NCT03131999

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jan 2018

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 24, 2017

Completed
3 days until next milestone

First Posted

Study publicly available on registry

April 27, 2017

Completed
9 months until next milestone

Study Start

First participant enrolled

January 23, 2018

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 7, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 7, 2019

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

March 26, 2020

Completed
Last Updated

June 16, 2020

Status Verified

June 1, 2020

Enrollment Period

1.1 years

First QC Date

April 24, 2017

Results QC Date

February 25, 2020

Last Update Submit

June 4, 2020

Conditions

Lymphangioleiomyomatosis

Keywords

VEGF-DImatinib mesylateSirolimus

Outcome Measures

Primary Outcomes (1)

  • Serum VEGF-D

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

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

Secondary Outcomes (1)

  • Adverse Events

    3 months

Other Outcomes (2)

  • Lung Function

    2 months

  • SGRQ

    2 months

Study Arms (2)

Imatinib Mesylate 400mg capsule

EXPERIMENTAL

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

Placebo Capsule

PLACEBO COMPARATOR

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

Interventions

Imatinib Mesylate 400Mg CapsuleDRUG

Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

Also known as: Gleevec
Imatinib Mesylate 400mg capsule
Placebo - CapsuleDRUG

Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

Also known as: Placebo
Placebo Capsule

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Definite or Probable LAM
  • FVC or Postbronchodilator FEV1 \<90% predicted

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Columbia University

New York, New York, 10032, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

MeSH Terms

Conditions

Lymphangioleiomyomatosis

Interventions

Imatinib Mesylate

Condition Hierarchy (Ancestors)

LymphangiomyomaNeoplasm, Lymphatic TissueNeoplasms by Histologic TypeNeoplasmsPerivascular Epithelioid Cell NeoplasmsNeoplasms, Connective and Soft TissueLymphoproliferative DisordersLymphatic DiseasesHemic and Lymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Intervention Hierarchy (Ancestors)

BenzamidesAmidesOrganic ChemicalsBenzoatesAcids, CarbocyclicCarboxylic AcidsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsPiperazinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsPyrimidines

Results Point of Contact

Title
Charlton Strange, MD
Organization
Medical University of South Carolina
strangec@musc.edu
843-792-3174

Study Officials

  • Christopher Meinberg

    Congressionally Directed Medical Research Programs

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor of Pulmonary and Critical Care Medicine

Study Record Dates

First Submitted

April 24, 2017

First Posted

April 27, 2017

Study Start

January 23, 2018

Primary Completion

March 7, 2019

Study Completion

March 7, 2019

Last Updated

June 16, 2020

Results First Posted

March 26, 2020

Record last verified: 2020-06

Data Sharing

IPD Sharing
Will not share

Locations

US(2)