Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC) - Full Text View

Sponsored by:	Novartis
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00789828

Purpose

This study will evaluate the safety and efficacy of RAD001 in treating patients with Subependymal Giant Cell Astrocytomas associated with Tuberous Sclerosis Complex.

Condition	Intervention	Phase
Tuberous Sclerosis Lymphangioleiomyomatosis	Drug: RAD001 (Everolimus) Drug: Placebo	Phase III

Genetics Home Reference related topics: familial encephalopathy with neuroserpin inclusion bodies tuberous sclerosis

MedlinePlus related topics: Tuberous Sclerosis

Drug Information available for: Everolimus Sirolimus

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Crossover Assignment, Safety/Efficacy Study
Official Title:	A Randomized, Double-Blind, Placebo-Controlled Study of RAD001 in the Treatment of Patients With Subependymal Giant Cell Astrocytomas (SEGA) Associated With Tuberous Sclerosis Complex (TSC)

Further study details as provided by Novartis:

Primary Outcome Measures:

• Subependymal Giant Cell Astrocytoma response rate through MRIs at screening, 12, 24 and 48 weeks and annually thereafter [ Time Frame: Screening, 12, 24, 48 weeks and annually for 4-5 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

• Time to Subependymal Giant Cell Astrocytoma progression through MRIs at screening, 12, 24 and 48 weeks and annually thereafter [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
• Skin lesion response rate tracked by digital photographs [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
• Change from baseline in biomarkers collected during the first years of study [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
• Changes in renal function by assessing creatinine clearance levels throughout the study [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
• Safety assessed on a continuous basis throughout study [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
• Change from baseline in frequency of epileptiform events monitored by 24 hour EEG at baseline and 6 months [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	99
Study Start Date:	February 2009
Estimated Primary Completion Date:	February 2014 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental	Drug: RAD001 (Everolimus) 1 mg in tablet form
2: Placebo Comparator	Drug: Placebo placebo

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

All Ages
Definite diagnosis of Tuberous Sclerosis
At least one Subependymal Giant Cell Astrocytoma of at least 1 cm in diameter
Evidence of SEGA progression as compared to prior MRI scans
Females of child bearing potential must use birth control

Exclusion Criteria:

Recent heart attack, cardiac related chest pain or stroke
Severely impaired lung function
Prior brain surgery
Severe liver dysfunction
Severe kidney dysfunction
Pregnancy or breast feeding
Current infection
History of organ transplant
Surgery within two months prior to study enrollment
Prior therapy with a medication in the same class as Everolimus
Uncontrolled high cholesterol
Uncontrolled diabetes
HIV
Patients with metal implants thus prohibiting MRI evaluations

Other protocol-defined inclusion/exclusion criteria may apply

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00789828

Locations

United States, Ohio
Cincinnati Children's Hospital
Cincinnati, Ohio, United States, 45229
United Kingdom
Novartis Invstigative Site
Brighton, United Kingdom

Sponsors and Collaborators

Novartis

More Information

Responsible Party:	Novartis ( External Affairs )
Study ID Numbers:	CRAD001M2301
Study First Received:	November 12, 2008
Last Updated:	November 12, 2008
ClinicalTrials.gov Identifier:	NCT00789828
Health Authority:	United States: Food and Drug Administration; Belgium: Pharmaceutical Inspectorate; Germany: Federal Institute for Drugs and Medical Devices; Italy: Ministry of Health; Netherlands: Medicines Evaluation Board; Poland: Drug Institute; United Kingdom: Medicine and Healthcare products Regulatory Agency; Australia: Department of Health and Ageing Therapeutic Goods Administration

Keywords provided by Novartis:

SEGA
Tuberous Sclerosis
Subependymal Giant Cell Astrocytoma
mTOR

RAD001
Mamalian Target Rapamycin
Everolimus
TSC

Study placed in the following topic categories:

Everolimus
Sirolimus
Immunoproliferative Disorders
Astrocytoma
Nervous System Malformations
Lymphangiomyoma
Sclerosis
Neurodegenerative Diseases
Bourneville syndrome
Lymphatic Diseases
Lymphangioleiomyomatosis
Neuroectodermal Tumors

Tuberous Sclerosis
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Tuberous sclerosis
Neoplasms, Germ Cell and Embryonal
Neuroepithelioma
Glioma
Lymphoproliferative Disorders
Malformations of Cortical Development
Congenital Abnormalities
Neoplasms, Glandular and Epithelial
Neurocutaneous Syndromes

Additional relevant MeSH terms:

Neoplasms by Histologic Type
Immune System Diseases
Immunologic Factors
Neoplasms, Nerve Tissue
Nervous System Diseases
Physiological Effects of Drugs
Hamartoma

Immunosuppressive Agents
Pharmacologic Actions
Lymphatic Vessel Tumors
Neoplasms
Pathologic Processes
Neoplasms, Neuroepithelial

ClinicalTrials.gov processed this record on January 16, 2009