Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

Tuberous Sclerosis Clinical Trial

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

Status Completed

Clinical Trial Summary

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

Clinical Trial 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. ;

Study Design

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

Related Conditions & MeSH terms

• Lymphangioleiomyomatosis
• Sclerosis
• Tuberous Sclerosis

• NCT number: NCT00457808
• Study type: Interventional
• Source: Children's Hospital Medical Center, Cincinnati
• Status: Completed
• Phase: Phase 2
• Start date: December 2002
• Completion date: March 2006