A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)

Novartis

Status and phase: Enrolling; Phase 4

Conditions: Renal Angiomyolipoma

Treatments: Drug: Everolimus

Study type: Interventional

Funder types: Industry

Identifiers

NCT05252585

CRAD001M2402

Details and patient eligibility

About

The purpose of this prospective study is to assess the safety and efficacy of everolimus in Taiwanese patients with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC) . Only patients who fulfil the local reimbursement criteria of everolimus for TSC-AML will be included in this study.

Full description

This open-label, prospective, single-arm, multicenter Phase IV post approval commitment (PAC) study is planned to be conducted in approximately 10 patients with confirmed diagnosis of TSC-AML and who fulfil the local reimbursement criteria of everolimus for TSC-AML treatment.

The study will have a 30-day screening phase, and each patient will be on treatment up to 52 weeks. Enrollment will end at the latest within 52 weeks from Day 1 of the study, regardless of the number of patients actually recruited. After completion of the treatment phase/end of treatment (EOT), eligible patients will enter a 4-week safety follow up (FU) phase. Patients who continue to be on treatment beyond 52 weeks, based on the investigator's judgment will not be included in the 4-week safety FU phase.

Enrollment: 10 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Adult male or female patients from ≥ 18 years of age.
2. Signed informed consent must be obtained prior to participation in the study.
3. Participants with TSC associated with renal AML which is eligible for treatment with everolimus per local reimbursement criteria.

Exclusion criteria

1. Patients with severe hepatic impairment (Child-Pugh class C)
2. Any severe and/or uncontrolled medical conditions.
3. Pregnant or breast-feeding females.
4. Patients with hypersensitivity to the active substance, to other rapamycin derivatives, or to any of the excipients.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Everolimus

Experimental group

Description:

Participants with confirmed diagnosis of TSC-AML and who fulfil the local (Taiwan) reimbursement criteria of everolimus for TSC-AML treatment

Treatment:

Drug: Everolimus