Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibro… (NCT01365468) | Clinical Trial Compass
TerminatedPhase 2
Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)
Stopped: Poor patients' accrual
Israel9 participantsStarted 2012-04
Plain-language 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.
Who can participate
Age range6 Years
SexALL
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Inclusion criteria
✓. Clinically definite diagnosis of NF1 according to the NIH consensus conference criteria.
✓. 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
✓. Measurable disease: patient must have at least one measurable PN amenable to volumetric MRI analysis.
Exclusion criteria
✕. Chronic treatment with systemic steroids or another immunosuppressive agent.
✕. Evidence of an active optic glioma, malignant glioma, malignant peripheral nerve sheath tumor, or other cancer requiring treatment with chemotherapy or radiation therapy.
✕. Clinical evidence of significantly impaired lung function
✕. Pregnancy or breast feeding.
✕. Prior therapy with mTOR inhibitors (e.g.sirolimus, temsirolimus, everolimus).
What they're measuring
1
Time to Disease Progression (TTP) Based on Change in Volumetric MRI Measurements in Children and Adults (In Stratum I Only)
Timeframe: Screening, after course #6, #12, #18, #24, End of Treatment(1 course=28days)
2
Number of Patients With Objective Radiographic Responses Based on Volumetric MRI Measurements (In Stratum 2 Only)
Timeframe: Screening, after course #6, then every 6 months and end of treatment(1 course=28days)
3
Number of Patients With Adverse Events Assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04
Timeframe: From the time ICF was signed until 28 days after End of Treatment (up to a maximum of 25 months)