Long-term safety of Imatinib for patients with Lymphangioleiomyomatosis

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]

Phase 1 Interventional Columbia University · NCT06889168

This study is testing if the cancer drug Imatinib is safe and can help women with Lymphangioleiomyomatosis by seeing how it affects their lung health over time.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment20 (estimated)
Ages18 Years to 64 Years
SexFemale
SponsorColumbia University Academic / other
Drugs / interventionsCobimetinib, Imatinib, nilotinib
Locations2 sites (New York, New York and 1 other locations)
Trial IDNCT06889168 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the long-term safety and tolerability of Imatinib mesylate in patients diagnosed with Lymphangioleiomyomatosis (LAM), a rare lung disease. The study aims to determine if Imatinib, an FDA-approved drug for leukemia, can effectively induce cell death in LAM cells, potentially offering a new treatment option. Participants will be monitored for their pulmonary function and overall health while receiving either Imatinib or a placebo. The trial seeks to include women aged 18 to 64 with specific pulmonary function test results and confirmed or possible LAM diagnosis.

Who should consider this trial

Good fit: Ideal candidates for this study are women aged 18 to 64 with a confirmed or possible diagnosis of LAM and specific pulmonary function test results.

Not a fit: Patients who are currently pregnant, breastfeeding, or have undergone a transplant or surgery may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new therapeutic option for patients with LAM, improving their lung function and quality of life.

How similar studies have performed: Previous studies have shown promising results with similar approaches, particularly the pilot trial LAMP-1, which documented the safety of tyrosine kinase inhibitors in LAM patients.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Women 18 through 64 years of age (inclusive)
* Pulmonary Function Test (PFT) with following criteria:

  1. DLCO \>20% predicted and FVC \<90% OR
  2. 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.

Where this trial is running

New York, New York and 1 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions LymphangioleiomyomatosisLAMLAMP-2Imatinib
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.