LAM Pilot Study With Imatinib Mesylate (LAMP-1)
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ClinicalTrials.gov Identifier: NCT03131999 |
Recruitment Status :
Completed
First Posted : April 27, 2017
Results First Posted : March 26, 2020
Last Update Posted : June 16, 2020
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Condition or disease | Intervention/treatment | Phase |
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Lymphangioleiomyomatosis | Drug: Imatinib Mesylate 400Mg Capsule Drug: Placebo - Capsule | Phase 1 Phase 2 |
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 (Clinical Trial) |
Actual Enrollment : | 18 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | LAM Pilot Study With Imatinib Mesylate |
Actual Study Start Date : | January 23, 2018 |
Actual Primary Completion Date : | March 7, 2019 |
Actual Study Completion Date : | March 7, 2019 |

Arm | Intervention/treatment |
---|---|
Experimental: Imatinib Mesylate 400mg capsule
56 days of Imatinib mesylate 400 mg oral daily with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction to 200 mg daily is allowed for toxicity.
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Drug: Imatinib Mesylate 400Mg Capsule
Sirolimus or everolimus will be withdrawn after 28 days if used at baseline
Other Name: Gleevec |
Placebo Comparator: Placebo Capsule
56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity.
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Drug: Placebo - Capsule
Sirolimus or everolimus will be withdrawn after 28 days if used at baseline
Other Name: Placebo |
- Serum VEGF-D [ Time Frame: Before and 1 month after initiation of monotherapy imatinib mesylate or placebo ]Change in the square root of the intrasubject plasma VEGF-D
- Adverse Events [ Time Frame: 3 months ]Adverse event and Serious Adverse Event numbers using the CTCAE Version 4.03 definitions
- Lung Function [ Time Frame: 2 months ]FEV1 % predicted change
- SGRQ [ Time Frame: 2 months ]Saint Georges Respiratory Questionnaire change

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03131999
United States, New York | |
Columbia University | |
New York, New York, United States, 10032 | |
United States, South Carolina | |
Medical University of South Carolina | |
Charleston, South Carolina, United States, 29425 |
Study Director: | Christopher Meinberg | Congressionally Directed Medical Research Programs |
Documents provided by Charlie Strange, Medical University of South Carolina:
Responsible Party: | Charlie Strange, Professor of Pulmonary and Critical Care Medicine, Medical University of South Carolina |
ClinicalTrials.gov Identifier: | NCT03131999 |
Other Study ID Numbers: |
PRO00044389 |
First Posted: | April 27, 2017 Key Record Dates |
Results First Posted: | March 26, 2020 |
Last Update Posted: | June 16, 2020 |
Last Verified: | June 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | Yes |
VEGF-D Imatinib mesylate Sirolimus |
Lymphangioleiomyomatosis Lymphangiomyoma Lymphatic Vessel Tumors Neoplasms by Histologic Type Neoplasms Perivascular Epithelioid Cell Neoplasms Neoplasms, Connective and Soft Tissue Lymphoproliferative Disorders |
Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Imatinib Mesylate Antineoplastic Agents Protein Kinase Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action |