Treatment for Lymphangioleiomyomatosis

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Lymphangioleiomyomatosis+1 More
Sirolimus - Drug
Eligibility
18+
Female
What conditions do you have?
Select

Study Summary

This trial will test if sirolimus can prevent kidney disease from getting worse.

Eligible Conditions
  • Lymphangioleiomyomatosis
  • LAM

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Other trials for Lymphangioleiomyomatosis

Study Objectives

1 Primary · 2 Secondary · Reporting Duration: 2 years

2 years
Diffusing Capacity for Carbon Monoxide (DLCO)
Forced Expiratory Volume in 1 Second (FEV1 slope)
Total Lung Capacity (TLC)

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Lymphangioleiomyomatosis

Trial Design

2 Treatment Groups

Treatment
1 of 2
Placebo
1 of 2
Active Control
Non-Treatment Group

60 Total Participants · 2 Treatment Groups

Primary Treatment: Treatment · Has Placebo Group · Phase 3

Placebo
Drug
PlaceboComparator Group · 1 Intervention: Sirolimus · Intervention Types: Drug
Treatment
Drug
ActiveComparator Group · 1 Intervention: Sirolimus · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 2 years

Who is running the clinical trial?

National Center for Advancing Translational Sciences (NCATS)NIH
156 Previous Clinical Trials
101,980 Total Patients Enrolled
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,576 Previous Clinical Trials
46,968,521 Total Patients Enrolled
12 Trials studying Lymphangioleiomyomatosis
3,373 Patients Enrolled for Lymphangioleiomyomatosis
The LAM FoundationOTHER
4 Previous Clinical Trials
640 Total Patients Enrolled
4 Trials studying Lymphangioleiomyomatosis
640 Patients Enrolled for Lymphangioleiomyomatosis
University of CincinnatiLead Sponsor
385 Previous Clinical Trials
630,438 Total Patients Enrolled
5 Trials studying Lymphangioleiomyomatosis
679 Patients Enrolled for Lymphangioleiomyomatosis
Francis X. McCormack, M.D.Study ChairUniverisity of Cincinnati

Eligibility Criteria

Age 18+ · Female Participants · 7 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
This text is about a female person who is 18 years or older.
: 1) a) demonstration of diffuse opacities on CT consistent with LAM; or 1) b) elevated serum levels of HGF and/or c-met protein
The individual has a lung biopsy, abdominal mass biopsy, lymph node biopsy, or kidney biopsy that reveals LAM, tuberous sclerosis, angiomyolipomata (diagnosed by CT, MRI by the site radiologist or biopsy), or chylous pleural effusion (verified by tap), or VEGF-D level ≥ 800 pg/ml.
, low hemoglobin level (As high as 11.0 g/dL in women), or history of venous thromboembolism The text discusses a pre-trial measure of decline in FEV 1 rate
predicted suggests that the patient does not have asthma If the post-bronchodilator forced expiratory volume in one second is above 70% predicted, it suggests that the patient does not have asthma.
scan The individual has a baseline residualvolume of 120% of predicted, desaturation of 4% or more on six minute walk testing on room air, and more than 20 cysts on the carinal cut of theCT scan.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 20th, 2021

Last Reviewed: October 26th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.