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

Part of paid clinical trials in New York, New York.

Sponsor
Columbia University
Study ID
NCT06889168
Phase
PHASE1
Status
Recruiting
Apply to this trial

Conditions

  • Lymphangioleiomyomatosis
  • Lymphangioleiomyomatosis (LAM)

Eligibility Criteria

Sex
FEMALE
Age
18 Years - 64 Years
Healthy Volunteers
Not accepted

Interventions

  • Imatimib Mesylate — DRUG
    Participants will take Imatinib mesylate (imatinib), an FDA approved drug for leukemia, orally 400 mg (twice daily)
  • Placebo — DRUG
    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 Details

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.

Key Dates

Start date
Oct 20, 2025
Status verified
Jan 2026
Primary completion
Dec 31, 2026
Completion
Mar 31, 2027

Study Design

Enrollment
20 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

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.

Primary Outcome Measure

Incidence of Adverse Events [ Time Frame: 1 year ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
Columbia University Irving Medical CenterNew YorkNew York10032
Katarina Koumbourlis
[email protected]
212-305-3745
Sabrina Palumbo, BS
[email protected]
212-305-3745
Jeanine D'Armiento, MD, PhD (PRINCIPAL_INVESTIGATOR)
Monica Goldklang, MD (SUB_INVESTIGATOR)
Medical University of South CarolinaCharlestonSouth Carolina29425
Seth Polk
[email protected]
7573341517
Charlie Strange, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in New York, NY

By research site
Columbia University Irving Medical Center· New York, NYMedical University of South Carolina· Charleston, SC

Related Studies