Clinical trial NCT06695897: 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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Eosinophilic Esophagitis
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: Regeneron Pharmaceuticals
First posted: 2024-11-19
Enrollment: 150
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients suffering from EoE, ≥ 12 years of age with at least 40 kg bodyweight who are planned to be initiated with dupilumab for the treatment of EoE according to the prescribing information, i.e., the current Summary of Product Characteristics approved in the European Union (EU-SmPC).

Brief summary: 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.

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 patient´s 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 patient´s 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)

Primary outcomes:
measure: Descriptive statistical analysis of medical history characteristics
timeFrame: At baseline
measure: Descriptive statistical analysis of socio-demographic characteristics
timeFrame: At baseline
measure: Descriptive statistical analysis of disease characteristics
timeFrame: At baseline
Secondary outcomes:
measure: Absolut change from baseline in endoscopic reference score (EREFS)
timeFrame: After 3, 6 (optional), 12 and 24 months of dupilumab treatment
measure: Proportion of patients with eosinophils per high power field (EOS/hpf) < 15 in esophageal biopsies
timeFrame: After 3, 6 (optional), 12 and 24 months of dupilumab treatment
measure: Change in clinical symptoms from baseline
timeFrame: From baseline until 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment
measure: Behavioral changes regarding food intake from baseline
timeFrame: From baseline until 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment
measure: Absolute change from baseline in Dysphagia Numerical Rating Scale (NRS)
timeFrame: After 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment
measure: Absolute change from baseline in Hospital Anxiety and Depression Scale (HADS)
timeFrame: After 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment
measure: Absolute change from baseline Pediatric Eosinophilic Esophagitis Symptom Scores (PEESS v2.0)
timeFrame: After 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment
measure: Absolute change from baseline Pediatric Quality of Life Inventory (PEDS-QL)
timeFrame: After 1, 3, 6, 9, 12, 18 and 24 months of dupilumab treatment
measure: Number of hospitalizations due to EoE
timeFrame: During the last 12 months before baseline and after 12 and 24 months of dupilumab treatment
measure: Number of sick leave days at work due to EoE
timeFrame: During the last 12 months before baseline and after 12 and 24 months of dupilumab treatment
measure: Reason(s) for initiation of dupilumab treatment
timeFrame: At baseline
measure: Descriptive statistical analysis of details on dupilumab treatment
timeFrame: Throughout the study, for approximately 24 months after dupilumab treatment
measure: Analysis of treatment emergent adverse events (TEAEs)
timeFrame: Throughout the study, for approximately 24 months after initiation of dupilumab treatment
Other outcomes:
Locations:
city: Dachau
country: Germany
facility: Investigational Site Number: 0000001
state: Bayern
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: