on signs or symptoms of organ system involvement and/or dysfunction that could be directly related to blood eosinophilia of >1500 eosinophils/μL on ≥2 occasions, and/or tissue eosinophilia.
‡HES flare defined as a worsening of clinical signs/symptoms or increased eosinophils (on ≥ 2 occasions), resulting in an escalation/addition of oral corticosteroids (OCS) or cytotoxic or immunosuppressive therapy.