MedTrace Logo

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

NCT06889168 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2026-01-21

No results posted yet for this study

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. 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.

Conditions

  • Lymphangioleiomyomatosis (LAM)
  • Lymphangioleiomyomatosis

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.

Sponsors & Collaborators

  • United States Department of Defense

    collaborator FED

  • Medical University of South Carolina

    collaborator OTHER

  • Columbia University

    lead OTHER

Principal Investigators

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

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
64 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-20
Primary Completion
2026-12-31
Completion
2027-03-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06889168 on ClinicalTrials.gov