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

• Status: Completed
• Trial end date: 2015-11-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Treatments: Everolimus; Sirolimus

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