Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

2014-07-23 21:29:48 | BioPortfolio


The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.


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.

Study Design

Allocation: Non-Randomized, Control: Active Control, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Tuberous Sclerosis


Rapamycin, sirolimus


Cincinnati Childrens Hospital Medical Center
United States




Children's Hospital Medical Center, Cincinnati

Results (where available)

View Results


Published on BioPortfolio: 2014-07-23T21:29:48-0400

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Medical and Biotech [MESH] Definitions

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