Imatinib for LAM
(LAMP-2 Trial)
Trial Summary
Will I have to stop taking my current medications?
You may need to stop taking certain medications to participate in this trial. Specifically, if you are taking any antifungal medications, certain antibiotics, migraine treatments, HIV medications, anti-seizure drugs, some antidepressants, targeted cancer drugs, or other specified medications, you will not be eligible to join the study.
What data supports the effectiveness of the drug Imatinib Mesylate for LAM?
Imatinib Mesylate is known to be effective in treating certain types of leukemia, such as chronic myeloid leukemia (CML) and acute lymphoblastic leukemia (ALL), by targeting specific proteins involved in cancer cell growth. While this data is not directly related to LAM, it suggests that Imatinib's ability to inhibit specific proteins could potentially be beneficial in other conditions.12345
Is Imatinib safe for humans?
Imatinib mesylate is generally considered safe and well-tolerated, but it can cause side effects like nausea, vomiting, diarrhea, muscle cramps, and skin reactions. Some rare but serious side effects include bone marrow problems and skin pigmentation changes. Most side effects are mild and manageable, allowing patients to continue treatment.678910
How is the drug Imatinib Mesylate unique for treating LAM?
Imatinib Mesylate is unique because it is a tyrosine kinase inhibitor, which means it blocks specific enzymes (tyrosine kinases) that promote cell growth, and it is primarily used for treating certain types of leukemia. Its use for LAM (lymphangioleiomyomatosis) is novel as there are no standard treatments specifically approved for this condition.123411
What is the purpose of this trial?
Lymphangioleiomyomatosis (LAM) is a rare cystic lung disease that appears to behave like a slowly growing cancer. Since clinical progression is very slow, new blood tests have been used to speed the time required to find safe and effective medications. A large National Institute of Health study called MILES showed that sirolimus (also known as Rapamycin) improved lung function in individuals with LAM. Since most individuals with LAM and impaired lung function are now on sirolimus, future studies may prove more difficult. Laboratory studies suggested that Imatinib mesylate (imatinib), an FDA-approved drug for leukemia, initiates LAM cell death. A pilot trial with imatinib titled "Imatinib Mesylate for the treatment of Lymphangioleiomyomatosis" - (LAMP-1) was funded by the Department of Defense in 2016, and documented (1) the safety of use of tyrosine kinase inhibitors in patients with LAM; (2) the safety of concurrent use of tyrosine kinase and mTOR inhibitors; and, (3) short term variability in vascular endothelial growth factor D (VEGF-D) - a LAM biomarker, as a response to therapies. Due to the short-term LAMP-1 trial, LAMP-2 will be a longer-term 6-month clinical study evaluating the safety and tolerability of imatinib in patients with LAM. Patients that participate in the trial will come in for 5 office visits and check-up phone calls every 2 weeks over the course of 6 months.
Research Team
Jeanine D'Armiento, MD, PhD
Principal Investigator
Columbia University
Eligibility Criteria
This trial is for individuals with a rare lung disease called Lymphangioleiomyomatosis (LAM). Participants must have been previously treated with sirolimus, as the study examines the effects of Imatinib Mesylate, a drug used in leukemia treatment that may cause LAM cell death. The trial involves office visits and bi-weekly phone check-ups over 6 months.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive imatinib mesylate or placebo over the course of the trial
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term follow-up
Participants are monitored for long-term safety and changes in biomarkers
Treatment Details
Interventions
- Imatinib Mesylate
Find a Clinic Near You
Who Is Running the Clinical Trial?
Columbia University
Lead Sponsor
United States Department of Defense
Collaborator
Medical University of South Carolina
Collaborator