Rapamycin In Angiomyolipomas In Patients With Tuberous Sclerosis

CLINICAL TRIAL TO DETERMINE THE EFFICACY AND SAFETY OF RAPAMYCIN IN ANGIOMYOLIPOMAS IN PATIENTS WITH TUBEROUS SCLEROSIS

Status

Completed

Phases

Phase 4

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01217125

Enrollment

Registered

2010-10-08

Start date

2008-10-31

Completion date

2011-05-31

Last updated

2012-03-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Angiomyolipoma

Keywords

tuberousclerosis, angiomyolipoma, rapamycin

Brief summary

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

Interventions

DRUGSirolimus

plasma levels between 4 and 8

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

10 Years to No maximum

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

Design outcomes

Primary

Measure	Time frame	Description
Angiomyolipoma volume	2 years	Measured by MRI

Secondary

Measure	Time frame	Description
skin lesions, AML complications,	2 years	Evaluate skin lesions Collect event realted to AML

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026