A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas

Completed · Phase 2

Conditions studied: Neurofibromatosis Type 1

In brief

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.

Key facts

Study ID

NCT00634270

Run by

University of Alabama at Birmingham

People needed

58

Starts

2008-04-01

Expected to finish

2015-12-01

Last updated by the study team

2017-11-17

Who can join

Age: 3 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

Where it is running

• The University of Alabama at Birmingham — Birmingham, Alabama, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• University of Chicago — Chicago, Illinois, United States
• National Cancer Institute (NCI) — Bethesda, Maryland, United States
• Children's Hospital Boston — Boston, Massachusetts, United States
• Washington University — St Louis, Missouri, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• University of Utah — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

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