Clinical trial NCT06039241: Real-world Effectiveness Study of Long-term Treatment With Dupilumab in Participants ≥6 Years With Atopic Dermatitis
Official title: Prospective, Non-interventional, Observational Study for Multi-dimensional Assessment of Signs, Symptoms, Quality of Life and Disease Control of Long-term Treatment With Dupilumab in Patients With Atopic Dermatitis (≥6 Years)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Atopic Dermatitis
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2023-09-15
Enrollment: 900
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients ≥6 years of age in whom dupilumab therapy was initiated to treat their severe AD (6-11 years) or moderate to severe AD (adult and adolescent patients ≥12 years of age) according to the Summary of Product Characteristics. The decision to initiate treatment with dupilumab will be made by the attending physician based on the patient's medical requirements and standards of best medical practice. This decision will be made independent of and prior to data collection in this non-interventional study (NIS).

Brief summary: This is a Prospective, non-interventional (NIS) observational study in patients (≥6 years) with atopic dermatitis (AD) receiving dupilumab for the prospective evaluation of signs and symptoms, quality of life and disease control. The aim of this NIS is the characterization of the AD patient population in Germany, receiving dupilumab under everyday conditions in terms of their medical history, socio-demographic and disease-related characteristics, associated atopic comorbidities and type 2 inflammation diseases, concomitant therapy as well as previous systemic and ongoing AD treatments. In addition to the therapeutic response rate at Month 6, the long-term efficacy of dupilumab at Month 12 and Month 24 will be assessed by additional outcomes by measuring disease control in AD patients using questionnaires such as Atopic Dermatitis Control Tool (ADCT) and Recap of Atopic Eczema (RECAP). In addition, this NIS aims to assess the dosing pattern of dupilumab for AD, including variations in dosing regimen, reason for dupilumab treatment initiation or discontinuation, or change in therapy and concomitant therapies and duration of treatment. In addition, the effect of dupilumab in adult and pediatric AD patients with associated atopic comorbidities or type-2 inflammation diseases are observed, which corresponds to the clinical care situation. Finally, this NIS aims to collect long-term safety data in adult, adolescent and pediatric AD patients treated with dupilumab. Individual observation period is 2 years or until dupilumab is discontinued. Visits will be scheduled according to standard of care.


Eligibility criteria: Inclusion Criteria:

- Patients are at least 6 years of age at the baseline visit. - Initial treatment with dupilumab was initiated in adults and adolescents 12 years of age and older with moderate to severe AD, or in children 6 to 11 years of age with severe AD according to the Summary of Product Characteristics. - Patients or their guardians are able to understand and complete the study-related questionnaires. - Signing a written informed consent form by the patients before the initiation of documentation within the framework of this NIS or informed consent of parents/guardian, if applicable.

Exclusion Criteria:

- Patients who have a contraindication for dupilumab based on the current Summary of Product Characteristics. - Patients who have already been treated with dupilumab for more than 7 days. - Any acute or chronic diseases, which, in the opinion of the attending physician, would impair the patient's ability to complete questionnaires or participate in this study or could affect the interpretation of the results. - Participation in an ongoing interventional or observational study, which, in the opinion of the attending physician, could affect the assessment of the current study. "The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial."

Primary outcomes:
measure: Number of patients who maintain Eczema Area and Severity Index (EASI) ≤ 7 OR peak pruritus Numerical Rating Scale (NRS) ≤ 4 OR Dermatology Life Quality Index (DLQI) ≤ 5
timeFrame: From Month 6 to Month 12
measure: Mean percent change from baseline in Atopic Dermatitis Control Tools (ADCT) score (≥12 years)
timeFrame: From baseline to Week 52
measure: Mean percent change from baseline in Recap of atopic eczema (RECAP) score (6-11 years)
timeFrame: From baseline to Week 52
Secondary outcomes:
measure: Incidence of treatment emergent adverse events (TEAEs) during the observation period
timeFrame: From baseline up to 24 months
measure: Incidence of dupilumab-related TEAEs during the observation period.
timeFrame: From baseline up to 24 months
measure: Event rate (per patient year) by type of TEAEs during the observation period.
timeFrame: From baseline up to 24 months
measure: Event rate (per patient year) by type of dupilumab related TEAEs during the observational period
timeFrame: From Baseline up to 24 months
measure: Treating physician reported effectiveness data: Change from baseline in IGA score after 6, 12, and 24 months dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Treating physician reported effectiveness data.: Number of patients achieving EASI-75 (improvement in EASI score from baseline of ≥ 75%) after 6, 12 and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Treating physician reported effectiveness data: Absolute change from baseline in EASI score after 6, 12 and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Treating physician reported effectiveness data: Percentage change from baseline in EASI score after 6, 12, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Treating physician reported effectiveness data: Absolute change from baseline in IGA score after 6, 12, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Treating physician reported effectiveness data: Change from baseline in body surface area (BSA) after 6, 12, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on quality of life (QoL) data: Mean absolute change from baseline in Atopic Dermatitis Control Test (ADCT) score after 3, 6, 9, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean percent change from baseline in ADCT score after 3, 6, 9, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean absolute change from baseline in Recap of atopic eczema (RECAP) score after 3, 6, 9, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean percent change from baseline in RECAP score after 3, 6, 9, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean absolute change from baseline in Patient Oriented Eczema Measure (POEM) score after 6, 12, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean percent change from baseline in POEM score after 6, 12, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean absolute change from baseline in Dermatology Life Quality Index/Children's Dermatology Life Quality Index (DLQI/CDLQI) score after 6, 12, and 24 months of dupilumab therapy.
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean percent change from baseline in Dermatology Life Quality Index (DLQI)/Children's Dermatology Life Quality Index (CDLQI) score after 6, 12, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean Absolute Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) sleep disturbance score after 6, 12, and 24 months of dupilumab therapy.
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean Percent Change from baseline in PROMIS sleep disturbance score after 6, 12, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean absolute change from baseline in At baseline Questionnaire on Experience with Skin complaints (QES) score after 6, 12, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean percent change from baseline in At baseline Questionnaire on Experience with Skin complaints (QES) score after 6, 12, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean absolute change from baseline in Patient Global Assessment (PtGA) score after 3, 6, 9, 12, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean percent change from baseline in Patient Global Assessment (PtGA) score after 3, 6, 9, 12, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: : Mean percent change from baseline in Asthma control questionnaire-5 (ACQ-5)-Scores, assessed only in patients with co-existing Asthma after 3, 6, 9,12, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean absolute change from baseline in Asthma control questionnaire-5 (ACQ-5)-Scores, assessed only in patients with co-existing Asthma) after 3, 6, 9,12, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Mean absolute change from baseline in Allergic Visual analogue Scales ([Allergic-VAS] only patients with co-existing overall allergic symptoms) after 3, 6, 9, 12, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Mean percent change from baseline in Allergic Visual Analogue Scales ([Allergic-VAS] only patients with co-existing overall allergic symptoms) after 3, 6, 9, 12, 15, 18, and 24 months of dupilumab therapy
timeFrame: From Baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean absolute change from baseline in MACVIA-ARIA Sentinel Network Rhinitis Visual Analog Score (MASK-Rhinitis VAS score).
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean percent change from baseline in MACVIA-ARIA Sentinel Network Rhinitis Visual Analog Score (MASK-Rhinitis VAS score)
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean absolute change from baseline in MACVIA-ARIA Sentinel Network conjunctivitis Visual Analog Score (MASK-conjunctivitis VAS score), after 3, 6, 9, 12, 15, 18, and 24 months of dupilumab therapy.
timeFrame: From baseline up to 24 months
measure: Mean percent change from baseline in MACVIA-ARIA Sentinel Network conjunctivitis Visual Analog Score (MASK-conjunctivitis VAS score) after 3, 6, 9, 12, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean absolute change from baseline in Sinus Control Test (SCT) score, only assessed in patients with co-existing chronic rhinosinusitis after 3, 6, 9, 12, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient reported effectiveness and impact on QoL data: Mean percent change from baseline in SCT score, only assessed in patients with co-existing chronic rhinosinusitis after 3, 6, 9, 12, 15, 18, and 24 months of dupilumab therapy
timeFrame: From baseline up to 24 months
measure: Patient and treatment characteristics reported by the physician or patient: Socio -demographic data
timeFrame: At baseline
measure: Patient and treatment characteristics reported by the physician or patient: Medical history of disease
timeFrame: At baseline
measure: Patient and treatment characteristics reported by the physician or patient: Comorbidities/smoker status/alcohol usage
timeFrame: At baseline
measure: Patient and treatment characteristics reported by the physician or patient: Prior systemic AD treatment (including balneophototherapy and/or UV therapy)
timeFrame: At baseline
measure: Patient and treatment characteristics reported by the physician or patient: Current topical and/or systemic AD treatment of the last 4 weeks before baseline visit (dosage, type)
timeFrame: At baseline
measure: Patient and treatment characteristics reported by the physician or patient: Current treatment for co-existing type 2 inflammatory and atopic diseases
timeFrame: At baseline
measure: Patient and treatment characteristics reported by the physician or patient: Reasons for initiating dupilumab treatment
timeFrame: At baseline
measure: Patient and treatment characteristics reported by the physician or patient: Reasons for discontinuation of dupilumab treatment (if the discontinuation was before end of study)
timeFrame: Up to 24 months
measure: Patient and treatment characteristics reported by the physician or patient: Number of sick-leave days away from work/school due to AD
timeFrame: From baseline up to 24 months
measure: Patient and treatment characteristics reported by the physician or patient: Number of medical appointments due to AD
timeFrame: From baseline up to 24 months
measure: Patient and treatment characteristics reported by the physician or patient: Number of visits to alternative practitioners and complementary care physicians
timeFrame: From baseline upto 24 months
measure: Patient and treatment characteristics reported by the physician or patient: Cost of non-reimbursable supplementary therapy [€/month]
timeFrame: From baseline up to 24 months
Other outcomes:
Locations:
city: Aachen
country: Germany
facility: Investigational Site Number: 061
city: Ahaus
country: Germany
facility: Investigational Site Number: 092
city: Andernach
country: Germany
facility: Investigational Site Number: 066
city: Berlin
country: Germany
facility: Investigational Site Number: 002
city: Berlin
country: Germany
facility: Investigational Site Number: 003
city: Berlin
country: Germany
facility: Investigational Site Number: 176
city: Berlin
country: Germany
facility: Investigational Site Number: 077
city: Berlin
country: Germany
facility: Investigational Site Number: 004
city: Berlin
country: Germany
facility: Investigational Site Number: 174
city: Berlin
country: Germany
facility: Investigational Site Number: 073
city: Braunschweig
country: Germany
facility: Investigational Site Number: 059
city: Buxtehude
country: Germany
facility: Investigational Site Number: 057
city: Chemnitz
country: Germany
facility: Investigational Site Number: 028
city: Dresden
country: Germany
facility: Investigational Site Number: 072
city: Dresden
country: Germany
facility: Investigational Site Number: 026
city: Düren
country: Germany
facility: Investigational Site Number: 032
city: Erlangen
country: Germany
facility: Investigational Site Number: 043
city: Essen
country: Germany
facility: Investigational Site Number: 064
city: Friedberg
country: Germany
facility: Investigational Site Number: 184
city: Gera
country: Germany
facility: Investigational Site Number: 055
city: Gießen
country: Germany
facility: Investigational Site Number: 007
city: Gladbeck
country: Germany
facility: Investigational Site Number: 031
city: Göttingen
country: Germany
facility: Investigational Site Number: 078
city: Hamburg
country: Germany
facility: Investigational Site Number: 087
city: Hamburg
country: Germany
facility: Investigational Site Number: 088
city: Hamburg
country: Germany
facility: Investigational Site Number: 020
city: Hamburg
country: Germany
facility: Investigational Site Number: 086
city: Heidelberg
country: Germany
facility: Investigational Site Number: 169
city: Heilbad Heiligenstadt
country: Germany
facility: Investigational Site Number: 053
city: Jülich
country: Germany
facility: Investigational Site Number: 063
city: Karlsruhe
country: Germany
facility: Investigational Site Number: 186
city: Kiel
country: Germany
facility: Investigational Site Number: 016
city: Kiel
country: Germany
facility: Investigational Site Number: 017
city: Köln
country: Germany
facility: Investigational Site Number: 069
city: Landsberg
country: Germany
facility: Investigational Site Number: 183
city: Langenau
country: Germany
facility: Investigational Site Number: 038
city: Leipzig
country: Germany
facility: Investigational Site Number: 179
city: Mainz
country: Germany
facility: Investigational Site Number: 178
city: Mainz
country: Germany
facility: Investigational Site Number: 185
city: Mainz
country: Germany
facility: Investigational Site Number: 014
city: Mannheim
country: Germany
facility: Investigational Site Number: 013
city: Mölln
country: Germany
facility: Investigational Site Number: 018
city: Mönchengladbach
country: Germany
facility: Investigational Site Number: 035
city: München
country: Germany
facility: Investigational Site Number: 050
city: Neubrandenburg
country: Germany
facility: Investigational Site Number: 076
city: Nürnberg
country: Germany
facility: Investigational Site Number: 045
city: Nürnberg
country: Germany
facility: Investigational Site Number: 042
city: Oelde
country: Germany
facility: Investigational Site Number: 024
city: Potsdam
country: Germany
facility: Investigational Site Number: 082
city: Remscheid
country: Germany
facility: Investigational Site Number: 172
city: Rostock
country: Germany
facility: Investigational Site Number: 177
city: Wasserburg
country: Germany
facility: Investigational Site Number: 047
city: Wismar
country: Germany
facility: Investigational Site Number: 019
city: Wittlich
country: Germany
facility: Investigational Site Number: 168
city: Wuppertal
country: Germany
facility: Investigational Site Number: 193
Study officials:
affiliation: Sanofi
name: Clinical Sciences &amp; Operations
role: STUDY_DIRECTOR
References: