Eosinophilic Esophagitis (EoE)
Conditions
Keywords
Type 2 Inflammatory, Allergic/immune-mediated, Esophageal dysfunction
Brief summary
This study is researching an experimental drug called dupilumab (called "study drug"). The study is focused on children with active eosinophilic esophagitis (EoE; an inflammatory disease of the esophagus) which impacts feeding and nourishment. The aim of the study is to see how safe, tolerable, and effective the study drug is when given for 24 weeks to children with active EoE. The study is looking at several other research questions, including: * What side effects may happen from taking the study drug * How much study drug is in the blood at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
Interventions
DRUGdupilumab
Administered as defined in the protocol
Sponsors
Regeneron Pharmaceuticals
Sanofi
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Caregiver)
Eligibility
Sex/Gender
ALL
Age
6 Months to 6 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Pediatric patients aged ≥6 months and weighing ≥5 kg and \<15 kg at screening with active EoE 2. History of symptom(s) determined by the investigator to be the result of EoE in the month prior to screening, as defined in the protocol 3. Baseline endoscopic biopsies, performed during the screening period, with a demonstration on central reading of intraepithelial eosinophilic infiltration in at least 2 of the 3 biopsied esophageal regions, as defined in the protocol Key
Exclusion criteria
1. Prior participation in a dupilumab clinical trial or past or current treatment with dupilumab 2. Initiation or change of a food-elimination diet regimen or re-introduction of a previously eliminated food group in the 6 weeks prior to screening. Patients on a food-elimination diet must remain on the same diet throughout the study 3. Other causes of esophageal eosinophilia or the following conditions: eosinophilic gastroenteritis, hypereosinophilic syndrome, and eosinophilic granulomatosis with polyangiitis (Churg-Strauss syndrome) 4. Active Helicobacter pylori infection 5. History of Crohn's disease, ulcerative colitis, celiac disease, or prior esophageal surgery 6. Any esophageal stricture unable to be passed with a standard, diagnostic, upper endoscope or any critical esophageal stricture that requires dilation at screening 7. History of bleeding disorders or esophageal varices that, in the opinion of the investigator, would put the patient at undue risk for significant complications from an endoscopic procedure 8. Treatment with swallowed topical corticosteroids within 8 weeks prior to baseline standard of care endoscopy NOTE: Other Protocol-Defined Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment-emergent Serious Adverse Events (SAEs) | Up to 24 Weeks |
Secondary
| Measure | Time frame |
|---|---|
| Incidence of Anti-Drug Antibody (ADA) to dupilumab | Up to Week 36 |
| Titer of ADA to dupilumab | Up to Week 36 |
| Incidence of Neutralizing Antibody (NAb) to dupilumab | Up to Week 36 |
| Incidence of Treatment-Emergent Adverse Events (TEAEs) leading to permanent discontinuation of study treatment | Up to 24 Weeks |
| Incidence of treatment-emergent Adverse Event of Special Interest (AESIs) | Up to 24 Weeks |
| Incidence of TEAEs | Up to 24 Weeks |
| Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤6 eosinophils per high power field (eos/hpf) (400×) | At Week 24 |
| Proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf (400×) | At Week 24 |
| Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf) | Baseline to Week 24 |
| Concentrations of functional dupilumab in serum | Baseline up to Week 24 |
| Change in body weight for age percentile | Baseline up to Week 24 |
Countries
United States
Contacts
CONTACTClinical Trials Administrator
STUDY_DIRECTORClinical Trial Management
Regeneron Pharmaceuticals
Outcome results
None listed