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

NCT02334930 | Completed

• 1 other identifier: CHUBX 2013/34
• Study Type: observational
• Target: 13
• Locations: 1 country
• Sites: 1

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

Conditions

PEComa; Hemangioma

Keywords

propranolol; betablocker; angiogenesis

Outcome Measures

Primary Outcomes (1)

• YAP cellular expression by immunohistochemistry study on collected tissues

  At inclusion (day 0)]

Secondary Outcomes (4)

• 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)]

Study Arms (1)

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

Interventions

Biopsy OTHER

Patients

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients who will have biopsy or surgery for PEComa or vascular pediatric tumor (RICH, NICH, hemangioma or pyogenic granuloma)

You may qualify if:

• 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

You may not qualify if:

• none

Sponsors & Collaborators

• University Hospital, Bordeaux: lead
• Ministry for Health and Solidarity, France: collaborator
• National Cancer Institute, France: collaborator

Study Sites (1)

CHU de Bordeaux

Bordeaux, 33000, France

Location

MeSH Terms

Conditions

Perivascular Epithelioid Cell Neoplasms; Hemangioma

Interventions

Biopsy

Condition Hierarchy (Ancestors)

Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Vascular Tissue

Intervention Hierarchy (Ancestors)

Cytodiagnosis; Cytological Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Specimen Handling; Diagnostic Techniques, Surgical; Surgical Procedures, Operative; Investigative Techniques

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2014
• First Posted: January 8, 2015
• Study Start: April 1, 2015
• Primary Completion: January 1, 2016
• Study Completion: January 1, 2016
• Last Updated: August 22, 2016

Record last verified: 2016-08

Locations

FR (1)