Trial record 10 of 39 for:    Tuberous Sclerosis

Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers (ST BETA)

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified March 2014 by University Hospital, Bordeaux
Sponsor:
Collaborator:
Association Sclérose Tubéreuse de Bourneville
Information provided by (Responsible Party):
University Hospital, Bordeaux
ClinicalTrials.gov Identifier:
NCT02104011
First received: April 1, 2014
Last updated: April 3, 2014
Last verified: March 2014
  Purpose

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 Intervention Phase
Renal Angiomyolipomas
Tuberous Sclerosis
Drug: Propranolol
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers: Pilot Trial

Resource links provided by NLM:


Further study details as provided by University Hospital, Bordeaux:

Primary Outcome Measures:
  • Evolution of angiomyolipomas volume [ Time Frame: 6 months and 1 year after inclusion ] [ Designated as safety issue: No ]
    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 [ Time Frame: 6 months and 1 year after inclusion ] [ Designated as safety issue: No ]
    Improvement of renal function after 6 months and 1 year of treatment

  • Effect on the potential haemorraghic transformation [ Time Frame: 6 months and 1 year after inclusion ] [ Designated as safety issue: No ]
    Haemorraghic transformation of angiomyolipomas is diagnosed by Scanner or MRI.

  • Improvement of the quality of life [ Time Frame: 6 months and 1 year after inclusion. ] [ Designated as safety issue: No ]
    Th evolution of the quality of life is assessed by an EVA scale and by QOL scale.

  • Effect on face angiofibromas [ Time Frame: 6 months and 1 year after inclusion ] [ Designated as safety issue: No ]
    Evolution of the face angiofibromas by a dermatologic assessment.


Estimated Enrollment: 15
Study Start Date: March 2014
Estimated Study Completion Date: March 2016
Estimated Primary Completion Date: March 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Patient Drug: Propranolol

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02104011

Contacts
Contact: Claire RIGOTHIER, Dr claire.rigothier@chu-bordeaux.fr
Contact: Christian COMBE, Pr christian.combe@chu-bordeaux.fr

Locations
France
Nephrology department Not yet recruiting
Bordeaux, France
Sponsors and Collaborators
University Hospital, Bordeaux
Association Sclérose Tubéreuse de Bourneville
Investigators
Principal Investigator: Claire RIGOTHIER, Dr UH Bordeaux
  More Information

No publications provided

Responsible Party: University Hospital, Bordeaux
ClinicalTrials.gov Identifier: NCT02104011     History of Changes
Other Study ID Numbers: CHUBX 2011/35
Study First Received: April 1, 2014
Last Updated: April 3, 2014
Health Authority: France: Agence Nationale de Sécurité du Médicament et des produits de santé

Keywords provided by University Hospital, Bordeaux:
Angiomyolipomas in tuberous sclerosis
Beta-blockers
Anti-angiogenic therapies

Additional relevant MeSH terms:
Sclerosis
Tuberous Sclerosis
Angiomyolipoma
Pathologic Processes
Hamartoma
Neoplasms
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Neoplasms, Adipose Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Perivascular Epithelioid Cell Neoplasms
Adrenergic beta-Antagonists
Propranolol
Adrenergic Antagonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Anti-Arrhythmia Agents
Cardiovascular Agents

ClinicalTrials.gov processed this record on August 18, 2014