Renal Angiomyolipomas, Tuberous Sclerosis
Conditions
Keywords
Angiomyolipomas in tuberous sclerosis, Beta-blockers, Anti-angiogenic therapies
Brief summary
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.
Interventions
DRUGPropranolol
Sponsors
University Hospital, Bordeaux
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evolution of angiomyolipomas volume | 6 months and 1 year after inclusion | Stabilization or even regression of angiomyolipomas volume after 6 months and 1 year of treatment with a quantification of the vascular component. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Renal function evolution | 6 months and 1 year after inclusion | Improvement of renal function after 6 months and 1 year of treatment |
| Effect on the potential haemorraghic transformation | 6 months and 1 year after inclusion | Haemorraghic transformation of angiomyolipomas is diagnosed by Scanner or MRI. |
| Improvement of the quality of life | 6 months and 1 year after inclusion. | Th evolution of the quality of life is assessed by an EVA scale and by QOL scale. |
| Effect on face angiofibromas | 6 months and 1 year after inclusion | Evolution of the face angiofibromas by a dermatologic assessment. |
Countries
France
Contacts
PRINCIPAL_INVESTIGATORClaire RIGOTHIER, Dr
University Hospital, Bordeaux
Outcome results
None listed