Neoplasms

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Nervous System Diseases

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Astrocytoma

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Tuberous Sclerosis

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Neoplasms, Neuroepithelial

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Tuberous Sclerosis Complex

Subependymal Giant Cell Astrocytoma

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Everolimus

Sirolimus

Temsirolimus

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Immunologic Factors

As this was a non-randomized, open-label, single arm study, all patients in the study received treatment with everolilmus

Initial everolimus dosage will be 3 mg/m2/d taken daily or every other day, with titration to achieve a trough serum level of 5-15 ng/ml. Patients unable to tolerate levels in this range will have doses held or reduced 25% to achieve trough serum levels of 5-10 ng/ml. If trough serum level of 5-15 ng/ml is not achieved at a dosage of 3 mg/m2/d, then dosage escalation by 25% will be undertaken as tolerated. Everolimus will be similarly adjusted during the extension phase. If a subject misses more than 10 consecutive everolimus doses, additional days of everolimus treatment will be added so total duration of treatment is 6 months. If this occurs, dates of subsequent study events will be adjusted according to number of missed doses. This process will also be followed in the extension phase.

David N Franz, M.D.

The purpose of the study is to evaluate the safety and potential side effects of everolimus (an experimental drug) on a person with Tuberous Sclerosis Complex who also has been diagnosed with a brain tumor (astrocytoma)

The hypothesis is that the drug will cause the tumor size to decrease, and may have beneficial activity separate from effects on tumors in patients.

Tuberous Sclerosis Complex (TSC)is a genetic disorder with a birth incidence of approximately one in six thousand. Five to twenty percent of patients with TSC will develop astrocytoma, a slowly progressive tumor. They grow and cause damage to surrounding brain tissue, blockage of spinal fluid (hydrocephalus), blindness, trouble walking, seizures, and brain damage. If untreated, they can be fatal. Standard treatment involves surgery to remove the tumor; however surgery may itself cause brain damage, bleeding, or infection, as well as other complications. Studies have shown that everolimus suppresses the chemicals that cause tumors to grow in tuberous sclerosis, and may cause them to shrink.

The primary objective of this study is to find out the effects of everolimus on astrocytomas in a six month trial in patients with Tuberous Sclerosis who have been diagnosed with an astrocytoma

Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex

Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex

Novartis

Children's Hospital Medical Center, Cincinnati

This was a non-randomized, open-label, single arm study; all patients in the study received treatment with everolimus.

Age, Categorical

Sex: Female, Male

Race (NIH/OMB)

United States

Region of Enrollment

Dr. David Franz

Upper Respiratory Tract Infection

Stomatitis

Sinusitis

Mouth Ulceration

Cellulitis

Diarrhoea

Gastroenteritis

Otitis Media

Pyrexia

Vomiting

Acne

Convulsion

Nasopharyngitis

Rhinitis Allergic

Conjunctivitis

Pharyngitis

Rash

Constipation

Cough

Dermatitis Acneiform

Dermatitis Contact

Excoriation

Laceration

Nasal Congestion

Otitis Externa

Abnormal Behaviour

Body Tinea

Dry Skin

Pneumonia

Skin Infection

Urinary Tract Infection

This was a non-randomized, open-label, single arm study; all patients in the study received treatment with everolimus. The initial starting dose was to be 3.0 mg/m2/day taken either daily or every other day with titration to achieve target trough concentrations of 5 to 15 ng/mL, subject to tolerability. Study drug was self-administered orally (or administered by a caregiver) at the same time each day.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex	NCT00411619	Tuberous Sclero... Subependymal Gi...	Everolimus	3 Years -	Children's Hospital Medical Center, Cincinnati
Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex	NCT00411619	Tuberous Sclero... Subependymal Gi...	Everolimus	3 Years -	Children's Hospital Medical Center, Cincinnati

Spots Global Cancer Trial Database for subendymal giant cell astrocytomas (sega's)