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Discovery of Sirolimus Sensitive Biomarkers in Blood

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ClinicalTrials.gov Identifier: NCT03304678
Recruitment Status : Recruiting
First Posted : October 9, 2017
Last Update Posted : July 29, 2022
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Study Description
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Brief 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 3 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.

Participant samples will be stored in a secure place. No personal data will be connected to them.


Condition or disease
Lymphangioleiomyomatosis

Detailed 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
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Study Type : Observational
Estimated Enrollment : 26 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Discovery of Sirolimus Sensitive Biomarkers in Blood
Actual Study Start Date : December 4, 2017
Estimated Primary Completion Date : September 30, 2023
Estimated Study Completion Date : September 30, 2023

Resource links provided by the National Library of Medicine

Drug Information available for: Sirolimus

Groups and Cohorts
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Group/Cohort
taking sirolimus
Subjects will come to the NIH and begin taking sirolimus 2mg


Outcome Measures
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Primary Outcome Measures :
  1. to identify if miRNA are responsive to sirolimus in patients with LAM [ Time Frame: 9 months ]
    identify if miRNA are responsive to sirolimus in patients with LAM


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 90 Years   (Adult, Older Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
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.@@@
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03304678


Contacts
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Contact: Tatyana Worthy, R.N. (301) 827-1376 worthyt@mail.nih.gov
Contact: Joel Moss, M.D. (301) 496-1597 mossj@nhlbi.nih.gov

Locations
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United States, Maryland
National Institutes of Health Clinical Center Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)    800-411-1222 ext TTY8664111010    prpl@cc.nih.gov   
Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
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Principal Investigator: Joel Moss, M.D. National Heart, Lung, and Blood Institute (NHLBI)
More Information
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Additional Information:

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Responsible Party: National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT03304678    
Other Study ID Numbers: 180003
18-H-0003
First Posted: October 9, 2017    Key Record Dates
Last Update Posted: July 29, 2022
Last Verified: July 26, 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) ):
mTORC1 Inhibition
VEGF-D
MicroRNA
Natural History
Additional relevant MeSH terms:
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Lymphangioleiomyomatosis
Immune System Diseases
Lymphangiomyoma
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Neoplasms
 Perivascular Epithelioid Cell Neoplasms
Neoplasms, Connective and Soft Tissue
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders