Plexiform Neurofibroma Associated With Neurofibromatosis Type 1
Conditions
Interventions
Trade Name: Votubia
Product Name: Everolimus
Product Code: RAD001
Pharmaceutical Form: Tablet
Sponsors
Novartis Pharmaceuticals
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Clinically definite diagnosis of NF1 according to the NIH consensus conference criteria. 2. Patients must have PN that have the potential to cause significant morbidity, such as lesions that could compromise the airway or the great vessels, lesions that could cause nerve compression, lesions that could result in major deformity or significant cosmetic problems 3. Measurable disease: patient must have at least one measurable PN amenable to volumetric MRI analysis. Are the trial subjects under 18? yes Number of subjects for this age range: 6 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 3 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Chronic treatment with systemic steroids or another immunosuppressive agent. 2. Evidence of an active optic glioma, malignant glioma, malignant peripheral nerve sheath tumor, or other cancer requiring treatment with chemotherapy or radiation therapy. 3. Clinical evidence of significantly impaired lung function 4. Pregnancy or breast feeding. 5. Prior therapy with mTOR inhibitors (e.g.sirolimus, temsirolimus, everolimus). 6. No contraindications for MRI assessments
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1. Determine whether RAD001, administrated orally daily on a continuous dosing schedule might: a. Increases time to disease progression (TTP) based on volumetric MRI measurements in children and adults with NF1 in inoperable documented progressive PN (stratum 1). b. 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 study entry (stratum 2). 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.;Secondary Objective: Not applicable;Primary end point(s): Efficacy endpoints Stratum 1: PN TTP will be determined by volumetric MRIs. TTP in stratum 1 will be referred to the the placebo arm of a NCI POB placebo-controlled, double-blind, cross-over, phase II trial of the farnesyltransferase inhibitor tipifarnib for children and young adults with NF1 and progressive PN (NCT00021541). Stratum 2: PN response rate will be determined by volumetric MRIs. PN response rate is defined as the proportion of patients with a reduction in PN volume of at least 20% relative to baseline, where PN volume is the sum of the volumes of all target PN lesions identified at baseline, and confirmed with a second scan at least 1 course later. Safety endpoint Safety will be assessed by the National Cancer Institute's (NCI) Common Toxicity Criteria for Adverse Events (CTCAE), version 4.0;Timepoint(s) of evaluation of this end point: Hematology and chemistry assessment will be done at screening and at each scheduled visit thereafter throughout the study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): n.a.;Timepoint(s) of evaluation of this end point: n.a. | — |
Countries
Israel
Contacts
Public ContactClinical Trial Information Desk
Novartis Pharma AG
Outcome results
None listed