A Non-interventional Observational Study to Assess Long-term Efficacy and Safety of Dupilumab for the Treatment of Patients (>= 12 Years) With Eosinophilic Esophagitis Under Real-world-conditions in Germany

Official title

Prospective, Non-interventional Observational Study to Characterize Dupilumab in the Long-term Management of Eosinophilic Esophagitis, Safety and Patient Reported Outcomes (Quality of Life) in Clinical Routine

Overview

This is a longitudinal, observational non-interventional study (NIS) designed to characterize the patient population who receives dupilumab for eosinophilic esophagitis (EoE) treatment in a real-world setting in Germany with respect to their medical history, socio-demographic and disease characteristics, comorbidities (including type 2 inflammatory and atopic conditions), concomitant therapies and prior EoE treatments. Furthermore, this NIS aims to describe the long-term effectiveness of dupilumab for the treatment of EoE as assessed by the treating physician and reported by the patient, and its effect on health-related quality of life (HRQoL) during an individual observational period of two years. In addition, data on real-world use patterns and long-term safety data of dupilumab treatment for EoE will be collected.

Detailed description

The study duration for each participant is planned to be 2 years. Total study duration is approximately 4 years.

Interventions

• Drug Dupilumab
  This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.

Primary outcome measures

• Descriptive statistical analysis of medical history characteristics [Time frame: At baseline]
• Descriptive statistical analysis of socio-demographic characteristics [Time frame: At baseline]
• Descriptive statistical analysis of disease characteristics [Time frame: At baseline]

Secondary outcome measures (12)

• Absolut change from baseline in endoscopic reference score (EREFS) [Time frame: After 3, 6 (optional), 12 and 24 months of dupilumab treatment]
• Proportion of patients with eosinophils per high power field (EOS/hpf) < 15 in esophageal biopsies [Time frame: After 3, 6 (optional), 12 and 24 months of dupilumab treatment]
• Change in clinical symptoms from baseline [Time frame: From baseline until 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment]
• Behavioral changes regarding food intake from baseline [Time frame: From baseline until 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment]
• Absolute change from baseline in Dysphagia Numerical Rating Scale (NRS) [Time frame: After 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment]
• Absolute change from baseline in Hospital Anxiety and Depression Scale (HADS) [Time frame: After 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment]
• Absolute change from baseline Pediatric Eosinophilic Esophagitis Symptom Scores (PEESS v2.0) [Time frame: After 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment]
• Absolute change from baseline Pediatric Quality of Life Inventory (PEDS-QL) [Time frame: After 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment]
• Number of hospitalizations due to EoE [Time frame: During the last 12 months before baseline and after 12 and 24 months of dupilumab treatment]
• Number of sick leave days at work due to EoE [Time frame: During the last 12 months before baseline and after 12 and 24 months of dupilumab treatment]
• Reason(s) for initiation of dupilumab treatment [Time frame: At baseline]
• Descriptive statistical analysis of details on dupilumab treatment [Time frame: Throughout the study, for approximately 24 months after dupilumab treatment]

Eligibility criteria

Inclusion criteria

• Patients diagnosed with EoE who are inadequately controlled by, are intolerant to, or who are not candidates for conventional medicinal therapy
• Patients for whom the treating physician has decided to initiate new treatment with dupilumab for EoE according to EU-SmPC independently from entry in study or for whom treatment with dupilumab for EoE according to EU-SmPC has been started within the last 7 days. The decision to initiate dupilumab treatment is made by the treating physician and the patient according to the patients' medical need and to the standard of best medical practice. This decision is made independently and before inclusion in this NIS.
• ≥ 12 years of age at baseline visit
• ≥ 40 kg bodyweight
• Voluntary study participation and signed written informed consent by patient and parent(s)/guardian(s), if applicable

Exclusion criteria

• Patients who have a contraindication to dupilumab according to the current EU-SmPC.
• Patients who have been treated with dupilumab for more than 7 days
• Any acute or chronic condition that, in the treating physician's opinion, would limit the patients' ability to complete questionnaires or to participate in this study or impact the interpretation of the results.
• Participation in an ongoing interventional or observational study that might, in the treating physician's opinion, influence the assessments for the current study (parallel inclusion in another Sanofi-independent registry might be possible if the patient gives consent)

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

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

Healthy volunteers: Yes

Study design

Observational model

Cohort

Study locations

Germany · 1 center

• Investigational Site Number: 0000001 — Dachau

Identifiers

NCT: NCT06695897 · OBS18402 · U1111-1306-7545

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗