Discovery of Sirolimus Sensitive Biomarkers in Blood

Status Recruiting

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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Lymphangioleiomyomatosis

Clinical Trial Details

NCT number	NCT03304678
Study type	Interventional
Source	National Institutes of Health Clinical Center (CC)
Contact	Tatyana Worthy, R.N.
Phone	(301) 827-1376
Email	worthyt@mail.nih.gov
Status	Recruiting
Phase	Phase 2
Start date	December 4, 2017
Completion date	September 30, 2025

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT01687179` - Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis	Phase 1
Completed	`NCT00366509` - Role of Helicobacter Pylori and Its Toxins in Lung and Digestive System Diseases
Completed	`NCT00552955` - Effect of Fasting on the Size of Abdominal Lymphatic Tumors in Women
Unknown status	`NCT00490789` - Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM	Phase 2
Not yet recruiting	`NCT05087134` - Characterizing LAM With 11C-Choline PET/CT	Early Phase 1
Completed	`NCT02009241` - Pulmonary Rehabilitation in Lymphangioleiomyomatosis	N/A
Recruiting	`NCT05190627` - Effect of Loratadine in Lymphangioleiomyomatosis	Phase 2
Completed	`NCT05323370` - Lymphangioleiomyomatosis, a Study on Cathepsin K
Completed	`NCT02859194` - The Effect of Lt to Rt Shunt Using Veno-veno-arterial Extracorporeal Membrane Oxygenation (ECMO) on Coronary Oxygenation in Lung Transplantation Patients	N/A
Completed	`NCT01353209` - Letrozole for Lymphangioleiomyomatosis	Phase 2
Completed	`NCT03253913` - Resveratrol and Sirolimus in Lymphangioleiomyomatosis Trial	Phase 2
Recruiting	`NCT02432560` - Safety and Durability of Sirolimus for Treatment of LAM
Completed	`NCT02061397` - Safety of Simvastatin in LAM and TSC	Phase 1/Phase 2
Active, not recruiting	`NCT01552434` - Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease	Phase 1
Recruiting	`NCT04577937` - Sleep Patterns in Patients Affected by Lymphangioleiomiomatosis	N/A
Completed	`NCT00005906` - Treatment With Octreotide in Patients With Lymphangioleiomyomatosis	Phase 2
Recruiting	`NCT01799538` - Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis	Phase 1/Phase 2
Completed	`NCT00989742` - Doxycycline In Lymphangioleiomyomatosis (LAM)	Phase 4
Recruiting	`NCT06160310` - Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)
Enrolling by invitation	`NCT05727852` - Breath Analysis and Arterial Stiffness in Patients With Respiratory Diseases

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.