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RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

Phase 1/Phase 2
18 Years
65 Years
Open (Enrolling)
Tuberous Sclerosis, Lymphangioleiomyomatosis

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Trial Information

RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

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

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

Inclusion Criteria:

- Diagnosed with angiomyolipomas and have either Tuberous Sclerosis Complex or

- 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.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

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

Outcome Time Frame:

12 months

Safety Issue:


Principal Investigator

John J. Bissler, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Children's Hospital Medical Center, Cincinnati


United States: Food and Drug Administration

Study ID:

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



Start Date:

August 2005

Completion Date:

July 2013

Related Keywords:

  • Tuberous Sclerosis
  • Lymphangioleiomyomatosis
  • Tuberous Sclerosis Complex (TSC)
  • Lymyphangioleiomyomatosis
  • Mammalian Target of Rapamycin (mTOR)
  • RAD001
  • Angiomyolipmas
  • Sclerosis
  • Tuberous Sclerosis
  • Angiomyolipoma
  • Lymphangioleiomyomatosis



Cincinnati Children's Hospital Medical Center Cincinnati, Ohio  45229-3039