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

NCT01997255 | Withdrawn Phase 2 DMC

• 2 other identifiers: H-33251CRAD001MUS214T
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Sturge Weber Syndrome

Keywords

Everolimus; Epilepsy; Sturge Weber Syndrome; Port wine stain; Glaucoma

Outcome Measures

Primary Outcomes (1)

• Evaluate the clinical effectiveness of Everolimus as an adjunct treatment to reduce the seizure activity

  The primary efficacy parameter will be the percentage of subjects classified as responders (greater than 50% reduction in seizure frequency during the maintenance phase as compared to the baseline phase) or near-responders (25-50% reduction in seizure frequency during the maintenance phase as compared to the baseline phase), as reported by caregivers via seizure diaries.

  2 years

Secondary Outcomes (2)

• Clinical progression of facial and/or body port-wine hemangioma

  2 years

• Clinical progression of glaucoma

  2 years

Study Arms (1)

Everolimus

EXPERIMENTAL

Afinitor tablets will be administered at a starting dosage of 5 mg/ m2/ day, dosed once per day in the morning. To achieve appropriate doses for all subjects 2mg, 3mg, 5mg of everolimus Disperz tablets will be used. Subjects will take one or more of these tablets in combination to achieve the required dose. Dosages will be rounded to the nearest 2 mg when calculating doses for individual subjects.

Drug: Everolimus

Interventions

Everolimus DRUG

Afinitor tablets will be administered at a starting dosage of 5 mg/ m2/ day, dosed once per day in the morning. To achieve appropriate doses for all subjects 2mg, 3mg, 5mg of everolimus Disperz tablets will be used. Subjects will take one or more of these tablets in combination to achieve the required dose. Dosages will be rounded to the nearest 2 mg when calculating doses for individual subjects.

Also known as: Afinitor, RAD001

Everolimus

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Baylor College of Medicine: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (1)

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Sturge-Weber Syndrome; Epilepsy; Port-Wine Stain; Glaucoma

Interventions

Everolimus

Condition Hierarchy (Ancestors)

Hemangioma; Neoplasms, Vascular Tissue; Neoplasms by Histologic Type; Neoplasms; Neurocutaneous Syndromes; Nervous System Diseases; Angiomatosis; Vascular Diseases; Cardiovascular Diseases; Brain Diseases; Central Nervous System Diseases; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Ocular Hypertension; Eye Diseases

Intervention Hierarchy (Ancestors)

Sirolimus; Macrolides; Lactones; Organic Chemicals

Study Officials

• Angus A Wilfong, MD

  Baylor College of Medicine - Texas Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Pediatrics-Neurology

Study Record Dates

• First Submitted: November 15, 2013
• First Posted: November 28, 2013
• Study Start: April 1, 2014
• Primary Completion: April 1, 2016
• Study Completion: April 1, 2016
• Last Updated: May 5, 2016

Record last verified: 2015-12

Locations

US (1)