Plexiform Neurofibroma Associated With Neurofibromatosis Type 1 Clinical Trial
Official title:
A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)
This study will evaluate the antitumor activity and safety of RAD001 in patients with
Plexiform neurofibromas (PN) associated with Neurofibromatosis Type 1 (NF1).
The aim of the study is to :
1. determine whether RAD001, administrated orally daily on a continuous dosing schedule
might:
1. Increases time to disease progression (TTP) based on volumetric MRI measurements
in children and adults with NF1 in inoperable documented progressive PN (stratum
1).
2. Results in objective radiographic responses based on volumetric MRI measurements
in children and adults with NF1 and inoperable PN in the absence of documented
radiographic progression at the trail entry (stratum
2. To evaluate the tolerability and toxicity of chronic RAD001 administration in this
patient population as assessed by the NCI Common Toxicity Criteria, version 4.0.
n/a
Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment