Clinical trial NCT06191315: Efficacy and Safety of Subcutaneous Dupilumab in Participants With Asthma/Asthmatic Wheeze Aged 2 to <6 Years (LIBERTY ASTHMA TREKIDS)
Official title: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Efficacy and Longterm Safety of Dupilumab in Children 2 to <6 Years of Age With Uncontrolled Asthma and/or Recurrent Severe Asthmatic Wheeze
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Wheezing
Asthma
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: Regeneron Pharmaceuticals
First posted: 2024-01-05
Enrollment: 90
Sex: ALL
Minimum age (years): 2
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a parallel, Phase 3, 2-arm study to evaluate the efficacy and long-term safety of dupilumab treatment in children 2 to \<6 years of age with uncontrolled asthma and/or recurrent severe asthmatic wheeze. The study will be conducted in 2 parts. Part A will be a 52-week, randomized, double-blind, placebo-controlled study to assess the safety and efficacy of dupilumab in children aged 2 to \<6 years old with uncontrolled asthma and/or recurrent severe asthmatic wheeze. At the end of Part A, all eligible participants will be offered participation in Part B, an optional open-label extension phase.

Study details include:

Part A:

The study duration of part A will be up to 68 weeks consisting of a 4-week Screening, a 52week treatment period, and a 12-week post-treatment follow-up period. For participants who will chose to participate in Part B, the study duration will be up to 120 weeks (additional 52-week treatment period).

Part B:

For participants who will choose to participate in Part B, the study duration will be up to 120 weeks (Part A \[4-week Screening and a 52-week treatment period\] plus additional 52-week treatment period and a 12-week post-treatment follow-up period).

Detailed description: The duration per participant is up to 120 Weeks.

Eligibility criteria: Inclusion Criteria:

* Participant must be 2 to \<6 years of age
* Diagnosis of asthma or recurrent severe asthmatic wheeze that is not controlled with chronic 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:

  1. Physician diagnosed Atopic Dermatitis,
  2. Allergic sensitization to at least 1 aeroallergen (with a positive serum IgE defined as a value ≥0.35 kU/L).

     OR 2 minor criteria:
  3. Wheezing unrelated to colds,
  4. Peripheral blood eosinophilia ≥4%,
  5. 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.

Exclusion Criteria:

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).

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

Primary outcomes:
measure: Part A: Annualized rate of severe asthma exacerbations during the 52-week treatment period
timeFrame: Baseline through Week 52
measure: Part B: Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), and AEs leading to permanent treatment discontinuation
timeFrame: Week 52 through Week 116
Secondary outcomes:
measure: Part A: Annualized rate of hospitalization, ER or urgent care visit for asthma exacerbation during the 52week treatment period.
timeFrame: Baseline through Week 52
measure: Part A: Annualized rate of moderate asthma exacerbations during the 52-week treatment period
timeFrame: Baseline through Week 52
measure: Part A: Cumulative ICS dose during the 52-week treatment period
timeFrame: Baseline through Week 52
measure: Part A: Change from baseline in weekly average use of reliever medication during the 52-week treatment period
timeFrame: Baseline to Week 52
measure: Part A: Mean number of days without asthma symptoms (DWAS) using the Pediatric Asthma Caregiver Diary (PACD) during the 52-week treatment period
timeFrame: Baseline through Week 52
measure: Part A: Change from baseline to Week 52 in daytime symptom score using the daytime record of PACD
timeFrame: Baseline to Week 52
measure: Part A: Incidence of TEAEs, SAEs, AESIs, and AEs leading to permanent treatment discontinuation
timeFrame: Baseline through Week 52
measure: Part A: Change from baseline to Week 52 in Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Scale
timeFrame: Baseline to Week 52
measure: Part A: Caregiver Global Impression of Change in their child's asthma control (CGI-change in asthma control) at Week 52
timeFrame: Week 52
measure: Part A: Physician Global Assessment of Change of the child's asthma control (PGA-change in asthma control) at Week 52
timeFrame: Week 52
measure: Part A: Change from baseline to Week 52 in Caregiver Global Impression of their child's asthma control (CGI-asthma control)
timeFrame: Baseline to Week 52
measure: Part A: Change from baseline to Week 52 in Caregiver Global Impression of their child's asthma severity (CGI-asthma severity)
timeFrame: Baseline to Week 52
measure: Part A: Change from baseline to Week 52 in Physician Global Assessment of the child's asthma control (PGA-asthma control).
timeFrame: Baseline to Week 52
measure: Part A: Change from baseline to Week 52 in Test for Respiratory and Asthma Control in Kids (TRACK)
timeFrame: Baseline to Week 52
measure: Part A: Change from baseline in blood eosinophil level at Weeks 24 and 52
timeFrame: Baseline to Week 24 and 52
measure: Part A: Concentration of dupilumab in serum over time during the 52-week treatment period
timeFrame: Baseline through Week 52
measure: Part A: Incidence of treatment-emergent anti-drug antibody (ADA) against dupilumab over time
timeFrame: Baseline through Week 52
measure: Part A: IgG response to any vaccination for tetanus, diphtheria and pertussis and antibody for influenza (HAI antibody titers) vaccine administered according to vaccination schedule during the 52-week treatment period
timeFrame: Baseline through Week 52
measure: Part B: Annualized rate of severe asthma exacerbations events during the 52-week Part B treatment period
timeFrame: Week 52 through Week 104
measure: Part B Concentration of dupilumab in serum over time during the 52-week Part B treatment period
timeFrame: Week 52 through Week 104
measure: Part B: Incidence of treatment-emergent anti-drug antibodies (ADAs) against dupilumab over time
timeFrame: Week 52 through Week 104
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital- Site Number : 8400001
state: Arizona
city: Newport Beach
country: United States
facility: Newport Children's Medical Group - Newport Beach- Site Number : 8400023
state: California
city: Destin
country: United States
facility: Allervie Clinical Research - Destin- Site Number : 8400016
state: Florida
city: Miami
country: United States
facility: EMDA Clinical Research- Site Number : 8400026
state: Florida
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400011
state: Illinois
city: Owensboro
country: United States
facility: Allergy and Asthma Specialist- Site Number : 8400002
state: Kentucky
city: Rochester
country: United States
facility: Mayo Clinic Hospital Rochester- Site Number : 8400008
state: Minnesota
city: Buffalo
country: United States
facility: UBMD Pediatrics- Site Number : 8400013
state: New York
city: Hawthorne
country: United States
facility: Boston Children's Health Physicians - Hawthorne- Site Number : 8400010
state: New York
city: Chapel Hill
country: United States
facility: UNC Children's Hospital- Site Number : 8400005
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center- Site Number : 8400004
state: Ohio
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center- Site Number : 8400015
state: Ohio
city: Nashville
country: United States
facility: Vanderbilt University Medical Center- Site Number : 8400024
state: Tennessee
city: Boerne
country: United States
facility: South Texas Medical Research Institute - TTS Research- Site Number : 8400022
state: Texas
city: Buenos Aires
country: Argentina
facility: Investigational Site Number : 0320008
state: Ciudad De Buenos Aires
city: Rosario
country: Argentina
facility: Investigational Site Number : 0320005
state: Santa Fe
city: Buenos Aires
country: Argentina
facility: Investigational Site Number : 0320003
city: Buenos Aires
country: Argentina
facility: Investigational Site Number : 0320002
city: Buenos Aires
country: Argentina
facility: Investigational Site Number : 0320001
city: Corrientes
country: Argentina
facility: Investigational Site Number : 0320009
city: Córdoba
country: Argentina
facility: Investigational Site Number : 0320006
city: Mendoza
country: Argentina
facility: Investigational Site Number : 0320004
city: Recife
country: Brazil
facility: Inst de Medicina Integral Professor Fernando Figueira- Site Number : 0760006
state: Pernambuco
city: Porto Alegre
country: Brazil
facility: Hospital Ernesto Dornelles Site Number : 0760004
state: Rio Grande Do Sul
city: Porto Alegre
country: Brazil
facility: Hospital Ernesto Dornelles- Site Number : 0760004
state: Rio Grande Do Sul
city: Sorocaba
country: Brazil
facility: Clinica de Alergia Martti Antila- Site Number : 0760002
state: São Paulo
city: São Paulo
country: Brazil
facility: Hospital das Clinicas FMUSP- Site Number : 0760001
city: Edmonton
country: Canada
facility: Investigational Site Number : 1240002
state: Alberta
city: Vancouver
country: Canada
facility: Investigational Site Number : 1240001
state: British Columbia
city: Hamilton
country: Canada
facility: Investigational Site Number : 1240007
state: Ontario
city: Montreal
country: Canada
facility: Investigational Site Number : 1240006
state: Quebec
city: Sherbrooke
country: Canada
facility: Investigational Site Number : 1240005
state: Quebec
city: Prague
country: Czechia
facility: Investigational Site Number : 2030001
city: Créteil
country: France
facility: Investigational Site Number : 2500005
city: Lille
country: France
facility: Investigational Site Number : 2500001
city: Nice
country: France
facility: Investigational Site Number : 2500004
city: Paris
country: France
facility: Investigational Site Number : 2500002
city: Paris
country: France
facility: Investigational Site Number : 2500003
city: Düsseldorf
country: Germany
facility: Investigational Site Number : 2760002
city: Frankfurt
country: Germany
facility: Investigational Site Number : 2760004
city: Leipzig
country: Germany
facility: Investigational Site Number : 2760001
city: Athens
country: Greece
facility: Investigational Site Number : 3000001
city: Budapest
country: Hungary
facility: Investigational Site Number : 3480005
city: Debrecen
country: Hungary
facility: Investigational Site Number : 3480004
city: Szigetvár
country: Hungary
facility: Investigational Site Number : 3480001
city: Székesfehérvár
country: Hungary
facility: Investigational Site Number : 3480002
city: Milan
country: Italy
facility: Buzzi Children's Hospital - Investigational Site Number : 3800003
state: Lombardia
city: Padua
country: Italy
facility: Investigational Site Number : 3800005
state: Padova
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesù - Investigational Site Number : 3800002
state: Roma
city: Naples
country: Italy
facility: AOU Luigi Vanvitelli - Investigational Site Number : 3800004
city: Pavia
country: Italy
facility: Fondazione IRCCS Policlinico San Matteo - Investigational Site Number : 3800001
city: Fukuoka
country: Japan
facility: Investigational Site Number : 3920002
city: Fukuoka
country: Japan
facility: Investigational Site Number : 3920001
city: TSU
country: Japan
facility: Investigational Site Number : 3920004
city: Ureshino
country: Japan
facility: Investigational Site Number : 3920003
city: San Juan Del Río
country: Mexico
facility: Investigational Site Number : 4840001
state: Querétaro
city: Durango
country: Mexico
facility: Investigational Site Number : 4840004
city: Veracruz
country: Mexico
facility: Investigational Site Number : 4840002
city: Rotterdam
country: Netherlands
facility: Investigational Site Number : 5280001
city: Lodz
country: Poland
facility: Investigational Site Number : 6160001
state: Lódzkie
city: Tarnow
country: Poland
facility: Investigational Site Number : 6160004
city: Warsaw
country: Poland
facility: Investigational Site Number : 6160003
city: Barcelona
country: Spain
facility: Investigational Site Number : 7240001
state: Barcelona [Barcelona]
city: Sabadell
country: Spain
facility: Investigational Site Number : 7240002
state: Castilla Y León
city: Esplugues de Llobregat
country: Spain
facility: Investigational Site Number : 7240007
state: Catalunya [Cataluña]
city: Jerez de La Frontera
country: Spain
facility: Investigational Site Number : 7240006
state: Cádiz
city: Santiago de Compostela
country: Spain
facility: Investigational Site Number : 7240003
state: Galicia [Galicia]
city: Madrid
country: Spain
facility: Investigational Site Number : 7240005
state: Madrid, Comunidad De
city: Valencia
country: Spain
facility: Investigational Site Number : 7240004
city: Leicester
country: United Kingdom
facility: Investigational Site Number : 8260002
state: Leicestershire
city: Birmingham
country: United Kingdom
facility: Investigational Site Number : 8260003
city: Bradford
country: United Kingdom
facility: Investigational Site Number : 8260004
city: London
country: United Kingdom
facility: Investigational Site Number : 8260001
Study officials:
References: