NCT00457808

Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (planned)
• Sponsor: Children's Hospital Medical Center, Cincinnati · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

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.

Conditions

• Tuberous Sclerosis
• Lymphangioleiomyomatosis

Interventions

Type	Name	Description
DRUG	Rapamycin, sirolimus

Timeline

• Start date: 2002-12-01
• Completion: 2006-03-01
• First posted: 2007-04-09
• Last updated: 2007-04-09

Locations

1 site across 1 country: United States