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 | 17.1 |
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 characterize the safety and pharmacokinetics (PK) of dupilumab in pediatric patients with moderate-to-severe atopic dermatitis (AD) (for adolescents ≥12 to <18 years of age) or severe AD (for children ≥6 to <12 years of age). |
|
E.2.2 | Secondary objectives of the trial |
The secondary objective of the study is to explore the immunogenicity and efficacy of dupilumab in pediatric patients with moderate-to-severe AD (for adolescents ≥12 to <18 years of age) or severe AD (for children ≥6 to <12 years of age). |
|
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 |
The purpose of the genomic analyses is to identify genomic associations with clinical or biomarker response to target modulation, disease prognosis and progression, or other clinical outcome measures. |
|
E.3 | Principal inclusion criteria |
1. Male or female patients ≥6 to <18 years of age with a diagnosis of 1. Atopic Dermatitis whose disease cannot be adequately controlled with topical medications
2. Minimum disease severity, as defined by Investigator’s Global Assessment (IGA)
a. IGA = 3 or 4 in adolescents ≥12 to <18 year of age
b. IGA = 4 in children ≥6 to <12 years of age
|
|
E.4 | Principal exclusion criteria |
1. Recent treatment (within specific time windows before the baseline visit) with systemic immunosuppressive agents for eg. Systemic corticosteroids, live (attenuated) vaccines and other investigational drugs including biologics
2. History of any of the following infections:
a. Any systemic infection requiring treatment within 4 weeks before the baseline visit
b. Superficial skin infections within 1 week before the baseline visit
c. Known history of HIV infection
d. History of seropositivity to hepatitis B or C screening tests
e. History of clinical endoparasitosis (ie, helminthic infection) within 12 months before the baseline visit, or high risk of helminthic infection, unless subsequent medical assessments (e.g. stool exam, blood tests, etc.) have ruled out the possibility of parasite infection/infestation
3. History of malignancy within 5 years before the baseline visit
4. Persistent (confirmed by repeated tests ≥2 weeks apart) elevated transaminases (alanine aminotransferase [ALT] and/or aspartate aminotransferase [AST]) more than 3 times the upper limit of normal (ULN) during the screening period
5. Presence of any severe concomitant illness(es) that, in the investigator’s judgment, would adversely affect the patient’s participation in the study
6. Presence of skin comorbidities that may interfere with study assessments
7. Females patients who are pregnant or breastfeeding
8. Female patients who are of reproductive potential and are sexually active, who are unwilling to use adequate methods of contraception
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
The primary objective of characterizing the PK profiles of dupilumab in pediatric
AD patients aged ≥6 to <18 years will be addressed by PK parameters. |
|
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) |
The secondary objectives are:
- Incidence of treatment-emergent adverse events (TEAEs)
- Percent change from baseline in Eczema Area and Severity Index (EASI)
- Percent change from baseline in SCORing Atopic Dermatitis (SCORAD)
score
- Percent change from baseline in Pruritus Numerical Rating Scale (NRS)
- Percentage of patients with an Investigator Global Assessment (IGA) score
of 0 or 1 |
|
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) | Yes |
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.8.2.4 | 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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 20 |
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 |
|
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 5 |