Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers

Study Overview
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Brief Summary
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Treatment of angiomyolipomas is based on invasive techniques such as surgery or embolization. Development of anti-angiogenic therapies is a major and growing field of research in hypervascularized tumors. Angiomyolipomas have been shown to regress after prolonged treatment with mTOR inhibitors (Sirolimus), but with a large proportion of secondary effects. We showed recently that beta-blockers were able to induce regression of infantile hemagiomas. Consequently, we looked for and found, histologically, in a few cases of angiomyolipomas the presence of beta2 receptors. The aim of the study is to estimate if beta-blockers could induce regression or stabilization of renal angiomyolipomas in tuberous sclerosis in a pilot study.

Official Title
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Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers: Pilot Trial

Conditions
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Renal Angiomyolipomas, Tuberous Sclerosis

Intervention / Treatment
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* Drug: Propranolol


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Tuberous sclerosis patients with one or several angiomyolipomas of a size of at least 4 cms. Exclusion Criteria: Patients whom CT or MR scan shows one or several intra-lesional aneurisms requiring a preventive embolization. Patients with a retroperitoneal hemorragic complication requiring a preventive embolization. Patients whom biopsy will show an adenocarcinoma, hypertension non controlled, renal failure and severe liver. Diabetic subjects insufficiently controlled. Beta-blockers contra-indication. Psychosis, severe mental disorder. Patient already treated with beta-blockers or mTOR inhibitors. Pregnant or nursing women.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Patient<br> | Drug: Propranolol<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evolution of angiomyolipomas volume | Stabilization or even regression of angiomyolipomas volume after 6 months and 1 year of treatment with a quantification of the vascular component. | 6 months and 1 year after inclusion | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Renal function evolution | Improvement of renal function after 6 months and 1 year of treatment | 6 months and 1 year after inclusion | 
| Effect on the potential haemorraghic transformation | Haemorraghic transformation of angiomyolipomas is diagnosed by Scanner or MRI. | 6 months and 1 year after inclusion | 
| Improvement of the quality of life | Th evolution of the quality of life is assessed by an EVA scale and by QOL scale. | 6 months and 1 year after inclusion. | 
| Effect on face angiofibromas | Evolution of the face angiofibromas by a dermatologic assessment. | 6 months and 1 year after inclusion | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Angiomyolipomas in tuberous sclerosis, Beta-blockers, Anti-angiogenic therapies