Development, Validation and Evaluation of an Adult and Pediatric Eosinophilic Esophagitis Activity Index

NCT00939263 | Unknown DMC

• 2 other identifiers: 231/080901
• Study Type: observational
• Target: 650
• Locations: 4 countries
• Sites: 25

Brief Summary

Eosinophilic Esophagitis (EoE) is a chronic inflammatory disease of the esophagus affecting children and adults. The most frequent symptoms are swallowing difficulties and thoracic pain. The disease has first been described in the 1980s and is found with rapidly increasing frequency mainly in industrialized countries. The factors that lead to EoE are until now incompletely understood, of importance, the disease is found more frequently in men and patients suffering from allergic diseases (e.g., Asthma). So far there exists no activity index to define the severity of EoE; such an index is urgently needed for future clinical trials to determine the efficacy of current and future therapies. The investigators' 3-year project, carried out in cooperation with international EoE experts, aims at the development of an activity index for adult and pediatric EoE patients that will be used in future clinical trials as well as observational studies.

Conditions

Deglutition Disorders; Esophageal Diseases; Eosinophilia

Keywords

deglutition disorders; esophageal diseases; eosinophilia

Outcome Measures

Primary Outcomes (1)

• Development of an EE Activity Index

  12 months

Secondary Outcomes (1)

• Evaluation and validation of the activity index

  2 years

Study Arms (2)

1

cohorts 1 (item weighting phase): 100 children with Eosinophilic Esophagitis, 150 adults with Eosinophilic Esophagitis

Other: assessment of disease activity using the EEsAI

2

cohorts 2 (evaluation phase): 200 children with Eosinophilic Esophagitis, 200 adults with Eosinophilic Esophagitis

Other: assessment of disease activity using the EEsAI

Interventions

assessment of disease activity using the EEsAI OTHER

disease activity index

1; 2

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult and pediatric patients with the diagnosis Eosinophilic Esophagitis, who are treated in the participating hospitals

You may qualify if:

• every patient with the diagnosis Eosinophilic Esophagitis
• pediatric and adult EoE patients

Sponsors & Collaborators

• Insel Gruppe AG, University Hospital Bern: lead
• University of Bern: collaborator
• Kantonsspital Olten: collaborator
• Luzerner Kantonsspital: collaborator
• Mayo Clinic: collaborator
• University of Cincinnati: collaborator
• University of Colorado, Denver: collaborator
• Northwestern University: collaborator
• Indiana University School of Medicine: collaborator
• Philadelphia University: collaborator

Study Sites (25)

Gastroenterology and Hepatology : Mayo Clinic, Scottsdale

Scottsdale, Arizona, 85261, United States

RECRUITING

Department of Pediatrics : University of California

San Diego, California, 92123, United States

RECRUITING

The Children's Hospital : University of Colorado Denver

Aurora, Colorado, 80045, United States

RECRUITING

Gastroenterology and Hepatology : Mayo Clinic, Jacksonville

Jacksonville, Florida, 32224, United States

RECRUITING

Division of Gastroenterology : Northwestern University Chicago

Chicago, Illinois, 60611, United States

RECRUITING

Gastroenterology, hepatology and nutrition : Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60614, United States

RECRUITING

Riley Hospital for Children : Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

RECRUITING

Institute for Clinical Research and Health Policy Studies: Tufts Medical Center

Boston, Massachusetts, 02112, United States

RECRUITING

Division of Allergy and Inflammation : Harvard Medical School Boston

Brookline, Massachusetts, 02112, United States

NOT YET RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, United States

RECRUITING

Pediatrics : Mount Sinai School of Medicine

New York, New York, 10029, United States

RECRUITING

Gastroenterology, Hepatology and Nutrition : University of Cincinnati

Cincinnati, Ohio, 45229, United States

WITHDRAWN

Children's Hospital of Philadelphia : University of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Inova Fairfax Hospital for Children

Fairfax, Virginia, United States

NOT YET RECRUITING

Clinical Epidemiology & Biostatistics : Mc Master University

Hamilton, Ontario, Canada

NOT YET RECRUITING

Ste-Justine Hospital : University of Montreal

Montreal, Quebec, Canada

NOT YET RECRUITING

Department of Gastroenterology, Inselspital/Bern University Hospital

Bern, 3010, Switzerland

WITHDRAWN

Pädiatrische Gastroenterologie Medizinische Universitäts-Kinderklinik Bern

Bern, 3010, Switzerland

NOT YET RECRUITING

Institute for Social and Preventive Medicine, University of Bern

Bern, 3012, Switzerland

RECRUITING

University Hospital Lausanne (CHUV)

Lausanne, 1011, Switzerland

RECRUITING

Department of Gastroenterology and Hepatology, Kantonsspital Lucerne

Lucerne, 6000, Switzerland

RECRUITING

Institute for Pathology, Kantonsspital Lucerne

Lucerne, 6000, Switzerland

NOT YET RECRUITING

Praxis Römerhof, Römerstr. 7, Olten

Olten, 4600, Switzerland

RECRUITING

Ostschweizer Kinderspital

Sankt Gallen, 9006, Switzerland

NOT YET RECRUITING

Northumbria Healthcare Foundation Trust North Tyneside Hospital

North Shields, United Kingdom

WITHDRAWN

Related Publications (12)

• U.S. Department of Health and Human Services FDA Center for Drug Evaluation and Research; U.S. Department of Health and Human Services FDA Center for Biologics Evaluation and Research; U.S. Department of Health and Human Services FDA Center for Devices and Radiological Health. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes. 2006 Oct 11;4:79. doi: 10.1186/1477-7525-4-79.

  PMID: 17034633 BACKGROUND

• Patrick DL, Burke LB, Powers JH, Scott JA, Rock EP, Dawisha S, O'Neill R, Kennedy DL. Patient-reported outcomes to support medical product labeling claims: FDA perspective. Value Health. 2007 Nov-Dec;10 Suppl 2:S125-37. doi: 10.1111/j.1524-4733.2007.00275.x.

  PMID: 17995471 BACKGROUND

• Furuta GT, Liacouras CA, Collins MH, Gupta SK, Justinich C, Putnam PE, Bonis P, Hassall E, Straumann A, Rothenberg ME; First International Gastrointestinal Eosinophil Research Symposium (FIGERS) Subcommittees. Eosinophilic esophagitis in children and adults: a systematic review and consensus recommendations for diagnosis and treatment. Gastroenterology. 2007 Oct;133(4):1342-63. doi: 10.1053/j.gastro.2007.08.017. Epub 2007 Aug 8.

  PMID: 17919504 BACKGROUND

• Straumann A, Simon HU. Eosinophilic esophagitis: escalating epidemiology? J Allergy Clin Immunol. 2005 Feb;115(2):418-9. doi: 10.1016/j.jaci.2004.11.006. No abstract available.

  PMID: 15696105 BACKGROUND

• Noel RJ, Putnam PE, Rothenberg ME. Eosinophilic esophagitis. N Engl J Med. 2004 Aug 26;351(9):940-1. doi: 10.1056/NEJM200408263510924. No abstract available.

  PMID: 15329438 BACKGROUND

• Straumann A, Spichtin HP, Grize L, Bucher KA, Beglinger C, Simon HU. Natural history of primary eosinophilic esophagitis: a follow-up of 30 adult patients for up to 11.5 years. Gastroenterology. 2003 Dec;125(6):1660-9. doi: 10.1053/j.gastro.2003.09.024.

  PMID: 14724818 BACKGROUND

• Liacouras CA, Spergel JM, Ruchelli E, Verma R, Mascarenhas M, Semeao E, Flick J, Kelly J, Brown-Whitehorn T, Mamula P, Markowitz JE. Eosinophilic esophagitis: a 10-year experience in 381 children. Clin Gastroenterol Hepatol. 2005 Dec;3(12):1198-206. doi: 10.1016/s1542-3565(05)00885-2.

  PMID: 16361045 BACKGROUND

• Guyatt GH, Deyo RA, Charlson M, Levine MN, Mitchell A. Responsiveness and validity in health status measurement: a clarification. J Clin Epidemiol. 1989;42(5):403-8. doi: 10.1016/0895-4356(89)90128-5.

  PMID: 2659745 BACKGROUND

• Schoepfer AM, Hirano I, Coslovsky M, Roumet MC, Zwahlen M, Kuehni CE, Hafner D, Alexander JA, Dellon ES, Gonsalves N, Leung J, Bussmann C, Collins MH, Newbury RO, Smyrk TC, Woosley JT, Yang GY, Romero Y, Katzka DA, Furuta GT, Gupta SK, Aceves SS, Chehade M, Spergel JM, Falk GW, Meltzer BA, Comer GM, Straumann A, Safroneeva E; International EEsAI Study Group. Variation in Endoscopic Activity Assessment and Endoscopy Score Validation in Adults With Eosinophilic Esophagitis. Clin Gastroenterol Hepatol. 2019 Jul;17(8):1477-1488.e10. doi: 10.1016/j.cgh.2018.11.032. Epub 2018 Nov 23.

  PMID: 30476587 DERIVED

• Safroneeva E, Straumann A, Coslovsky M, Zwahlen M, Kuehni CE, Panczak R, Haas NA, Alexander JA, Dellon ES, Gonsalves N, Hirano I, Leung J, Bussmann C, Collins MH, Newbury RO, De Petris G, Smyrk TC, Woosley JT, Yan P, Yang GY, Romero Y, Katzka DA, Furuta GT, Gupta SK, Aceves SS, Chehade M, Spergel JM, Schoepfer AM; International Eosinophilic Esophagitis Activity Index Study Group. Symptoms Have Modest Accuracy in Detecting Endoscopic and Histologic Remission in Adults With Eosinophilic Esophagitis. Gastroenterology. 2016 Mar;150(3):581-590.e4. doi: 10.1053/j.gastro.2015.11.004. Epub 2015 Nov 14.

  PMID: 26584601 DERIVED

• Safroneeva E, Coslovsky M, Kuehni CE, Zwahlen M, Haas NA, Panczak R, Taft TH, Hirano I, Dellon ES, Gonsalves N, Leung J, Bussmann C, Woosley JT, Yan P, Romero Y, Furuta GT, Gupta SK, Aceves SS, Chehade M, Straumann A, Schoepfer AM; International EEsAI Study Group. Eosinophilic oesophagitis: relationship of quality of life with clinical, endoscopic and histological activity. Aliment Pharmacol Ther. 2015 Oct;42(8):1000-10. doi: 10.1111/apt.13370. Epub 2015 Aug 14.

  PMID: 26271642 DERIVED

• Schoepfer AM, Straumann A, Panczak R, Coslovsky M, Kuehni CE, Maurer E, Haas NA, Romero Y, Hirano I, Alexander JA, Gonsalves N, Furuta GT, Dellon ES, Leung J, Collins MH, Bussmann C, Netzer P, Gupta SK, Aceves SS, Chehade M, Moawad FJ, Enders FT, Yost KJ, Taft TH, Kern E, Zwahlen M, Safroneeva E; International Eosinophilic Esophagitis Activity Index Study Group. Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis. Gastroenterology. 2014 Dec;147(6):1255-66.e21. doi: 10.1053/j.gastro.2014.08.028. Epub 2014 Aug 23.

  PMID: 25160980 DERIVED

MeSH Terms

Conditions

Deglutition Disorders; Esophageal Diseases; Eosinophilia

Condition Hierarchy (Ancestors)

Gastrointestinal Diseases; Digestive System Diseases; Pharyngeal Diseases; Otorhinolaryngologic Diseases; Leukocyte Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Alain Schoepfer, MD, PD+MER1

  Department of Gastroenterology and Hepatology, Centre Hospitalier Universitaire Vaudois / CHUV

  PRINCIPAL INVESTIGATOR

• Alex Straumann, MD, Prof.

  Praxis Römerhof, Olten, Switzerland

  STUDY CHAIR

• Marcel Zwahlen, PhD, Prof.

  Institute for Social and Preventive Medicine, University of Bern

  STUDY DIRECTOR

• Claudia Kuehni, MD MSc, Prof.

  Institute for Social and Preventive Medicine, University of Bern

  STUDY DIRECTOR

• Christian Bussmann, MD

  Viollier AG

  STUDY DIRECTOR

Central Study Contacts

Ekaterina Safroneeva, PhD

CONTACT

+41 31 631 59 71; esafroneeva@ispm.unibe.ch

Claudia Kuehni, MD MSc, Prof.

CONTACT

+41 31 631 35 07; kuehni@ispm.unibe.ch

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 13, 2009
• First Posted: July 14, 2009
• Study Start: January 1, 2011
• Primary Completion: July 1, 2016
• Study Completion: December 1, 2016
• Last Updated: September 16, 2015

Record last verified: 2015-09

Locations

CH (8); CA (2); US (14); GB (1)