NCT01814059

Sirolimus for Eosinophil-Associated Gastrointestinal Disorders

Terminated Phase 1 Last updated 5 July 2018

What this trial tests

Phase 1 trial testing sirolimus in Eosinophilic Gastroenteritis in 4 participants. Terminated before completion.

Timeline

7 March 2013

Primary endpoint
12 May 2015

12 May 2015

Quick facts

Lead sponsor	National Institute of Allergy and Infectious Diseases (NIAID)
Phase	Phase 1
Status	Terminated
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	4
Start date	7 March 2013
Primary completion	12 May 2015
Estimated completion	12 May 2015
Sites	1 location across United States

Drugs / interventions tested

• sirolimus (sirolimus) — full drug profile →

Conditions studied

• Eosinophilic Gastroenteritis — all drugs for Eosinophilic Gastroenteritis →
• Eosinophilic Esophagitis — all drugs for Eosinophilic Esophagitis →

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Who can join

Adults 18 to 65, any sex, with Eosinophilic Gastroenteritis or Eosinophilic Esophagitis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. The Therapeutic Pipeline for Eosinophilic Esophagitis: Current Landscape and Future Directions.
   Pasta A, Bertin L, Mari A, Calabrese F, et al · · 2025 · cited 1× · PMID 41471371 · DOI 10.3390/ph18121882