Actively Recruiting
A Phase 1, Randomized, Double-blinded, Placebo Controlled Trial Evaluating the Long-term Safety and Tolerability of Imatinib for Lymphangioleiomyomatosis
Led by Columbia University · Updated on 2026-01-21
20
Participants Needed
2
Research Sites
12 weeks
Total Duration
On this page
Sponsors
C
Columbia University
Lead Sponsor
U
United States Department of Defense
Collaborating Sponsor
AI-Summary
What this Trial Is About
Lymphangioleiomyomatosis (LAM) is a rare lung disease that behaves like a slow-growing cancer caused by unregulated activity of the mTOR pathway. Researchers are studying the long-term safety and tolerability of imatinib mesylate, a drug approved for leukemia that may induce death of LAM cells. This Phase 1 trial follows a previous short-term pilot study and aims to provide more information on imatinib's effects in patients with LAM. Participants will be randomly assigned to receive either imatinib mesylate or a placebo over a six-month period. Imatinib is taken orally at a dose of 400 mg twice daily. The trial includes five office visits and check-up phone calls every two weeks to monitor participants. The study is designed as a double-blinded, placebo-controlled trial to compare safety and tolerability between the two groups. During the study, participants will undergo assessments including lung function tests such as Forced Vital Capacity (FVC) and Forced Expiratory Volume in One Second (FEV1), as well as measurement of the biomarker VEGF-D. Quality of life will be evaluated using the St. George Respiratory Questionnaire. The main outcome measured is the incidence of adverse events over one year. Participants will be monitored regularly for safety and adherence throughout the trial duration.
CONDITIONS
Brief Title
Evaluating the Long-term Safety and Tolerability of Imatinib in Patients With Lymphangioleiomyomatosis (LAM)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Women 18 through 64 years of age (inclusive)
- Confirmed or possible diagnosis of lymphangioleiomyomatosis (LAM)
- Pulmonary Function Test results with either DLCO >20% predicted and FVC <90% OR post bronchodilator FEV1 between 30% and 90% predicted
- Willingness to avoid grapefruit juice and St. John's wort during the study
- Ability and willingness to comply with study procedures
You will not qualify if you...
- Women who have undergone or will undergo a transplant
- Women planning to have surgery during the study
- Women who are currently pregnant or planning pregnancy
- Women who are currently breastfeeding or lactating
- Dementia or cognitive dysfunction preventing consent or study completion
- Currently taking any excluded medications such as certain antifungals, antibiotics, antiretrovirals, anti-epileptic drugs, antidepressants, targeted cancer drugs, and others listed
- Non-English speaking, illiterate, or vulnerable persons
- Any condition that might negatively affect study outcomes as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1 year
Participants take either imatinib mesylate or placebo orally twice daily to evaluate the long-term safety and tolerability of imatinib for LAM.
Visits at baseline and approximately every 6 months for assessments
Trial Site Locations
Total: 2 locations
1
Columbia University Irving Medical Center
New York, New York, United States, 10032
Actively Recruiting
2
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Actively Recruiting
Research Team
K
Katarina Koumbourlis, BS
S
Sabrina Palumbo, BS
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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