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RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM
This study is currently recruiting participants.
Study NCT00457964   Information provided by Children's Hospital Medical Center, Cincinnati
First Received: April 6, 2007   Last Updated: June 25, 2007   History of Changes
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April 6, 2007
June 25, 2007
August 2005
  • The primary outcome measures will be the volumetrically determined angiomyolipoma burden
  • Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline
  • Patients will be considered complete responders if their angiomyolipomas decreases by 75% or more.
  • The primary outcome measures will be the volumetrically determined angiomyolipoma burden
  • Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline
  • Patients will be considered complete responders if their angiomyolipomas decreases by 75% or more.
Complete list of historical versions of study NCT00457964 on ClinicalTrials.gov Archive Site
 
 
 
RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM
RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

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.

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

Phase I, Phase II
Interventional
Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
  • Tuberous Sclerosis
  • Lymphangioleiomyomatosis
Drug: RAD001
 
 

*   Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
 
Recruiting
30
August 2009
 

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.
Both
18 Years to 65 Years
No
Contact: Ashwini Roy-Chaudhury, MPH 513-636-2073 ashwini.roy-chaudhury@cchmc.org
United States
 
 
NCT00457964
 
 
Children's Hospital Medical Center, Cincinnati
Novartis
Principal Investigator: John J. Bissler, M.D. Children's Hospital Medical Center, Cincinnati
Children's Hospital Medical Center, Cincinnati
April 2007

 †    Required WHO trial registration data element.
††   WHO trial registration data element that is required only if it exists.