Sirolimus for Eosinophil-Associated Gastrointestinal Disorders

Stopped early · Phase 1

Conditions studied: Eosinophilic Gastroenteritis, Eosinophilic Esophagitis

In brief

Background: * Eosinophil-associated gastrointestinal disorders (EGID) are a group of related disorders that affect the esophagus, stomach, and bowel. There are two major types of EGID, eosinophilic esophagitis and eosinophilic gastroenteritis. They are caused by the body's immune system being activated by food allergens, which then damages the gut wall. People with EGID have large numbers of eosinophils (a type of white blood cell) in their gut. EGID can cause difficulty swallowing, abdominal pain, or nausea. * At present, there are no drugs specifically approved to treat EGID. Most adults who have EGID receive steroid therapy to manage the symptoms. However, long-term steroid use may cause other problems in the body. Researchers want to see if low-dose sirolimus can be used to treat EGID. Sirolimus is a drug used to prevent rejection of transplanted organs. It may be able to keep the body's immune cells from being activated by food allergens and decrease the eosinophils. Objectives: \- To see if low dose sirolimus is safe and decreases blood or gut eosinophils in EGID. Eligibility: * Individuals between 18 to 65 years of age who have EGID. * Participants must also have an elevated blood eosinophil count and positive blood tests for IgE antibodies to foods. Design: * Participants who are on medicine for EGID or related symptoms must be on a stable dose for 1 month before screening and stay on that dose throughout the study. * Participants will be screened with a medical history and physical exam, and review their symptoms. They will provide blood and urine samples. They will also have heart and lung function tests. Some participants may have allergy skin tests. * At the first study visit, participants will have 2 days of inpatient tests. They will repeat the tests from the screening visit. They will also have a full analysis of the esophagus, stomach, and small intestine. On the second day, they will start to take sirolimus as a liquid with orange juice or water. * Participants will continue to take sirolimus at home. They will record their doses and any symptoms. They will also have a visit to provide blood samples about 2 weeks after the first study visit. * At the second study visit (about a month after the first visit), participants will repeat the tests from the screening visit. The sirolimus dose may be adjusted as needed. * Participants will take sirolimus for at least another 28 days. Depending on the dose of the drug and the blood test results, some participants may need to take it for up to 112 days. Those who take the drug for a longer period will have additional study visits with tests. * There will be another study visit when participants stop taking the drug. The last visit will be a final follow-up visit.

Key facts

Study ID: NCT01814059
Run by: National Institute of Allergy and Infectious Diseases (NIAID)
People needed: 4
Starts: 2013-03-07
Expected to finish: 2015-05-12
Last updated by the study team: 2018-07-05

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

Individuals will not be eligible to participate in this study if they meet any of the following criteria:
Are breast feeding.
Are HIV positive or have any other known immunodeficiency.
Have used any investigational agent within 6 months of the screening visit.
Express the FIP1L1-PDGF-R fusion gene.
Have evidence of preexisting proteinuria with urine albumin-to-creatinine ratio >200 mg/g (men) or >300 mg/g (women).
Have serum creatinine with estimated GFR <60 mL/min/1.73 m2.
Have any chronic liver disease, including hepatitis B or C.
Use of medications that have CYP3A4 or P-glycoprotein (P-gp) inhibitor or inducer activity.
Are Taking ACE inhibitor medicatons
Drug allergy or intolerance to allergies to sirolimus, other rapalogs, or excipients in the preparation.
Have any condition that, as determined by the investigator, places the patient at undue risk by participating in the study.

Where it is running

National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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