Adjunctive Everolimus (RAD 001) Therapy for Epilepsy in Children With Sturge-Weber Syndrome (SWS)


Phase 2 Results N/A

Eligibility Criteria

Inclusion Criteria

- 2-18 years of age, male and female.
- Subject weights greater than or equal to 6 kg at study entry.
- Subjects clinically diagnosed with SWS, inclusive of cerebral and dermatologic involvement. Ophthalmic involvement will be monitored if present, but is not necessary for enrollment.
- History of epilepsy with a history of at least 4 seizures in the month prior to screening.
- Medically refractory epilepsy defined as failure of two or more approved antiepileptic therapies.
- Females of child-bearing potential must use highly effective contraception during the study and for 8 weeks after stopping treatment.
- Sexually active males must use a condom during intercourse while taking study drug, and for 8 weeks after stopping study treatment.
- Adequate bone marrow function.
- Adequate liver function.
- Adequate renal function.
- Acceptable fasting serum cholesterol and fasting triglycerides levels.

Exclusion Criteria

- Patients currently receiving anticancer therapies or who have received anticancer therapies within 4 weeks of the start of Everolimus (including chemotherapy, radiation therapy, antibody based therapy, etc.).
- Known intolerance or hypersensitivity to Everolimus or other rapamycin analogs.
- Known impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral Everolimus.
- Uncontrolled diabetes mellitus despite adequate therapy.
- Patients who have any severe and/or uncontrolled medical conditions.
- Chronic treatment with corticosteroids or other immunosuppressive agents. Topical or inhaled corticosteroids are allowed.
- Known history of HIV seropositivity.
- Patients who have received live attenuated vaccines within 1 week of start of Everolimus and during the study.
- Patients who have a history of another primary malignancy, with the exceptions of: non-melanoma skin cancer, and carcinoma in situ of the cervix, uteri, or breast from which the patient has been disease free for ≥3 years.
- Patients with a history of non-compliance to medical regimens or who are considered potentially unreliable or will not be able to complete the entire study.
- Patients who are currently part of or have participated in any clinical investigation with an investigational drug within 1 month prior to dosing.
- Pregnant or nursing (lactating) women.