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

NCT05252585 | Active Not Recruiting Phase 4

• 1 other identifier: CRAD001M2402
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 4

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.

Conditions

Renal Angiomyolipoma

Keywords

Tuberous Sclerosis Complex; TSC; Angiomyolipoma; AML; Everolimus

Outcome Measures

Primary Outcomes (1)

• Percentage of participants with adverse events (AEs), Serious AEs (SAEs) and AEs of special interest (AESI)

  Percentage of participants with AEs, SAEs and AESIs.

  From first dose of study treatment up to 56 weeks

Secondary Outcomes (3)

• Angiomyolipoma (AML) response rate

  Up to 52 weeks

• AML progression rate

  Up to 52 weeks

• Percentage of participants with laboratory abnormalities

  From screening up to 56 weeks

Study Arms (1)

Everolimus

EXPERIMENTAL

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

Drug: Everolimus

Interventions

Everolimus DRUG

Everolimus tablets for oral use. The recommended everolimus starting dose will be 10 mg orally taken once daily for all patients, except for those with impaired liver function, for whom the everolimus dose will be: * Child-Pugh grade A: 7.5 mg once daily (for patients with mild hepatic impairment) * Child-Pugh grade B: 5.0 mg once daily (for patients with moderate hepatic impairment)

Also known as: RAD001

Everolimus

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (4)

Novartis Investigative Site

Taichung, Taiwan ROC, 40201, Taiwan

Location

Novartis Investigative Site

Taoyuan District, Taiwan ROC, 33305, Taiwan

Location

Novartis Investigative Site

Taipei, 10002, Taiwan

Location

Novartis Investigative Site

Taoyuan District, 33305, Taiwan

Location

MeSH Terms

Conditions

Angiomyolipoma; Tuberous Sclerosis

Interventions

Everolimus

Condition Hierarchy (Ancestors)

Neoplasms, Adipose Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Perivascular Epithelioid Cell Neoplasms; Hamartoma; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Sirolimus; Macrolides; Lactones; Organic Chemicals

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 16, 2022
• First Posted: February 23, 2022
• Study Start: May 1, 2023
• Primary Completion (Estimated): October 10, 2026
• Study Completion (Estimated): October 10, 2026
• Last Updated: April 2, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

TW (4)