Clinical Trial: Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM

Study Status: Active, not recruiting
Recruit Status: Unknown status
Study Type: Interventional

Official Title: A Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatos

Brief Summary: The purpose of this study is to determine the safety and efficacy of the mTOR inhibitor sirolimus as a treatment for renal angiomyolipomas in patients with tyberous sclerosis complex or sporadic lymphangioleiomyomatosis.

Detailed Summary: Inherited mutations of the TSC1 or TSC2 gene cause tuberous sclerosis while acquired (somatic) mutations of either gene are associated with sporadic lymphangioleiomyomatosis (LAM). Renal angiomyolipomas are a feature of both disorders. TSC1 and TSC2 regulate signalling through the mammalian target of rapamycin (mTOR) pathway. Inhibition of mTOR may result in a decrease in size of TSC 1/2 assciated lesions. We are treating patients with tuberous sclerosis or sporadic LAM with the mTOR inhibitor rapamycin in a non-randomised, open label pilot study of safety and efficacy. Change in size of renal angiomyolipomas is the primary end point
Sponsor: Cardiff University

Current Primary Outcome:

  • longest diameter of renal angiomyolipomas assessed by MRI scan, toxicity graded by National Cancer Institute's Common Terminology Criteria for Adverse Events v3.0 [ Time Frame: assessments at baseline and 2,6,12 and 24 months ]
  • toxicity graded by National Cancer Institute's Common Terminology Criteria for Adverse Events [ Time Frame: throughout study ]


Original Primary Outcome: longest diameter of renal angiomyolipomas assessed by MRI scan [ Time Frame: assessments at baseline and 2,6,12 and 24 months ]

Current Secondary Outcome: respiratory function tests (FEV1, FVC, DLCO), cognitive function (memory, executive skills) [ Time Frame: 2 years ]

Original Secondary Outcome: Same as current

Information By: Cardiff University

Dates:
Date Received: June 21, 2007
Date Started: October 2005
Date Completion: September 2009
Last Updated: April 29, 2008
Last Verified: April 2008