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

• Register: ClinicalTrials.gov
• Last refreshed on: 17 May 2016
• Main ID: NCT01997255
• Date of registration: 15/11/2013
• Prospective Registration: Yes
• Primary sponsor: Baylor College of Medicine
• Public title: Adjunctive Everolimus (RAD 001) Therapy for Epilepsy in Children With Sturge-Weber Syndrome (SWS)
• Scientific title: An Open-Label Controlled Study of Adjunctive Everolimus (RAD 001) Therapy for Epilepsy in Children With Sturge-Weber Syndrome
• Date of first enrolment: April 2014
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT01997255
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Angus A Wilfong, MD
• Affiliation: Baylor College of Medicine/Texas Children's Hospital

Key inclusion & exclusion 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.



Age minimum: 2 Years
Age maximum: 18 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Sturge Weber Syndrome

Intervention(s)

Drug: Everolimus

Primary Outcome(s)

Evaluate the clinical effectiveness of Everolimus as an adjunct treatment to reduce the seizure activity [Time Frame: 2 years]

Secondary Outcome(s)

Clinical progression of facial and/or body port-wine hemangioma [Time Frame: 2 years]

Clinical progression of glaucoma [Time Frame: 2 years]

Secondary ID(s)

CRAD001MUS214T

H-33251

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Novartis Pharmaceuticals