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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03560466




Registration number
NCT03560466
Ethics application status
Date submitted
22/03/2018
Date registered
18/06/2018
Date last updated
5/10/2022

Titles & IDs
Public title
Assessment of the Safety and Efficacy of Dupilumab in Children With Asthma (Liberty Asthma Excursion)
Scientific 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
Secondary ID [1] 0 0
2017-003317-25
Secondary ID [2] 0 0
LTS14424
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Asthma 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Asthma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Dupilumab (SAR231893/REGN668)
Treatment: Drugs - Asthma controller therapies (incl. prednisone/prednisolone)
Treatment: Drugs - Asthma reliever therapies

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


Treatment: Drugs: Dupilumab (SAR231893/REGN668)
Pharmaceutical form: solution for injection
Route of administration: subcutaneous (sc)

Treatment: Drugs: Asthma controller therapies (incl. prednisone/prednisolone)
Pharmaceutical form: powder, or solution, or pill
Route of administration: inhaled, oral or parenteral

Treatment: Drugs: Asthma reliever therapies
Pharmaceutical form: powder or solution
Route of administration: inhaled
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The number of patients experiencing any treatment emergent adverse event (TEAE)
Timepoint [1] 0 0
From Day 1 up to Week 64
Primary outcome [2] 0 0
Japan sub-study: Change from baseline in pre-bronchodilator percentage (%) predicted FEV1 at Week 12
Timepoint [2] 0 0
Baseline to Week 12
Secondary outcome [1] 0 0
Annualized rate of severe asthma exacerbation events during the treatment period
Timepoint [1] 0 0
From Day 1 up to Week 52
Secondary outcome [2] 0 0
Change from baseline in % predicted FEV1
Timepoint [2] 0 0
Baseline to Week 64
Secondary outcome [3] 0 0
Change from baseline in absolute FEV1
Timepoint [3] 0 0
Baseline to Week 64
Secondary outcome [4] 0 0
Change from baseline in FVC
Timepoint [4] 0 0
Baseline to Week 64
Secondary outcome [5] 0 0
Change from baseline in FEF 25 to 75%
Timepoint [5] 0 0
Baseline to Week 64
Secondary outcome [6] 0 0
Serum dupilumab concentrations
Timepoint [6] 0 0
From Day 1 up to Week 64
Secondary outcome [7] 0 0
Incidence of treatment-emergent antidrug antibodies (ADA) against dupilumab
Timepoint [7] 0 0
From Day 1 up to Week 64
Secondary outcome [8] 0 0
Blood eosinophil counts
Timepoint [8] 0 0
From Day 1 up to Week 64
Secondary outcome [9] 0 0
Serum total IgE
Timepoint [9] 0 0
From Day 1 up to Week 64
Secondary outcome [10] 0 0
Japan sub-study: Annualized rate of severe asthma exacerbation events during the treatment period
Timepoint [10] 0 0
From Day 1 up to Week 52
Secondary outcome [11] 0 0
Japan sub-study: Change from baseline in pre-bronchodilator % predicted FEV1 at Weeks 2, 4, 8, 24, 52, and 64
Timepoint [11] 0 0
Baseline to Week 2, 4, 8, 24, 52 and 64
Secondary outcome [12] 0 0
Japan sub-stud: Change from baseline in absolute FEV1
Timepoint [12] 0 0
Baseline to Week 2, 4, 8, 12, 24, 52 and 64
Secondary outcome [13] 0 0
Japan sub-study: Change from baseline in FVC
Timepoint [13] 0 0
Baseline to Week 2, 4, 8, 12, 24, 52 and 64
Secondary outcome [14] 0 0
Japan sub-study: Change from baseline in FEF 25-75%
Timepoint [14] 0 0
Baseline to Week 2, 4, 8, 12, 24, 52 and 64
Secondary outcome [15] 0 0
Japan sub-study: Change from baseline in ACQ-IA
Timepoint [15] 0 0
Baseline to Week 2, 4, 8, 12, 24, 36, 52, and 64
Secondary outcome [16] 0 0
Japan sub-study: The number of patients experiencing any TEAEs
Timepoint [16] 0 0
From Day 1 up to Week 64
Secondary outcome [17] 0 0
Japan sub-study: Serum dupilumab concentrations
Timepoint [17] 0 0
From Day 1 up to week 64
Secondary outcome [18] 0 0
Japan sub-study: Incidence of treatment-emergent ADA against dupilumab
Timepoint [18] 0 0
From Day 1 up to Week 64
Secondary outcome [19] 0 0
Japan sub-study: Serum total immunoglobulin E (IgE)
Timepoint [19] 0 0
From Day 1 up to Week 64
Secondary outcome [20] 0 0
Japan sub-study: Change from Baseline in FeNO
Timepoint [20] 0 0
Baseline to Weeks 2, 4, 8, 12, 24, 52, and 64

Eligibility
Key inclusion 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).
Minimum age
6 Years
Maximum age
11 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/06/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/03/2025
Actual
Sample size
Target
Accrual to date
Final
365
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Investigational Site Number :0360005 - North Adelaide
Recruitment postcode(s) [1] 0 0
5006 - North Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Missouri
Country [4] 0 0
United States of America
State/province [4] 0 0
Nebraska
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Oklahoma
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
Argentina
State/province [10] 0 0
Buenos Aires
Country [11] 0 0
Argentina
State/province [11] 0 0
Mendoza
Country [12] 0 0
Brazil
State/province [12] 0 0
Rio Grande Do Sul
Country [13] 0 0
Brazil
State/province [13] 0 0
São Paulo
Country [14] 0 0
Canada
State/province [14] 0 0
Quebec
Country [15] 0 0
Chile
State/province [15] 0 0
Los Ríos
Country [16] 0 0
Chile
State/province [16] 0 0
Reg Metropolitana De Santiago
Country [17] 0 0
Chile
State/province [17] 0 0
Valparaíso
Country [18] 0 0
Colombia
State/province [18] 0 0
Antioquia
Country [19] 0 0
Colombia
State/province [19] 0 0
Cali
Country [20] 0 0
Hungary
State/province [20] 0 0
Budapest
Country [21] 0 0
Hungary
State/province [21] 0 0
Gyula
Country [22] 0 0
Hungary
State/province [22] 0 0
Mezökövesd
Country [23] 0 0
Hungary
State/province [23] 0 0
Szigetvár
Country [24] 0 0
Hungary
State/province [24] 0 0
Székesfehérvár
Country [25] 0 0
Hungary
State/province [25] 0 0
Töröbálint
Country [26] 0 0
Hungary
State/province [26] 0 0
Zalaegerszeg
Country [27] 0 0
Italy
State/province [27] 0 0
Firenze
Country [28] 0 0
Italy
State/province [28] 0 0
Padova
Country [29] 0 0
Italy
State/province [29] 0 0
Roma
Country [30] 0 0
Japan
State/province [30] 0 0
Aichi
Country [31] 0 0
Japan
State/province [31] 0 0
Chiba
Country [32] 0 0
Japan
State/province [32] 0 0
Gunma
Country [33] 0 0
Japan
State/province [33] 0 0
Hokkaido
Country [34] 0 0
Japan
State/province [34] 0 0
Kanagawa
Country [35] 0 0
Japan
State/province [35] 0 0
Saga
Country [36] 0 0
Japan
State/province [36] 0 0
Shiga
Country [37] 0 0
Japan
State/province [37] 0 0
Shizuoka
Country [38] 0 0
Japan
State/province [38] 0 0
Tokyo
Country [39] 0 0
Japan
State/province [39] 0 0
Habikino-shi
Country [40] 0 0
Lithuania
State/province [40] 0 0
Siauliai
Country [41] 0 0
Lithuania
State/province [41] 0 0
Utena
Country [42] 0 0
Lithuania
State/province [42] 0 0
Vilnius
Country [43] 0 0
Mexico
State/province [43] 0 0
Chihuahua
Country [44] 0 0
Mexico
State/province [44] 0 0
Durango, Durango
Country [45] 0 0
Mexico
State/province [45] 0 0
Monterrey, Nuevo León
Country [46] 0 0
Mexico
State/province [46] 0 0
Veracruz
Country [47] 0 0
Poland
State/province [47] 0 0
Lódzkie
Country [48] 0 0
Poland
State/province [48] 0 0
Wielkopolskie
Country [49] 0 0
Russian Federation
State/province [49] 0 0
Perm
Country [50] 0 0
Russian Federation
State/province [50] 0 0
Saint Petersburg
Country [51] 0 0
Russian Federation
State/province [51] 0 0
Saint-Petersburg
Country [52] 0 0
Russian Federation
State/province [52] 0 0
St Petersburg
Country [53] 0 0
Russian Federation
State/province [53] 0 0
St. Petersburg
Country [54] 0 0
South Africa
State/province [54] 0 0
Cape Town
Country [55] 0 0
Spain
State/province [55] 0 0
Barcelona [Barcelona]
Country [56] 0 0
Turkey
State/province [56] 0 0
Adana
Country [57] 0 0
Turkey
State/province [57] 0 0
Ankara
Country [58] 0 0
Turkey
State/province [58] 0 0
Istanbul
Country [59] 0 0
Ukraine
State/province [59] 0 0
Chernivtsi
Country [60] 0 0
Ukraine
State/province [60] 0 0
Dnipro
Country [61] 0 0
Ukraine
State/province [61] 0 0
Kharkiv
Country [62] 0 0
Ukraine
State/province [62] 0 0
Kryvyi Rig
Country [63] 0 0
Ukraine
State/province [63] 0 0
Kyiv
Country [64] 0 0
Ukraine
State/province [64] 0 0
Zaporizhzhya

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Sanofi
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Regeneron Pharmaceuticals
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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
Trial website
https://clinicaltrials.gov/ct2/show/NCT03560466
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Sanofi
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03560466