Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Completed · Phase 3 · Has a placebo group

Conditions studied: Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)

In brief

This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.

Key facts

Study ID

NCT00790400

Run by

Novartis Pharmaceuticals

People needed

118

Starts

2009-04-01

Expected to finish

2015-11-01

Last updated by the study team

2017-02-17

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Barrow Tuberous Sclerosis Center — Phoenix, Arizona, United States
• Massachusetts General Hospital Massachussetts General Hospita — Boston, Massachusetts, United States
• Minnesota Epilepsy Group — Saint Paul, Minnesota, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• LeBonheur Childrens Medical Group SC-2 — Memphis, Tennessee, United States
• Novartis Investigative Site — Torono, Ontario, Canada
• Novartis Investigative Site — Lyon, France, France
• Novartis Investigative Site — Berlin, Germany
• Novartis Investigative Site — München, Germany
• Novartis Investigative Site — Siena, SI, Italy
• Novartis Investigative Site — Torino, TO, Italy
• Novartis Investigative Site — Roma, Italy
• Novartis Investigative Site — Sapporo, Hokkaido, Japan
• Novartis Investigative Site — Suita, Osaka, Japan
• Novartis Investigative Site — Yamagata, Yamagata, Japan
• Novartis Investigative Site — Utrecht, Netherlands, Netherlands
• Novartis Investigative Site — Warsaw, Poland
• Novartis Investigative Site — Warsaw, Poland
• Novartis Investigative Site — Moscow, Russia
• Novartis Investigative Site — Barcelona, Catalonia, Spain
• Novartis Investigative Site — Brighton, East Sussex, United Kingdom
• Novartis Investigative Site — Craigavon, Northern Ireland, United Kingdom
• Novartis Investigative Site — Cardiff, Wales, United Kingdom
• Novartis Investigative Site — London, United Kingdom

Full record on ClinicalTrials.gov

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