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Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Severe Atopic Dermatitis (Liberty AD PRESCHOOL)

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ClinicalTrials.gov Identifier: NCT03346434
Recruitment Status : Recruiting
First Posted : November 17, 2017
Last Update Posted : June 14, 2019
Sponsor:
Collaborator:
Sanofi
Information provided by (Responsible Party):
Regeneron Pharmaceuticals

Brief Summary:
This study is a 2-part (parts A and B) phase 2/3 study to evaluate the safety, pharmacokinetics (PK) and efficacy of dupilumab in participants 6 months to less than 6 years of age with severe atopic dermatitis (AD).

Condition or disease Intervention/treatment Phase
Dermatitis, Atopic Drug: Dupilumab Drug: Matching placebo Phase 2 Phase 3

Detailed Description:
  1. Part A (open-label, single-ascending-dose, sequential cohort phase 2 study):

    • Primary objective is to characterize the safety and PK of dupilumab administered as a single dose in pediatric participants, 6 months to less than 6 years of age, with severe AD.
    • Secondary objective is to evaluate the efficacy and immunogenicity of a single dose of dupilumab in participants 6 months to less than 6 years of age with severe AD
  2. Part B (randomized, double-blind, parallel-group, placebo-controlled phase 3 study):

    • Primary objective is to demonstrate the efficacy of multiple doses of dupilumab over 16 weeks of treatment when administered concomitantly with topical corticosteroids (TCS) in pediatric participants, 6 months to less than 6 years of age, with severe AD.
    • Secondary objective is to assess the safety and immunogenicity of multiple doses of dupilumab over 16 weeks of treatment when administered concomitantly with TCS in participants 6 months to less than 6 years of age with severe AD

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 280 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description:

Part A: Single-ascending-dose cohorts staggered by age;

Part B: Parallel Group

Masking: None (Open Label)
Masking Description:

Part A: Open Label;

Part B: Masked, Randomized

Primary Purpose: Treatment
Official Title: A Phase 2/3 Study Investigating the Pharmacokinetics, Safety, and Efficacy of Dupilumab in Patients Aged ≥6 Months to <6 Years With Severe Atopic Dermatitis
Actual Study Start Date : November 30, 2017
Estimated Primary Completion Date : June 3, 2021
Estimated Study Completion Date : August 27, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Eczema
Drug Information available for: Dupilumab

Arm Intervention/treatment
Experimental: Part A (Open label Dupilumab): Age cohorts 1 & 2

Age cohort 1: ≥2 years old to <6 years old

Age cohort 2: ≥6 months to <2 years old

Drug: Dupilumab
Solution for injection, subcutaneous (SC)
Other Names:
  • DUPIXENT®
  • REGN668
  • SAR231893

Experimental: Part B (Double-blind): Dupilumab dose 1
The results of part A will be used to guide the selection of dose levels and dosing frequency for part B.
Drug: Dupilumab
Solution for injection, subcutaneous (SC)
Other Names:
  • DUPIXENT®
  • REGN668
  • SAR231893

Experimental: Part B (Double-blind): Dupilumab dose 2
The results of part A will be used to guide the selection of dose levels and dosing frequency for part B.
Drug: Dupilumab
Solution for injection, subcutaneous (SC)
Other Names:
  • DUPIXENT®
  • REGN668
  • SAR231893

Experimental: Part B (Double-Blind): Placebo Drug: Matching placebo
Solution for injection, subcutaneous (SC)




Primary Outcome Measures :
  1. Part A: Pharmacokinetic (PK) parameter - Maximum observed concentration (Cmax) [ Time Frame: Baseline to week 4 ]
    Peak dupilumab concentration in serum following single-dose administration

  2. Part A: PK parameter - Time to reach maximum observed concentration (Tmax) time [ Time Frame: Baseline to week 4 ]
  3. Part A: PK parameter - Area under the concentration-time curve (AUC) from time zero to the time of the last measurable concentration (AUClast) [ Time Frame: Baseline to week 4 ]
    AUC computed from time zero to the time of the last positive concentration

  4. Part A: Incidence of Treatment-Emergent Adverse Events (TEAEs) [Safety and Tolerability] [ Time Frame: Baseline to week 8 ]
    TEAEs include adverse events (AEs), serious adverse events (SAEs), deaths, and laboratory abnormalities

  5. Part B: Proportion of participants with an Investigator Global Assessment (IGA) score of 0 to 1 (on a 5-point scale) at week 16 [ Time Frame: At week 16 ]

Secondary Outcome Measures :
  1. Part A: Incidence of Serious Adverse Events (SAEs) [ Time Frame: Baseline to week 4 ]
  2. Part A: Incidence of severe TEAEs [ Time Frame: Baseline to week 4 ]
  3. Part A: Percent change in EASI score [ Time Frame: Baseline to week 4 ]
  4. Part A: Percent change in SCORing Atopic Dermatitis (SCORAD) score [ Time Frame: Baseline to week 4 ]
  5. Part A: Proportion of participants with an IGA score of either 0 or 1 (on a 5-point scale) [ Time Frame: At week 4 ]
  6. Part A: Determine immunogenicity titer [ Time Frame: Baseline to week 4 ]
    Assessed by measurement of anti-drug antibodies

  7. Part B: Proportion of participants with EASI-75 (≥75% improvement from baseline) [ Time Frame: At week 16 ]
  8. Part B: Percent change in EASI score [ Time Frame: Baseline to week 16 ]
  9. Part B: Reduction in pruritus (appropriate measure and endpoint definition in this patient population to be determined) [ Time Frame: At week 16 ]
  10. Part B: Proportion of participants with EASI-50 (≥50% improvement from baseline) [ Time Frame: At week 16 ]
  11. Part B: Proportion of participants with EASI-90 (≥90% improvement from baseline) [ Time Frame: At week 16 ]
  12. Part B: Change in percent Body Surface Area (BSA) affected by AD [ Time Frame: Baseline to week 16 ]
  13. Part B: Percent change in SCORAD [ Time Frame: Baseline to week 16 ]
  14. Part B: Change in Children's Dermatology Life Quality Index (CDLQI) for participants ≥4 years of age [ Time Frame: Baseline to week 16 ]
  15. Part B: Change in Infants' Dermatology Quality of Life Index (IDQOL) for participants <4 years of age [ Time Frame: Baseline to week 16 ]
  16. Part B: Change in Dermatitis Family Index (DFI) [ Time Frame: Baseline to week 16 ]
  17. Part B: Change in Patient Oriented Eczema Measure (POEM) [ Time Frame: Baseline to week 16 ]
  18. Part B: Topical treatment for AD - proportion of medication-free days [ Time Frame: Baseline to week 16 ]
  19. Part B: Mean weekly dose of topical corticosteroids (TCS) [ Time Frame: Baseline to week 16 ]
  20. Part B: Mean of caregiver missed workdays [ Time Frame: Baseline to week 16 ]
  21. Part B: Incidence of skin infection TEAEs [ Time Frame: Baseline to week 16 ]
  22. Part B: Incidence of SAEs through week 16 [ Time Frame: Baseline to week 16 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria

  1. Diagnosis of AD according to the American Academy of Dermatology consensus criteria at the screening visit
  2. Participants with documented recent history (within 6 months before the screening visit) of inadequate response to topical AD medication(s)
  3. IGA = 4 at screening and baseline visits
  4. EASI ≥21 at screening and baseline visits
  5. Body Surface Area (BSA) ≥15% at screening and baseline visits

Key Exclusion Criteria

  1. Participation in a prior dupilumab clinical study
  2. History of important side effects of medium potency topical corticosteroids (only applicable for part B of the study)
  3. Having used immunosuppressive/immunomodulating drugs within 4 weeks before the baseline visit
  4. Treatment with a live (attenuated) vaccine within 4 weeks before the baseline visit
  5. Active chronic or acute infection requiring treatment with systemic antibiotics, antivirals, antiprotozoals, or antifungals within 2 weeks before the baseline visit.
  6. Known history of human immunodeficiency virus (HIV) infection or HIV seropositivity at the screening visit
  7. History of malignancy at any time before the baseline visit
  8. Diagnosed active endoparasitic infections or at high risk of these infections
  9. Severe concomitant illness(es) that, in the investigator's judgment, would adversely affect the patient's participation in the study

Note: Other protocol defined Inclusion/ Exclusion criteria apply


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03346434


Contacts
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Contact: Clinical Trials Administrator 844-734-6643 clinicaltrials@regeneron.com

  Hide Study Locations
Locations
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United States, Alabama
Regeneron Investigational Site Recruiting
Birmingham, Alabama, United States, 35209
United States, California
Regeneron Investigational site Recruiting
Los Angeles, California, United States, 90027
Regeneron Investigational site Recruiting
Orange, California, United States, 92868
Regeneron Investigational Site Recruiting
Palo Alto, California, United States, 94304
Regeneron Investigational Site Recruiting
San Diego, California, United States, 92123
United States, Florida
Regeneron Investigational Site Recruiting
Coral Gables, Florida, United States, 33146
Regeneron Investigational Site Recruiting
Tampa, Florida, United States, 33612-3807
United States, Georgia
Regeneron Investigational Site Recruiting
Columbus, Georgia, United States, 31904
Regeneron Investigational Site Recruiting
Macon, Georgia, United States, 31217
United States, Illinois
Regeneron Investigational Site Recruiting
Chicago, Illinois, United States, 60611
United States, Massachusetts
Regeneron Investigational Site Recruiting
Boston, Massachusetts, United States, 02115
United States, Michigan
Regeneron Investigational Site Recruiting
Ann Arbor, Michigan, United States, 48109-5314
United States, Minnesota
Regeneron Investigational Site Recruiting
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Regeneron Investigational Site Recruiting
Saint Louis, Missouri, United States, 63104-1003
United States, New Hampshire
Regeneron Investigational Site Recruiting
Lebanon, New Hampshire, United States, 03756
United States, New York
Regeneron Investigational Site Recruiting
Forest Hills, New York, United States, 113756
United States, Oregon
Regeneron Investigational Site Recruiting
Portland, Oregon, United States, 97239
United States, South Carolina
Regeneron Investigational Site Recruiting
Charleston, South Carolina, United States, 29425
United States, Texas
Regeneron Investigational Site Recruiting
San Antonio, Texas, United States, 78218
United States, Virginia
Regeneron Investigational Site Recruiting
Norfolk, Virginia, United States, 23502
Germany
Regeneron Investigational site Recruiting
Muenster, North Rhine-Westphal, Germany, 48149
Regeneron Investigational Site Recruiting
Dresden, Sachsen, Germany, 01307
Regeneron Investigational site Recruiting
Frankfurt/ Main, Germany, 60590
Regeneron Investigational site Recruiting
Hamburg, Germany, 22149
Regeneron Investigational site Recruiting
Muenchen, Germany, 80802
United Kingdom
Regeneron Investigational Site Recruiting
Manchester, Lancashire, United Kingdom, M13 9WL
Regeneron Investigational Site Recruiting
Sheffield, South Yorkshire, United Kingdom, S10 2TH
Regeneron Investigational Site Recruiting
Newcastle, United Kingdom, NEB 4LP
Sponsors and Collaborators
Regeneron Pharmaceuticals
Sanofi
Investigators
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Study Director: Clinical Trial Management Regeneron Pharmaceuticals

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Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03346434     History of Changes
Other Study ID Numbers: R668-AD-1539
2016-000955-28 ( EudraCT Number )
First Posted: November 17, 2017    Key Record Dates
Last Update Posted: June 14, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Regeneron Pharmaceuticals:
Eczema
Additional relevant MeSH terms:
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Dermatitis, Atopic
Dermatitis
Skin Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases, Eczematous
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs