STBETA: Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers

Sponsor

University Hospital, Bordeaux (Other)

Overall Status

Completed

CT.gov ID

NCT02104011

Collaborator

(none)

Enrollment

Location

Arm

30.1

Actual Duration (Months)

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Renal Angiomyolipomas Tuberous Sclerosis	Drug: Propranolol	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

2 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

May 22, 2015

Actual Primary Completion Date :

Nov 22, 2017

Actual Study Completion Date :

Nov 22, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Patient	Drug: Propranolol

Arm

Intervention/Treatment

Experimental: Patient

Drug: Propranolol

Outcome Measures

Primary Outcome Measures

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

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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.