drugset / Trial / NCT00634270
A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas
Phase 2
Completed
58 enrolled
University of Alabama at Birmingham
Boston Children's Hospital · collabChildren's Hospital Medical Center, Cincinnati · collabChildren's Hospital of Philadelphia · collabChildren's National Research Institute · collabNational Cancer Institute (NCI) · collabUniversity of Chicago · collabUniversity of Utah · collabWashington University School of Medicine · collab
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2008-04
- Primary completion
- 2014-11
- Completion
- 2015-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Sirolimus | Small molecule | 0.8 mg/m2 | Oral |