RECRUITING

A Phase 1 Trial Evaluating the Long-term Safety and Tolerability of Imatinib for the Treatment of Lymphangioleiomyomatosis (LAM)

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

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]

Quick Facts

Study Start:2025-04-01
Study Completion:2026-08
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06889168

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 64 Years
Sexes Eligible for Study:FEMALE
Accepts Healthy Volunteers:No
Standard Ages:ADULT
Inclusion CriteriaExclusion Criteria
  1. * Women 18 through 64 years of age (inclusive)
  2. * Pulmonary Function Test (PFT) with following criteria:
  3. 1. DLCO \>20% predicted and FVC \<90% OR
  4. 2. Post bronchodilator FEV1 between 30% and 90% predicted.
  5. * Confirmed or possible diagnosis of LAM
  6. * Willing to avoid grapefruit juice and St. John's wort while in the study
  7. * Able and willing to comply with the study procedures
  1. * Women who have or will undergo a transplant
  2. * Women who will undergo surgery
  3. * Women who are currently pregnant or plan on a pregnancy
  4. * Women who are currently breast feeding or lactating
  5. * 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
  6. * Currently taking any of the following medications:
  7. * Antifungal Medications: Ketoconazole; Itraconazole ; Voriconazole.
  8. * Antibiotics for bacterial infections: Clarithromycin.
  9. * Analgesics to treat headaches/migraines: Dihydroergotamine; Dihydroergotamine intranasal
  10. * Antiretroviral protease inhibitors used in human immunodeficiency virus (HIV) infections: Atazanavir ; Nelfinavir; Indinavir; Ritonavir; Saquinavir
  11. * Anti-epileptic or seizure medications: Carbamazepine, Fosphenytoin; Oxcarbamazepine; Phenobarbital ; Phenytoin; Primidone
  12. * Anti-depressant medications: Nefazodone; St. John's wort
  13. * Targeted cancer drugs: Regorafenib; Venetoclax ; Cobimetinib
  14. * 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).
  15. * Non English speaking, illiterate, or other vulnerable persons will not be included among study subjects.
  16. * Any condition that in the opinion of the investigator might adversely influence the study outcome.

Contacts and Locations

Study Contact

Sydney Harris, MBS
CONTACT
2123053745
sh4514@cumc.columbia.edu
Sabrina Palumbo, BS
CONTACT
2123053745
sp4461@cumc.columbia.edu

Principal Investigator

Jeanine D'Armiento, MD, PhD
PRINCIPAL_INVESTIGATOR
Columbia University

Study Locations (Sites)

Columbia University Irving Medical Center
New York, New York, 10032
United States
Medical University of South Carolina
Charleston, South Carolina, 29425
United States

Collaborators and Investigators

Sponsor: Columbia University

  • Jeanine D'Armiento, MD, PhD, PRINCIPAL_INVESTIGATOR, Columbia University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-04-01
Study Completion Date2026-08

Study Record Updates

Study Start Date2025-04-01
Study Completion Date2026-08

Terms related to this study

Keywords Provided by Researchers

  • LAM
  • LAMP-2
  • Imatinib

Additional Relevant MeSH Terms

  • Lymphangioleiomyomatosis (LAM)
  • Lymphangioleiomyomatosis