Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) (EXIST-2)
Status and phase
Completed
Phase 3
Conditions
Tuberous Sclerosis Complex (TSC)
Lymphangioleiomyomatosis (LAM)
Treatments
Drug: Everolimus (RAD001)
Drug: Everolimus Placebo
Study type
Interventional
Funder types
Industry
Identifiers
Details and patient eligibility
About
This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.
Enrollment
118 patients
Sex
All
Ages
18+ years old
Volunteers
No Healthy Volunteers
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
Trial design
Primary purpose
Treatment
Allocation
Randomized
Interventional model
Parallel Assignment
Masking
Quadruple Blind
118 participants in 2 patient groups, including a placebo group
Everolimus
Experimental group
Description:
Study drug was given by continuous oral daily dosing of two 5 mg tablets.
Treatment:
Drug: Everolimus (RAD001)
Placebo
Placebo Comparator group
Description:
Placebo was given by continuous oral daily dosing of two 5 mg tablets.
Treatment:
Drug: Everolimus Placebo
Trial contacts and locations
25
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Data sourced from clinicaltrials.gov
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