drugset

drugset / Trial / NCT00457808

Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

NCT00457808 ↗

Phase 2 Completed 25 enrolled Children's Hospital Medical Center, Cincinnati The LAM Foundation · collab Tuberous Sclerosis Alliance · collab

Non-randomizedSingle-groupOpen-labelTreatment

Summary

The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.

Timeline

Start: 2002-12
Primary completion: —
Completion: 2006-03

Publications

Bissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, Schmithorst VJ, Laor T, Brody AS, Bean J, Salisbury S, Franz DN. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med. 2008 Jan 10;358(2):140-51. doi: 10.1056/NEJMoa063564.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Sirolimus	Small molecule	—	—

Indications