A Study of Nintedanib for LymphAngioleioMyomatosis (LAM) (NCT03062943) | Clinical Trial Compass
CompletedPhase 2
A Study of Nintedanib for LymphAngioleioMyomatosis (LAM)
Italy30 participantsStarted 2016-12-06
Plain-language summary
This trial is conducted locally. The aim of this trial is assess the efficacy and a favorable benefit-risk ratio for nintedanib in the treatment of LAM at the dose of 150 mg bid
Who can participate
Age range
18 Years
Sex
FEMALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Written Informed Consent for participating to trial.
* Patient aged ≥ 18 years at visit 1.
* Sporadic or TSC associated LAM, classified as ''definite'' by the European Respiratory Society criteria and /or serum VEGFD level \>/= 800 mg/ml, and evidence of a 10% deterioration in FEV1 and /or loss of 80 ml of FEV1 or more in the last year (post bronchodilator). Also LAM patients with proven side effects and/or toxicities/ contraindications to sirolimus therapy will be eligible for this study.
Exclusion Criteria:
Laboratory parameters have to satisfy entry criteria as shown below:
* Laboratory parameters (screening)
* AST, ALT \> 1.5 x ULN
* Bilirubin \> 1.5 x ULN
* Positivity for HIV or Hepatitis.
* Chylous effusions.
* Relapsing pneumothorax.
* Angiomyolipoma \> 5 cm.
* Treatment with mTOR inhibitors in the last month.
* Patient eligible for Lung Transplantation.
* Hormone therapy.
* Patients are excluded if they are post lung transplant or had previously been diagnosed with a pneumothorax, chylous effusion, bleeding angiomyolipoma within the previous 6 months.
* Current smokers.
* Other diseases:
* Cardiac disease.
* Myocardial infarction within 6 months of visit 2.
* Unstable angina within 1 month of visit 2.
* Bleeding Risk:
* Known genetic predisposition to bleeding.
* Patients who require fibrinolysis, full-dose therapeutic anticoagulation (e.g. vitamin K antagonists, heparin, NOA) or high dose antiplatelet therapy2.
* History of ha…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.