Assessing the safety and effectiveness of Everolimus for kidney tumors in patients with Tuberous Sclerosis Complex in Taiwan
Phase IV, Prospective Single Arm Study of Safety and Efficacy of Votubia (Everolimus) in Taiwanese Adults With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma
This study is testing if the medication Everolimus is safe and effective for treating kidney tumors in patients with Tuberous Sclerosis Complex in Taiwan.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Novartis Industry-sponsored |
| Locations | 3 sites (Taichung, Taiwan ROC and 2 other locations) |
| Trial ID | NCT05252585 on ClinicalTrials.gov |
What this trial studies
This Phase IV study aims to evaluate the safety and efficacy of Everolimus in Taiwanese patients diagnosed with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC). The study will include approximately 10 patients who meet the local reimbursement criteria for Everolimus treatment. Participants will undergo a 30-day screening phase followed by up to 52 weeks of treatment, with a subsequent 4-week safety follow-up for eligible patients. The study is open-label and multicenter, focusing on real-world effectiveness post-approval.
Who should consider this trial
Good fit: Ideal candidates are adult patients aged 18 and older with a confirmed diagnosis of TSC associated with renal AML who meet local reimbursement criteria for Everolimus.
Not a fit: Patients with severe hepatic impairment, uncontrolled medical conditions, or those who are pregnant or breastfeeding may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new effective treatment option for patients suffering from renal angiomyolipoma associated with Tuberous Sclerosis Complex.
How similar studies have performed: Other studies have shown positive outcomes with Everolimus in treating TSC-related conditions, indicating a promising approach.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Taichung, Taiwan ROC and 2 other locations
- Novartis Investigative Site — Taichung, Taiwan ROC, Taiwan (Recruiting)
- Novartis Investigative Site — Taoyuan, Taiwan ROC, Taiwan (Recruiting)
- Novartis Investigative Site — Taipei, Taiwan (Recruiting)
Study contacts
- Study coordinator: Novartis Pharmaceuticals
- Email: novartis.email@novartis.com
- Phone: +41613241111
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.