Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study to Determine the Efficacy and Safety of Dupilumab in Adult and Adolescent Patients With Eosinophilic Esophagitis (EoE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03633617
Recruitment Status : Recruiting
First Posted : August 16, 2018
Last Update Posted : October 29, 2019
Sponsor:
Collaborator:
Sanofi
Information provided by (Responsible Party):
Regeneron Pharmaceuticals

Brief Summary:

The primary objectives of the study by study part are:

Part A:

To determine the treatment effect of dupilumab compared with placebo in adult and adolescent patients with EoE after 24 weeks of treatment as assessed by histological and clinical measures, and to inform/confirm the final sample size determination for Part B.

Part B:

To demonstrate the efficacy of dupilumab treatment compared with placebo in adult and adolescent patients with EoE after 24 weeks of treatment as assessed by histological and clinical measures.

Part C:

To assess the safety and efficacy of dupilumab treatment in adult and adolescent patients with EoE after up to 52 weeks of treatment as assessed by histological and clinical measures.

The secondary objectives of the study are:

  • To evaluate the safety, tolerability, and immunogenicity of dupilumab treatment for up to 52 weeks in adult and adolescent patients with EoE
  • To explore the relationship between dupilumab concentration and responses in adult and adolescent patients with EoE, using descriptive analyses

Condition or disease Intervention/treatment Phase
Eosinophilic Esophagitis Drug: Dupilumab Drug: Placebo Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 425 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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)
Actual Study Start Date : September 24, 2018
Estimated Primary Completion Date : May 18, 2022
Estimated Study Completion Date : March 14, 2023

Resource links provided by the National Library of Medicine

Drug Information available for: Dupilumab

Arm Intervention/treatment
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.
Drug: Dupilumab
Solution for injection administered subcutaneously
Other Names:
  • DUPIXENT
  • REGN668
  • SAR231893

Drug: Placebo
Matching placebo

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.
Drug: Dupilumab
Solution for injection administered subcutaneously
Other Names:
  • DUPIXENT
  • REGN668
  • SAR231893

Drug: Placebo
Matching placebo

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.
Drug: Dupilumab
Solution for injection administered subcutaneously
Other Names:
  • DUPIXENT
  • REGN668
  • SAR231893




Primary Outcome Measures :
  1. Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤6 eosinophils per high-power field (eos/hpf) [ Time Frame: At week 24 ]
  2. Absolute change in Dysphagia Symptom Questionnaire (DSQ) score [ Time Frame: Baseline to week 24 ]
    The DSQ is used to measure the frequency and intensity of dysphagia. DSQ scores can range from 0 to 84, with a lower score indicating less-frequent or less-severe dysphagia.


Secondary Outcome Measures :
  1. Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤6 eosinophils per high-power field (eos/hpf) [ Time Frame: At week 52 ]
  2. Absolute change in Dysphagia Symptom Questionnaire (DSQ) score [ Time Frame: Baseline to week 52 ]
    The DSQ is used to measure the frequency and intensity of dysphagia. DSQ scores can range from 0 to 84, with a lower score indicating less-frequent or less-severe dysphagia.

  3. Absolute change in EoE endoscopic reference score (EREFS) [ Time Frame: Baseline to week 24 ]
    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. 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.

  4. Absolute change in EoE endoscopic reference score (EREFS) [ Time Frame: Baseline to week 52 ]
    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. 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.

  5. Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf) [ Time Frame: Baseline to week 24 ]
  6. Percent change in peak esophageal intraepithelial eosinophil count (eos/hpf) [ Time Frame: Baseline to week 52 ]
  7. Absolute change in EoE Grade Score from the EoE Histology Scoring System (EoEHSS) [ Time Frame: Baseline to week 24 ]
    The EoEHSS assesses the severity (grade) and extent (stage) of abnormalities using a 4-point scale (0 normal; 3 maximum change).

  8. Absolute change in EoE Grade Score from the EoEHSS [ Time Frame: Baseline to week 52 ]
    The EoEHSS assesses the severity (grade) and extent (stage) of abnormalities using a 4-point scale (0 normal; 3 maximum change).

  9. Absolute change in EoE Stage Score from the EoEHSS [ Time Frame: Baseline to week 24 ]
    The EoEHSS assesses the severity (grade) and extent (stage) of abnormalities using a 4-point scale (0 normal; 3 maximum change).

  10. Absolute change in EoE Stage Score from the EoEHSS [ Time Frame: Baseline to week 52 ]
    The EoEHSS assesses the severity (grade) and extent (stage) of abnormalities using a 4-point scale (0 normal; 3 maximum change).

  11. Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤15 eos/hpf [ Time Frame: At week 24 ]
  12. Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤15 eos/hpf [ Time Frame: At week 52 ]
  13. Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤1 eos/hpf [ Time Frame: At week 24 ]
  14. Proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤1 eos/hpf [ Time Frame: At week 52 ]
  15. Percent change in DSQ [ Time Frame: Baseline to week 24 ]
    The DSQ is used to measure the frequency and intensity of dysphagia. DSQ scores can range from 0 to 84, with a lower score indicating less-frequent or less-severe dysphagia.

  16. Percent change in DSQ [ Time Frame: Baseline to week 52 ]
    The DSQ is used to measure the frequency and intensity of dysphagia. DSQ scores can range from 0 to 84, with a lower score indicating less-frequent or less-severe dysphagia.

  17. Absolute change in health-related quality of life (QOL) as measured by EoE Impact Questionnaire (EoE-IQ) [ Time Frame: Baseline to week 24 ]
    The EoE-IQ is a disease-specific measure of health-related QOL in EoE patients. The EoE-IQ measures EoE impact on emotional, social, work and school, and sleep aspect of a patient

  18. Absolute change in health-related QOL as measured by EoE Impact Questionnaire (EoE-IQ) [ Time Frame: Baseline to week 52 ]
    The EoE-IQ is a disease-specific measure of health-related QOL in EoE patients. The EoE-IQ measures EoE impact on emotional, social, work and school, and sleep aspect of a patient.

  19. Absolute change in severity and/or frequency of EoE symptoms other than dysphagia [ Time Frame: Baseline to week 24 ]
  20. Absolute change in severity and/or frequency of EoE symptoms other than dysphagia [ Time Frame: Baseline to week 52 ]
  21. Portion of patients with use of rescue medications or procedures during the placebo-controlled treatment period [ Time Frame: At week 24 ]
  22. Portion of patients with use of rescue medications or procedures during the placebo-controlled treatment period [ Time Frame: At week 52 ]
  23. Absolute change in esophageal distensibility plateau measured by functional lumen imagining [ Time Frame: Baseline to week 24 ]
    A catheter-based procedure that measures the cross-sectional area at multiple sites along the esophagus.

  24. Absolute change in esophageal distensibility plateau measured by functional lumen imagining [ Time Frame: Baseline to week 52 ]
    A catheter-based procedure that measures the cross-sectional area at multiple sites along the esophagus.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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


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): NCT03633617


Contacts
Layout table for location contacts
Contact: Clinical Trials Administrator 844-734-6643 clinicaltrials@regeneron.com

  Hide Study Locations
Locations
Layout table for location information
United States, Arizona
Regeneron Study Site Recruiting
Phoenix, Arizona, United States, 85016
Regeneron Study Site Recruiting
Scottsdale, Arizona, United States, 85259
United States, Arkansas
Regeneron Study Site Recruiting
Little Rock, Arkansas, United States, 72202
United States, California
Regeneron Study Site Recruiting
La Jolla, California, United States, 92037
Regeneron Study Site Recruiting
Los Angeles, California, United States, 90025
Regeneron Study Site Recruiting
Mountain View, California, United States, 94305
Regeneron Study Site Recruiting
Rolling Hills Estates, California, United States, 90274
Regeneron Study Site Recruiting
San Diego, California, United States, 92123
United States, Colorado
Regeneron Study Site Recruiting
Aurora, Colorado, United States, 80045
Regeneron Study Site Recruiting
Aurora, Colorado, United States, 80220
Regeneron Study Site Recruiting
Colorado Springs, Colorado, United States, 80907
Regeneron Study Site Recruiting
Lone Tree, Colorado, United States, 80124
United States, Connecticut
Regeneron Study Site Recruiting
Bristol, Connecticut, United States, 06010
United States, Florida
Regeneron Study Site Recruiting
Miami, Florida, United States, 33156
United States, Idaho
Regeneron Study Site Recruiting
Idaho Falls, Idaho, United States, 83404
United States, Illinois
Regeneron Study Site Recruiting
Chicago, Illinois, United States, 60611
Regeneron Study Site Recruiting
Park Ridge, Illinois, United States, 60068
Regeneron Study Site Recruiting
Urbana, Illinois, United States, 61801
United States, Indiana
Regeneron Study Site Recruiting
Indianapolis, Indiana, United States, 46202
United States, Iowa
Regeneron Study Site Recruiting
Iowa City, Iowa, United States, 52242
United States, Maryland
Regeneron Study Site Recruiting
Hagerstown, Maryland, United States, 21742
United States, Massachusetts
Regeneron Study Site Recruiting
Boston, Massachusetts, United States, 02111
Regeneron Study Site Recruiting
Worcester, Massachusetts, United States, 01655
United States, Michigan
Regeneron Study Site Recruiting
Ann Arbor, Michigan, United States, 48109
Regeneron Study Site Recruiting
Chesterfield, Michigan, United States, 48047
United States, Minnesota
Regeneron Study Site Recruiting
Plymouth, Minnesota, United States, 55446
Regeneron Study Site Recruiting
Rochester, Minnesota, United States, 55905
United States, Nebraska
Regeneron Study Site Recruiting
Lincoln, Nebraska, United States, 68505
Regeneron Study Site Recruiting
Omaha, Nebraska, United States, 68130
United States, New York
Regeneron Study Site Recruiting
Bronx, New York, United States, 10461
Regeneron Study Site Recruiting
Great Neck, New York, United States, 11023
Regeneron Study Site Recruiting
New York, New York, United States, 10016
Regeneron Study Site Recruiting
New York, New York, United States, 10029
United States, North Carolina
Regeneron Study Site Recruiting
Chapel Hill, North Carolina, United States, 27599
United States, Ohio
Regeneron Study Site Recruiting
Cincinnati, Ohio, United States, 45229
Regeneron Study Site Recruiting
Dublin, Ohio, United States, 43016
Regeneron Study Site Recruiting
Englewood, Ohio, United States, 45415
Regeneron Study Site Recruiting
Mentor, Ohio, United States, 44060
United States, Pennsylvania
Regeneron Study Site Recruiting
Hershey, Pennsylvania, United States, 17033
Regeneron Study Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, South Carolina
Regeneron Study Site Recruiting
Greenville, South Carolina, United States, 29615
United States, Tennessee
Regeneron Study Site Recruiting
Memphis, Tennessee, United States, 38103
United States, Texas
Regeneron Study Site Recruiting
Dallas, Texas, United States, 75207
Regeneron Study Site Recruiting
Fort Worth, Texas, United States, 76104
Regeneron Study Site Recruiting
Garland, Texas, United States, 75044
Regeneron Study Site Recruiting
Houston, Texas, United States, 77030
Regeneron Study Site Recruiting
San Antonio, Texas, United States, 78229
United States, Utah
Regeneron Study Site Recruiting
Salt Lake City, Utah, United States, 84132
United States, Virginia
Regeneron Study Site Recruiting
Charlottesville, Virginia, United States, 22903
Regeneron Study Site Recruiting
Roanoke, Virginia, United States, 24013
United States, Wisconsin
Regeneron Study Site Recruiting
Milwaukee, Wisconsin, United States, 53226
Australia, New South Wales
Regeneron Study Site Recruiting
Camperdown, New South Wales, Australia, 2050
Australia, Queensland
Regeneron Study Site Recruiting
Woolloongabba, Queensland, Australia, 4102
Australia, South Australia
Regeneron Study Site Recruiting
Elizabeth Vale, South Australia, Australia, 5112
Australia, Victoria
Regeneron Study Site Recruiting
Melbourne, Victoria, Australia, 3052
Belgium
Regeneron Study Site Recruiting
Bruges, Belgium, 8310
Regeneron Study Site Recruiting
Leuven, Belgium, 03000
Canada, Ontario
Regeneron Study Site Recruiting
London, Ontario, Canada, N6A5W9
Canada, Quebec
Regeneron Study Site Recruiting
Montreal, Quebec, Canada, H3T1C5
France
Regeneron Study Site Recruiting
Pessac, France, 33604 Cedex
Regeneron Study Site Recruiting
Toulouse, France, TSA 50032-31059
Italy
Regeneron Study Site Recruiting
Milano, Italy, 20122
Regeneron Study Site Recruiting
Pisa, Italy, 56124
Netherlands
Regeneron Study Site Recruiting
Amsterdam, Netherlands, 1105 AZ
Regeneron Study Site Recruiting
Maastricht, Netherlands, 6229HX
Regeneron Study Site Recruiting
Nijmegen, Netherlands, 6525GA
Spain
Regeneron Study Site Recruiting
Tomelloso, Ciudad Real, Spain, 13700
United Kingdom
Regeneron Study Site Recruiting
Barnsley, Yorkshire, United Kingdom, S752EP
Sponsors and Collaborators
Regeneron Pharmaceuticals
Sanofi
Investigators
Layout table for investigator information
Study Director: Clinical Trial Management Regeneron Pharmaceuticals

Layout table for additonal information
Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03633617     History of Changes
Other Study ID Numbers: R668-EE-1774
2018-000844-25 ( EudraCT Number )
First Posted: August 16, 2018    Key Record Dates
Last Update Posted: October 29, 2019
Last Verified: June 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Esophagitis
Eosinophilic Esophagitis
Esophageal Diseases
Gastrointestinal Diseases
Digestive System Diseases
Gastroenteritis
Eosinophilia
Leukocyte Disorders
Hematologic Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs