A randomized, double-blind, placebo-controlled, parallel group study to assess the efficacy and long-term safety of dupilumab in children 2 to <6 years of age with uncontrolled asthma and/or recurrent severe asthmatic wheeze
Efficacy and safety of subcutaneous dupilumab in participants with asthma/asthmatic wheeze aged 2 to <6 years (LIBERTY ASTHMA TREKIDS)
Obara Kentaro
Sanofi K.K.
Tokyo Opera City Tower, 3-20-2, Nishi Shinjuku, Shinjuku-ku, Tokyo 163-1488, Japan
+81-3-6301-3670
clinical-trials-jp@sanofi.com
Clinical Study Unit
Sanofi K.K.
Tokyo Opera City Tower, 3-20-2, Nishi Shinjuku, Shinjuku-ku, Tokyo 163-1488, Japan
+81-3-6301-3670
clinical-trials-jp@sanofi.com
Recruiting
May. 30, 2025
90
Interventional
randomized controlled trial
double blind
placebo control
parallel assignment
treatment purpose
- Participant must be 2 to <6 years of age.
- Diagnosis of asthma or recurrent severe asthmatic wheeze that is not controlled with chronic inhaled corticosteroids (ICS) for at least 3 months with stable use of at least low dose ICS for >=1 month prior to Screening Visit 1 with evidence of uncontrolled asthma and/or recurrent severe asthmatic wheeze.
- At least one additional major criterion from the modified asthma predictive index:
a) Physician diagnosed Atopic Dermatitis,
b) Allergic sensitization to at least 1 aeroallergen (with a positive serum immunoglobulin E [IgE] defined as a value >=0.35 kU/L).
OR 2 minor criteria:
c) Wheezing unrelated to colds,
d) Peripheral blood eosinophilia >=4%,
e) Allergic sensitization to milk, eggs, or peanuts (defined by serum specific IgE >0.35 kU/L).
- Parent(s)/caregiver(s)/legal guardian(s) willing and able to comply with clinic visits and study-related procedures.
- Parent(s)/caregiver(s)/legal guardian(s) able to understand the study requirements.
- Participants/parent(s)/caregiver(s)/legal guardian(s), as appropriate, must be able to understand and complete study-related questionnaires.
- Body weight at screening and randomization >5 kg and <30 kg.
- Parents or caregivers or legal guardian capable of giving signed informed consent.
Participants are excluded from the study if any of the following criteria apply:
- Severe asthma with the need for chronic oral/systemic corticosteroid use (>1 month continuous) at the time of screening enrollment.
- History of a systemic hypersensitivity reaction or anaphylaxis to biologic therapy, including any excipient.
- History of prematurity (<34 weeks gestation).
- Any other chronic lung disease that would impair lung function (eg, cystic fibrosis, bronchopulmonary dysplasia) or chronic lung disease of prematurity or need for oxygen for more than 5 days in the neonatal period.
- History of life-threatening asthma (eg, requiring intubation).
Study Arms:
Experimental: Dupilumab (double-blind period)
- Dupilumab subcutaneous injection as per protocol.
Placebo Comparator: Placebo
- Placebo matching dupilumab subcutaneous injection as per protocol.
1. Part A: Annualized rate of severe asthma exacerbations during the 52-week treatment period
[Time Frame: Baseline through Week 52]
Annualized rate of severe asthma exacerbations during the 52-week treatment period.
2. Part B: Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), and adverse events (AEs) leading to permanent treatment discontinuation
[Time Frame: Week 52 through Week 116]
Incidence of TEAEs, SAEs, AESIs, and AEs leading to permanent treatment discontinuation.
1. Part A: Annualized rate of hospitalization, emergency room (ER) or urgent care visit for asthma exacerbation during the 52-week treatment period
[Time Frame: Baseline through Week 52]
2. Part A: Annualized rate of moderate asthma exacerbations during the 52-week treatment period
[Time Frame: Baseline through Week 52]
Annualized rate of moderate asthma exacerbations during the 52-week treatment period.
3. Part A: Cumulative ICS dose during the 52-week treatment period
[Time Frame: Baseline through Week 52]
4. Part A: Change from baseline in weekly average use of reliever medication during the 52-week treatment period
[Time Frame: Baseline to Week 52]
5. Part A: Mean number of days without asthma symptoms (DWAS) using the Pediatric Asthma Caregiver Diary (PACD) during the 52-week treatment period
[Time Frame: Baseline through Week 52]
A day without asthma symptoms (DWAS) is defined as a day without any asthma symptoms, beta-agonist use, oral steroid use, or need for urgent asthma care. The PACD consists of a daytime and overnight record. The daytime asthma symptom questions are scored on a 6-point scale from 0 (no symptoms/interference) to 5 (very severe symptoms/interference). The overnight questions are scored on a 5-point scale from 0 (no overnight cough and caregiver not disturbed at all) to 4 (coughed all night and disturbed all night).
6. Part A: Change from baseline to Week 52 in daytime symptom score using the daytime record of PACD
[Time Frame: Baseline to Week 52]
The PACD consists of a daytime and overnight record. The daytime asthma symptom questions are scored on a 6-point scale from 0 (no symptoms/interference) to 5 (very severe symptoms/interference).
7. Part A: Incidence of TEAEs, SAEs, AESIs, and AEs leading to permanent treatment discontinuation
[Time Frame: Baseline through Week 52]
8. Part A: Change from baseline to Week 52 in Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Scale
[Time Frame: Baseline to Week 52]
PedsQL measures health-related quality of life (HRQOL) in healthy children and adolescents and those with acute and chronic health conditions. Scores range 0-100 with higher scores indicate better HRQOL.
9. Part A: Caregiver Global Impression of Change in their child's asthma control (CGI-change in asthma control) at Week 52
[Time Frame: Week 52]
The Caregiver Global Impression of Change of Asthma (CGI-change in asthma control) is a 1-item questionnaire that asks the caregiver to provide the overall assessment of change of their child's asthma control on a 7-point scale, compared to just before he/she started taking the study treatment. Response choices are: 0 = "Very much better", 1 = "Moderately better", 2 = "A little better", 3 = "No change", 4 = "A little worse", 5 = "Moderately worse", 6 = "Very much worse".
10. Part A: Physician Global Assessment of Change of the child's asthma control (PGA-change in asthma control) at Week 52
[Time Frame: Week 52]
The Physician Global Assessment of Change of Asthma (PGA-change in asthma control) is a 1-item questionnaire that asks the physician to provide the overall assessment of change in the child's asthma control on a 7-point scale, compared to just before he/she started taking the study treatment. Response choices are: 0 = "Very much better", 1 = "Moderately better", 2 = "A little better", 3 = "No change", 4 = "A little worse", 5 = "Moderately worse", 6 = "Very much worse".
11. Part A: Change from baseline to Week 52 in Caregiver Global Impression of their child's asthma control (CGI-asthma control)
[Time Frame: Baseline to Week 52]
The Caregiver Global Impression of asthma control (CGI-Asthma Control) is a 1-item questionnaire that asks caregiver to provide the assessment of their child's asthma control over the past 7 days, on a 4-point scale for the past week. Response choices are: 1 = "Not controlled", 2 = "Somewhat controlled", 3 = "Moderately controlled", 4 = "Well controlled".
12. Part A: Change from baseline to Week 52 in Caregiver Global Impression of their child's asthma severity (CGI-asthma severity)
[Time Frame: Baseline to Week 52]
The Caregiver Global Impression of asthma severity (CGI-Asthma Severity) is a 1-item questionnaire that asks caregivers to provide the assessment of their child's asthma control over the past 7 days, on a 4-point scale for the past week. Response choices are: 1 = "None", 2 = "Mild", 3 = "Moderate", 4 = "Severe".
13. Part A: Change from baseline to Week 52 in Physician Global Assessment of the child's asthma control (PGA-asthma control)
[Time Frame: Baseline to Week 52]
The Physician Global Assessment of asthma control (PGA-asthma Control) is a 1-item questionnaire that asks physicians to assess the control of the child's asthma in the past 7 days on a 4-point scale for the past week. Response choices are: 1 = "Not controlled", 2 = "Somewhat controlled", 3 = "Moderately controlled", 4 = "Well controlled".
14. Part A: Change from baseline to Week 52 in Test for Respiratory and Asthma Control in Kids (TRACK)
[Time Frame: Baseline to Week 52]
TRACK is a validated questionnaire for caregiver completion for preschool aged children with symptoms consistent with asthma. The total score range of 0 to 100. Higher scores indicated better asthma control.
15. Part A: Change from baseline in blood eosinophil level at Weeks 24 and 52
[Time Frame: Baseline to Week 24 and 52]
16. Part A: Concentration of dupilumab in serum over time during the 52-week treatment period
[Time Frame: Baseline through Week 52]
17. Part A: Incidence of treatment-emergent anti-drug antibody (ADA) against dupilumab over time
[Time Frame: Baseline through Week 52]
18. Part A: Immunoglobulin G (IgG) response to any vaccination for tetanus, diphtheria and pertussis and antibody for influenza (hemagglutination inhibition [HAI] antibody titers) vaccine administered according to vaccination schedule during the 52-week treatment period
[Time Frame: Baseline through Week 52]
19. Part B: Annualized rate of severe asthma exacerbations events during the 52-week Part B treatment period
[Time Frame: Week 52 through Week 104]
Annualized rate of severe asthma exacerbations events during the 52-week Part B treatment period.
20. Part B: Concentration of dupilumab in serum over time during the 52-week Part B treatment period
[Time Frame: Week 52 through Week 104]
21. Part B: Incidence of treatment-emergent ADAs against dupilumab over time
[Time Frame: Week 52 through Week 104]
5-1-1 Kashii Teriha, Higashi-ku, Fukuoka City, Fukuoka
+81-92-682-7000
Approval
April. 08, 2025
Yes
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
NCT06191315
ClinicalTrials.gov
2023-504331-41
CTIS
Argentina/Brazil/Canada/Czechia/France/Germany/Greece/Hungary/Italy/Mexico/Netherlands/Poland/Spain/United Kingdom/United States
