E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
Atopic dermatitis / Eczema |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10003639 |
E.1.2 | Term | Atopic dermatitis |
E.1.2 | System Organ Class | 100000004858 |
|
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 |
The primary objective of the study is to assess the long-term safety of dupilumab in pediatric patients with AD. |
|
E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study are: - To assess the long-term efficacy of dupilumab in pediatric patients with AD - To assess the trough concentrations of functional dupilumab in serum and the immunogenicity in pediatric patients with AD after re-treatment with dupilumab. |
|
E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Vaccine Response: Parents/caregivers will be encouraged to provide vaccination plans for patients for the following vaccines (alone or combined): tetanus, diphtheria, pertussis, meningococcal, Haemophilus influenzae type b, and influenza virus, which are in line with the patient’s age and local medical practice during the study. In case any of the above listed vaccines is planned during the study, parents/caregivers may optionally sign a separate informed consent for the collection of 2 blood samples for assay of vaccine IgG in serum for each vaccination. Live attenuated viruses are excluded during the study. Vaccinations should preferably occur after completion of at least 12 weeks of treatment while remaining in compliance with the patient’s recommended immunization schedule. |
|
E.3 | Principal inclusion criteria |
1. Male or female, ≥6 months to <18 years of age at the time of screening. 2. Participated in a prior dupilumab study in pediatric patients with AD and adequately completed the visits and assessments required for both the treatment and follow-up periods, as defined in the prior study protocol.
|
|
E.4 | Principal exclusion criteria |
1. Patients who, during their participation in a prior dupilumab study in pediatric patients with AD, developed a serious adverse event (SAE) deemed related to study drug, which in the opinion of the investigator or of the medical monitor could indicate that continued treatment with study drug may present an unreasonable risk for the patient. 2. Patients, who during their participation in a prior dupilumab study in pediatric patients with AD, developed an AE that was deemed related to study drug and led to study treatment discontinuation, which in the opinion of the investigator or of the medical monitor could indicate that continued treatment with study drug may present an unreasonable risk for the patient. 3. Treatment with an investigational drug, other than dupilumab, within 8 weeks or within 5 half-lives (if known), whichever is longer, before the baseline visit. 4. History of human immunodeficiency virus (HIV) infection or HIV seropositivity at the screening visit. 5. Known or suspected immunodeficiency. 6. Recent infections requiring anti-infectious treatment 7. Recent history or high risk of clinical endoparasitoses 8. High risk populations (low life expectancy, severe concomitant diseases, etc.) 9. Pregnant or breast-feeding women 10. Women of childbearing potential who are unwilling to practice highly effective contraception 11. Treatment with a live (attenuated) vaccine within 4 weeks before the baseline visit |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence and rate of treatment-emergent adverse events (TEAEs) from baseline through the last study visit. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Throughout the duration of the study |
|
E.5.2 | Secondary end point(s) |
1. Incidence and rate (events per patient-year) of SAEs and AEs of special interest 2. Proportion of patients who achieve and maintain remission (IGA 0-1) over time 3. EASI-75: Proportion of patients achieving and maintaining at least 75% reduction in EASI score over time.
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Throughout the duration of the study |
|
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 | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
|
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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 40 |
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 |
Canada |
Czech Republic |
Germany |
Hungary |
Poland |
United Kingdom |
United States |
|
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
|
The end of study is defined as the date that the last patient completes the last study visit, withdraws from the study, or is lost to follow-up (ie, the patient can no longer be contacted by the investigator). |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 12 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 12 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |