A Study of Dupilumab in Small Children With an Allergic Condition of the Esophagus (Food Pipe): Eosinophilic Esophagitis

Official title

A Randomized Study Evaluating 2 Dose Regimens of Dupilumab in Children ≥6 Months of Age Weighing ≥5 to <15 kg With Active Eosinophilic Esophagitis

Overview

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

• Drug dupilumab
  Administered as defined in the protocol

Primary outcome measures

• Incidence of treatment-emergent Serious Adverse Events (SAEs) [Time frame: Up to 24 Weeks]

Secondary outcome measures (11)

• Incidence of Treatment-Emergent Adverse Events (TEAEs) leading to permanent discontinuation of study treatment [Time frame: Up to 24 Weeks]
• Incidence of treatment-emergent Adverse Event of Special Interest (AESIs) [Time frame: Up to 24 Weeks]
• Incidence of TEAEs [Time frame: Up to 24 Weeks]
• Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤6 eosinophils per high power field (eos/hpf) (400×) [Time frame: At Week 24]
• Proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf (400×) [Time frame: At Week 24]
• Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf) [Time frame: Baseline to Week 24]
• Change in body weight for age percentile [Time frame: Baseline up to Week 24]
• Concentrations of functional dupilumab in serum [Time frame: Baseline up to Week 24]
• Incidence of Anti-Drug Antibody (ADA) to dupilumab [Time frame: Up to Week 36]
• Titer of ADA to dupilumab [Time frame: Up to Week 36]
• Incidence of Neutralizing Antibody (NAb) to dupilumab [Time frame: Up to Week 36]

Eligibility criteria

Inclusion criteria

• Pediatric patients aged ≥6 months and weighing ≥5 kg and <15 kg at screening with active EoE
• 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
• 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

Exclusion criteria

• Prior participation in a dupilumab clinical trial or past or current treatment with dupilumab
• 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
• Other causes of esophageal eosinophilia or the following conditions: eosinophilic gastroenteritis, hypereosinophilic syndrome, and eosinophilic granulomatosis with polyangiitis (Churg-Strauss syndrome)
• Active Helicobacter pylori infection
• History of Crohn's disease, ulcerative colitis, celiac disease, or prior esophageal surgery
• Any esophageal stricture unable to be passed with a standard, diagnostic, upper endoscope or any critical esophageal stricture that requires dilation at screening
• 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
• Treatment with swallowed topical corticosteroids within 8 weeks prior to baseline standard of care endoscopy

NOTE: Other Protocol-Defined Inclusion/Exclusion Criteria Apply

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Single blind

Primary purpose

Treatment

Study locations

United States · 12 centers

• Phoenix Childrens Hospital — Phoenix
• University of California San Francisco (UCSF) — San Francisco
• Children's Hospital Colorado — Aurora
• GI Care for Kids LLC — Atlanta
• Massachusetts General Hospital — Boston
• Weill Cornell Medicine — New York
• University of North Carolina, Bioinformatics Building — Chapel Hill
• Children's Hospital of Philadelphia — Philadelphia
• … and 4 more centers

Identifiers

NCT: NCT07112378 · R668-EE-2423

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗