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| Sponsors and Collaborators: |
Children's Hospital Medical Center, Cincinnati Novartis |
| Information provided by: | Children's Hospital Medical Center, Cincinnati |
| ClinicalTrials.gov Identifier: | NCT00457964 |
Purpose
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 |
|
Tuberous Sclerosis Lymphangioleiomyomatosis |
Drug: RAD001 |
Phase I Phase II |
| Genetics Home Reference related topics: | familial encephalopathy with neuroserpin inclusion bodies tuberous sclerosis |
| MedlinePlus related topics: | Tuberous Sclerosis |
| ChemIDplus related topics: | Everolimus Sirolimus |
| 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
Eligibility
| Ages Eligible for Study: | 18 Years to 65 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| 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. | |||||
| Children's Hospital Medical Center, Cincinnati |
| Novartis |
| Principal Investigator: | John J. Bissler, M.D. | Children's Hospital Medical Center, Cincinnati |
More Information
| Study ID Numbers: | CCHMC IRB # 04-07-22 |
| First Received: | April 6, 2007 |
| Last Updated: | June 25, 2007 |
| ClinicalTrials.gov Identifier: | NCT00457964 |
| Health Authority: | United States: Food and Drug Administration |
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