Back to resultsTreatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers (STBETA)
Primary Purpose
Renal Angiomyolipomas, Tuberous Sclerosis
Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Propranolol
Sponsored by
About this trial
This is an interventional treatment trial for Renal Angiomyolipomas focused on measuring Angiomyolipomas in tuberous sclerosis, Beta-blockers, Anti-angiogenic therapies
Eligibility Criteria
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.
Sites / Locations
- Nephrology department
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Patient
Arm Description
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Renal function evolution
Improvement of renal function after 6 months and 1 year of treatment
Effect on the potential haemorraghic transformation
Haemorraghic transformation of angiomyolipomas is diagnosed by Scanner or MRI.
Improvement of the quality of life
Th evolution of the quality of life is assessed by an EVA scale and by QOL scale.
Effect on face angiofibromas
Evolution of the face angiofibromas by a dermatologic assessment.
Full Information
NCT ID
NCT02104011
First Posted
April 1, 2014
Last Updated
January 19, 2018
Sponsor
University Hospital, Bordeaux
1. Study Identification
Unique Protocol Identification Number
NCT02104011
Brief Title
Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers
Acronym
STBETA
Official Title
Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers: Pilot Trial
Study Type
Interventional
2. Study Status
Record Verification Date
January 2018
Overall Recruitment Status
Completed
Study Start Date
May 22, 2015 (Actual)
Primary Completion Date
November 22, 2017 (Actual)
Study Completion Date
November 22, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Bordeaux
4. Oversight
Data Monitoring Committee
Yes
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Renal Angiomyolipomas, Tuberous Sclerosis
Keywords
Angiomyolipomas in tuberous sclerosis, Beta-blockers, Anti-angiogenic therapies
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Patient
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Propranolol
Primary Outcome Measure Information:
Title
Evolution of angiomyolipomas volume
Description
Stabilization or even regression of angiomyolipomas volume after 6 months and 1 year of treatment with a quantification of the vascular component.
Time Frame
6 months and 1 year after inclusion
Secondary Outcome Measure Information:
Title
Renal function evolution
Description
Improvement of renal function after 6 months and 1 year of treatment
Time Frame
6 months and 1 year after inclusion
Title
Effect on the potential haemorraghic transformation
Description
Haemorraghic transformation of angiomyolipomas is diagnosed by Scanner or MRI.
Time Frame
6 months and 1 year after inclusion
Title
Improvement of the quality of life
Description
Th evolution of the quality of life is assessed by an EVA scale and by QOL scale.
Time Frame
6 months and 1 year after inclusion.
Title
Effect on face angiofibromas
Description
Evolution of the face angiofibromas by a dermatologic assessment.
Time Frame
6 months and 1 year after inclusion
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Claire RIGOTHIER, Dr
Organizational Affiliation
UH Bordeaux
Official's Role
Principal Investigator
Facility Information:
Facility Name
Nephrology department
City
Bordeaux
Country
France
12. IPD Sharing Statement
Learn more about this trial
Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers
We'll reach out to this number within 24 hrs