Clinical Trial: Evaluation of New Biomarkers Predictive of Efficacy Betablockers in PEComa and Vascular Pediatric Tumors

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: Evaluation of New Biomarkers Predictive of Efficacy Betablockers in PEComa and Vascular Pediatric Tumors

Brief Summary:

PEComa is rare tumor affecting particularly patients with Tuberous Sclerosis. Biological similarities were seen between PEComa and infantile hemangioma. Propranolol is highly efficient to treat infantile hemangioma and we believe that this drug can also be useful for the treatment of PEComa.

Purpose : to understand the mechanism of action of propranolol in PEComas and related pediatric vascular lesions and to select possible novel targets of betablockers in vascular tumors related to PEComas by using YAP oncogene activation as a molecular marker


Detailed Summary:
Sponsor: University Hospital, Bordeaux

Current Primary Outcome: YAP cellular expression by immunohistochemistry study on collected tissues [ Time Frame: At inclusion (day 0)] ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Beta adrenergic receptors expression by immunohistochemistry study on collected tissues [ Time Frame: At inclusion (day 0)] ]
  • Study of Hippo/YAP pathway expression by western-blot, and correlation with propranolol efficacy [ Time Frame: At inclusion (day 0)] ]
  • Study of MAP/kinases pathway expression by western-blot, and correlation with propranolol efficacy [ Time Frame: At inclusion (day 0)] ]
  • Study of PKA/AMPc pathway expression by western-blot, and correlation with propranolol efficacy [ Time Frame: At inclusion (day 0)] ]


Original Secondary Outcome: Same as current

Information By: University Hospital, Bordeaux

Dates:
Date Received: December 22, 2014
Date Started: April 2015
Date Completion:
Last Updated: August 19, 2016
Last Verified: August 2016