Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
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.
Interventional
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Volume of Angiomyolipomata
John Bissler, MD
Principal Investigator
Cincinnati Childrens Hospital Medical Center
United States: Food and Drug Administration
CCHMC IRB # 02-10-16
NCT00457808
December 2002
March 2006
Name | Location |
---|---|
Cincinnati Childrens Hospital Medical Center | Cincinnati, Ohio 45229-3039 |