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Completed

NCT Number: NCT03560466

Assessment of the Safety and Efficacy of Dupilumab in Children With Asthma (Liberty Asthma Excursion)

Primary Objective:

* To evaluate the long-term safety and tolerability of dupilumab in pediatric patients with asthma who participated in a previous dupilumab asthma clinical study. * To evaluate the efficacy of dupilumab in children of 6 to <12 years of age with uncontrolled persistent asthma in the Japan sub-study.

Secondary Objectives:

* To evaluate the long-term efficacy of dupilumab in pediatric patients with asthma who participated in a previous dupilumab asthma clinical study. * To evaluate dupilumab in pediatric patients with asthma who participated in a previous dupilumab asthma clinical study with regard to:

* Systemic exposure. * Anti-drug antibodies (ADAs). * Biomarkers. * To evaluate the safety and tolerability of dupilumab in pediatric patients with asthma in the Japan sub-study * To evaluate dupilumab in pediatric patients with asthma in the Japan substudy with regard to:

* Systemic exposure, * Anti-drug antibodies (ADAs), * Biomarkers

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Key information

Conditions

Asthma Bronchial Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Lung Diseases Lung Diseases, Obstructive Respiratory Hypersensitivity Respiratory Tract Diseases

Age range

6 year–11 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Investigational Site Number : 0320001, CABA, Buenos Aires, Argentina

Loading trial locations.

About this study

Study duration per participant is approximatively 64 weeks including a 52 weeks treatment period and 12 weeks post treatment follow-up.

Japan substudy:

Study duration per participant is approximately 68 weeks including 3-5 weeks screening period, 52 weeks treatment period and 12 weeks post treatment follow-up period.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Pediatric patients with asthma who completed the treatment in a dupilumab asthma trial (EFC14153).
  • Signed written informed consent/assent. Specific for Brazil: EFC14153 patients from Brazil, who prematurely discontinued Investigational Medicinal Product (IMP) to receive Yellow Fever vaccine (a live attenuated vaccine) during Yellow Fever outbreak, are allowed to be enrolled in LTS14424 after completing the required procedures in EFC14153 (completion of remaining visits and procedures until end of treatment (EOT) V28, considered as V1 for LTS14424).

Patients who are not able to complete their treatment in Study EFC14153 due to the COVID-19 pandemic will be allowed to enroll into Study LTS14424. Patients who enroll in LTS14424 after completing the EFC14153 EOS visit should have eligibility for LTS14424 reevaluated including background medication check and laboratory assessments (including complete blood count [CBC] with differential and basic chemistry) within 1 month prior to LTS14424 Visit 1.

For Japan sub-study

  • Signed written inform consent/assent
  • Children 6 to <12 years of age, with a physician diagnosis of persistent asthma for ≥12 months prior to screening
  • Blood eosinophil count ≥150 cells/μL or fractional exhaled nitric oxide (FeNO) ≥20 parts per billion (ppb) at screening visit (Visit 0).

Exclusion criteria

  • Any chronic lung disease other than asthma (eg, cystic fibrosis, bronchopulmonary dysplasia) which may impair lung function.
  • Inability to follow the procedures of the study/noncompliance (eg, due to language problems or psychological disorders).
  • Patients receiving concomitant treatment or required a new concomitant treatment prohibited in the study.
  • Patients or his/her parent(s)/caregiver(s)/legal guardian(s) is related to the Investigator or any Sub-Investigator, research assistant, pharmacist, study coordinator, other staff thereof directly involved in the conduct of the study.
  • Patients who experienced any hypersensitivity reactions to dupilumab in a previous dupilumab study, which, in the opinion of the Investigator, could indicate that continued treatment with dupilumab may present an unreasonable risk for the patient.
  • Any abnormalities or adverse events at screening (last treatment visit in the study EFC14153 will be the screening visit) that per Investigator judgment would adversely affect patient's participation in this study or would require permanent IMP discontinuation.
  • For female patients who have commenced menstruating at any time during the study and are either:
  • Found to have a positive urine pregnancy test, or
  • Sexually active, not using an established acceptable contraceptive method.
  • Planned live, attenuated vaccinations during the study.
  • Patients with active autoimmune disease or patients using immunosuppressive therapy for autoimmune disease (eg, juvenile idiopathic arthritis, inflammatory bowel disease, systemic lupus erythematosus) at enrollment.

For Japan sub-study:

  • Any chronic lung disease other than asthma (eg, cystic fibrosis, bronchopulmonary dysplasia) which may impair lung function.
  • Inability to follow the procedures of the study/noncompliance (eg, due to language problems or psychological disorders).
  • Patients receiving concomitant treatment or required a new concomitant treatment prohibited in the study at the screening and enrollment visits.
  • Patients who previously have been treated with dupilumab
  • Diagnosed with active parasitic infection (helminthes); suspected or high risk of parasitic infection, unless clinical and (if necessary) laboratory assessments have ruled out active infection before randomization
  • Known or suspected history of immunosuppression, including history of invasive opportunistic infections (eg, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystosis, aspergillosis), despite infection resolution; or unusually frequent, recurrent, or prolonged infections, per Investigator's judgment.

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

Treatment and study plan

Dupilumab (SAR231893/REGN668)

Drug

Pharmaceutical form: solution for injection

Route of administration: subcutaneous (sc)

Asthma controller therapies (incl. prednisone/prednisolone)

Drug

Pharmaceutical form: powder, or solution, or pill

Route of administration: inhaled, oral or parenteral

Asthma Reliever Therapies

Drug

Pharmaceutical form: powder or solution

Route of administration: inhaled

Primary outcomes

  1. Main Study: Number of Participants With Any Treatment-Emergent Adverse Events (TEAEs)

    Time frame: From first dose of study treatment (Day 1) up to 112 days post last dose of study treatment, approximately 64 weeks

    An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which did not necessarily have to have a causal relationship with this treatment. TEAEs were AEs that developed or worsened or became serious during the TEAE period.

  2. Japan Sub-study: Change From Baseline to Week 12 in Pre-Bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)

    Time frame: Baseline (Day 1) to Week 12

    FEV1 was the volume of air (in liters) exhaled from the lungs in the first second of a forced expiration as measured by spirometer. Spirometry was performed after a wash out period of bronchodilators according to their action duration. Baseline was defined as the last available measurement prior to the first study treatment dose if the participant was treated, or the last available value up to enrollment if the participant was not exposed to study treatment in the current study.

Secondary outcomes

  1. Japan Sub-study: Number of Participants With Any Treatment-Emergent Adverse Events

    Time frame: From first dose of study treatment (Day 1) up to 112 days post last dose of study treatment, approximately 64 weeks

    An AE was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which did not necessarily have to have a causal relationship with this treatment. TEAEs were AEs that developed or worsened or became serious during the TEAE period.

  2. Annualized Rate of Severe Asthma Exacerbation Events During the 52-Week Treatment Period

    Time frame: Up to Week 52

    A severe exacerbation event during study was defined as a deterioration of asthma requiring use of systemic corticosteroid (SCS) for ≥3 days or hospitalization/emergency room visit because of asthma, requiring SCS. Annualized severe exacerbation event rate was defined as total number of events that occurred during 52-week treatment period divided by total number of participant-years followed in 52-week treatment period.

  3. Change From Baseline in Pre-Bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second Over Time

    Time frame: Baseline (Day 1) and Weeks 2, 4 (Japan sub-study), 8, 12, 24, 52 and 64

    FEV1 was volume of air (in liters) exhaled from the lungs in first second of a forced expiration as measured by spirometer. Spirometry was performed after a wash out period of bronchodilators according to their action duration. Baseline (main study): original baseline of parent study EFC14153. Baseline (sub-study): last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in current study.

  4. Change From Baseline in Absolute Forced Expiratory Volume in 1 Second Over Time

    Time frame: Baseline (Day 1) and Weeks 2, 4 (Japan sub-study), 8, 12, 24, 52 and 64

    FEV1 was volume of air (in liters) exhaled from the lungs in first second of a forced expiration as measured by spirometer. Spirometry was performed after a wash out period of bronchodilators according to their action duration. Baseline (main study): original baseline of parent study EFC14153. Baseline (sub-study): last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in current study.

  5. Change From Baseline in Forced Vital Capacity (FVC) Over Time

    Time frame: Baseline (Day 1) and Weeks 2, 4 (Japan sub-study), 8, 12, 24, 52 and 64

    FVC was defined as volume of air (in liters) that could be forcibly blown out after full inspiration in upright position as measured by spirometer. Spirometry was performed after wash out period of bronchodilators according to their action duration. Baseline (main study):original baseline of parent study EFC14153. Baseline (sub-study): last available measurement prior to first study treatment dose if participant was treated or last available value up to enrollment if participant was not exposed to study treatment in current study.

  6. Change From Baseline in Forced Expiratory Flow [FEF] 25% to 75% Over Time

    Time frame: Baseline (Day 1) and Weeks 2, 4 (Japan sub-study), 8, 12, 24, 52 and 64

    FEF was defined as amount of air which can be forcibly exhaled from lungs in first second of forced exhalation. FEF 25-75% was the mean FEF between 25% and 75% of FVC. FVC was defined as volume of air that could be forcibly blown out after full inspiration in upright position. Spirometry was performed after wash out period of bronchodilators. Baseline (main study): original baseline of parent study EFC14153. Baseline (sub-study): last available measurement prior to first study treatment dose if participant was treated or last available value up to enrollment if participant was not exposed to study treatment in current study.

  7. Main Study: Change From Baseline in Percent Predicted Forced Vital Capacity Over Time

    Time frame: Baseline (Day 1) and Weeks 2, 8, 12, 24, 52 and 64

    FVC was defined as volume of air (in liters) that could be forcibly blown out after full inspiration in upright position as measured by spirometer. Spirometry was performed after wash out period of bronchodilators according to their action duration. Baseline was defined as the original baseline of parent study EFC14153.

  8. Japan Sub-study: Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-Question Version (ACQ-7-IA) Over Time

    Time frame: Baseline (Day 1) and Weeks 2, 4, 8, 12, 24, 36, 52 and 64

    ACQ-7-IA had 7 questions with first 5 items of ACQ-7 addressing most common asthma symptoms: frequency in past week awoken by asthma during night, severity of asthma symptoms in morning, limitation of daily activities due to asthma, shortness of breath due to asthma, wheeze; 2 questions on short-acting bronchodilator use and predicted bronchodilator use of FEV1. Participants/parents/guardians recalled child's previous week asthma and responded to questions on 7-point scale: 0=no impairment, 6=maximum impairment. Global ACQ-7-IA score was the mean of 7 questions, ranging from 0 (totally controlled) to 6 (severely uncontrolled). Higher scores indicated lower asthma control. Baseline: last available measurement prior to first study treatment dose if participant was treated or last available value up to enrollment if participant was not exposed to study treatment in current study.

  9. Japan Sub-study: Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 5-Question Version (ACQ-5-IA) Over Time

    Time frame: Baseline (Day 1) and Weeks 2, 4, 8, 12, 24, 36, 52 and 64

    The ACQ-5-IA had 5 questions addressing most common asthma symptoms: frequency in past week awoken by asthma during night, severity of asthma symptoms in morning, limitation of daily activities due to asthma, shortness of breath due to asthma and wheeze. Participants/parents/guardians recalled the child's previous week asthma and responded to symptom and bronchodilator use questions on a 7-point scale: 0=no impairment, 6=maximum impairment. Global ACQ-5-IA score was the mean of 5 questions, ranging from 0 (totally controlled) to 6 (severely uncontrolled). Higher score indicated lower asthma control. Baseline: last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in current study.

  10. Serum Concentrations of Dupilumab

    Time frame: Weeks 12, 24, 52 and 64

    Blood samples were collected at the specified timepoints for determination of functional dupilumab concentration in serum.

  11. Number of Participants With Anti-Drug Antibodies (ADAs) Against Dupilumab

    Time frame: From first dose of study treatment (Day 1) to Week 64

    ADA positive was defined as either treatment-boosted or treatment-emergent response in the ADA assay. Treatment-boosted response was defined as a positive response in the ADA assay post first dose in the current study that was greater than or equal to 4-fold of the baseline titer levels, when baseline results were positive. Treatment-emergent response was defined as a positive response in the ADA assay post first dose in the current study, when baseline results were negative or missing. Baseline (main study): original baseline of parent study EFC14153. Baseline (sub-study): last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in current study.

  12. Main Study: Percent Change From Baseline in Blood Eosinophil Count at Week 64

    Time frame: Baseline (Day 1) to Week 64

    Blood samples were collected at specified timepoints to assess percent change from baseline in eosinophil count. Baseline was defined as the original baseline of parent study EFC14153.

  13. Percent Change From Baseline in Total Immunoglobulin (Ig) E in Serum at Week 64

    Time frame: Baseline (Day 1) to Week 64

    Blood samples were collected at specified timepoints to assess percent change from baseline in total IgE in serum. Baseline (main study): the original baseline of parent study EFC14153. Baseline (sub-study): last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in current study.

  14. Japan Sub-study: Change From Baseline in Fractional Exhaled Nitric Oxide (FeNO) at Week 64

    Time frame: Baseline (Day 1) to Week 64

    FeNO was analyzed using a NIOX instrument or similar analyzer using a flow rate of 50 milliliter per second. FeNO assessment was conducted prior to spirometry and following a fast of ≥1 hour. Baseline was defined as the last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in the current study.

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Collaborators

  • Regeneron Pharmaceuticals

Registry information

Official study title

One Year Study to Evaluate the Long-term Safety and Tolerability of Dupilumab in Pediatric Patients With Asthma Who Participated in a Previous Dupilumab Asthma Clinical Study

Important dates

Study start
2018
Primary completion
2025
Study completion
2025
First posted
Jun 18, 2018
Registry last updated
Nov 21, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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