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

• Status: Withdrawn
• Trial end date: 2016-04-01
• Target enrollment: 0
• Participant gender: All

Summary

Sturge Weber Syndrome (SWS) is a rare disease that affects the patient's brain and causes benign (non-cancerous) tumors to grow in the brain. One of the symptoms of SWS is epilepsy. People with epilepsy have seizures. Some patients may also have eye problems and a red mark on their facial skin.

This study is being done to find out if the study drug, everolimus, is safe and has helpful effects in patients with SWS who have seizures and are not responding to their current anti-epileptic medication.

The study drug, everolimus (Afinitor®), is supplied by Novartis Pharmaceuticals Corporation.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Baylor College of Medicine
• Collaborator: Novartis Pharmaceuticals
• Treatments: Everolimus; Sirolimus

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.