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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)

Phase 3
18 Years
Open (Enrolling)
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)

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Trial Information

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

- 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


- 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

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Outcome Measure:

Angiomyolipoma Response Rate as Per Central Radiology Review (Double-blind Period)

Outcome Description:

Angiomyolipoma response defined as the combination of the following criteria: reduction in angiomyolipoma volume of ≥ 50% relative to baseline, where angiomyolipoma volume was sum of volumes of all target lesions identified at baseline, and with a confirmatory scan performed approximately 12 weeks later (no sooner than 8 weeks later)• No new angiomyolipoma lesions ≥ 1.0 cm in longest diameter were identified.• There were no kidney increases in volume > 20% from nadir. The patient did not have any angiomyolipoma-related bleeding of ≥ grade 2

Outcome Time Frame:

From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or analysis cut-off date (30-June-2011).

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

April 2009

Completion Date:

December 2014

Related Keywords:

  • Tuberous Sclerosis Complex (TSC)
  • Lymphangioleiomyomatosis (LAM)
  • Angiomyolipoma
  • AML
  • Tuberous Sclerosis Complex
  • TSC
  • mTOR
  • RAD001
  • Mammalian Target of Rapamycin
  • Everolimus
  • Afinitor
  • SEGA
  • Subependymal Giant Cell Astrocytoma
  • Seizures
  • Sclerosis
  • Tuberous Sclerosis
  • Angiomyolipoma
  • Lymphangioleiomyomatosis



Novartis Investigative Site Boston, Massachusetts  02115
Novartis Investigative Site Cleveland, Ohio  44195
Novartis Investigative Site Birmingham, Alabama  35294
Novartis Investigative Site Tucson, Arizona  85724
Novartis Investigative Site Minneapolis, Minnesota  55455