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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
Sponsor:
Collaborator:
Columbia University
Information provided by (Responsible Party):
Charlie Strange, Medical University of South Carolina

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

Condition or disease Intervention/treatment Phase
Lymphangioleiomyomatosis Drug: Imatinib Mesylate 400Mg Capsule Drug: Placebo - Capsule Phase 1 Phase 2

Detailed Description:

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.

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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.
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Sirolimus or everolimus will be withdrawn after 28 days if used at baseline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.
Drug: Placebo - Capsule
Sirolimus or everolimus will be withdrawn after 28 days if used at baseline
Other Name: Placebo




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Adverse Events [ Time Frame: 3 months ]
    Adverse event and Serious Adverse Event numbers using the CTCAE Version 4.03 definitions


Other Outcome Measures:
  1. Lung Function [ Time Frame: 2 months ]
    FEV1 % predicted change

  2. SGRQ [ Time Frame: 2 months ]
    Saint Georges Respiratory Questionnaire change



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Medical University of South Carolina
Columbia University
Investigators
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Study Director: Christopher Meinberg Congressionally Directed Medical Research Programs
  Study Documents (Full-Text)

Documents provided by Charlie Strange, Medical University of South Carolina:
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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

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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
Keywords provided by Charlie Strange, Medical University of South Carolina:
VEGF-D
Imatinib mesylate
Sirolimus
Additional relevant MeSH terms:
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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