A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Inclusion Criteria

Inclusion Criteria

- Male or Female 18 years or older

- Clinically definite diagnosis of Tuberous Sclerosis Complex according to the modified
Gomez criteria or sporadic LAM (biopsy-proven or compatible chest CT scan)

- Clinically definite diagnosis of renal angiomyolipoma

- At least one Angiomyolipoma of ≥ 3 cm in its longest diameter using CT or MRI

- Females of child bearing potential must use birth control and have documentation of
negative pregnancy test

- Written informed consent according to local guidelines

Exclusion Criteria:

- Recent heart attack, cardiac related chest pain or stroke

- Severely impaired lung function

- Bleeding related to angiomyolipoma or embolization during 6 months prior to
randomization

- Clinically significant chylous ascites

- Clinically significant hematological or hepatic abnormality

- Severe liver dysfunction

- Severe kidney dysfunction

- Pregnancy or breast feeding

- Current infection

- History of organ transplant

- Surgery within two months prior to study enrollment

- Prior therapy with a medication in the same class as Everolimus

- Recent use of an investigational drug

- Bleeding diathesis or on oral anti-vitamin K medication

- Uncontrolled high cholesterol

- Uncontrolled diabetes

- HIV

- Inability to attend scheduled clinic visits

- Patients with metal implants thus prohibiting MRI evaluations

- Angiomyolipoma which requires surgery at the time of randomization

- History of malignancy

- Severe or uncontrolled medical conditions which would cause an unacceptable safety
risk or compromise compliance with the protocol

Other protocol-defined inclusion/exclusion criteria may apply