Clinical trial NCT03001180: Identification of Biomarkers for Patients with Vascular Anomalies
Official title: Identification of Biomarkers for Patients with Vascular Anomalies
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Vascular Anomaly
Generalized Lymphatic Anomaly
Kaposiform Hemangioendothelioma
Kaposiform Lymphangiomatosis
Gorham-Stout Disease
Klippel Trenaunay Syndrome
Congenital Lipomatous Overgrowth, Vascular Malformations, and Epidermal Nevi
Keywords: Generalized Lymphatic Anomaly
Vascular Anomaly
Kaposiform Hemangioendothelioma
Kaposiform Lymphangiomatosis
Vascular Endothelial Growth Factor
Biomarkers
GLA
KLA
KHE
GSD
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
Collaborators: name: Lymphangiomatosis and Gorham's Disease Alliance (LGDA)
name: Klippel Trenaunay (KT) Support Group
name: CLOVES Syndrome Community Support Group
First posted: 2016-12-22
Enrollment: 1000
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Any participant having labs drawn as standard of care will have blood drawn for the study if consented/assented. All participants who are undergoing a surgical procedure or sclerotherapy are currently consented for participation in the tissue bank. The principle investigator will be looking at tissue from these participants. There is no further recruitment for the study.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Correlation of Biomarkers with Differential Diagnosis
timeFrame: An Average of Every 2 Years
measure: Correlation of Biomarkers with Disease Severity
timeFrame: An Average of Every 2 Years
measure: Correlation of Biomarkers with Response to Therapies
timeFrame: An Average of Every 2 Years
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Timothy LeCras, PhD
role: PRINCIPAL_INVESTIGATOR
References: