RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM
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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 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis |
- Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline [ Time Frame: 12 months ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 30 |
| Study Start Date: | August 2005 |
| Estimated Study Completion Date: | July 2013 |
| Estimated Primary Completion Date: | July 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Administration of RAD001 |
Drug: RAD001
5 and 10 mg/day or 30, 50, 70mg/week
|
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
Eligibility| Ages Eligible for Study: | 18 Years to 65 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Diagnosed with angiomyolipomas and have either Tuberous Sclerosis Complex or lymphangioleiomyomatosis
- Angiomyolipoma one centimeter or greater in largest diameter
- Between the ages of 18 and 65 years old.
- If female, documentation of negative pregnancy test prior to enrollment and, where applicable, use of appropriate non-estrogen containing birth control contraceptive regimen while on study.
- Adequate renal function (creatinine < 3 mg/dl)
Exclusion Criteria:
- Pregnant or lactating women
- Continuous requirement for supplemental oxygen
- Surgery within past 2 months
- Use of an investigational drug within last 30 days.
Contacts and Locations| United States, Ohio | |
| Cincinnati Children's Hospital Medical Center | |
| Cincinnati, Ohio, United States, 45229 | |
| Principal Investigator: | John J. Bissler, M.D. | Children's Hospital Medical Center, Cincinnati |
More Information
No publications provided
| Responsible Party: | Children's Hospital Medical Center, Cincinnati |
| ClinicalTrials.gov Identifier: | NCT00457964 History of Changes |
| Other Study ID Numbers: | CCHMC IRB #2008-0812(04-07-22) |
| Study First Received: | April 6, 2007 |
| Last Updated: | January 3, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Children's Hospital Medical Center, Cincinnati:
|
Tuberous Sclerosis Complex (TSC) Lymyphangioleiomyomatosis Mammalian Target of Rapamycin (mTOR) RAD001 Angiomyolipmas |
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 Sirolimus Everolimus Antibiotics, Antineoplastic Antineoplastic Agents Therapeutic Uses |
ClinicalTrials.gov processed this record on May 23, 2013