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

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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.
    

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
    

Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline


Condition

Tuberous Sclerosis

Intervention

RAD001

Study Arms / Comparison Groups

 Administration of RAD001
Description:  

Publications

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

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

36

Start Date

August 2005

Completion Date

July 2013

Primary Completion Date

July 2013

Eligibility 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.
      

Gender

All

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

John J. Bissler, M.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00457964

Organization ID

CCHMC IRB #2008-0812(04-07-22)


Responsible Party

Sponsor

Study Sponsor

Children's Hospital Medical Center, Cincinnati

Collaborators

 Novartis Pharmaceuticals

Study Sponsor

John J. Bissler, M.D., Principal Investigator, LaBonheur Children's Hospital


Verification Date

September 2013