NCT03131999

LAM Pilot Study With Imatinib Mesylate

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Medical University of South Carolina · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

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.

Conditions

• Lymphangioleiomyomatosis

Interventions

Type	Name	Description
DRUG	Imatinib Mesylate 400Mg Capsule	Sirolimus or everolimus will be withdrawn after 28 days if used at baseline
DRUG	Placebo - Capsule	Sirolimus or everolimus will be withdrawn after 28 days if used at baseline

Timeline

• Start date: 2018-01-23
• Primary completion: 2019-03-07
• Completion: 2019-03-07
• First posted: 2017-04-27
• Last updated: 2020-06-16
• Results posted: 2020-03-26

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study