E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Mild, moderate or severe asthma |
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E.1.1.1 | Medical condition in easily understood language |
Mild, moderate or severe asthma |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
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 describe the pharmacokinetic parameters following a single subcutaneous administration of tezepelumab in children with mild, moderate, or severe asthma |
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E.2.2 | Secondary objectives of the trial |
• To evaluate the immunogenicity of tezepelumab |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1 Written informed consent and written informed assent and any locally required authorisation obtained from the subject and legal representative prior to any study related procedure taking place.
Age 2 Age 5 to 11 years (inclusive) at Visit 1 and Visit 2 (Day 1).
Type of Subject and Disease Characteristics 3 Documented physician diagnosed asthma for at least 6 months prior to Visit 1. 4 Documented treatment with total daily dose of either low, medium, or high dose ICS for at least 6 months, as described in Step 2 to Step 4 of GINA guidelines (GINA 2020) with stable dose for at least 3 months prior to Visit 1. 6 Evidence of asthma as documented by either: (a) Historical airway reversibility, or (b) Airway reversibility after use of an inhaled short-acting β2 agonist (SABA) (FEV1 ≥ 12%) demonstrated at Visit 1a, or at Visit 2a if not achieved at Visit 1a and historical airway reversibility is not available. 7 Pre bronchodilator (BD) FEV1 of ≥ 70% of predicted normal value at Visit 1a.
Weight 11 Body weight ≥ 16 kg at Visit 1 and Visit 2 (Day 1). 12 Body mass index for age at both screening and Day 1 that is between 5th and 95th percentile (Centers for Disease Control Growth Charts). |
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E.4 | Principal exclusion criteria |
Subjects are excluded from the study if any of the following criteria apply: Medical Conditions 1 History of any clinically significant disease or disorder other than asthma which, in the opinion of the Investigator, may either put the subject at risk because of participation in the study, or influence the results or the subject’s ability to participate in the study. 2 History of a deterioration in asthma or asthma exacerbation that required a burst of systemic corticosteroids within 3 months of Visit 1, up to and including Visit 2 (Day 1). 3 Use of systemic or intra-articular glucocorticosteroids for conditions other than asthma is not allowed within 3 months prior to Visit 2 and is discouraged until EOS. 4 History of hospitalisation (overnight admission) for asthma within 6 months of Visit 1, up to and including Visit 2 (Day 1). 5 History of a life threatening asthma exacerbation requiring intubation or mechanical ventilation. 6 History of systemic corticosteroid use for the maintenance treatment of asthma within 3 months of Visit 1, up to and including Visit 2 (Day 1) and discouraged until EOS. 11 History of cancer.
Prior/Concurrent Clinical Study Experience 17 History of hypersensitivity or anaphylactic reaction to any biologic therapy.
Diagnostic assessments 22 Any clinical signs, symptoms, or abnormal findings during screening that may be indicative of past or present MIS-C. |
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E.5 End points |
E.5.1 | Primary end point(s) |
• Maximum concentration (Cmax) • Time to Cmax (tmax) • Area under the concentration-time curve (AUC) • Terminal phase elimination half-life (t1/2) • Apparent clearance (CL/F) • Apparent steady-state volume of distribution (Vss/F) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Various timepoints throughout the study |
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E.5.2 | Secondary end point(s) |
• Presence of anti-drug antibodies (ADA) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Various timepoints throughout the study |
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
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) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | No |
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.8.3 |
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 | 5 |
E.8.5 | The trial involves multiple Member States | No |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
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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The end of study is defined as the date of the last visit of the last subject in the study. |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 5 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 5 |