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Sponsors and Collaborators: |
Children's Hospital Medical Center, Cincinnati Novartis |
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Information provided by: | Children's Hospital Medical Center, Cincinnati |
ClinicalTrials.gov Identifier: | NCT00457964 |
The purpose of this research study is to find out what effects RAD001 has on angiomyolipomas of a person with Tuberous Sclerosis Complex and to determine the safe dose of RAD001 without toxicity.
The hypothesis is that the drug will inhibit the growth of the angiomyolipomas and possibly even cause regression.
Condition | Intervention | Phase |
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Tuberous Sclerosis Lymphangioleiomyomatosis |
Drug: RAD001 |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis |
Estimated Enrollment: | 30 |
Study Start Date: | August 2005 |
Estimated Study Completion Date: | August 2009 |
Tuberous sclerosis complex is a genetic disorder with a birth incidence of approximately one in six thousand.
Angiomyolipomas occur in eighty percent of persons with TS, and in up to 60% of persons with LAM. They are fatty tumors that often occur in kidney or liver as well as other parts of the body. They can grow and cause damage to surrounding kidney tissue and even renal failure. They may also leak blood causing potentially life-threatening hemorrhage.
Laboratory studies have shown that RAD001 suppresses the chemical that cause tumors to grow in tuberous sclerosis and sporadic LAM.
The primary goal of this study is to evaluate the clinical effectiveness of RAD001 in a 24 month trial. Although the primary goal is to determine if the drug RAD001 has effects on angiomyolipomas, other diseases associated with tuberous sclerosis will be monitored too, specifically any change in involvement of your brain or lungs with tuberous sclerosis. The use of RAD001 to treat angiomyolipomas in tuberous sclerosis or sporadic LAM is considered experimental
Ages Eligible for Study: | 18 Years to 65 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Ashwini Roy-Chaudhury, MPH | 513-636-2073 | ashwini.roy-chaudhury@cchmc.org |
United States, Ohio | |
Cincinnati Children's Hospital Medical Center | Recruiting |
Cincinnati, Ohio, United States, 45229 | |
Principal Investigator: John J Bissler, M.D. |
Principal Investigator: | John J. Bissler, M.D. | Children's Hospital Medical Center, Cincinnati |
Study ID Numbers: | CCHMC IRB # 04-07-22 |
Study First Received: | April 6, 2007 |
Last Updated: | June 25, 2007 |
ClinicalTrials.gov Identifier: | NCT00457964 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Tuberous Sclerosis Complex (TSC) Lymyphangioleiomyomatosis Mammalian Target of Rapamycin (mTOR) RAD001 Angiomyolipmas |
Everolimus Sirolimus Immunoproliferative Disorders Immunologic Factors Nervous System Malformations Lymphangiomyoma Sclerosis Angiomyolipoma Neurodegenerative Diseases Immunosuppressive Agents Lymphatic Diseases |
Lymphangioleiomyomatosis Neoplasms, Connective and Soft Tissue Tuberous Sclerosis Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn Bourneville Syndrome Lymphoproliferative Disorders Malformations of Cortical Development Congenital Abnormalities Neurocutaneous Syndromes |
Immunologic Factors Nervous System Malformations Physiological Effects of Drugs Angiomyolipoma Neurodegenerative Diseases Neoplasms, Connective and Soft Tissue Heredodegenerative Disorders, Nervous System Pathologic Processes Tuberous Sclerosis Congenital Abnormalities Neurocutaneous Syndromes Everolimus Neoplasms by Histologic Type Immunoproliferative Disorders Immune System Diseases |
Nervous System Diseases Lymphangiomyoma Sclerosis Immunosuppressive Agents Hamartoma Pharmacologic Actions Lymphatic Vessel Tumors Lymphangioleiomyomatosis Lymphatic Diseases Neoplasms Genetic Diseases, Inborn Malformations of Cortical Development Lymphoproliferative Disorders Neoplasms, Adipose Tissue |