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

• Status: Not yet recruiting
• Trial end date: 2024-04-30
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Treatments: Everolimus

Criteria

Inclusion Criteria:

1. Signed informed consent must be obtained prior to participation in the study.

2. With TSC associated with renal AML which is eligible for treatment with everolimus per locally approved label (at least one of below):

• Tumor size of ≥ 4 cm with history of clinically meaningful hemorrhage.

• Aneurysm lesion ≥ 5 mm, ineligible for trans-arterioemolization or surgery.

• Recurrence after/refractory to trans-arterioemolization or surgery.

3. Presence of at least one AML lesion ≥ 1 cm in its longest diameter via CT or MRI imaging.

Exclusion Criteria:

1. AML related bleeding or embolization during the 6 months prior to enrollment.

2. History of myocardial infarction, angina or stroke related to atherosclerosis in the previous 12 months.

3. Significant hematological or hepatic abnormality (e.g. hemoglobin < 9 g/dL, platelets < 100×109/L, transaminase levels > 2.5 × the upper limit of normal (ULN), serum bilirubin > 2 × ULN), or absolute neutrophil count (ANC) < 1.5×109/L without supportive treatment of hematopoietic growth factor.

4. Prior therapy with systemic mTOR inhibitors (sirolimus, temsirolimus, everolimus).

5. Fasting serum cholesterol > 300 mg/dL (or > 7.75 mmol/L) AND fasting triglycerides > 2.5 × ULN. NOTE: In case one or both of these thresholds are exceeded, the patient can only be included after initiation of appropriate lipid lowering medication.

6. Inadequate renal function: serum creatinine > 1.5 × ULN.

7. Any severe and/or uncontrolled medical conditions.

8. Pregnant or breast feeding females.