A Study of Nintedanib for LymphAngioleioMyomatosis (LAM)

Summary

This trial is conducted locally. The aim of this trial is assess the efficacy and a favorable benefit-risk ratio for nintedanib in the treatment of LAM at the dose of 150 mg bid

Timeline

Start

2016-12-06

Primary completion

2021-12

Completion

2021-12

Publications

• Background Ma L, Chen Z, Erdjument-Bromage H, Tempst P, Pandolfi PP. Phosphorylation and functional inactivation of TSC2 by Erk implications for tuberous sclerosis and cancer pathogenesis. Cell. 2005 Apr 22;121(2):179-93. doi: 10.1016/j.cell.2005.02.031.
• Results Wollin L, Maillet I, Quesniaux V, Holweg A, Ryffel B. Antifibrotic and anti-inflammatory activity of the tyrosine kinase inhibitor nintedanib in experimental models of lung fibrosis. J Pharmacol Exp Ther. 2014 May;349(2):209-20. doi: 10.1124/jpet.113.208223. Epub 2014 Feb 20.
• Results Inoki K, Li Y, Zhu T, Wu J, Guan KL. TSC2 is phosphorylated and inhibited by Akt and suppresses mTOR signalling. Nat Cell Biol. 2002 Sep;4(9):648-57. doi: 10.1038/ncb839.
• Results Crino L, Metro G. Therapeutic options targeting angiogenesis in nonsmall cell lung cancer. Eur Respir Rev. 2014 Mar 1;23(131):79-91. doi: 10.1183/09059180.00008913.
• Harari S, Elia D, Caminati A, Geginat J, Luisi F, Pelosi G, Specchia C, Torre O, Trevisan R, Vasco C, Zompatori M, Cassandro R. Nintedanib for patients with lymphangioleiomyomatosis: a phase 2, open-label, single-arm study. Lancet Respir Med. 2024 Dec;12(12):967-974. doi: 10.1016/S2213-2600(24)00217-0. Epub 2024 Oct 25.

Drugs

Indications