A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas
Status:
Completed
Trial end date:
2015-12-01
Target enrollment:
Participant gender:
Summary
Treatment Overview
This phase II study will evaluate the activity of sirolimus in children and adults with NF1
and inoperable plexiform neurofibromas that have the potential to cause significant
morbidity. The following disease strata will be studied:
Stratum 1: Progressive plexiform neurofibroma(s) that have the potential to cause significant
morbidity. The endpoint will be time to tumor progression based on volumetric tumor
measurements.
Stratum 2: Plexiform neurofibromas without documented radiographic progression at trial
entry. The endpoint will be radiographic response. As of May 2009, Stratum 2 was closed to
enrollment. Stratum 1 is active.
Phase:
Phase 2
Details
Lead Sponsor:
University of Alabama at Birmingham
Collaborators:
Boston Children's Hospital Boston Children’s Hospital Children's Hospital Medical Center, Cincinnati Children's Hospital of Philadelphia Children's National Research Institute Children's Research Institute National Cancer Institute (NCI) University of Chicago University of Utah Washington University School of Medicine