Clinical trial

Discovery of Sirolimus Sensitive Biomarkers in Blood

Name

180003

Description

Background: Lymphangioleiomyomatosis (LAM) is a rare, progressive disease. It usually affects women in the prime of their lives. It typically results in lung destruction. Studies have shown that a drug called sirolimus stabilizes lung function in people with LAM. But researchers do not know what drug dose and blood serum levels are needed to reach this stability. Researchers want to learn more about the right dose of sirolimus for people with LAM. Objective: To determine if blood and urine markers after 1 dose and again after 9 months can be used to evaluate the correct dose of sirolimus for people with LAM. Eligibility: Women ages 18-90 with LAM whose doctors have decided they should start taking sirolimus to treat it. Design: At visit 1, participants will take their first dose of sirolimus by mouth at the clinic. They will have blood and urine collected. Participants will take 1 tablet of the study drug each day. Visit 2 will be 3 months after visit 1. Participants will have blood and urine collected. Visit 3 will be 9 months after visit 1. Participants will have blood and urine collected. Participant samples will be stored in a secure place. No personal data will be connected to them.

Trial arms

Trial start

2017-12-04

Estimated PCD

2025-09-30

Trial end

2025-09-30

Status

Recruiting

Phase

Early phase I

Treatment

Sirolimus 2mg

Patients with LAM, whose treating physicians have decided that they need to start treatment with sirolimus will be referred to the NIH Clinical Center for these studies.

Arms:

Treatment with sirolimus

Size

Primary endpoint

to identify if miRNA are responsive to sirolimus in patients with LAM

9 months

Eligibility criteria

* INCLUSION CRITERIA * Female 18 to 90 years of age * Diagnosis of LAM * Initiation of sirolimus therapy (2mg daily) based on standard-of-care pulmonary indications and the advice of the patient s local physician EXCLUSION CRITERIA * Unable to travel to the NIH * Unable to provide informed consent * Advanced stage of a pulmonary or a systemic illness in which the risk of the study is judged to be significant even in the absence of a clear contraindication to the procedures * Women who are pregnant or lactating

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'OTHER', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 26, 'type': 'ESTIMATED'}}

Updated at

2024-03-26

1 organization

1 product

1 indication

Organization

National Heart, Lung, and Blood Institute

Product

Sirolimus

Indication

Lymphangioleiomyomatosis