Identification of Biomarkers for Patients With Vascular Anomalies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.

ClinicalTrials.gov Identifier: NCT03001180

Recruitment Status : Recruiting

First Posted : December 22, 2016

Last Update Posted : February 9, 2021

See Contacts and Locations

Sponsor:

Collaborators:

Lymphangiomatosis and Gorham's Disease Alliance (LGDA)

Klippel Trenaunay (KT) Support Group

CLOVES Syndrome Community Support Group

Information provided by (Responsible Party):

Children's Hospital Medical Center, Cincinnati

Study Description

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.

Condition or disease
Vascular Anomaly Generalized Lymphatic Anomaly Kaposiform Hemangioendothelioma Kaposiform Lymphangiomatosis Gorham-Stout Disease Klippel Trenaunay Syndrome Congenital Lipomatous Overgrowth, Vascular Malformations, and Epidermal Nevi

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 Design

Layout table for study information

Study Type :	Observational
Estimated Enrollment :	1000 participants
Observational Model:	Case-Control
Time Perspective:	Cross-Sectional
Official Title:	Identification of Biomarkers for Patients With Vascular Anomalies
Study Start Date :	April 2015
Estimated Primary Completion Date :	January 2050

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Klippel-Trenaunay syndrome Capillary malformation-arteriovenous malformation syndrome Parkes Weber syndrome

Genetic and Rare Diseases Information Center resources: Klippel-Trenaunay Syndrome Parkes Weber Syndrome Weber Syndrome Hemangioendothelioma Kaposiform Hemangioendothelioma Lymphangiomatosis Kaposiform Lymphangiomatosis Gorham's Disease CLOVES Syndrome Sturge-Weber Syndrome

U.S. FDA Resources

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :

Correlation of Biomarkers with Differential Diagnosis [ Time Frame: An Average of Every 2 Years ]
Correlation of Biomarkers with Disease Severity [ Time Frame: An Average of Every 2 Years ]
Correlation of Biomarkers with Response to Therapies [ Time Frame: An Average of Every 2 Years ]

Biospecimen Retention: Samples With DNA

Tissue, Blood, Plasma, and Serum Samples

Eligibility Criteria

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information

Ages Eligible for Study:	Child, Adult, Older Adult
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No
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.

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:

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03001180

Contacts

Layout table for location contacts

Contact: Timothy LeCras, PhD	5138034862	hvmcresearch@cchmc.org

Locations

Layout table for location information

United States, Massachusetts
Boston Children's Hospital	Terminated
Boston, Massachusetts, United States, 02115
United States, Ohio
Cincinnati Children's Hospital Medical Center	Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Megan Metcalf 513-803-2606 hvmcresearch@cchmc.org
Principal Investigator: Timothy LeCras, PhD
Sub-Investigator: Adrienne Hammill, MD, PhD

Sponsors and Collaborators

Children's Hospital Medical Center, Cincinnati

Lymphangiomatosis and Gorham's Disease Alliance (LGDA)

Klippel Trenaunay (KT) Support Group

CLOVES Syndrome Community Support Group

Investigators

Layout table for investigator information

Principal Investigator:	Timothy LeCras, PhD	Children's Hospital Medical Center, Cincinnati

More Information

Layout table for additonal information

Responsible Party:	Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:	NCT03001180
Other Study ID Numbers:	CCHMC-LMI
First Posted:	December 22, 2016 Key Record Dates
Last Update Posted:	February 9, 2021
Last Verified:	February 2021

Keywords provided by Children's Hospital Medical Center, Cincinnati:


Generalized Lymphatic Anomaly Vascular Anomaly Kaposiform Hemangioendothelioma Kaposiform Lymphangiomatosis Vascular Endothelial Growth Factor	Biomarkers GLA KLA KHE GSD

Additional relevant MeSH terms:

Layout table for MeSH terms

Hemangioendothelioma Osteolysis, Essential Vascular Malformations Klippel-Trenaunay-Weber Syndrome Congenital Abnormalities Cardiovascular Abnormalities Cardiovascular Diseases Hemangioma Neoplasms, Vascular Tissue	Neoplasms by Histologic Type Neoplasms Angiomatosis Vascular Diseases Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Osteolysis Bone Resorption

To Top