NCT03525834

Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.

Phase IV, Single Arm Study of Safety and Efficacy of Everolimus in Chinese Adults With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma Not Requiring Immediate Surgery

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 40 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to assess the safety and efficacy of everolimus (Afinitor®) in Chinese patients with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC).

Detailed description

This was an open label, single arm, multi-center, Phase IV Post-Approval Commitment (PAC) study with once daily oral dose of 10 mg everolimus in participants with renal AML associated with TSC. There were three separate phases in this study: a Screening phase, an Open-label treatment phase where participants received everolimus for 48 weeks or until disease progression, and a Treatment discontinuation follow-up phase for patients who discontinue study drug for reasons other than disease progression. Every participant had an End of Treatment visit within 28 days after last dose and a safety follow-up visit 30 days after last dose.

Conditions

• Renal Angiomyolipoma

Interventions

Type	Name	Description
DRUG	Everolimus	Everolimus 2.5 mg and 5 mg tablets with dosage regimen of 10 mg orally once daily.

Timeline

• Start date: 2018-11-09
• Primary completion: 2020-09-25
• Completion: 2020-09-25
• First posted: 2018-05-16
• Last updated: 2021-09-09
• Results posted: 2021-09-09

Locations

5 sites across 1 country: China