Rapamycin In Angiomyolipomas In Patients With Tuberous Sclerosis

Puigvert Foundation

Status and phase: Completed; Phase 4

Conditions: Angiomyolipoma

Treatments: Drug: Sirolimus

Study type: Interventional

Funder types: Other

Identifiers

NCT01217125

EudraCT 2007-005978-30

Details and patient eligibility

About

The purpose of this study is to determine whether rapamycin is safe and effective in the treatment of renal angiomyolipomas in patients with tuberousclerosis.

Enrollment: 18 patients

Sex: All

Ages: 10+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

Diagnosis of tuberousclerosis Angiomyolipoma >2cm age>10 years

Exclusion criteria

Creatinine >4 No recent AML bleeding No hepatic abnormalities (liver tests 2fold) Hematocrit<27% Thrombocytopenia (<100.000/mm3) Leukopenia (<3000/mm3) Ischemic cardiopathy Recent surgery (2 months prior to enrollment) Pregnancy Serum cholesterol over 7.8 mmol/l or hypertriglyceridemia fasten(>4.6 mmol/l) non controlled with drugs malignancy in the previous 2 years allergy to macrolides

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)