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

Completed

• Conditions: PEComa; Hemangioma
• Interventions: Other: Biopsy

• Registration Number: NCT02334930
• Lead Sponsor: University Hospital, Bordeaux

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 Description

Not available

Study Timeline

• Study First Submitted: 2014-12-22
• Study First Submitted QC: 2015-01-06
• Study First Posted: 2015-01-08
• Study Start: 2015-04
• Primary Completion: 2016-01
• Study Completion: 2016-01
• Status Verified: 2016-08
• Last Update Submitted: 2016-08-19
• Last Update Posted: 2016-08-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 13

Inclusion Criteria

• patients who will have biopsy or surgery for PEComa or vascular pediatric tumor (RICH, NICH, hemangioma or pyogenic granuloma)
• A free, informed and written consent will be established

Exclusion Criteria

• none

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients	Biopsy	Patients who will have biopsy or surgery for Perivascular epithelioid cell tumor (PEComa) or vascular pediatric tumor (Rapidly Involuting Congenital Hemangioma (RICH), Non Involuting Congenital Hemangioma (NICH), hemangioma or pyogenic granuloma)

Primary Outcome Measures

Name	Time	Method
YAP cellular expression by immunohistochemistry study on collected tissues	At inclusion (day 0)]

Secondary Outcome Measures

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

Trial Locations

Locations (1)

CHU de Bordeaux; 🇫🇷 Bordeaux, France