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LAM Pilot Study With Imatinib Mesylate (LAMP-1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Lymphangioleiomyomatosis
Interventions Drug: Imatinib Mesylate 400Mg Capsule
Drug: Placebo - Capsule
Enrollment 18
Recruitment Details 18 Subjects signed consent, 2 subjects failed baseline evaluation and were not randomized. 1 subject was randomized, took 4 placebo pills and stopped (without side effects) and is deemed not evaluable.
Pre-assignment Details  
Arm/Group Title Imatinib Mesylate 400mg Capsule Placebo Capsule
Hide Arm/Group Description

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.

Imatinib Mesylate 400Mg Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity.

Placebo - Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline
Period Title: Overall Study
Started 8 8
Completed 8 7
Not Completed 0 1
Reason Not Completed
Withdrawal by Subject             0             1
Arm/Group Title Imatinib Mesylate 400mg Capsule Placebo Capsule Total
Hide Arm/Group Description

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.

Imatinib Mesylate 400Mg Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity.

Placebo - Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

 Total of all reporting groups
Overall Number of Baseline Participants 8 7 15
Hide Baseline Analysis Population Description
Withdrawal after 4 pills of placebo is not included in baseline population
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants 7 participants 15 participants
45  (12) 50  (11) 47.7  (12)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 7 participants 15 participants
Female
8
 100.0%
7
 100.0%
15
 100.0%
Male
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 7 participants 15 participants
American Indian or Alaska Native
1
  12.5%
0
   0.0%
1
   6.7%
Asian
1
  12.5%
1
  14.3%
2
  13.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
  12.5%
1
  14.3%
2
  13.3%
White
5
  62.5%
5
  71.4%
10
  66.7%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Forced Expiratory Volume in 1 Second  
Geometric Mean (Standard Deviation)
Unit of measure:  Percent predicted
Number Analyzed 8 participants 7 participants 15 participants
59  (23.1) 60  (15.4) 59.5  (18.8)
1.Primary Outcome
Title Serum VEGF-D
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Imatinib Mesylate 400mg Capsule Placebo Capsule
Hide Arm/Group Description:

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.

Imatinib Mesylate 400Mg Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity.

Placebo - Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline
Overall Number of Participants Analyzed 8 7
Mean (Standard Deviation)
Unit of Measure: Log transformed VEGF-D change pg/dL
0.05  (0.10) 0.07  (0.13)
2.Secondary Outcome
Title Adverse Events
Hide Description Adverse event and Serious Adverse Event numbers using the CTCAE Version 4.03 definitions
Time Frame 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Imatinib Mesylate 400mg Capsule Placebo Capsule
Hide Arm/Group Description:

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.

Imatinib Mesylate 400Mg Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity.

Placebo - Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline
Overall Number of Participants Analyzed 8 7
Measure Type: Number
Unit of Measure: number of events
Adverse Events 10 12
Serious Adverse Events 0 0
3.Other Pre-specified Outcome
Title Lung Function
Hide Description FEV1 % predicted change
Time Frame 2 months
Outcome Measure Data Not Reported
4.Other Pre-specified Outcome
Title SGRQ
Hide Description Saint Georges Respiratory Questionnaire change
Time Frame 2 months
Outcome Measure Data Not Reported
Time Frame 3 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Imatinib Mesylate 400mg Capsule Placebo Capsule
Hide Arm/Group Description

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.

Imatinib Mesylate 400Mg Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity.

Placebo - Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline
All-Cause Mortality
Imatinib Mesylate 400mg Capsule Placebo Capsule
Affected / at Risk (%) Affected / at Risk (%)
Total   0/8 (0.00%)      0/7 (0.00%)    
Hide Serious Adverse Events
Imatinib Mesylate 400mg Capsule Placebo Capsule
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/8 (0.00%)      0/7 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Imatinib Mesylate 400mg Capsule Placebo Capsule
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   8/8 (100.00%)      5/7 (71.43%)    
Gastrointestinal disorders     
Nausea and/or vomitting †  4/8 (50.00%)  4 4/7 (57.14%)  4
Diarrhea †  1/8 (12.50%)  1 2/7 (28.57%)  2
General disorders     
Fatigue †  3/8 (37.50%)  3 0/7 (0.00%)  0
Insomnia †  0/8 (0.00%)  2/7 (28.57%)  2
Nervous system disorders     
Headache †  0/8 (0.00%)  0 3/7 (42.86%)  3
Respiratory, thoracic and mediastinal disorders     
Elevated Trough Sirolimus †  2/8 (25.00%)  2 1/7 (14.29%)  1
Hypoxemia †  1/8 (12.50%)  1 0/7 (0.00%)  0
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Charlton Strange, MD
Organization: Medical University of South Carolina
Phone: 843-792-3174
EMail: strangec@musc.edu
Layout table for additonal information
Responsible Party: Charlie Strange, Medical University of South Carolina
ClinicalTrials.gov Identifier: NCT03131999    
Other Study ID Numbers: PRO00044389
First Submitted: April 24, 2017
First Posted: April 27, 2017
Results First Submitted: February 25, 2020
Results First Posted: March 26, 2020
Last Update Posted: June 16, 2020