Sirolimus for Eosinophil-Associated Gastrointestinal Disorders
Status:
Terminated
Trial end date:
2015-05-12
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)