Identification of Biomarkers for Patients with Vascular Anomalies

Recruiting

• Conditions: Kaposiform Lymphangiomatosis; Generalized Lymphatic Anomaly; Gorham-Stout Disease; Congenital Lipomatous Overgrowth, Vascular Malformations, and Epidermal Nevi; Vascular Anomaly; Kaposiform Hemangioendothelioma; Klippel Trenaunay Syndrome

• Registration Number: NCT03001180
• Lead Sponsor: Children's Hospital Medical Center, Cincinnati

Brief Summary

The study will use blood (serum and plasma) and tissue obtained from participants undergoing prescribed surgical resection of vascular anomalies of interest proposed in this study. The study will also use blood (serum and plasma) and tissue collected and stored in a tissue bank maintained by the Department of Hematology/Oncology.

Detailed Description

While vascular anomalies are rare diseases, they can be life-threatening and devastating to affected children and their families. Advances in diagnosis, monitoring and therapies will be significantly improved if non-invasive biomarkers that are sensitive and specific can be identified. Obtaining a tissue biopsy to help in diagnosis can actually worsen the disease and so identification of specific blood biomarkers is highly desirable. Studies will measure angiogenic factors in serum and plasma samples at baseline and on therapy. Tissue removed during surgical resection or blood removed prior to sclerotherapy will be used to obtain cells and tissue for the assessment of where biomarkers are coming from and to identify disease-causing pathways for new therapeutic targeting.

Study Timeline

• Study Start: 2015-04
• Study First Submitted: 2016-12-15
• Study First Submitted QC: 2016-12-20
• Study First Posted: 2016-12-22
• Status Verified: 2024-10
• Last Update Submitted: 2024-11-05
• Last Update Posted: 2024-11-06
• Primary Completion: 2050-12
• Study Completion: 2050-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 1000

Inclusion Criteria

• Any patient having labs drawn as standard of care will have blood drawn for the study if consented/ assented.
• All patients who are undergoing a surgical procedure or sclerotherapy are currently consented for participation in the tissue bank.

Exclusion Criteria

• N/A

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Correlation of Biomarkers with Disease Severity	An Average of Every 2 Years
Correlation of Biomarkers with Differential Diagnosis	An Average of Every 2 Years
Correlation of Biomarkers with Response to Therapies	An Average of Every 2 Years

Trial Locations

Locations (2)

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States