Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers

Phase 2

Completed

• Conditions: Renal Angiomyolipomas; Tuberous Sclerosis
• Interventions: Drug: Propranolol

• Registration Number: NCT02104011
• Lead Sponsor: University Hospital, Bordeaux

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-04-01
• Study First Submitted QC: 2014-04-03
• Study First Posted: 2014-04-04
• Study Start: 2015-05-22
• Primary Completion: 2017-11-22
• Study Completion: 2017-11-22
• Status Verified: 2018-01
• Last Update Submitted: 2018-01-19
• Last Update Posted: 2018-01-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 2

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Patient	Propranolol	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Nephrology department; 🇫🇷 Bordeaux, France