Clinical trial NCT02407756: A Study to Determine the Safety and Tolerability of Dupilumab (REGN668/SAR231893) in Patients Aged ≥6 to <18 Years With Atopic Dermatitis (Eczema)
Official title: A Phase 2a Study Investigating the Safety, Pharmacokinetics, Immunogenicity, and Exploratory Efficacy of Dupilumab in Patients Aged ≥6 to <18 Years With Atopic Dermatitis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Atopic Dermatitis
Keywords: Eczema
Lead sponsor: Regeneron Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: Sanofi
First posted: 2015-04-03
Enrollment: 78
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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).

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).


Eligibility criteria: Key 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)

   1. IGA = 3 or 4 in adolescents ≥12 to \<18 year of age
   2. IGA = 4 in children ≥6 to \<12 years of age

Key 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:

   1. Any systemic infection requiring treatment within 4 weeks before the baseline visit
   2. Superficial skin infections within 1 week before the baseline visit
   3. Known history of HIV infection
   4. History of seropositivity to hepatitis B or C screening tests
   5. 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

Primary outcomes:
measure: Pharmacokinetics (PK) of Dupilumab: Maximum Plasma Concentration Observed (Cmax) After Single Administration
timeFrame: Day 2, 4, 8, 15, 22, 29, 36, 43, and 50
measure: PK of Dupilumab: Area Under the Plasma Concentration Versus Time Curve (AUClast) After Single Administration
timeFrame: Day 2, 4, 8, 15, 22, 29, 36, 43, and 50
measure: PK of Dupilumab: Trough Dupilumab Concentration in Serum (Ctrough) Before 3rd and 4th Repeated Dose
timeFrame: Pre-dose on Day 71 and Day 85
Secondary outcomes:
measure: Percent Reduction From Baseline in Eczema Area and Severity Index (EASI) at Week 12
timeFrame: Baseline to Week 12 (one week after last dose)
measure: Percent Reduction From Baseline in SCORing Atopic Dermatitis (SCORAD) Score at Week 12
timeFrame: Baseline to Week 12 (one week after last dose)
measure: Percent Reduction From Baseline in Pruritus Numerical Rating Scale (NRS) at Week 12
timeFrame: Baseline to Week 12 (one week after last dose)
measure: Percentage of Subjects With Investigator Global Assessment (IGA) Score of "0" or "1" (Clear or Almost Clear) at Week 12
timeFrame: Week 12
measure: Percent Reduction From Baseline in Body Surface Area (BSA) at Week 12
timeFrame: Baseline to Week 12
Other outcomes:
Locations:
city: Markham
country: Canada
state: Ontario
city: Peterborough
country: Canada
state: Ontario
city: Waterloo
country: Canada
state: Ontario
city: Windsor
country: Canada
state: Ontario
city: Kutna Hora
country: Czechia
city: Prague
country: Czechia
city: Dresden
country: Germany
city: Frankfurt
country: Germany
city: Gera
country: Germany
city: Hamburg
country: Germany
city: Kiel
country: Germany
city: Luebeck
country: Germany
city: Muenster
country: Germany
city: Munich
country: Germany
city: Tuebingen
country: Germany
city: Kaposvar
country: Hungary
city: Miskolc
country: Hungary
city: Szeged
country: Hungary
city: Szolnok
country: Hungary
city: Katowice
country: Poland
city: Krakow
country: Poland
city: Lodz
country: Poland
city: Warszawa
country: Poland
city: Wroclaw
country: Poland
city: Manchester
country: United Kingdom
city: Sheffield
country: United Kingdom
Study officials:
affiliation: Regeneron Pharmaceuticals
name: Clinical Trial Management
role: STUDY_DIRECTOR
References:
citation: Blauvelt A, Guttman-Yassky E, Paller AS, Simpson EL, Cork MJ, Weisman J, Browning J, Soong W, Sun X, Chen Z, Kosloski MP, Kamal MA, Delevry D, Chuang CC, O'Malley JT, Bansal A. Long-Term Efficacy and Safety of Dupilumab in Adolescents with Moderate-to-Severe Atopic Dermatitis: Results Through Week 52 from a Phase III Open-Label Extension Trial (LIBERTY AD PED-OLE). Am J Clin Dermatol. 2022 May;23(3):365-383. doi: 10.1007/s40257-022-00683-2. Epub 2022 May 14.
citation: Cork MJ, Thaci D, Eichenfield LF, Arkwright PD, Hultsch T, Davis JD, Zhang Y, Zhu X, Chen Z, Li M, Ardeleanu M, Teper A, Akinlade B, Gadkari A, Eckert L, Kamal MA, Ruddy M, Graham NMH, Pirozzi G, Stahl N, DiCioccio AT, Bansal A. Dupilumab in adolescents with uncontrolled moderate-to-severe atopic dermatitis: results from a phase IIa open-label trial and subsequent phase III open-label extension. Br J Dermatol. 2020 Jan;182(1):85-96. doi: 10.1111/bjd.18476. Epub 2019 Oct 8.