EXIST-2: Angiomyolipoma Associated With Either TSC or Sporadic Lymphangioleiomyomatosis
EXIST-2 (EXamining everolimus In a Study of TSC-2) is a prospective, randomized, double-blind, parallel group, placebo-controlled phase III multicenter clinical trial of everolimus (RAD001) in the treatment of patients with angiomyolipoma associated with either tuberous sclerosis complex (TSC) or sporadic lymphangioleiomyomatosis.
Patients are randomized to receive continuous therapy with either everolimus (RAD001) 10 mg/day orally or placebo. The trial will enroll 99 TSC or sporadic lymphangioleiomyomatosis patients with angiomyolipoma, in a 2:1 randomization to everolimus (RAD001) or placebo, at multiple sites in the United States and Europe.
EXIST-2 Key facts
Primary endpoint: Angiomyolipoma response rate
Secondary endpoints:
Time to angiomyolipoma progression
Skin lesion response rate
Change from baseline in angiogenic molecules
Duration of angiomyolipoma response (in treatment arm)
Exposure to everolimus (in treatment arm)
Safety
Target enrollment: 99
*Treatment until angiomyolipoma progression, unacceptable toxicity, or discontinuation for any other reason.
†When the final analysis is complete, an extension phase will be launched if the results are favorable to everolimus. All participants will be offered the option
of receiving open-label everolimus for 4 years after the last participant was randomized.
Click here to learn more about this clinical trial and participating centers from Clinicaltrials.gov
Disclaimer : These are investigational studies; there is no guarantee that everolimus will become commercially available for these indications
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