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A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Trial Profile

A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 06 Nov 2021

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At a glance

  • Drugs Everolimus (Primary)
  • Indications Angiomyolipoma; Lymphangioleiomyomatosis; Tuberous sclerosis
  • Focus Registrational; Therapeutic Use
  • Acronyms EXIST-2
  • Sponsors Novartis
  • Most Recent Events

    • 19 Jul 2018 Results assessing the effect of everolimus on renal function in patients with tuberous sclerosis complex from EXIST-1 and EXIST-2 studies, published in the Nephrology Dialysis Transplantation.
    • 10 May 2016 Long-term results of extension study (n=112) presented at the 111th Annual Meeting of the American Urological Association
    • 21 Apr 2016 Results (n=119) of pooled analysis of 3 studies (NCT00411619, NCT00789828 and NCT00790400) analysing endocrine and menstrual cycle-related events in females presented at the 68th Annual Meeting of the American Academy of Neurology

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