The study will evaluate the safety of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC).
Conditions
Interventions
Product Name: RAD001 2,5 mg
Product Code: RAD001
Pharmaceutical Form: Tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Male or female of age 3 years or older • Clinically definite diagnosis of tuberous sclerosis according to the modified Gomez criteria (Roach 1998, Hyman 2000, Table 5-1). Clinically definite diagnosis is defined as either of the following: • Two Major Features from Table 5-1. • One Major Feature plus two Minor Features from Table 5-1. • Presence of at least one SEGA lesion identified by MRI or CT (according to local requirements). Note: SEGA lesions are only diagnosed in patients with TSC. They arise in the subependymal layer of the lateral ventricle and are usually located near the foramen of Monroe and enhance homogeneously with contrast on MRI with no evidence of surrounding edema. • If female and of child-bearing potential, documentation of negative pregnancy test prior to enrollment. Sexually active pre-menopausal female patients (and female partners of male patients) must use adequate contraceptive measures while on study and for up to 8 weeks after ending treatment. • Written informed consent according to local guidelines Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Patients for whom SEGA-related surgery is required and planned • History of myocardial infarction, angina or stroke related to atherosclerosis • Known impaired lung function (e.g. FEV1 or DLCO = 70% of predicted) • Significant hematological or hepatic abnormality (i.e. transaminase levels > 2.5 x ULN or serum bilirubin > 1.5 x ULN, hemoglobin 300 mg/dL OR >7.75 mmol/L AND fasting triglycerides > 2.5 x ULN • Uncontrolled diabetes mellitus as defined by fasting serum glucose > 1.5 x ULN • Patients with bleeding diathesis or on oral anti-vitamin K medication • Serum creatinine > 1.5 x ULN. • Any severe and/or uncontrolled medical conditions which could cause unacceptable safety risks: • uncontrolled hypercholesterolemia/hypertriglyceridemia • Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of study drug (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome). • Patients with a known hypersensitivity to RAD001 or other rapamycin analogs (sirolimus, temsirolimus), or to its excipients • For the purpose of MRI assessments (if applicable): • Ferromagnetic metal implants other than those approved as safe for use in MR scanner (e.g., braces, some types of aneurysm clips, shrapnel) • Patients suffering from uncontrollable claustrophobia or physically unable to fit into the machine (e.g. obesity) • Patients with known history of HIV seropositivity • Inability to attend scheduled clinic visits and noncompliance with scheduled clinic visits required for dose monitoring, dose adjustment and toxicity managment • Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods. If barrier contraceptives are used, they must be continued throughout the study by both sexes
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate safety of RAD001 in patients with SEGA associated with TSC.;Secondary Objective: To evaluate the investigator’s best overall response rate of RAD001 in patients with SEGA associated with TSC.;Primary end point(s): Safety: Grade 3 and 4 Adverse Events, Serious Adverse Events | — |
Countries
Belgium, Czech Republic, Germany, Greece, Hungary, Italy, Spain
Outcome results
None listed