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

NCT ID: NCT03560466

Last Updated: 2025-11-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 378 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-06-21
• Study Completion Date: 2025-04-01

Brief Summary

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

Detailed Description

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.

Conditions

Asthma

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Dupilumab

Doses of dupilumab will be administered every 2 weeks or every 4 weeks added to current controller medications for 52 weeks

Group Type: EXPERIMENTAL

Dupilumab (SAR231893/REGN668)

Intervention Type: DRUG

Pharmaceutical form: solution for injection

Route of administration: subcutaneous (sc)

Asthma controller therapies (incl. prednisone/prednisolone)

Intervention Type: DRUG

Pharmaceutical form: powder, or solution, or pill

Route of administration: inhaled, oral or parenteral

Asthma reliever therapies

Intervention Type: DRUG

Pharmaceutical form: powder or solution

Route of administration: inhaled

Interventions

Dupilumab (SAR231893/REGN668)

Pharmaceutical form: solution for injection

Route of administration: subcutaneous (sc)

Intervention Type: DRUG

Asthma controller therapies (incl. prednisone/prednisolone)

Pharmaceutical form: powder, or solution, or pill

Route of administration: inhaled, oral or parenteral

Intervention Type: DRUG

Asthma reliever therapies

Pharmaceutical form: powder or solution

Route of administration: inhaled

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 11 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role: collaborator

Sanofi

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

Arizona Allergy and Immunology Research- Site Number : 8400002

Gilbert, Arizona, United States

Asthma and Airway Disease Research Center Site Number : 8400012

Tucson, Arizona, United States

Peninsula Research Associates Site Number : 8400001

Rolling Hills Estates, California, United States

Division of Pulmonary Medicine and Allergy Medicine- Site Number : 8400006

St Louis, Missouri, United States

Somnos Clinical Research Site Number : 8400022

Lincoln, Nebraska, United States

Northwell Health- Site Number : 8400023

Great Neck, New York, United States

Columbia University Medical Center- Site Number : 8400013

New York, New York, United States

Rochester Regional Health- Site Number : 8400007

Rochester, New York, United States

Immunocarolina LLC Site Number : 8400004

Charlotte, North Carolina, United States

Cincinnati Children's Hospital Medical Center Site Number : 8400008

Cincinnati, Ohio, United States

OK Clinical Research, LLC- Site Number : 8400024

Edmond, Oklahoma, United States

Allergy & Asthma Research Center- Site Number : 8400003

San Antonio, Texas, United States

Investigational Site Number : 0320002

CABA, Buenos Aires, Argentina

Investigational Site Number : 0320001

CABA, Buenos Aires, Argentina

Investigational Site Number : 0320004

Vicente Lopez, Buenos Aires F.D., Argentina

Investigational Site Number : 0320003

Buenos Aires, , Argentina

Investigational Site Number : 0320006

Mendoza, , Argentina

Investigational Site Number : 0360005

North Adelaide, South Australia, Australia

Irmandade da Santa Casa de Misericordia de Porto Alegre- Site Number : 0760001

Porto Alegre, Rio Grande do Sul, Brazil

Hospital Sao Lucas da PUCRS- Site Number : 0760007

Porto Alegre, Rio Grande do Sul, Brazil

Unidade Ambulatorial de Pesquisa Clinica I - NGP - UNIFESP- Site Number : 0760002

São Paulo, São Paulo, Brazil

Instituto da Criança of Hospital das Clínicas de São Paulo- Site Number : 0760004

São Paulo, São Paulo, Brazil

Clinica de Alergia Martti Antila Site Number : 0760006

Sorocaba, São Paulo, Brazil

Investigational Site Number : 1240003

Québec, , Canada

Investigational Site Number : 1520001

Valdivia, Los Ríos Region, Chile

Investigational Site Number : 1520005

Santiago, Reg Metropolitana de Santiago, Chile

Investigational Site Number : 1520009

Santiago, Reg Metropolitana de Santiago, Chile

Investigational Site Number : 1520007

Viña del Mar, Región de Valparaíso, Chile

Investigational Site Number : 1520002

Viña del Mar, Región de Valparaíso, Chile

Investigational Site Number : 1700004

Antioquia, , Colombia

Investigational Site Number : 1700002

Cali, , Colombia

Investigational Site Number : 3480006

Budapest, , Hungary

Investigational Site Number : 3480002

Gyula, , Hungary

Investigational Site Number : 3480012

Mezőkövesd, , Hungary

Investigational Site Number : 3480001

Székesfehérvár, , Hungary

Investigational Site Number : 3480008

Szigetvár, , Hungary

Investigational Site Number : 3480003

Töröbálint, , Hungary

Investigational Site Number : 3480007

Zalaegerszeg, , Hungary

Investigational Site Number : 3800003

Florence, , Italy

Investigational Site Number : 3800004

Padua, , Italy

Investigational Site Number : 3800005

Roma, , Italy

Investigational Site Number : 3920013

Chiba, Chiba, Japan

Investigational Site Number : 3920016

Chuo-ku, Chiba, Japan

Investigational Site Number : 3920005

Yotsukaido-shi, Chiba, Japan

Investigational Site Number : 3920015

Fukuoka, Fukuoka, Japan

Investigational Site Number : 3920011

Fukuoka, Fukuoka, Japan

Investigational Site Number : 3920008

Shibukawa, Gunma, Japan

Investigational Site Number : 3920014

Onomichi-shi, Hiroshima, Japan

Investigational Site Number : 3920003

Sapporo, Hokkaido, Japan

Investigational Site Number : 3920012

Tsu, Mie-ken, Japan

Investigational Site Number : 3920010

Ureshino-shi, Saga-ken, Japan

Investigational Site Number : 3920009

Habikino-Shi, , Japan

Investigational Site Number : 4400005

Šiauliai, , Lithuania

Investigational Site Number : 4400003

Utena, , Lithuania

Investigational Site Number : 4400001

Vilnius, , Lithuania

Investigational Site Number : 4400004

Vilnius, , Lithuania

Investigational Site Number : 4840006

Chihuahua City, , Mexico

Investigational Site Number : 4840004

Chihuahua City, , Mexico

Investigational Site Number : 4840003

Durango, Durango, , Mexico

Investigational Site Number : 4840001

Monterrey, Nuevo León, , Mexico

Investigational Site Number : 4840002

Veracruz, , Mexico

Investigational Site Number : 6160002

Poznan, Greater Poland Voivodeship, Poland

Investigational Site Number : 6160001

Lodz, Lódzkie, Poland

Investigational Site Number : 6430004

Perm, , Russia

Investigational Site Number : 6430002

Saint Petersburg, , Russia

Investigational Site Number : 6430001

Saint Petersburg, , Russia

Investigational Site Number : 6430003

Saint Petersburg, , Russia

Investigational Site Number : 6430005

Saint Petersburg, , Russia

Investigational Site Number : 7100001

Cape Town, , South Africa

Investigational Site Number : 7240001

Barcelona, Barcelona [Barcelona], Spain

Investigational Site Number : 7920005

Adana, , Turkey (Türkiye)

Investigational Site Number : 7920001

Ankara, , Turkey (Türkiye)

Investigational Site Number : 7920003

Istanbul, , Turkey (Türkiye)

Investigational Site Number : 7920004

Istanbul, , Turkey (Türkiye)

Investigational Site Number : 8040007

Chernivtsi, , Ukraine

Investigational Site Number : 8040004

Dnipro, , Ukraine

Investigational Site Number : 8040005

Kharkiv, , Ukraine

Investigational Site Number : 8040008

Kryvyi Rig, , Ukraine

Investigational Site Number : 8040001

Kyiv, , Ukraine

Investigational Site Number : 8040002

Zaporizhzhya, , Ukraine

Investigational Site Number : 8040003

Zaporizhzhya, , Ukraine

Countries

United States; Argentina; Australia; Brazil; Canada; Chile; Colombia; Hungary; Italy; Japan; Lithuania; Mexico; Poland; Russia; South Africa; Spain; Turkey (Türkiye); Ukraine

References

Bacharier LB, Maspero JF, Katelaris CH, Fiocchi AG, Gagnon R, de Mir I, Guilbert TW, Jackson DJ, Staudinger HW, Laws E, Mannent LP, Akinlade B, Maloney J, Tawo K, Khokhar FA, Li N, Hardin M, Abdulai RM, Lederer DJ, Robinson LB; Liberty Asthma EXCURSION Investigators. Assessment of long-term safety and efficacy of dupilumab in children with asthma (LIBERTY ASTHMA EXCURSION): an open-label extension study. Lancet Respir Med. 2024 Jan;12(1):45-54. doi: 10.1016/S2213-2600(23)00303-X. Epub 2023 Nov 10.

Reference Type: DERIVED

PMID: 37956679 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

U1111-1200-1757

Identifier Type: REGISTRY

Identifier Source: secondary_id

2017-003317-25

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

LTS14424

Identifier Type: -

Identifier Source: org_study_id