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Tracking Information | |||||
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First Received Date † | April 6, 2007 | ||||
Last Updated Date | June 25, 2007 | ||||
Start Date † | August 2005 | ||||
Current Primary Outcome Measures † |
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Original Primary Outcome Measures † |
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Change History | Complete list of historical versions of study NCT00457964 on ClinicalTrials.gov Archive Site | ||||
Current Secondary Outcome Measures † | |||||
Original Secondary Outcome Measures † | |||||
Descriptive Information | |||||
Brief Title † | RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM | ||||
Official Title † | RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis | ||||
Brief Summary | 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. |
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Detailed Description | 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 |
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Study Phase | Phase I, Phase II | ||||
Study Type † | Interventional | ||||
Study Design † | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study | ||||
Condition † |
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Intervention † | Drug: RAD001 | ||||
Study Arms / Comparison Groups | |||||
Publications * | |||||
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline. |
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Recruitment Information | |||||
Recruitment Status † | Recruiting | ||||
Estimated Enrollment † | 30 | ||||
Estimated Completion Date | August 2009 | ||||
Primary Completion Date | |||||
Eligibility Criteria † | Inclusion Criteria:
Exclusion Criteria:
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Gender | Both | ||||
Ages | 18 Years to 65 Years | ||||
Accepts Healthy Volunteers | No | ||||
Contacts †† |
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Location Countries † | United States | ||||
Expanded Access Status | |||||
Administrative Information | |||||
NCT ID † | NCT00457964 | ||||
Responsible Party | |||||
Secondary IDs †† | |||||
Study Sponsor † | Children's Hospital Medical Center, Cincinnati | ||||
Collaborators †† | Novartis | ||||
Investigators † |
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Information Provided By | Children's Hospital Medical Center, Cincinnati | ||||
Verification Date | April 2007 | ||||
† Required WHO trial registration data element. †† WHO trial registration data element that is required only if it exists. |