ClinicalTrials.gov record NCT06889168

Evaluating the Long-term Safety and Tolerability of Imatinib in Patients With Lymphangioleiomyomatosis (LAM)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

A Phase 1, Randomized, Double-blinded, Placebo Controlled, Trial Evaluating the Long-term Safety and Tolerability of Imatinib for the Treatment of Lymphangioleiomyomatosis [LAMP-2 Trial]

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.
Age18 years old to 64 years old
SexFemale
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 1, randomized, triple-blind, placebo-controlled trial tests imatinib in people with lymphangioleiomyomatosis (LAM). It is aimed mainly at measuring safety and tolerability over a longer 6-month treatment period (with adverse events tracked up to one year). The study enrolls women with confirmed or possible LAM (brief gist: adult women within the study's age range) and compares oral imatinib to placebo, with scheduled office visits and phone check-ins.

What it is studying. The study is testing the long-term safety and tolerability of imatinib taken orally compared with placebo in people with LAM. It also compares effects on a LAM biomarker (VEGF-D), lung function measures (FVC and FEV1), and a respiratory quality-of-life questionnaire (SGRQ) between the two groups.

What the researchers hope to learn. To evaluate the long-term safety and tolerability of imatinib in people with LAM and to compare changes between imatinib and placebo in VEGF-D levels, lung function (FVC and FEV1), and St. George Respiratory Questionnaire (SGRQ) scores.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Imatimib Mesylate
  • Placebo

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Women 18 through 64 years of age (inclusive)
  • Pulmonary Function Test (PFT) with following criteria:
  • DLCO >20% predicted and FVC <90% OR
  • Post bronchodilator FEV1 between 30% and 90% predicted.
  • Confirmed or possible diagnosis of LAM
  • Willing to avoid grapefruit juice and St. John's wort while in the study
  • Able and willing to comply with the study procedures

Exclusion criteria

  • Women who have or will undergo a transplant
  • Women who will undergo surgery
  • Women who are currently pregnant or plan on a pregnancy
  • Women who are currently breast feeding or lactating
  • Dementia or other cognitive dysfunction that, in the opinion of the investigator, would prevent the participant from consenting to the study or completing study procedures
  • Currently taking any of the following medications:
  • Antifungal Medications: Ketoconazole; Itraconazole ; Voriconazole.
  • Antibiotics for bacterial infections: Clarithromycin.
  • Analgesics to treat headaches/migraines: Dihydroergotamine; Dihydroergotamine intranasal
  • Antiretroviral protease inhibitors used in human immunodeficiency virus (HIV) infections: Atazanavir ; Nelfinavir; Indinavir; Ritonavir; Saquinavir
  • Anti-epileptic or seizure medications: Carbamazepine, Fosphenytoin; Oxcarbamazepine; Phenobarbital ; Phenytoin; Primidone
  • Anti-depressant medications: Nefazodone; St. John's wort
  • Targeted cancer drugs: Regorafenib; Venetoclax ; Cobimetinib
  • Ivabradine (used to treat chronic heart failure); Telithromycin (used to treat community acquired pneumonia); Lomitapide (treatment of familial hypercholesterolemia); Lonafarnib (Hutchinson-Gilford progeria syndrome); conivaptan (treat low sodium levels); flibanserin (management of hypoactive sexual desire disorder (HSDD)); Naloxegol (opioid-induced constipation); Warfarin (prevent blood clots); Lurasidone (schizophrenia and bipolar depression); Eliglustat (treatment of Gaucher's disease).
  • Non English speaking, illiterate, or other vulnerable persons will not be included among study subjects.
  • Any condition that in the opinion of the investigator might adversely influence the study outcome.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

Lymphangioleiomyomatosis (LAM) is a rare cystic lung disease that is due to a very slow growing cancer that proliferates via unopposed activity of the mTOR pathway. A large NIH study (MILES) found that sirolimus (Rapamycin) that inhibits the mTOR pathway improved lung function and quality of life compared to placebo in LAM. Sirolimus has been shown to cause growth suppression but not apoptosis of LAM cells in culture. Additionally, not all patients with LAM have a clinical effect with sirolimus. Recently, imatinib mesylate has been shown to induce LAM cell apoptosis, raising the possibility of more lasting therapy for LAM. Currently, most LAM patients are on sirolimus and attempts to find sirolimus naive patients have not been successful for other studies. LAM cells use the mesenchymal PDGF receptor pathway for proliferation, similar to certain leukemias and slow growing neoplasms. Laboratory studies suggested that Imatinib mesylate (imatinib), an FDA approved drug for leukemia, initiates LAM cell death. A pilot trial with imatinib (LAMP-1) was funded by the DOD 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 VEGF-D as a response to therapies. Concurrent cell-based research studies during this time showed equally efficacious tumoricidal activity of nilotinib in vitro. Due to the short-term LAMP-1 trial, the investigators propose a longer-term clinical study evaluating the safety and tolerability of imatinib in patients with LAM.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomly assigned to take oral imatinib (imatinib mesylate) 400 mg twice daily or a matching placebo for six months. The study involves five clinic visits plus check‑up phone calls every two weeks, lung function tests (PFTs) and blood tests (including measurement of VEGF‑D) at baseline and 6 months, and completion of a respiratory quality‑of‑life questionnaire (SGRQ). Safety monitoring and reporting of any side effects will occur throughout participation. You must avoid grapefruit juice and St. John's wort while in the study.

How the study is structured. Participants are randomly assigned to one of two groups (imatinib or placebo) in a parallel design. The trial is triple-blinded (participants, investigators, and those assessing outcomes are blinded). Imatinib is given orally at the dose listed in the record (400 mg twice daily); placebo matches the study drug in appearance and administration. The main treatment period is 6 months with five office visits and regular phone check-ups every two weeks; adverse events are monitored through one year and key measurements are taken at baseline and 6 months.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on lymphangioleiomyomatosis

Clinical trials10 trials listed for lymphangioleiomyomatosisEvery trial the registry lists for lymphangioleiomyomatosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in lymphangioleiomyomatosis researchLatest monthly update: September 2026. Includes: Pleural fluid drainage. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Oct 20, 2025
Primary completion
December 2026 (estimated)
Study completion
March 2027 (estimated)
Target enrollment
20 participants (estimated)
Last updated on registry
Jan 21, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Columbia University
Principal investigator
Jeanine D'Armiento, Professor of Medicine in Anesthesiology
Columbia University
Collaborators
  • United States Department of Defense
  • Medical University of South Carolina

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (2)

Terms used on this page · explanations by MedicalUpdates.ai

biomarker
Something measurable in the body, such as a blood test result or an imaging finding, that indicates how a condition or treatment is behaving.
DLCO
A breathing test that measures how well the lungs pass oxygen from the air into the blood.
FVC
A breathing test that measures the total amount of air a person can blow out after a full breath in. It is often given as a percentage of what is expected for a person's age and size.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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