Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM

Phase 2 Unknown 14 enrolled Cardiff University Royal Sussex County Hospital · collab St Georges Hospital Medical School · collab The Tuberous Sclerosis Association · collab University of Nottingham · collab Wyeth is now a wholly owned subsidiary of Pfizer · collab

Non-randomizedSingle-groupOpen-labelTreatment

Summary

The purpose of this study is to determine the safety and efficacy of the mTOR inhibitor sirolimus as a treatment for renal angiomyolipomas in patients with tyberous sclerosis complex or sporadic lymphangioleiomyomatosis.

Timeline

Start: 2005-10
Primary completion: 2009-09
Completion: 2009-09

Publications

Davies DM, de Vries PJ, Johnson SR, McCartney DL, Cox JA, Serra AL, Watson PC, Howe CJ, Doyle T, Pointon K, Cross JJ, Tattersfield AE, Kingswood JC, Sampson JR. Sirolimus therapy for angiomyolipoma in tuberous sclerosis and sporadic lymphangioleiomyomatosis: a phase 2 trial. Clin Cancer Res. 2011 Jun 15;17(12):4071-81. doi: 10.1158/1078-0432.CCR-11-0445. Epub 2011 Apr 27.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Sirolimus	Small molecule	—	Oral

Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM

Summary

Timeline

Publications

Drugs

Indications