E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10003553 |
E.1.2 | Term | Asthma |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the long-term safety and tolerability of dupilumab in pediatric patients with asthma who participated in a previous dupilumab asthma clinical study. |
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E.2.2 | Secondary objectives of the trial |
-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. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-Pediatric patients with asthma who completed the treatment in a dupilumab asthma trial (EFC14153). -Signed written informed consent/assent. - 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 CBC with differential and basic chemistry) within 1 month prior to LTS14424 Visit 1. |
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E.4 | Principal 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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Treatment-emergent-adverse-event (TEAEs): The number (n) and percentage (%) of patients experiencing any TEAEs. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1) Severe asthma exacerbation events: Annualized rate of severe asthma exacerbation events, during the treatment period 2) Change in % predicted FEV1: Change in percentage (%) predicted forced expiratory volume in 1 second (FEV1) - Clinically significant changes from baseline 3) Change in absolute FEV1: Change in absolute FEV1 - Clinically significant changes from baseline 4) Change in FVC: Change in forced vital capacity (FVC) 5) Change in FEF: Change in forced expiratory flow [FEF] 25-75%) 6) Assessment of dupilumab concentration: Serum dupilumab concentrations - Changes from first dupilumab injection 7) Assessment of immunogenicity: Titers of Anti-dupilumab antibodies 8) Assessment of blood Eosinophil count: Blood: Eosinophil count 9) Assessment of immunoglobulins: Serum: Immunoglobulins (Ig)
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1) From Day 1 up to Week 52 2) to 9) From Day 1 up to Week 64 |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 34 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Argentina |
Australia |
Brazil |
Canada |
Chile |
Colombia |
Hungary |
Italy |
Lithuania |
Mexico |
Poland |
Romania |
Russian Federation |
South Africa |
Spain |
Turkey |
Ukraine |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 90 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 90 |