PEC-Hem: Evaluation of New Biomarkers Predictive of Efficacy Betablockers in PEComa and Vascular Pediatric Tumors

Sponsor

University Hospital, Bordeaux (Other)

Overall Status

Completed

CT.gov ID

NCT02334930

Collaborator

Ministry for Health and Solidarity, France (Other), National Cancer Institute, France (Other)

Enrollment

Location

Duration (Months)

1.4

Patients Per Site Per Month

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
PEComa Hemangioma	Other: Biopsy

Study Design

Study Type:

Observational

Actual Enrollment :

13 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Study Start Date :

Apr 1, 2015

Actual Primary Completion Date :

Jan 1, 2016

Actual Study Completion Date :

Jan 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
Patients 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)	Other: Biopsy

Arm

Intervention/Treatment

Patients

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)

Other: Biopsy

Outcome Measures

Primary Outcome Measures

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

Secondary Outcome Measures

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)]]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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