Discovery of Sirolimus Sensitive Biomarkers in Blood

Lymphangioleiomyomatosis Clinical Trial

Official title:

Discovery of Sirolimus Sensitive Biomarkers in Blood

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03304678
• Other study ID #: 180003
• Secondary ID: 18-H-0003
• Status: Recruiting
• Phase: Phase 2
• Start date: December 4, 2017
• Est. completion date: September 30, 2025

Study information

• Verified date: March 22, 2024
• Source: National Institutes of Health Clinical Center (CC)
• Contact: Tatyana Worthy, R.N.
• Phone: (301) 827-1376
• Email: worthyt[@]mail.nih.gov
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Description:

Sirolimus (rapamycin), which acts as a targeted inhibitor of the protein mechanistic target of rapamycin (mTOR), has been shown to be effective in patients with lymphangioleiomyomatosis (LAM). It stabilizes lung function, resolves chylous effusions and lymphangioleiomas and shrinks angiomyolipomas. The current study is to understand better the short-term action of the drug by following the effects on potential biomarkers in blood and urine. Patients with LAM will have samples taken prior to administration of first dose of the drug, at 1 hr and then at 23 hours after the drug (trough level). At 3 and 9 months, samples will be obtained at trough and 1 hour after the dose. Molecular and cellular analyses will be performed to look for potential biomarkers.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 26
• Est. completion date: September 30, 2025
• Est. primary completion date: September 30, 2025
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 18 Years to 90 Years

Eligibility

• 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

Related Conditions & MeSH terms

• Lymphangioleiomyomatosis

Intervention

Drug:
• 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.

Locations

Country	Name	City	State
United States	National Institutes of Health Clinical Center	Bethesda	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
National Heart, Lung, and Blood Institute (NHLBI)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	to identify if miRNA are responsive to sirolimus in patients with LAM	identify if miRNA are responsive to sirolimus in patients with LAM	9 months

External Links

•   NIH Clinical Center Detailed Web Page