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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 SiteBoston, Massachusetts  02115
Novartis Investigative SiteCleveland, Ohio  44195
Novartis Investigative SiteBirmingham, Alabama  35294
Novartis Investigative SiteTucson, Arizona  85724
Novartis Investigative SiteMinneapolis, Minnesota  55455