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Atopic Dermatitis (Dupilumab Study)

Study Description

Brief Summary

The primary objective of the study is to assess the long-term safety of dupilumab in pediatric participants with AD.

The secondary objectives of the study are:

  • To assess the long-term efficacy of dupilumab in pediatric participants with AD
  • To assess the trough concentrations of functional dupilumab in serum and the immunogenicity in pediatric participants with AD after re-treatment with dupilumab

Optional Pre-filled Pen (PFP) Sub-Study in pediatric patients ≥2 to <12 years of age with AD

Co-Primary Objectives are:

  • To evaluate the pharmacokinetic (PK) of dupilumab PFPs
  • To evaluate the safety of dupilumab PFPs

Secondary Objective is:

  • To evaluate the immunogenicity of dupilumab PFPs

Condition or Disease

Atopic Dermatitis

Study Design

Study Type: Interventional  (Clinical Trial)
Estimated Enrollment: 800 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Dupilumab in Patients ≥6 Months to <18 Years of Age With Atopic Dermatitis
Actual Study Start Date: October 15, 2015
Estimated Primary Completion Date: November 3, 2026
Estimated Study Completion Date: November 3, 2026

Outcome Measures

Primary Outcome Measures

  1. Rate of treatment-emergent adverse events (TEAEs) per participant year from baseline through the last study visit [ Time Frame: Baseline up to week 272 ]
  2. Number of participants with at least one TEAE per participant year from baseline through the last study visit [ Time Frame: Baseline up to week 272 ]
  3. OPTIONAL SUB-STUDY: Pharmacokinetic (PK) of dupilumab: Peak concentration (Cmax) [ Time Frame: Up to week 16 ]
    Peak serum concentration after multiple doses of dupilumab administered using the PFP (test) relative to the prefilled syringe (reference)
  4. OPTIONAL SUB-STUDY: PK of dupilumab: Trough concentration (Ctrough) [ Time Frame: Up to week 16 ]
    Drug concentration in serum after multiple doses of dupilumab administered using the PFP (test) relative to the prefilled syringe (reference)
  5. OPTIONAL SUB-STUDY: Incidence of TEAEs during the 12-week PFP treatment period and during entire sub-study [ Time Frame: Up to week 16 ]
  6. OPTIONAL SUB-STUDY: Incidence of SAEs during the 12-week PFP treatment period and during entire sub-study [ Time Frame: Up to week 16 ]

Secondary Outcome Measures

  1. Number of treatment-emergent serious adverse events (SAEs) from baseline through the last study visit [ Time Frame: Baseline up to week 272 ]
  2. Incidence of TEAEs of special interest from baseline through the last study visit [ Time Frame: Baseline up to week 272 ]
  3. Proportion of participants with an Investigator Global Assessment (IGA) score of 0 or 1 (clear or almost clear) at all in-clinic visits post-baseline [ Time Frame: Baseline up to week 272 ]
  4. Proportion of participants with Eczema Area and Severity Index (EASI)-75 (≥75% reduction in EASI from baseline of parent study) response at all in-clinic visits post-baseline [ Time Frame: Baseline up to week 272 ]
  5. Change from baseline in EASI score at all in-clinic visits post-baseline [ Time Frame: Baseline up to week 272 ]
  6. Percent change from baseline in EASI at all in-clinic visits post-baseline [ Time Frame: Baseline up to week 272 ]
  7. Change from baseline in Body Surface Area (BSA) affected by AD (BSA) at all in-clinic visits post-baseline [ Time Frame: Baseline up to week 272 ]
  8. Percent change from baseline in SCORing Atopic Dermatitis (SCORAD) at all in-clinic visits post-baseline [ Time Frame: Baseline up to week 272 ]
  9. Change from baseline in Children’s Dermatology Life Quality Index (CDLQI) for participants ≥4 years of age at all in-clinic visits post-baseline in which the assessments are planned to be performed [ Time Frame: Baseline up to week 272 ]
  10. Change from baseline in Infants’ Dermatology Quality of Life Index (IDQOL) for participants <4 years of age at all in-clinic visits post-baseline in which the assessments are planned to be performed [ Time Frame: Baseline up to week 272 ]
  11. Proportion of responders (defined as participants with IGA 0 or 1) who maintain IGA 0 or 1 during at least 75% of the subsequent* visits during the treatment period [ Time Frame: Baseline to week 260 ]
    *Subsequent refers to the visits following the first visit at which IGA 0 or 1 is achieved.
  12. For responders (defined as participants with IGA 0 or 1), median percentage of subsequent* visits during the treatment period, at which IGA 0 or 1 is maintained [ Time Frame: Baseline to week 260 ]
    *Subsequent refers to the visits following the first visit at which IGA 0 or 1 is achieved.
  13. Number of AD flares during the study [ Time Frame: Baseline to week 272 ]
    AD flares are defined as worsening of the disease that requires escalation/intensification of AD treatment
  14. Annualize event rate of AD flares during the study [ Time Frame: Baseline to week 272 ]
    AD flares are defined as worsening of the disease that requires escalation/intensification of AD treatment
  15. Proportion of participants with at least one flare during the study [ Time Frame: Baseline to week 272 ]
    AD flares are defined as worsening of the disease that requires escalation/intensification of AD treatment
  16. Proportion of well-controlled weeks [ Time Frame: Baseline to week 272 ]
    Well-controlled weeks are those for which participants or parents/caregivers answer “Yes” AND during which no rescue treatments were administered
  17. OPTIONAL SUB-STUDY: Incidence and titer of treatment-emergent anti-drug antibodies (ADA) (PFP Sub-Study) [ Time Frame: Up to 16 weeks ]

Eligibility Criteria

Ages Eligible for Study:   6 Months to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers: No

Inclusion Criteria

  • Participated in a prior dupilumab study in pediatric participants 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
  • PFP Sub-Study Only:
  • Age ≥2 to <12 years at time of screening
  • Body weight ≥5 kg and <60 kg at time of screening
  • Must have received the same dupilumab dose regimen to be used in the PFP sub-study during the previous 12 weeks in the main OLE study using the prefilled syringe, as defined in the protocol

Exclusion Criteria

  • Participants who, during their participation in a prior dupilumab study developed an adverse event (AE) or serious adverse event (SAE) deemed related to study drug which could indicate that continued treatment with study drug may present an unreasonable risk for the patient
  • Participants, who during the participation in a prior Dupilumab study, developed an AE that was deemed related to study drug and led to study treatment discontinuation, which in the opinion of the investigator or medical monitor could indicate that continued treatment with study drug may present an unreasonable risk for the patient
  • 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
  • Having used immunosuppressive/immunomodulating drugs within 4 weeks before the baseline visit
  • Treatment with a live (attenuated) vaccine within 4 weeks before the baseline visit
  • Diagnosed active endoparasitic infections or at high risk of these infections
  • Severe concomitant illness(es) that, in the investigator’s judgment, would adversely affect the participant’s participation in the study
  • PFP Sub-study Only:
  • Poor compliance as defined by having missed 1 or more of the planned last 3 injections in the main OLE study prior to entering the sub-study
  • Switched dupilumab doses within the past 12 weeks
  • Meet criteria for temporary/permanent discontinuation of study drug at time of screening into PFP sub-study, as defined in the protocol.

Note: Other protocol defined Inclusion / Exclusion criteria may apply

ADDITIONAL DETAILS