RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM - Full Text View

Sponsor:	Children's Hospital Medical Center, Cincinnati
Collaborator:	Novartis Pharmaceuticals
Information provided by (Responsible Party):	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

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized 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

Resource links provided by NLM:

Genetics Home Reference related topics: lymphangioleiomyomatosis tuberous sclerosis complex

MedlinePlus related topics: Tuberous Sclerosis

Drug Information available for: Sirolimus Everolimus CCI 779

U.S. FDA Resources

Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome Measures:

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)

Intervention Details:

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

Criteria

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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00457964

Locations

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

Investigators

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 # 04-07-22
Study First Received:	April 6, 2007
Last Updated:	January 12, 2012
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 February 12, 2012