Clinical trial NCT05252585: A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)
Official title: Phase IV, Prospective Single Arm Study of Safety and Efficacy of Votubia (Everolimus) in Taiwanese Adults With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Renal Angiomyolipoma
Keywords: Tuberous Sclerosis Complex
TSC
Angiomyolipoma
AML
Everolimus
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2022-02-23
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Percentage of participants with adverse events (AEs), Serious AEs (SAEs) and AEs of special interest (AESI)
timeFrame: From first dose of study treatment up to 56 weeks
Secondary outcomes:
measure: Angiomyolipoma (AML) response rate
timeFrame: Up to 52 weeks
measure: AML progression rate
timeFrame: Up to 52 weeks
measure: Percentage of participants with laboratory abnormalities
timeFrame: From screening up to 56 weeks
Other outcomes:
Locations:
city: Taichung
country: Taiwan
facility: Novartis Investigative Site
state: Taiwan ROC
city: Taoyuan
country: Taiwan
facility: Novartis Investigative Site
state: Taiwan ROC
city: Taipei
country: Taiwan
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: