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RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00457964
Recruitment Status : Completed
First Posted : April 9, 2007
Last Update Posted : September 25, 2013
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

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.

Condition or disease Intervention/treatment Phase
Tuberous Sclerosis Lymphangioleiomyomatosis Drug: RAD001 Phase 1 Phase 2

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 36 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
Study Start Date : August 2005
Actual Primary Completion Date : July 2013
Actual Study Completion Date : July 2013

Arm Intervention/treatment
Experimental: Administration of RAD001 Drug: RAD001
5 and 10 mg/day or 30, 50, 70mg/week

Primary Outcome Measures :
  1. Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline [ Time Frame: 12 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00457964

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United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Novartis Pharmaceuticals
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Principal Investigator: John J. Bissler, M.D. LaBonheur Children's Hospital
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Responsible Party: Children's Hospital Medical Center, Cincinnati Identifier: NCT00457964    
Other Study ID Numbers: CCHMC IRB #2008-0812(04-07-22)
First Posted: April 9, 2007    Key Record Dates
Last Update Posted: September 25, 2013
Last Verified: September 2013
Keywords provided by Children's Hospital Medical Center, Cincinnati:
Tuberous Sclerosis Complex (TSC)
Mammalian Target of Rapamycin (mTOR)
Additional relevant MeSH terms:
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Tuberous Sclerosis
Pathologic Processes
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Perivascular Epithelioid Cell Neoplasms
Neoplasms, Connective and Soft Tissue
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Neoplasms, Adipose Tissue
Antineoplastic Agents