Lymphangioleiomyomatosis (LAM)Phase 1LAMP-2NCT06889168
Evaluating the Long-term Safety and Tolerability of Imatinib in Patients With Lymphangioleiomyomatosis (LAM)
A Phase 1 study testing Imatimib Mesylate in people with Lymphangioleiomyomatosis (LAM) and Lymphangioleiomyomatosis, run by Columbia University at 2 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 64 years, women only
- Conditions the study is for
- Lymphangioleiomyomatosis (LAM), Lymphangioleiomyomatosis
- What is being tested?
- Imatimib Mesylate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 2 sitesUnited States
- Study dates
- 20 Oct 2025to Mar 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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:
Show all 16Show fewer
- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
Seth Polk, 7573341517, polkse@musc.edu
Charlie Strange, MD
- Recruiting
Columbia University Irving Medical Center
New York, New York, 10032
Katarina Koumbourlis, 212-305-3745, kk3922@cumc.columbia.edu
Sabrina Palumbo, BS, 212-305-3745, sp4461@cumc.columbia.edu
About this study
Official title: 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]
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.
Read the detailed description
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.
- Being tested
- Imatimib Mesylate, Placebo
- Keywords
- LAM, LAMP-2, Imatinib
- Medical subject headings
- Lymphangioleiomyomatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Imatimib MesylateDrug
Also called Gleevec®, imatinib
Participants will take Imatinib mesylate (imatinib), an FDA approved drug for leukemia, orally 400 mg (twice daily)
PlaceboDrug
Placebo will be administered in the same dosage and manner as the study drug. The placebo looks like the study drug but contains no active ingredients.
| Study group | Role | What they receive |
|---|---|---|
| Imatinib Mesylate Group | Active comparator | This group will receive imatinib mesylate over the course of the trial. Receives: Drug: Imatimib Mesylate |
| Placebo Group | Placebo comparator | This group will receive placebo over the course of the trial. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events
To observe and compare the occurrence of adverse events experienced by participants receiving imatinib mesylate compared to placebo throughout the duration of the study.
Measured: 1 year
Secondary outcomes (4)
Change in VEGF-D between study groups
To compare the change in the log-transformed level of VEGF-D from baseline to 6 months between imatinib mesylate and placebo groups.
Measured: Baseline, 6 months
Change in Forced Vital Capacity (FVC) between study groups
To compare the change in Forced Vital Capacity (FVC) from baseline to 6 months between imatinib mesylate and placebo groups.
Measured: Baseline, 6 months
Change in percent Forced Expiratory Volume in One Second (FEV1) between study groups
To compare the change in the percent predicted FEV1 (Forced expiratory volume in one second) using "GLI other" standards from baseline to 6 months between imatinib mesylate and placebo groups.
Measured: Baseline, 6 months
Change in St. George Respiratory Questionnaire (SGRQ) score between study groups
To compare the change in the total score of the St. George Respiratory Questionnaire (SGRQ) from baseline to 6 months between imatinib mesylate and placebo groups. Scores range from 0 to 100, with 0 representing no health impairment and 100 representing maximum health impairment.
Measured: Baseline, 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2025
- Study start20 Oct 2025
- Primary completionDec 2026 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lymphangioleiomyomatosis (LAM)Studies of the same condition, open and under way.
- More trials run by Columbia UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Collaborators
- United States Department of Defense, Medical University of South Carolina
- Responsible party
- Principal investigator, Jeanine D'Armiento, Columbia University
- Investigators
- Jeanine D'Armiento, MD, PhD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06889168
- Sponsor's protocol ID
- AAAT1955
- Acronym
- LAMP-2
- Status last verified
- Jan 2026
- First submitted
- 15 Mar 2025
- Last updated
- 21 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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]
- ClinicalTrials.gov ID
- NCT06889168
- Acronym / sponsor ID
- LAMP-2 / AAAT1955
- Keywords
- LAM, LAMP-2, Imatinib
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Collaborators
- United States Department of Defense, Medical University of South Carolina
- Study start
- 20 Oct 2025 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 21 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Imatinib Mesylate Group | Active comparator | Drug: Imatimib MesylateThis group will receive imatinib mesylate over the course of the trial. |
| Placebo Group | Placebo comparator | Drug: PlaceboThis group will receive placebo over the course of the trial. |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Women 18 through 64 years of age (inclusive)
- Pulmonary Function Test (PFT) with following criteria:
- 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
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Imatimib MesylateAlso: Gleevec®, imatinibParticipants will take Imatinib mesylate (imatinib), an FDA approved drug for leukemia, orally 400 mg (twice daily) | Drug | Imatinib Mesylate Group |
| PlaceboPlacebo will be administered in the same dosage and manner as the study drug. The placebo looks like the study drug but contains no active ingredients. | Drug | Placebo Group |
Primary outcome
Incidence of Adverse Events
Time frame: 1 year
To observe and compare the occurrence of adverse events experienced by participants receiving imatinib mesylate compared to placebo throughout the duration of the study.
4 secondary outcomes
Change in VEGF-D between study groups
Time frame: Baseline, 6 months
Change in Forced Vital Capacity (FVC) between study groups
Time frame: Baseline, 6 months
Change in percent Forced Expiratory Volume in One Second (FEV1) between study groups
Time frame: Baseline, 6 months
Change in St. George Respiratory Questionnaire (SGRQ) score between study groups
Time frame: Baseline, 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jan 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jeanine D'Armiento, MD, PhD principal investigator
Columbia University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Imatimib Mesylate in people with Lymphangioleiomyomatosis (LAM) and Lymphangioleiomyomatosis, run by Columbia University at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 21 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Katarina Koumbourlis, BS (212-305-3745, kk3922@cumc.columbia.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Adverse Events (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jan 2026.