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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

 No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

 Interventional
Eligible Ages 18 Years - 64 Years
Gender Female
More 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.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

 NCT06889168
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

 Phase 1
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

 Columbia University
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

 Jeanine D'Armiento, MD, PhD
Principal Investigator Affiliation Columbia University
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

 Other, U.S. Fed
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

 Lymphangioleiomyomatosis (LAM), Lymphangioleiomyomatosis
Additional Details

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.

Arms & Interventions

Arms

Active Comparator: Imatinib Mesylate Group

This group will receive imatinib mesylate over the course of the trial.

Placebo Comparator: Placebo Group

This group will receive placebo over the course of the trial.

Interventions

Drug: - Imatimib Mesylate

Participants will take Imatinib mesylate (imatinib), an FDA approved drug for leukemia, orally 400 mg (twice daily)

Drug: - Placebo

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.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

New York, New York

Status

Recruiting

Address

Columbia University Irving Medical Center

New York, New York, 10032

Site Contact

Sydney Harris, MBS

[email protected]

2123053745

Medical University of South Carolina, Charleston, South Carolina

Status

Recruiting

Address

Medical University of South Carolina

Charleston, South Carolina, 29425

Site Contact

Seth Polk

[email protected]

7573341517

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