Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
Condition: Tuberous Sclerosis Complex (TSC); Lymphangioleiomyomatosis (LAM)
MeSH terms: Tuberous Sclerosis; Lymphangioleiomyomatosis; Angiomyolipoma; Sclerosis
Intervention: Everolimus (RAD001); Everolimus Placebo
Intervention description: Everolimus is used in 5 mg strength tablets, blister-packed under aluminum foil in units of ten tablets and dosed on a daily basis. 10mg daily dosing throughout the trial.; Matching placebo was provided as a matching tablet and was also blister-packed under aluminum foil in units of ten.
Intervention MeSH terms: Everolimus
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 118.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Angiomyolipoma; AML; Tuberous Sclerosis Complex; TSC; mTOR; RAD001; Mammalian Target of Rapamycin; Everolimus; Afinitor; SEGA; Subependymal Giant Cell Astrocytoma; Seizures; Tuberous sclerosis complex (TSC); Tuberous sclerosis; benign tumors of brain; kidney; heart; eyes; lungs; skinTSC1; TSC2; hamaratin; tuberin, tumor growth suppressors; gyri; tubers; Sporadic Lymphangioleiomyomatosis.; Lymphangioleiomyomatosis (LAM); rare lung disease

Summary:

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

Eligibility 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