58
Participants
Start Date
April 30, 2008
Primary Completion Date
November 30, 2014
Study Completion Date
December 31, 2015
Sirolimus
This phase II study will evaluate children and adults with neurofibromatosis type-1 (NF1) and plexiform neurofibromas treated with sirolimus. It is divided in two strata. The first stratum will evaluate time to progression (TTP) in children and adults with NF1 and progressive plexiform neurofibromas with the potential to cause significant morbidity treated with sirolimus. The second stratum will evaluate objective radiographic response to sirolimus in children and adults with NF1 and inoperable plexiform neurofibromas with the potential to cause significant morbidity that do not have documented progression of the PN at time of trial entry.
Children's Hospital of Philadelphia, Philadelphia
Children's National Medical Center, Washington D.C.
National Cancer Institute (NCI), Bethesda
The University of Alabama at Birmingham, Birmingham
University of Chicago, Chicago
Washington University, St Louis
University of Utah, Salt Lake City
Children's Hospital Boston, Boston
Cincinnati Children's Hospital Medical Center, Cincinnati
Collaborators (1)
Boston Children's Hospital
OTHER
Children's Hospital of Philadelphia
OTHER
Children's National Research Institute
OTHER
Children's Hospital Medical Center, Cincinnati
OTHER
National Cancer Institute (NCI)
NIH
University of Chicago
OTHER
University of Utah
OTHER
Washington University School of Medicine
OTHER
University of Alabama at Birmingham
OTHER