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

Sponsored by:	Novartis
Information provided by:	Novartis
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 Lymphangioleiomyomatosis	Drug: Everolimus (RAD001) Other: 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 RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Further study details as provided by Novartis:

Primary Outcome Measures:

• Angiomyolipoma 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 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

• Time to angiomyolipoma 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 (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 ]
• 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 (5 years). ] [ Designated as safety issue: Yes ]

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

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

Eligibility

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

Criteria

Inclusion Criteria:

18 years or older
Definite diagnosis of Tuberous Sclerosis or Sporadic Lymphangioleiomyomatosis
At least one Angiomyolipoma of at least 3 cm in diameter
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

Locations

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

Sponsors and Collaborators

Novartis

More Information

Responsible Party:	Novartis ( external Affairs )
Study ID Numbers:	CRAD001M2302
Study First Received:	November 10, 2008
Last Updated:	November 12, 2008
ClinicalTrials.gov Identifier:	NCT00790400
Health Authority:	United States: Food and Drug Administration; Belgium: Pharmaceutical Inspectorate; France: Ministry of Health; 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; Korea: Korea Food and Drug Administration

Keywords provided by Novartis:

Lymphangioleiomyomatosis
Tuberous Sclerosis
Angiomyolipoma
mTOR

RAD001
Mammalian Target of Rapamycin
Everolimus

Study placed in the following topic categories:

Everolimus
Sirolimus
Immunoproliferative Disorders
Nervous System Malformations
Lymphangiomyoma
Sclerosis
Angiomyolipoma
Neurodegenerative Diseases
Bourneville syndrome
Lymphatic Diseases

Lymphangioleiomyomatosis
Neoplasms, Connective and Soft Tissue
Tuberous Sclerosis
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Tuberous sclerosis
Lymphoproliferative Disorders
Malformations of Cortical Development
Congenital Abnormalities
Neurocutaneous Syndromes

Additional relevant MeSH terms:

Lymphatic Vessel Tumors
Neoplasms
Neoplasms by Histologic Type
Pathologic Processes
Immunologic Factors
Immune System Diseases

Physiological Effects of Drugs
Nervous System Diseases
Immunosuppressive Agents
Hamartoma
Pharmacologic Actions
Neoplasms, Adipose Tissue

ClinicalTrials.gov processed this record on January 16, 2009