Safety and Tolerability Study of Oral EUR-1100 to Treat Eosinophilic Esophagitis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Aptalis Pharma
ClinicalTrials.gov Identifier:
NCT01386112
First received: June 28, 2011
Last updated: October 31, 2012
Last verified: October 2012
  Purpose

Eosinophilic esophagitis (EoE) is an inflammatory disease of the esophagus, characterized by eosinophilic infiltration and gastrointestinal (GI) symptoms. There are no pharmacological treatments for EoE approved by the US Food and Drug Administration (FDA). Supported by published case series and controlled trials in children and adults, the most widely used drug treatment for EoE is off-label use of corticosteroids intended for local (esophageal mucosal) action.

This study will evaluate the safety and tolerability of orally administered EUR-1100 once or twice daily. Eligible subjects will be randomized into one of the 3 treatment groups. The Treatment Period will be 8 weeks during which subjects will visit the clinic at the screening visit, randomization, week 4, 8 and 1 week after end of treatment for clinical symptom assessment and safety evaluation. Additional phone visits will occur at week 2 and week 6.


Condition Intervention Phase
Eosinophilic Esophagitis
Drug: EUR-1100
Drug: placebo
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Safety and Tolerability Phase 1/2a Study of Two Dosing Regimens of EUR-1100 for Oral Use, in Subjects With Eosinophilic Esophagitis (EoE)

Resource links provided by NLM:


Further study details as provided by Aptalis Pharma:

Primary Outcome Measures:
  • Morning serum cortisol (change from baseline measure) [ Time Frame: Screening visit (up to 21 days), week 4, week 8 and follow-up ] [ Designated as safety issue: Yes ]
  • Standard safety laboratory tests [ Time Frame: Screening visit (up to 21 days), week 4, week 8 and follow-up ] [ Designated as safety issue: No ]
    Hematology, serum chemistry and liver function tests, urinalysis, urine chemistry

  • Treatment-emergent adverse events collection [ Time Frame: Screening visit (up to 21 days), Randomization day, week 2 and week 6(phone visit), week 4 and week 8 (office visit), follow-up (office visit, up to 11 days after week 8 visit) ] [ Designated as safety issue: No ]
  • Physical examination and vital signs collection [ Time Frame: Screening (up to 21 days), week4, week 8 and follow-up ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Esophagoduodenoscopy with multiple biopsies [ Time Frame: Screening (up to 21 days) and week 8 ] [ Designated as safety issue: No ]
  • Patient reported outcomes, and physician global assessment [ Time Frame: Screening (up to 21 days), week 4 and 8. ] [ Designated as safety issue: No ]

Enrollment: 24
Study Start Date: September 2011
Study Completion Date: October 2012
Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: EUR-1100 1.5 mg Drug: EUR-1100
Active oral medication
Experimental: EUR-1100 3.0 mg Drug: EUR-1100
Active oral medication
Placebo Comparator: placebo Drug: placebo
matching placebo

  Eligibility

Ages Eligible for Study:   12 Years to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Male and female subjects aged ≥ 12 and ≤ 55 years;
  • Written informed consent (parent or guardian must sign when applicable) and assent form, if required;
  • Evidence of eosinophilic esophagitis defined by esophageal mucosal eosinophil count greater than or equal to 24 per HPF (400x magnification) in at least 1 of the esophageal sites biopsied (proximal/middle and/or distal);
  • Lack of histological response to previously administered high dose proton pump inhibitor;
  • Clinical symptoms of eosinophilic esophagitis with at least one of the following symptoms: chest pain or discomfort, dysphagia (difficulty swallowing) or food impaction.

Key Exclusion Criteria:

  • Known contraindication, hypersensitivity or intolerance to corticosteroids;
  • Any physical, mental, or social condition, history of illness or laboratory abnormality that, in the investigator's judgment, might interfere with study procedures or the ability of the subject to adhere to and complete the study;
  • Oral or esophageal mucosal infection of any type;
  • Any condition affecting the esophageal mucosa or altering esophageal motility other than EoE;
  • Use of systemic (oral or parenteral) or inhaled, intranasal or high-potency dermal topical corticosteroids in the 30 days prior to the esophageal biopsy required for entrance to this study (or prior to EGD if done during the pre-Screening period) or at any time between the biopsy and the Randomization Visit;
  • Adrenal suppression;
  • Any medical condition in which the use of anti-inflammatory or immunosuppressant drugs are required or may be anticipated to be required during the study;
  • Contraindication to EGD or esophageal biopsy or narrowing of the esophagus precluding EGD;
  • History of esophageal or gastric surgery (history of esophageal dilatation is allowed);
  • Gastrointestinal bleeding;
  • Current chronic infection, immunosuppression, immunodeficiency;
  • History or presence of Crohn's disease, celiac disease, or other inflammatory disease of the gastrointestinal tract;
  • Alcohol or drug abuse;
  • Female subjects who are pregnant or breastfeeding;
  • Participation in a clinical study involving an investigational drug within 30 days of the Screening Visit.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01386112

Locations
United States, Georgia
Children's Center for Digestive Health
Atlanta, Georgia, United States, 30342
United States, Illinois
Northwestern University School of Medicine
Chicago, Illinois, United States, 60611-2951
United States, Indiana
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, New Jersey
South Jersey Pediatric Gastroenterology
Mays Landing, New Jersey, United States, 08330
United States, Pennsylvania
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
United States, Virginia
O&O Alpan LLC Center for Clinical Trials
Fairfax, Virginia, United States, 22030
Sponsors and Collaborators
Aptalis Pharma
Investigators
Principal Investigator: Ikuo Hirano, MD Northwestern University School of Medicine
  More Information

No publications provided

Responsible Party: Aptalis Pharma
ClinicalTrials.gov Identifier: NCT01386112     History of Changes
Other Study ID Numbers: PR-021
Study First Received: June 28, 2011
Last Updated: October 31, 2012
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Esophagitis
Eosinophilic Esophagitis
Esophageal Diseases
Gastrointestinal Diseases
Digestive System Diseases
Gastroenteritis
Eosinophilia
Leukocyte Disorders
Hematologic Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases

ClinicalTrials.gov processed this record on April 17, 2014