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

Last updated: April 1, 2026
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Not Recruiting

Phase

4

Condition

Scar Tissue

Wilms' Tumor

Treatment

Everolimus

Clinical Study ID

NCT05252585
CRAD001M2402
  • Ages 18-65
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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 witheverolimus per local reimbursement criteria.

Exclusion

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 rapamycinderivatives, or to any of the excipients.

Study Design

Total Participants: 4
Treatment Group(s): 1
Primary Treatment: Everolimus
Phase: 4
Study Start date:
May 01, 2023
Estimated Completion Date:
October 10, 2026

Study 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.

Connect with a study center

  • Novartis Investigative Site

    Taichung, Taiwan ROC 40201
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Taichung 1668399, Taiwan ROC 40201
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Taoyuan, Taiwan ROC 33305
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Taoyuan District, Taiwan ROC 33305
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Kuei Shan Chiang, Taoyuan Taiwan ROC 33305
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Taipei, 10002
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Taipei 1668341, 10002
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Taoyuan District 1667905, 33305
    Taiwan

    Site Not Available

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