Dupilumab as Add-On Therapy for Hypereosinophilic Syndrome With Partial Clinical Response to Eosinophil-Depleting Biologic Agents
NCT Number: NCT06477653
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2
Conditions: Hypereosinophilic Syndrome
Interventions: BIOLOGICAL: dupilumab
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 30
Study Design: Allocation: NON_RANDOMIZED|Intervention Model: PARALLEL|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-09-10
Completion Date: 2027-03-30
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States

Summary:
Background:

Hypereosinophilic syndrome (HES) is a blood disorder that causes high levels of white blood cells called eosinophils. HES can damage the lungs and airways, intestines, skin, and other organs. The current primary treatment for HES can cause serious side effects. Secondary treatments do not work in all people.

Objective:

To test an approved drug (dupilumab), combined with other drugs, in people with HES.

Eligibility:

People aged 18 years and older who take drugs (mepolizumab, reslizumab, or benralizumab) to treat HES.

Design:

Participants will have up to 6 clinic visits and 7 remote visits in up to 48 weeks.

Participants will be screened. They will have blood and urine tests. They will have a test of their heart function. They will take surveys about how HES affects their daily life. Some participants may have a bone marrow biopsy: A sample of tissue and fluid from inside a bone will be removed with a large needle.

Participants will have other tests specific to their symptoms. For example, those with symptoms affecting their lungs will have breathing tests. Others may have tests that target symptoms in their sinuses, gastrointestinal tract, or skin.

Dupilumab is injected under the skin once every 1 or 2 weeks. Dose and timing will vary among participants. They will be taught how to inject themselves at home between clinic visits. They will take dupilumab plus their current medications for 24 weeks. If the drug is helping them, they will continue taking it for another 24 weeks.

Participants will have a final visit 12 weeks after their last dose.

Primary Outcome Measures: Clinical improvement on dupilumab therapy as assessed by HES-MBS (HES-most bothersome symptom) and HES-SI (HES-Symptom Inventory)., To assess the efficacy of add-on dupilumab therapy in reducing residual asthma, skin, esophageal, and sinus symptoms in patients with HES in complete hematologic and partial clinical remission on eosinophil-lowering biologics., Baseline through week 24
Secondary Outcome Measures: Incidence of new or worsening symptoms or signs attributable to eosinophilia requiring therapeutic intervention., To determine the effect of eosinophil-lowering therapy on dupilumab-induced blood eosinophilia and eosinophil-associated AEs., During the first 24 weeks of study.|Peripheral blood absolute eosinophil counts., To determine the effect of eosinophil-lowering therapy on dupilumab-induced blood eosinophilia and eosinophil-associated AEs., At 4, 12 and 24 weeks.|Proportion of patients who maintain an eosinophil count below 0.5 x 10^9/L., To determine the effect of eosinophil-lowering therapy on dupilumab-induced blood eosinophilia and eosinophil-associated AEs., At 4, 12 and 24 weeks.