LAM Pilot Study With Imatinib Mesylate

Phase 2CompletedNCT03131999

Medical University of South Carolina18 enrolled

Overview

This is a phase 1 clinical trial comparing imatinib mesylate to placebo for individuals with lymphangioleiomyomatosis (LAM).

This is a double blind, adjusted parallel design, randomized clinical trial comparing imatinib mesylate 400 mg daily or matching placebo on the primary outcome of log transformed serum VEGF-D level in patients with LAM. Sirolimus using patients will have co-administration of Imatinib mesylate or placebo for 28 days prior to sirolimus discontinuation. The duration of 400 mg imatinib mesylate or placebo will be 56 days, a dose reduction is allowed for toxicity. The primary endpoint will be the change in the log transformed VEGF-D one month after monotherapy imatinib mesylate or placebo. Total trial duration is 2 months of drug administration.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Lymphangioleiomyomatosis

Interventions

Imatinib Mesylate 400Mg CapsuleDRUG

Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

Placebo - CapsuleDRUG

Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Definite or Probable LAM * FVC or Postbronchodilator FEV1 \<90% predicted Exclusion Criteria: * Current or planned pregnancy or lactation * Unwillingness to discontinue sirolimus * Change in the dose or use of sirolimus within the past month * Inability to perform spirometry * Allergy or intolerance of albuterol and/or ipratropium * Other serious illness that would impact the outcome of the study including cancer that has not received curative therapy, Grade III/IV cardiac problems as defined by the New York Heart Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6 months of study), uncontrolled diabetes, chronic renal disease, chronic liver disease, or active uncontrolled infection * Current lung transplant * Known diagnosis of human immunodeficiency virus (HIV) infection * Current cigarette smoking * Required use of warfarin, ketoconazole, itraconazole, clarithromycin, or rifampin during the 2 months of the study. * Unwillingness to avoid grapefruit juice or St. Johns Wort during the study. * Planned surgery during the 2 months of the study. * Patient with any significant history of non-compliance to medical regimens or with inability to grant reliable informed consent. * Patient has received and other investigational agents within 28 days of first day of study drug dosing.

Locations (2)

Columbia University

New York, New York, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Outcomes

Primary Outcomes

Serum VEGF-D

Change in the square root of the intrasubject plasma VEGF-D

Time frame: Before and 1 month after initiation of monotherapy imatinib mesylate or placebo

Secondary Outcomes

Adverse Events

Adverse event and Serious Adverse Event numbers using the CTCAE Version 4.03 definitions

Time frame: 3 months

Data from ClinicalTrials.gov

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