Conditions: Plexiform Neurofibroma Associated With Neurofibromatosis Type 1
Keywords: RAD001, Everolimus, Plexiform Neurofibroma, Neurofibromatosis Type 1
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 9
Healthy volunteers: no

Brief summary:
This study was to 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 was 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.

Detailed description:
Approximately 20 patients were to be enrolled to receive everolimus in an open label manner. A total of 9 patients were enrolled to either Stratum 1 or Stratum 2.

The study was open for enrollment up to 2 years. Because the target enrollment was not achieved in this period, study was terminated with less patient than planned.

Arms:
arm 1: enrolled patients received everolimus (RAD001) in an open label manner. Recommended starting dose of everolimus depend on body surface area, starting from 2.5 mg once daily to 7.5 mg once daily.

Interventions: intervention 1: Everolimus (RAD001)
Intervention descriptions: intervention 1: oral daily dosing of tablet starting with 2.5 mg

Protocol document: