Clinical Trial: Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
Brief Summary: The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.
Detailed Summary: The study design was an open label, phase I/II trial of sirolimus for one year followed by one year off therapy.Patients were seen at baseline,at two weeks to four weeks, and at 2,4,6,9,12,18 and 24 months. Angiomyolipomata imaging was performed at all but the two to four week visit. Complete pulmonary function tests and six-minute walk were obtained at baseline,6 or 9 month, 12 and 24 month visits, while simple spirometry only was performed at all other visits.
Sponsor: Children's Hospital Medical Center, Cincinnati
Current Primary Outcome: Volume of Angiomyolipomata
Original Primary Outcome: Same as current
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Children's Hospital Medical Center, Cincinnati
Dates:
Date Received: April 6, 2007
Date Started: December 2002
Date Completion:
Last Updated: April 6, 2007
Last Verified: April 2007