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NCT Number: NCT05252585

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

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

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Novartis Investigative Site, Taichung, Taiwan ROC, Taiwan

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

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)

Other names: RAD001

Primary outcomes

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

    Time frame: From first dose of study treatment up to 56 weeks

    Percentage of participants with AEs, SAEs and AESIs.

Secondary outcomes

  1. Angiomyolipoma (AML) response rate

    Time frame: Up to 52 weeks

    AML response rate is defined as the percentage of patients with an AML response. AML response will be defined as: A reduction in AML volume of at least 50% relative to screening, where AML volume is the sum of the volumes of all target AML identified at screening. In addition, AML response have to satisfy: no new AML ≥ 1 cm in longest diameter are identified, neither kidney increases in volume by more than 20% from nadir (where nadir is the lowest kidney volume at the screening), the participant does not have any angiomyolipoma-related bleeding of grade equal or over 2 (as defined by NCI CTCAE, version 5).

  2. AML progression rate

    Time frame: Up to 52 weeks

    AML progression rate is defined as the percentage of patients with an AML progression. AML progression status is defined as one or more of the following: an increase from nadir of 25% or more in AML volume to a value greater than screening AML (where nadir is the lowest AML volume obtained for the participant previously in the trial), the appearance of a new AML ≥ 1.0 cm in longest diameter, an increase from nadir of 20% or more in the volume of either kidney to a value greater than screening (where nadir is the lowest kidney volume obtained for the participant previously in the trial), angiomyolipoma-related bleeding grade ≥2 (as defined by NCI CTCAE, version 5)

  3. Percentage of participants with laboratory abnormalities

    Time frame: From screening up to 56 weeks

    The laboratory assessment (including hematology, coagulation, biochemistry, and urinalysis) will be recorded at baseline and during the study based on changes in grade of laboratory abnormality.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2023
Primary completion
2026
Study completion
2026
First posted
Feb 23, 2022
Registry last updated
Jun 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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