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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03633617




Registration number
NCT03633617
Ethics application status
Date submitted
14/08/2018
Date registered
16/08/2018
Date last updated
28/06/2023

Titles & IDs
Public title
Study to Determine the Efficacy and Safety of Dupilumab in Adult and Adolescent Patients With Eosinophilic Esophagitis (EoE)
Scientific title
A Phase 3, Randomized, 3-Part Study to Investigate the Efficacy and Safety of Dupilumab in Adult and Adolescent Patients With Eosinophilic Esophagitis (EoE)
Secondary ID [1] 0 0
2018-000844-25
Secondary ID [2] 0 0
R668-EE-1774
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Eosinophilic Esophagitis 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Inflammatory and Immune System 0 0 0 0
Allergies

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Dupilumab
Treatment: Drugs - Placebo

Experimental: Part A: Dupilumab or Placebo - Part A consists of a 24-week double-blind treatment period. Participants will be randomized to receive dupilumab or placebo. At the end of the double-blind treatment visit (week 24), eligible participants may enter Part C. Participants who do not enter Part C will enter a 12-week follow-up period.

Experimental: Part B: Dupilumab or Placebo - Part B consists of a 24-week double-blind treatment period. Participants will be randomized to receive dupilumab dosing regimen 1, dupilumab dosing regimen 2 or placebo. At the end of the double-blind treatment visit (week 24), eligible participants may enter Part C. Participants who do not enter Part C will enter a 12-week follow-up period.

Experimental: Part C: Dupilumab - Part C is a 28-week extended active treatment period. Participants will receive dupilumab dosing regimen 1, dupilumab dosing regimen 2. At the end of the treatment period (week 52), participants will enter a 12-week follow-up period.


Treatment: Drugs: Dupilumab
Solution for injection administered subcutaneously

Treatment: Drugs: Placebo
Matching placebo

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of =6 Eosinophils Per High-power Field (Eos/Hpf) in All Three Regions at Week 24
Timepoint [1] 0 0
At week 24
Primary outcome [2] 0 0
Absolute Change From Baseline in Dysphagia Symptom Questionnaire (DSQ) Total Score at Week 24
Timepoint [2] 0 0
Baseline and week 24
Secondary outcome [1] 0 0
Percent Change From Baseline in Peak Esophageal Intraepithelial Eosinophil Count (Eos/Hpf) in All Three Regions at Week 24
Timepoint [1] 0 0
Baseline and week 24
Secondary outcome [2] 0 0
Percent Change From Baseline in DSQ Total Score at Week 24
Timepoint [2] 0 0
Baseline and week 24
Secondary outcome [3] 0 0
Absolute Change From Baseline in Eosinophilic Esophagitis Histology Scoring System (EoEHSS) Mean Grade Score at Week 24
Timepoint [3] 0 0
Baseline and week 24
Secondary outcome [4] 0 0
Absolute Change From Baseline in EoEHSS Mean Stage Score at Week 24
Timepoint [4] 0 0
Baseline and week 24
Secondary outcome [5] 0 0
Absolute Change From Baseline in EoE Endoscopic Reference Total Score (EoE-EREFS) at Week 24
Timepoint [5] 0 0
Baseline and week 24
Secondary outcome [6] 0 0
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of <15 Eos/Hpf in All Three Regions at Week 24
Timepoint [6] 0 0
At week 24
Secondary outcome [7] 0 0
Normalized Enrichment Score (NES) for the Relative Change From Baseline in the EoE Diagnostic Panel (EDP) at Week 24
Timepoint [7] 0 0
Baseline and week 24
Secondary outcome [8] 0 0
NES for the Relative Change From Baseline in the Type 2 Inflammation Signature (T2INF) at Week 24
Timepoint [8] 0 0
Baseline and week 24
Secondary outcome [9] 0 0
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of =1 Eos/Hpf in All Three Regions at Week 24
Timepoint [9] 0 0
At week 24
Secondary outcome [10] 0 0
Absolute Change From Baseline in Health-related Quality of Life (QoL) Average Score as Measured by EoE Impact Questionnaire (EoE-IQ) at Week 24
Timepoint [10] 0 0
Baseline and week 24
Secondary outcome [11] 0 0
Absolute Change From Baseline in Severity of EoE Symptoms Other Than Dysphagia as Measured by EoE Symptom Questionnaire (EoE-SQ) at Week 24
Timepoint [11] 0 0
Baseline and week 24
Secondary outcome [12] 0 0
Absolute Change From Baseline in Frequency of EoE Symptoms Other Than Dysphagia as Measured by EoE-SQ at Week 24
Timepoint [12] 0 0
Baseline and week 24
Secondary outcome [13] 0 0
Percentage of Participants Who Received Rescue Treatment During the Placebo-controlled, Double-blind Treatment Period at Week 24
Timepoint [13] 0 0
At week 24
Secondary outcome [14] 0 0
Absolute Change From Baseline in Esophageal Distensibility Plateau Measured by Functional Lumen Imaging, if Collected, at Week 24
Timepoint [14] 0 0
At week 24
Secondary outcome [15] 0 0
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of =6 Eosinophils Per High-power Field (Eos/Hpf) in All Three Regions at Week 52
Timepoint [15] 0 0
At week 52
Secondary outcome [16] 0 0
Absolute Change in Dysphagia Symptom Questionnaire (DSQ) Total Score at Week 52
Timepoint [16] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [17] 0 0
Percent Change in DSQ Total Score at Week 52
Timepoint [17] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [18] 0 0
Absolute Change in EoE Endoscopic Reference Total Score (EoE-EREFS) at Week 52
Timepoint [18] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [19] 0 0
Percent Change in Peak Esophageal Intraepithelial Eosinophil Count (Eos/Hpf) in All Three Regions at Week 52
Timepoint [19] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [20] 0 0
Absolute Change in EoE Histology Scoring System (EoEHSS) Mean Grade Score at Week 52
Timepoint [20] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [21] 0 0
Absolute Change in EoEHSS Mean Stage Score at Week 52
Timepoint [21] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [22] 0 0
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of <15 Eos/Hpf in All Three Regions at Week 52
Timepoint [22] 0 0
At week 52
Secondary outcome [23] 0 0
Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of =1 Eos/Hpf in All Three Regions at Week 52
Timepoint [23] 0 0
At week 52
Secondary outcome [24] 0 0
Absolute Change in Health-related QOL as Measured by EoE-IQ at Week 52
Timepoint [24] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [25] 0 0
Absolute Change From Baseline in Severity of EoE Symptoms Other Than Dysphagia as Measured by EoE-SQ at Week 52
Timepoint [25] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [26] 0 0
Absolute Change From Baseline in Frequency of EoE Symptoms Other Than Dysphagia as Measured by EoE-SQ at Week 52
Timepoint [26] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [27] 0 0
Percentage of Participants Who Received Rescue Medication During the 28-week Extended Active Treatment Period
Timepoint [27] 0 0
Baseline (of Part C) to week 28
Secondary outcome [28] 0 0
NES for the Relative Change From Baseline in EoE Diagnostic Panel (EDP) at Week 52
Timepoint [28] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [29] 0 0
NES for the Relative Change in the Type 2 Inflammation Signature (T2INF) at Week 52
Timepoint [29] 0 0
Baseline (of previous study part) and week 52
Secondary outcome [30] 0 0
Concentration of Functional Dupilumab in Serum at Week 52
Timepoint [30] 0 0
Baseline (of Part C) up to week 52
Secondary outcome [31] 0 0
Incidence of Treatment-emergent Anti-drug Antibody (ADA) Response
Timepoint [31] 0 0
Baseline (of previous study part) up to week 52

Eligibility
Key inclusion criteria
Key Inclusion Criteria (Parts A & B):

* A documented diagnosis of EoE by endoscopic biopsy
* Baseline endoscopic biopsies with a demonstration on central reading of intraepithelial eosinophilic infiltration
* History (by patient report) of an average of at least 2 episodes of dysphagia (with intake of solids) per week in the 4 weeks prior to screening

Key
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria (Parts A & B):

* Body weight =40 kg
* Prior participation in a dupilumab clinical trial, or past or current treatment with dupilumab
* Initiation or change of a food-elimination diet regimen or re-introduction of a previously eliminated food group in the 6 weeks prior to screening.
* Other causes of esophageal eosinophilia or the following conditions: hypereosinophilic syndrome and eosinophilic granulomatosis with polyangiitis (Churg-Strauss syndrome)
* Active Helicobacter pylori infection
* History of achalasia, Crohn's disease, ulcerative colitis, celiac disease, and prior esophageal surgery
* Any esophageal stricture unable to be passed with a standard, diagnostic, 9 to10 mm upper endoscope or any critical esophageal stricture that requires dilation at screening
* History of bleeding disorders or esophageal varices
* Pregnant or breastfeeding women, or women planning to become pregnant or breastfeed during the study

Key Exclusion Criteria (Part C):

* Participants who, during Part A or Part B, developed a serious adverse event (SAE) and/or adverse event (AE) deemed related to study drug, which in the opinion of the investigator could indicate that continued treatment with study drug may present an unreasonable risk for the participant
* Participants who became pregnant during Part A or Part B
* Participants who are prematurely discontinued from study drug due to an AE (patients who are prematurely discontinued from study drug due to lack of efficacy are eligible to enter Part C)
* Patients who did not undergo endoscopy with biopsies prior to receiving rescue treatment

Note: Other inclusion/ exclusion criteria apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
Regeneron Study Site - Camperdown
Recruitment hospital [2] 0 0
Regeneron Study Site - Woolloongabba
Recruitment hospital [3] 0 0
Regeneron Study Site - Elizabeth Vale
Recruitment hospital [4] 0 0
Regeneron Study Site - Melbourne
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [3] 0 0
5112 - Elizabeth Vale
Recruitment postcode(s) [4] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Arkansas
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Connecticut
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Idaho
Country [8] 0 0
United States of America
State/province [8] 0 0
Illinois
Country [9] 0 0
United States of America
State/province [9] 0 0
Indiana
Country [10] 0 0
United States of America
State/province [10] 0 0
Iowa
Country [11] 0 0
United States of America
State/province [11] 0 0
Kansas
Country [12] 0 0
United States of America
State/province [12] 0 0
Maryland
Country [13] 0 0
United States of America
State/province [13] 0 0
Massachusetts
Country [14] 0 0
United States of America
State/province [14] 0 0
Michigan
Country [15] 0 0
United States of America
State/province [15] 0 0
Minnesota
Country [16] 0 0
United States of America
State/province [16] 0 0
Nebraska
Country [17] 0 0
United States of America
State/province [17] 0 0
New York
Country [18] 0 0
United States of America
State/province [18] 0 0
North Carolina
Country [19] 0 0
United States of America
State/province [19] 0 0
Ohio
Country [20] 0 0
United States of America
State/province [20] 0 0
Pennsylvania
Country [21] 0 0
United States of America
State/province [21] 0 0
South Carolina
Country [22] 0 0
United States of America
State/province [22] 0 0
Tennessee
Country [23] 0 0
United States of America
State/province [23] 0 0
Texas
Country [24] 0 0
United States of America
State/province [24] 0 0
Utah
Country [25] 0 0
United States of America
State/province [25] 0 0
Virginia
Country [26] 0 0
United States of America
State/province [26] 0 0
Washington
Country [27] 0 0
United States of America
State/province [27] 0 0
Wisconsin
Country [28] 0 0
Belgium
State/province [28] 0 0
Bruges
Country [29] 0 0
Belgium
State/province [29] 0 0
Edegem
Country [30] 0 0
Belgium
State/province [30] 0 0
Leuven
Country [31] 0 0
Canada
State/province [31] 0 0
Ontario
Country [32] 0 0
Canada
State/province [32] 0 0
Quebec
Country [33] 0 0
Canada
State/province [33] 0 0
Hamilton
Country [34] 0 0
France
State/province [34] 0 0
Pessac
Country [35] 0 0
France
State/province [35] 0 0
Toulouse
Country [36] 0 0
Germany
State/province [36] 0 0
Hannover
Country [37] 0 0
Germany
State/province [37] 0 0
Magdeburg
Country [38] 0 0
Germany
State/province [38] 0 0
Munich
Country [39] 0 0
Italy
State/province [39] 0 0
Genoa
Country [40] 0 0
Italy
State/province [40] 0 0
Milano
Country [41] 0 0
Italy
State/province [41] 0 0
Naples
Country [42] 0 0
Italy
State/province [42] 0 0
Pisa
Country [43] 0 0
Italy
State/province [43] 0 0
Rome
Country [44] 0 0
Italy
State/province [44] 0 0
Rozzano
Country [45] 0 0
Netherlands
State/province [45] 0 0
Amsterdam
Country [46] 0 0
Netherlands
State/province [46] 0 0
Maastricht
Country [47] 0 0
Netherlands
State/province [47] 0 0
Nijmegen
Country [48] 0 0
Spain
State/province [48] 0 0
Ciudad Real
Country [49] 0 0
Spain
State/province [49] 0 0
Barcelona
Country [50] 0 0
Spain
State/province [50] 0 0
Madrid
Country [51] 0 0
Sweden
State/province [51] 0 0
Stockholm
Country [52] 0 0
Switzerland
State/province [52] 0 0
Zurich
Country [53] 0 0
United Kingdom
State/province [53] 0 0
Whitechapel
Country [54] 0 0
United Kingdom
State/province [54] 0 0
Yorkshire

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Regeneron Pharmaceuticals
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Sanofi
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trial Management
Address 0 0
Regeneron Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.