Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - Full Text View

Sponsor:	Novartis Pharmaceuticals
Information provided by (Responsible Party):	Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:	NCT00790400

Purpose

This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipomas associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.

Condition	Intervention	Phase
Tuberous Sclerosis Complex (TSC) Lymphangioleiomyomatosis (LAM)	Drug: Everolimus (RAD001) Other: Placebo	Phase III

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Resource links provided by NLM:

Genetics Home Reference related topics: lymphangioleiomyomatosis pyridoxine-dependent epilepsy tuberous sclerosis complex

MedlinePlus related topics: Tuberous Sclerosis

Drug Information available for: Sirolimus Everolimus CCI 779

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:

• Angiomyolipoma response rate through CT/MRIs at screening, 12, 24 and 48 weeks and annually thereafter [ Time Frame: Screening, 12, 24, 48 weeks and annually for 4 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

• Time to angiomyolipoma progression through CT/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 (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 (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 (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 (5 years). ] [ Designated as safety issue: Yes ]
• Evaluate lung function in people with LAM only [ Time Frame: at baseline, 6, 12, 18, 24 weeks and every 12 weeks thereafter ] [ Designated as safety issue: Yes ]

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

Arms	Assigned Interventions
Experimental: 1: RAD001 5mg	Drug: Everolimus (RAD001) 5 mg in tablet form. 10mg daily dosing throughout the trial. Other Name: RAD001
Placebo Comparator: 2: RAD001 Placebo	Other: Placebo Placebo

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Male or Female 18 years or older
Definite diagnosis of Tuberous Sclerosis according to the modified Gomez criteria
Clinically definite diagnosis of renal angiomyolipoma
At least one Angiomyolipoma of ≥ 3 cm in its longest diameter using CT or MRI
Females of child bearing potential must use birth control

Exclusion Criteria:

Recent heart attack, cardiac related chest pain or stroke
Severely impaired lung function
Bleeding related to angiomyolipoma
Severe liver dysfunction
Severe kidney disfunction
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: NCT00790400

Show 29 Study Locations

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

Study Director:

Novartis Pharmaceuticals

More Information

Additional Information:

Visit EXIST-2 NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers. This link exits the ClinicalTrials.gov site

This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:	NCT00790400 History of Changes
Other Study ID Numbers:	CRAD001M2302
Study First Received:	November 10, 2008
Last Updated:	October 13, 2011
Health Authority:	United States: Food and Drug Administration; Canada: Health Canada; France: Ministry of Health; Germany: Federal Institute for Drugs and Medical Devices; Italy: Ministry of Health; Japan: Ministry of Health, Labour and Welfare, Pharmaceutical amd Medical Safety Bureau; Netherlands: Medical Ethics Review Committee (METC); Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products; Russia: Federal Service on Surveillance in Healthcare and Social Development of Russian Federation; Spain: Ministerio de Sanidad y Politica Social; United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Novartis:

Angiomyolipoma
AML
Tuberous Sclerosis Complex
TSC
mTOR
RAD001

Mammalian Target of Rapamycin
Everolimus
Afinitor
SEGA
Subependymal Giant Cell Astrocytoma
Seizures

Additional relevant MeSH terms:

Sclerosis
Tuberous Sclerosis
Angiomyolipoma
Lymphangioleiomyomatosis
Pathologic Processes
Hamartoma
Neoplasms
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn

Neoplasms, Adipose Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Perivascular Epithelioid Cell Neoplasms
Lymphangiomyoma
Lymphatic Vessel Tumors
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Everolimus
Sirolimus
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on February 09, 2012