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Study to Investigate the Efficacy and Safety of Dupilumab in Pediatric Patients With Active Eosinophilic Esophagitis (EoE) (EoE KIDS)

Primary Purpose

Eosinophilic Esophagitis (EoE)

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Dupilumab
Matching Placebo
Sponsored by
Regeneron Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Eosinophilic Esophagitis (EoE)

Eligibility Criteria

1 Year - 11 Years (Child)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  1. A documented diagnosis of eosinophilic esophagitis (EoE)
  2. Baseline endoscopic biopsies with a demonstration on central reading of intraepithelial eosinophilic infiltration

Key Exclusion Criteria:

  1. Body weight <5 kg or ≥60 kg at screening
  2. Other causes of esophageal eosinophilia
  3. Active Helicobacter pylori
  4. History of Crohn's disease, ulcerative colitis, celiac disease, or prior esophageal surgery
  5. Any esophageal stricture unable to be passed with a standard, diagnostic, upper endoscope or any critical esophageal stricture that requires dilation at screening
  6. Treatment with swallowed topical corticosteroids within 8 weeks prior to baseline standard of care endoscopy
  7. History of bleeding disorders or esophageal varices that, in the opinion of the investigator, would put the patient at undue risk for significant complications from an endoscopy procedure
  8. Active parasitic infection or suspected parasitic infection
  9. Known or suspected immunodeficiency disorder

Key Exclusion for Patients Re-Entering the Study (for Entry into Part C, as defined in protocol):

  1. Patients who are ≥12 years old, weigh ≥40 kg (or minimum weight for which dupilumab is approved for EoE), and dupilumab is commercially available for the treatment of EoE in their country
  2. Patients who, during their previous participation in this clinical trial, developed an SAE and/or AE deemed related to dupilumab, which in the opinion of the investigator or of the medical monitor could indicate that continued treatment with dupilumab may present an unreasonable risk for the patient
  3. Patients who did not undergo endoscopy with biopsies at week 16 and/or week 52 or prior to receiving rescue treatment Note: If the endoscopy with biopsies could not occur due to COVID-19 restrictions and rescue treatment was needed to be initiated without delay, these patients will be eligible to participate in Part C
  4. Patients who became pregnant during their previous participation in this dupilumab clinical trial
  5. Patients who, during their previous participation in this trial, were prematurely withdrawn because of a protocol violation, poor compliance, or inability to complete required study assessments

NOTE: Other protocol defined inclusion/exclusion criteria apply

Sites / Locations

  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site
  • Regeneron Study Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

Part A - High Dose

Part A - Low Dose

Part B - High Dose

Part B - Low Dose

Part C - High Dose

Arm Description

Part A consists of a 16-week double-blind treatment period. Patients will be randomized to receive dupilumab or placebo subcutaneous (SC) administration at tiered dosing regimens based on body weight

Part A consists of a 16-week double-blind treatment period. Patients will be randomized to receive dupilumab or placebo subcutaneous (SC) administration at tiered dosing regimens based on body weight

Part B consists of a 36-week extended active treatment period. All patients to receive subcutaneous (SC) administration at tiered dosing regimens based on body weight

Part B consists of a 36-week extended active treatment period. All patients to receive subcutaneous (SC) administration at tiered dosing regimens based on body weight

Part C consists of up to 108-week open-label extension period. All patients will receive higher exposure dupilumab subcutaneous (SC) administration at tiered dosing regimens based on body weight. No matching placebo administered in Part C.

Outcomes

Primary Outcome Measures

Proportion of patients achieving peak esophageal intraepithelial eosinophil count ≤6 eos/hpf (400×)

Secondary Outcome Measures

Proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf
Part A
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf
Part B
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf
Part C
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf
Part C
Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf)
Part A
Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf)
Part B
Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf)
Part C
Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf)
Part C
Absolute change in mean eosinophilic esophagitis (EoE) Histology Scoring System (EoE-HSS)
Part A The EoEHSS assesses the severity (grade) and extent (stage) of abnormalities using a 4-point scale (0 normal; 3 maximum change).
Absolute change in mean EoE-HSS
Part B The EoEHSS as stated above.
Absolute change in mean EoE-HSS
Part C The EoEHSS as stated above.
Absolute change in mean EoE-HSS
Part C The EoEHSS as stated above.
Absolute change in Eosinophilic Esophagitis-Endoscopic Reference (EoE EREFS)
Part A EoE esophageal characteristics will be analyzed based on the EoE-EREFS, a scoring system for inflammatory and remodeling features of disease using both overall scores and scores for each individual characteristic. The proximal and distal esophageal regions will be scored separately; The score for each region ranges from 0 to 9 and the overall score ranges from 0 to 18.
Absolute change in EoE-EREFS
Part B EoE esophageal characteristics will be analyzed based on the EoE-EREFS, as stated above.
Absolute change in EoE-EREFS
Part C EoE esophageal characteristics will be analyzed based on the EoE-EREFS, as stated above.
Absolute change in EoE-EREFS
Part C EoE esophageal characteristics will be analyzed based on the EoE-EREFS, as stated above.
Change in the type 2 inflammation transcriptional signature
Part A
Change in the type 2 inflammation transcriptional signature
Part B
Change in the proportion of days with 1 or more EoE signs as measured by the Pediatric EoE Sign/Symptom Questionnaire- Caregiver version (PESQ-C)
Part A: For patients aged ≥1 to <12 years The PESQ-C is an observer-reported outcome measure intended to be completed independently by caregivers. The PESQ-C will measure occurrence of signs of EoE and will be completed once daily via an electronic diary. Data from a 14-day period preceding the baseline visit and a 14-day period preceding the week 16 visit will be used to calculate the proportion of days or total time segments within a day (night, morning, afternoon, evening) with 1 or more EoE signs.
Change in the proportion of days with 1 or more EoE signs as measured by the PESQ-C
Part B: For patients aged ≥1 to <12 years The PESQ-C as stated above.
Number of sign-free days during the 14-day period preceding week 16 as measured by the PESQ-C
Part A: For patients aged ≥1 to <12 years The PESQ-C as stated above.
Number of sign-free days during the 14-day period preceding week 52 as measured by the PESQ-C
Part B: For patients aged ≥1 to <12 years The PESQ-C as stated above.
Change in the proportion of total segments within a day with 1 or more EoE signs as measured by the PESQ-C
Part A: For patients aged ≥1 to <12 years The PESQ-C as stated above.
Change in the proportion of total segments within a day with 1 or more EoE signs as measured by the PESQ-C
Part B: For patients aged ≥1 to <12 years The PESQ-C as stated above.
Change in the proportion of days with 1 or more EoE symptoms as measured by the Pediatric EoE Sign/Symptom Questionnaire- Patient version (PESQ-P)
Part A: For patients aged ≥8 to <12 years The PESQ-P is a patient-reported outcome measure intended to be completed independently by EoE patients. The PESQ-P will measure occurrence and severity of EoE symptoms and will be completed once daily via an electronic diary. Data from a 14-day period preceding the baseline visit and a 14-day period preceding the week 16 visit will be used to calculate the proportion of days or total time segments within a day (night, morning, afternoon, evening) with 1 or more EoE symptoms.
Change in the proportion of days with 1 or more EoE symptoms as measured by the PESQ-P
Part B: For patients aged ≥8 to <12 years The PESQ-P as stated above.
Number of symptom-free days during the 14-day period preceding week 16 as measured by the PESQ-P
Part A: For patients aged ≥8 to <12 years The PESQ-P as stated above.
Number of symptom-free days during the 14-day period preceding week 52 as measured by the PESQ-P
Part B: For patients aged ≥8 to <12 years The PESQ-P as stated above.
Change in the proportion of total segments within a day with 1 or more EoE symptoms as measured by the PESQ-P
Part A: For patients aged ≥8 to <12 years The PESQ-P as stated above.
Change in the proportion of total segments within a day with 1 or more EoE symptoms as measured by the PESQ-P
Part B: For patients aged ≥8 to <12 years The PESQ-P as stated above.
Change in total score as measured by the PEESSv2.0-caregiver version questionnaire
Part A: For patients aged ≥1 to <12 years The PEESSv2.0-caregiver version assesses the frequency and severity of EoE symptoms among pediatric patients (Franciosi, 2011). The PEESSv2.0 consists of 20 items and has a one-month recall period. The total score ranges from 0 to 100; higher scores indicate greater symptom burden of among pediatric EoE patients
Change in total score as measured by the PEESSv2.0- caregiver version questionnaire
Part C The PEESSv2.0-caregiver version as stated above.
Normalized Enrichment Scores (NES) for the relative change in the EoE diagnostic panel (EDP) transcriptome signature
Part A NES reflects the degree to which the activity level of a set of transcripts is overrepresented at the extremes (top or bottom) of the entire ranked list of transcripts within a sample and is normalized by accounting for the number of transcripts in the set (Barbie, 2009)(Subramanian, 2005). NES scores will be calculated for each transcriptome signature for each sample for statistical analyses.
NES for the relative change in the EDP transcriptome signature
Part B NES as stated above.
NES for the relative change in the EDP transcriptome signature
Part C NES as stated above.
NES for the relative change in the EDP transcriptome signature
Part C NES as stated above.
NES for the relative change in the type 2 inflammation transcriptome signature
Part A NES as stated above.
NES for the relative change in the type 2 inflammation transcriptome signature
Part B NES as stated above.
NES for the relative change in the type 2 inflammation transcriptome signature
Part C NES as stated above.
Change in body weight for age percentile
Part B
Change in body weight for age percentile
Part C
Change in body mass index for age z-score
Part B: For patients ≥2 years of age
Change in body mass index for age z-score
Part C: For patients ≥2 years of age
Change in weight for age z-score
Part B
Change in weight for age z-score
Part C
Change in weight for height z-score
Part B
Change in weight for height z-score
Part C
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤6 eos/hpf (400×)
Part C
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤6 eos/hpf (400×)
Part C
Proportion of patients (with food elimination diet regimens at baseline) that have a re-introduction of a previously eliminated food group
Part C
Proportion of patients (with food elimination diet regimens at baseline) that have a re-introduction of a previously eliminated food group
Part C
Incidence of treatment-emergent adverse events (TEAEs)
Part A
Incidence of TEAEs
Part B
Incidence of TEAEs
Part C
Incidence of treatment-emergent serious adverse events (SAEs)
Part A
Incidence of treatment-emergent SAEs
Part B
Incidence of treatment-emergent SAEs
Part C
Incidence of treatment-emergent adverse events of special interest (AESIs)
Part A
Incidence of treatment-emergent AESIs
Part B
Incidence of treatment-emergent AESIs
Part C
Incidence of TEAEs leading to permanent discontinuation of study treatment
Part A
Incidence of TEAEs leading to permanent discontinuation of study treatment
Part B
Incidence of TEAEs leading to permanent discontinuation of study treatment
Part C
Incidence of treatment-emergent Anti-drug antibody (ADA) responses and titer
Part A
Incidence of treatment-emergent ADA responses and titer
Part B
Incidence of treatment-emergent ADA responses and titer
Part C
Concentration of functional dupilumab in serum

Full Information

First Posted
May 7, 2020
Last Updated
June 1, 2023
Sponsor
Regeneron Pharmaceuticals
Collaborators
Sanofi
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1. Study Identification

Unique Protocol Identification Number
NCT04394351
Brief Title
Study to Investigate the Efficacy and Safety of Dupilumab in Pediatric Patients With Active Eosinophilic Esophagitis (EoE)
Acronym
EoE KIDS
Official Title
A Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Dupilumab in Pediatric Patients With Active Eosinophilic Esophagitis
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 1, 2020 (Actual)
Primary Completion Date
June 2, 2022 (Actual)
Study Completion Date
July 7, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Regeneron Pharmaceuticals
Collaborators
Sanofi

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The Primary objective is to demonstrate the efficacy of dupilumab treatment compared with placebo in pediatric patients with active eosinophilic esophagitis (EoE) based on histologic improvement meeting validated histologic criteria. The Secondary objectives are: To demonstrate the efficacy of dupilumab compared to placebo in pediatric patients with active EoE after 16 weeks of treatment as assessed by endoscopic visual measurements of disease activity using the Eosinophilic Esophagitis-Endoscopic Reference Score (EoE-EREFS) and histologic abnormalities as measured by the EoE Histology Scoring System (EoE-HSS) To evaluate the safety, tolerability, and immunogenicity of dupilumab treatment for up to 16 weeks in pediatric patients with active EoE To evaluate the effects of dupilumab on transcriptomic signatures associated with EoE and type 2 inflammation To study the effects of dupilumab on the type 2 inflammation gene expression signature To evaluate the concentration-time profile of functional dupilumab in serum in this population To assess efficacy of long-term (up to 160 weeks) dupilumab treatment To assess the impact of dupilumab treatment on changes in weight and growth during the extended active period and open-label extension period of the study To assess safety, tolerability, and immunogenicity of long-term (up to 160 weeks) dupilumab treatment To evaluate the impact of dupilumab treatment on EoE signs and symptoms
Detailed Description
This is a 3-part study: Part A: Double-blind 16-week treatment period Part B: 36-week extended active treatment period Part C: Up to108 weeks open-label extension period

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Eosinophilic Esophagitis (EoE)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
102 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Part A - High Dose
Arm Type
Experimental
Arm Description
Part A consists of a 16-week double-blind treatment period. Patients will be randomized to receive dupilumab or placebo subcutaneous (SC) administration at tiered dosing regimens based on body weight
Arm Title
Part A - Low Dose
Arm Type
Experimental
Arm Description
Part A consists of a 16-week double-blind treatment period. Patients will be randomized to receive dupilumab or placebo subcutaneous (SC) administration at tiered dosing regimens based on body weight
Arm Title
Part B - High Dose
Arm Type
Experimental
Arm Description
Part B consists of a 36-week extended active treatment period. All patients to receive subcutaneous (SC) administration at tiered dosing regimens based on body weight
Arm Title
Part B - Low Dose
Arm Type
Experimental
Arm Description
Part B consists of a 36-week extended active treatment period. All patients to receive subcutaneous (SC) administration at tiered dosing regimens based on body weight
Arm Title
Part C - High Dose
Arm Type
Experimental
Arm Description
Part C consists of up to 108-week open-label extension period. All patients will receive higher exposure dupilumab subcutaneous (SC) administration at tiered dosing regimens based on body weight. No matching placebo administered in Part C.
Intervention Type
Drug
Intervention Name(s)
Dupilumab
Other Intervention Name(s)
•DUPIXENT, •REGN668, •SAR231893
Intervention Description
Single-use, prefilled syringe
Intervention Type
Drug
Intervention Name(s)
Matching Placebo
Intervention Description
Matching formulation and regimen (depending on the weight tier) as dupilumab without the active substance
Primary Outcome Measure Information:
Title
Proportion of patients achieving peak esophageal intraepithelial eosinophil count ≤6 eos/hpf (400×)
Time Frame
At Week 16
Secondary Outcome Measure Information:
Title
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf
Description
Part A
Time Frame
At Week 16
Title
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf
Description
Part B
Time Frame
At Week 52
Title
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf
Description
Part C
Time Frame
At Week 100
Title
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf
Description
Part C
Time Frame
At Week 160
Title
Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf)
Description
Part A
Time Frame
Baseline to Week 16
Title
Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf)
Description
Part B
Time Frame
Baseline to Week 52
Title
Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf)
Description
Part C
Time Frame
Baseline to Week 100
Title
Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf)
Description
Part C
Time Frame
Baseline to Week 160
Title
Absolute change in mean eosinophilic esophagitis (EoE) Histology Scoring System (EoE-HSS)
Description
Part A The EoEHSS assesses the severity (grade) and extent (stage) of abnormalities using a 4-point scale (0 normal; 3 maximum change).
Time Frame
Baseline to Week 16
Title
Absolute change in mean EoE-HSS
Description
Part B The EoEHSS as stated above.
Time Frame
Baseline to Week 52
Title
Absolute change in mean EoE-HSS
Description
Part C The EoEHSS as stated above.
Time Frame
Baseline to Week 100
Title
Absolute change in mean EoE-HSS
Description
Part C The EoEHSS as stated above.
Time Frame
Baseline to Week 160
Title
Absolute change in Eosinophilic Esophagitis-Endoscopic Reference (EoE EREFS)
Description
Part A EoE esophageal characteristics will be analyzed based on the EoE-EREFS, a scoring system for inflammatory and remodeling features of disease using both overall scores and scores for each individual characteristic. The proximal and distal esophageal regions will be scored separately; The score for each region ranges from 0 to 9 and the overall score ranges from 0 to 18.
Time Frame
Baseline to Week 16
Title
Absolute change in EoE-EREFS
Description
Part B EoE esophageal characteristics will be analyzed based on the EoE-EREFS, as stated above.
Time Frame
Baseline to Week 52
Title
Absolute change in EoE-EREFS
Description
Part C EoE esophageal characteristics will be analyzed based on the EoE-EREFS, as stated above.
Time Frame
Baseline to Week 100
Title
Absolute change in EoE-EREFS
Description
Part C EoE esophageal characteristics will be analyzed based on the EoE-EREFS, as stated above.
Time Frame
Baseline to Week 160
Title
Change in the type 2 inflammation transcriptional signature
Description
Part A
Time Frame
Baseline at Week 16
Title
Change in the type 2 inflammation transcriptional signature
Description
Part B
Time Frame
Baseline at Week 52
Title
Change in the proportion of days with 1 or more EoE signs as measured by the Pediatric EoE Sign/Symptom Questionnaire- Caregiver version (PESQ-C)
Description
Part A: For patients aged ≥1 to <12 years The PESQ-C is an observer-reported outcome measure intended to be completed independently by caregivers. The PESQ-C will measure occurrence of signs of EoE and will be completed once daily via an electronic diary. Data from a 14-day period preceding the baseline visit and a 14-day period preceding the week 16 visit will be used to calculate the proportion of days or total time segments within a day (night, morning, afternoon, evening) with 1 or more EoE signs.
Time Frame
Baseline to Week 16
Title
Change in the proportion of days with 1 or more EoE signs as measured by the PESQ-C
Description
Part B: For patients aged ≥1 to <12 years The PESQ-C as stated above.
Time Frame
Baseline to Week 52
Title
Number of sign-free days during the 14-day period preceding week 16 as measured by the PESQ-C
Description
Part A: For patients aged ≥1 to <12 years The PESQ-C as stated above.
Time Frame
Up to Week 16
Title
Number of sign-free days during the 14-day period preceding week 52 as measured by the PESQ-C
Description
Part B: For patients aged ≥1 to <12 years The PESQ-C as stated above.
Time Frame
Up to Week 52
Title
Change in the proportion of total segments within a day with 1 or more EoE signs as measured by the PESQ-C
Description
Part A: For patients aged ≥1 to <12 years The PESQ-C as stated above.
Time Frame
Baseline to Week 16
Title
Change in the proportion of total segments within a day with 1 or more EoE signs as measured by the PESQ-C
Description
Part B: For patients aged ≥1 to <12 years The PESQ-C as stated above.
Time Frame
Baseline to Week 52
Title
Change in the proportion of days with 1 or more EoE symptoms as measured by the Pediatric EoE Sign/Symptom Questionnaire- Patient version (PESQ-P)
Description
Part A: For patients aged ≥8 to <12 years The PESQ-P is a patient-reported outcome measure intended to be completed independently by EoE patients. The PESQ-P will measure occurrence and severity of EoE symptoms and will be completed once daily via an electronic diary. Data from a 14-day period preceding the baseline visit and a 14-day period preceding the week 16 visit will be used to calculate the proportion of days or total time segments within a day (night, morning, afternoon, evening) with 1 or more EoE symptoms.
Time Frame
Baseline to Week 16
Title
Change in the proportion of days with 1 or more EoE symptoms as measured by the PESQ-P
Description
Part B: For patients aged ≥8 to <12 years The PESQ-P as stated above.
Time Frame
Baseline to Week 52
Title
Number of symptom-free days during the 14-day period preceding week 16 as measured by the PESQ-P
Description
Part A: For patients aged ≥8 to <12 years The PESQ-P as stated above.
Time Frame
Up to Week 16
Title
Number of symptom-free days during the 14-day period preceding week 52 as measured by the PESQ-P
Description
Part B: For patients aged ≥8 to <12 years The PESQ-P as stated above.
Time Frame
Up to Week 52
Title
Change in the proportion of total segments within a day with 1 or more EoE symptoms as measured by the PESQ-P
Description
Part A: For patients aged ≥8 to <12 years The PESQ-P as stated above.
Time Frame
Baseline to Week 16
Title
Change in the proportion of total segments within a day with 1 or more EoE symptoms as measured by the PESQ-P
Description
Part B: For patients aged ≥8 to <12 years The PESQ-P as stated above.
Time Frame
Baseline to Week 52
Title
Change in total score as measured by the PEESSv2.0-caregiver version questionnaire
Description
Part A: For patients aged ≥1 to <12 years The PEESSv2.0-caregiver version assesses the frequency and severity of EoE symptoms among pediatric patients (Franciosi, 2011). The PEESSv2.0 consists of 20 items and has a one-month recall period. The total score ranges from 0 to 100; higher scores indicate greater symptom burden of among pediatric EoE patients
Time Frame
Baseline to Week 16
Title
Change in total score as measured by the PEESSv2.0- caregiver version questionnaire
Description
Part C The PEESSv2.0-caregiver version as stated above.
Time Frame
Baseline to Week 160
Title
Normalized Enrichment Scores (NES) for the relative change in the EoE diagnostic panel (EDP) transcriptome signature
Description
Part A NES reflects the degree to which the activity level of a set of transcripts is overrepresented at the extremes (top or bottom) of the entire ranked list of transcripts within a sample and is normalized by accounting for the number of transcripts in the set (Barbie, 2009)(Subramanian, 2005). NES scores will be calculated for each transcriptome signature for each sample for statistical analyses.
Time Frame
Baseline to Week 16
Title
NES for the relative change in the EDP transcriptome signature
Description
Part B NES as stated above.
Time Frame
Baseline to Week 52
Title
NES for the relative change in the EDP transcriptome signature
Description
Part C NES as stated above.
Time Frame
Baseline to Week 100
Title
NES for the relative change in the EDP transcriptome signature
Description
Part C NES as stated above.
Time Frame
Baseline to Week 160
Title
NES for the relative change in the type 2 inflammation transcriptome signature
Description
Part A NES as stated above.
Time Frame
Baseline to Week 16
Title
NES for the relative change in the type 2 inflammation transcriptome signature
Description
Part B NES as stated above.
Time Frame
Baseline to Week 52
Title
NES for the relative change in the type 2 inflammation transcriptome signature
Description
Part C NES as stated above.
Time Frame
Baseline to Week 100
Title
Change in body weight for age percentile
Description
Part B
Time Frame
Baseline at Week 52
Title
Change in body weight for age percentile
Description
Part C
Time Frame
Baseline up to Week 160
Title
Change in body mass index for age z-score
Description
Part B: For patients ≥2 years of age
Time Frame
Baseline to Week 52
Title
Change in body mass index for age z-score
Description
Part C: For patients ≥2 years of age
Time Frame
Baseline to up to Week 160
Title
Change in weight for age z-score
Description
Part B
Time Frame
Baseline to Week 52
Title
Change in weight for age z-score
Description
Part C
Time Frame
Baseline up to Week 160
Title
Change in weight for height z-score
Description
Part B
Time Frame
Baseline to Week 52
Title
Change in weight for height z-score
Description
Part C
Time Frame
Baseline up to Week 160
Title
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤6 eos/hpf (400×)
Description
Part C
Time Frame
At Week 100
Title
Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤6 eos/hpf (400×)
Description
Part C
Time Frame
At Week 160
Title
Proportion of patients (with food elimination diet regimens at baseline) that have a re-introduction of a previously eliminated food group
Description
Part C
Time Frame
Baseline by Week 100
Title
Proportion of patients (with food elimination diet regimens at baseline) that have a re-introduction of a previously eliminated food group
Description
Part C
Time Frame
Baseline by Week 160
Title
Incidence of treatment-emergent adverse events (TEAEs)
Description
Part A
Time Frame
Up to Week 16
Title
Incidence of TEAEs
Description
Part B
Time Frame
Up to Week 52
Title
Incidence of TEAEs
Description
Part C
Time Frame
Up to Week 160
Title
Incidence of treatment-emergent serious adverse events (SAEs)
Description
Part A
Time Frame
Up to Week 16
Title
Incidence of treatment-emergent SAEs
Description
Part B
Time Frame
Up to Week 52
Title
Incidence of treatment-emergent SAEs
Description
Part C
Time Frame
Up to Week 160
Title
Incidence of treatment-emergent adverse events of special interest (AESIs)
Description
Part A
Time Frame
Up to Week 16
Title
Incidence of treatment-emergent AESIs
Description
Part B
Time Frame
Up to Week 52
Title
Incidence of treatment-emergent AESIs
Description
Part C
Time Frame
Up to Week 160
Title
Incidence of TEAEs leading to permanent discontinuation of study treatment
Description
Part A
Time Frame
Up to Week 16
Title
Incidence of TEAEs leading to permanent discontinuation of study treatment
Description
Part B
Time Frame
Up to Week 52
Title
Incidence of TEAEs leading to permanent discontinuation of study treatment
Description
Part C
Time Frame
Up to Week 160
Title
Incidence of treatment-emergent Anti-drug antibody (ADA) responses and titer
Description
Part A
Time Frame
Up to Week 16
Title
Incidence of treatment-emergent ADA responses and titer
Description
Part B
Time Frame
Up to Week 52
Title
Incidence of treatment-emergent ADA responses and titer
Description
Part C
Time Frame
Up to Week 160
Title
Concentration of functional dupilumab in serum
Time Frame
Baseline to end of study, Up to Week 160

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: A documented diagnosis of eosinophilic esophagitis (EoE) Baseline endoscopic biopsies with a demonstration on central reading of intraepithelial eosinophilic infiltration Key Exclusion Criteria: Body weight <5 kg or ≥60 kg at screening Other causes of esophageal eosinophilia Active Helicobacter pylori History of Crohn's disease, ulcerative colitis, celiac disease, or prior esophageal surgery Any esophageal stricture unable to be passed with a standard, diagnostic, upper endoscope or any critical esophageal stricture that requires dilation at screening Treatment with swallowed topical corticosteroids within 8 weeks prior to baseline standard of care endoscopy History of bleeding disorders or esophageal varices that, in the opinion of the investigator, would put the patient at undue risk for significant complications from an endoscopy procedure Active parasitic infection or suspected parasitic infection Known or suspected immunodeficiency disorder Key Exclusion for Patients Re-Entering the Study (for Entry into Part C, as defined in protocol): Patients who are ≥12 years old, weigh ≥40 kg (or minimum weight for which dupilumab is approved for EoE), and dupilumab is commercially available for the treatment of EoE in their country Patients who, during their previous participation in this clinical trial, developed an SAE and/or AE deemed related to dupilumab, which in the opinion of the investigator or of the medical monitor could indicate that continued treatment with dupilumab may present an unreasonable risk for the patient Patients who did not undergo endoscopy with biopsies at week 16 and/or week 52 or prior to receiving rescue treatment Note: If the endoscopy with biopsies could not occur due to COVID-19 restrictions and rescue treatment was needed to be initiated without delay, these patients will be eligible to participate in Part C Patients who became pregnant during their previous participation in this dupilumab clinical trial Patients who, during their previous participation in this trial, were prematurely withdrawn because of a protocol violation, poor compliance, or inability to complete required study assessments NOTE: Other protocol defined inclusion/exclusion criteria apply
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trial Management
Organizational Affiliation
Regeneron Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Regeneron Study Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Regeneron Study Site
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Regeneron Study Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Regeneron Study Site
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Regeneron Study Site
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Regeneron Study Site
City
Saint Petersburg
State/Province
Florida
ZIP/Postal Code
33701
Country
United States
Facility Name
Regeneron Study Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Regeneron Study Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Regeneron Study Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Regeneron Study Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Regeneron Study Site
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Regeneron Study Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02111
Country
United States
Facility Name
Regeneron Study Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Regeneron Study Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Regeneron Study Site
City
Lincoln
State/Province
Nebraska
ZIP/Postal Code
68510
Country
United States
Facility Name
Regeneron Study Site
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Regeneron Study Site
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Regeneron Study Site
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Regeneron Study Site
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Regeneron Study Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Regeneron Study Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Regeneron Study Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Regeneron Study Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75207
Country
United States
Facility Name
Regeneron Study Site
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Regeneron Study Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Regeneron Study Site
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Regeneron Study Site
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
All Individual Patient Data that underlie publicly available results will be considered for sharing
IPD Sharing Time Frame
Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.
IPD Sharing Access Criteria
Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).
IPD Sharing URL
https://vivli.org/

Learn more about this trial

Study to Investigate the Efficacy and Safety of Dupilumab in Pediatric Patients With Active Eosinophilic Esophagitis (EoE)

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