A Study in Male and Female Adolescent Participants With Severe Uncontrolled Asthma Starting Treatment With Dupilumab Injection (Dupixent®) (PEDIASTHMA)

National, Multicentre, Non-interventional, Prospective and Retrospective Study in Adolescent Patients With Severe Uncontrolled Asthma Starting Treatment With Dupilumab (Dupixent®)

Primary objective:

- Describe the characteristics of enrolled severe asthma patients

Secondary objectives:

• Assess the control of asthma under dupilumab (Dupixent®) treatment until 1 year
• Assess the clinical objectives of the asthma care
• Assess comorbidities associated with Type 2 inflammation
• Assess safety during the year of treatment

Study Overview

• Status: Active, not recruiting
• Conditions: Asthma

Detailed Description

52 weeks

• Study Type: Observational
• Enrollment (Actual): 105

Contacts and Locations

Study Locations

• France
  • France, France
    • Investigational site France

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 17 years (Child)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Adolescents, for whom initiation of dupilumab (Dupixent®) for the severe uncontrolled asthma indication was decided by the investigator before the inclusion in the study.

Description

Inclusion Criteria:

• Adolescents, for whom initiation of dupilumab (Dupixent®) for the severe uncontrolled asthma indication was decided by the investigator before the inclusion in the study
• Adolescents ≥ 12 and < 18 years of age at the time of the initiation of Dupixent® treatment

Exclusion Criteria:

- Adult participants ≥ 18 years of age

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Other

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Participants with severe asthma; Adolescents, for whom initiation of dupilumab (Dupixent®) for the severe uncontrolled asthma indication was decided by the investigator before the inclusion in the study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Medical history (including history of asthma)	Relevant medical history other than asthma according to the investigator (including documented comorbidities associated with type 2 inflammation). Asthma history includes patient age at the time of diagnosis and family history.	At the start of treatment with Dupixent® (day 1)
Previous treatments for asthma	Background asthma treatments (doses and international nonproprietary name (INN) of inhaled corticosteroids (ICS), long-acting beta-2 agonist (LABA) and oral corticosteroids (OCS), etc) including cumulative yearly dose of OCS. Use of oral corticosteroids due to exacerbation of asthma (dose, INN). Use of emergency treatments (short-acting beta-2 agonist (SABA)) (number of times per week).	At the start of treatment with Dupixent® (day 1)
Demographic characteristics	Age and gender.	At the start of treatment with Dupixent® (day 1)
Disease characteristics	Disease characteristics at the inclusion including severity (Global Initiative for Asthma (GINA) step), number of severe exacerbations during the past year, number of days in intensive care unit (ICU) for severe exacerbation since the diagnosis, forced expiratory volume per second (FEV1), Asthma Control Test (ACT) score.	At the start of treatment with Dupixent® (day 1)
Concomitant medication	Relevant concomitant medication other than medication for asthma according to the investigator.	At the start of treatment with Dupixent® (day 1)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline to week 52 in asthma control test (ACT) score at each subsequent visit over the year of treatment	ACT score range: 5-25; higher score indicates better asthma control.	From baseline (day 1) to week 52
Change from baseline to week 52 in number of annualized exacerbations and description of exacerbation setting	An exacerbation is defined as deterioration of asthma requiring use of systemic corticosteroids for ≥3 days; or increase in oral corticosteroids (OCS) dose at ≥2 times the current dose for ≥3 days; or hospitalization or emergency room visit because of asthma, requiring systemic corticosteroids.	From baseline (day 1) to week 52
Change from baseline to week 52 in Paediatric Asthma Quality of Life Questionnaire (PAQLQ) score	Questionnaire including 23 questions in 3 domains (symptoms, activity limitation, emotional function) and a 7-point scale (7 = not bothered at all; 1 = extremely bothered).	From baseline (day 1) to week 52
Change from baseline to week 52 in corticosteroid dose	Change in corticosteroid dose.	From baseline (day 1) to week 52
Change from baseline to week 52 in pre- bronchodilator forced expiratory volume per second (FEV1)	FEV1 was the volume of air exhaled in the first second of a forced expiration as measured by spirometer.	From baseline (day 1) to week 52
Change from baseline to week 52 in number of food allergic reactions	Following food allergic reactions will be recorded: allergic symptoms and anaphylaxis in real life or during an oral food challenge.	From baseline (day 1) to week 52
Change from baseline to week 52 in in visual analogue scale (VAS) score for allergic rhinitis	VAS score range: 0 ('no symptoms') to 100 ('severe symptoms').	From baseline (day 1) to week 52
Change from baseline to week 52 in in Score for atopic dermatitis (SCORAD) score for atopic dermatitis	SCORAD is a clinical tool for assessing the severity (i.e., extent, intensity) of atopic dermatitis as objectively as possible with scores ranging from 0-100. The higher the score indicates more severe atopic dermatitis.	From baseline (day 1) to week 52
Change from baseline to week 52 in sinonasal symptoms of nasal polyposis of food allergies	Following sinonasal symptoms will be recorded: nasal congestion, loss in smell, posterior nasal discharge, anterior nasal discharge.	From baseline (day 1) to week 52
Responder rates at week 52 for ACT (according to the minimal important difference (MID))	A participant is defined as responder if he/she has a change from baseline of 3 points or more at week 52 on the ACT score. ACT score range: 5-25; higher score indicates better asthma control.	At week 52
Responder rates at week 52 for PAQLQ (according to the MID))	A participant is defined as responder if he/she has a change from baseline of 0.5 points or more at week 52 on the PAQLQ score. The PAQLQ(S) overall score is calculated by adding points of individual responses to all questions, with the higher scores indicating less impairment that is, a better quality of life. Questionnaire including 23 questions in 3 domains (symptoms, activity limitation, emotional function) and a 7-point scale (7 = not bothered at all; 1 = extremely bothered).	At week 52
Reasons for Dupixent® discontinuation	All reasons for treatment discontinuation.	Day 1 to week 52
Collection of adverse events	Number of adverse events.	Day 1 to week 52
Collection of serious adverse events	Number of serious adverse events.	Day 1 to week 52

Collaborators and Investigators

• Sponsor: Sanofi
• Investigators: Study Director: Clinical Sciences & Operations, Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): November 4, 2021
• Primary Completion (Actual): March 14, 2024
• Study Completion (Estimated): April 28, 2025

Study Registration Dates

• First Submitted: September 27, 2021
• First Submitted That Met QC Criteria: September 27, 2021
• First Posted (Actual): October 7, 2021

Study Record Updates

• Last Update Posted (Actual): June 14, 2024
• Last Update Submitted That Met QC Criteria: June 13, 2024
• Last Verified: June 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Immune System Diseases; Lung Diseases; Hypersensitivity, Immediate; Bronchial Diseases; Lung Diseases, Obstructive; Respiratory Hypersensitivity; Hypersensitivity; Asthma
• Other Study ID Numbers: OBS17083; U1111-1256-9593 (Registry Identifier: ICTRP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org