Registry for Vascular Anomalies Associated With Coagulopathy

Hemangiomas Clinical Trial

— VAC  

Official title:

International Registry for Vascular Anomalies Associated With Coagulopathy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00576888
• Other study ID #: CHW07/226
• Status: Completed
• Start date: November 2007
• Est. completion date: April 5, 2019

Study information

• Verified date: September 2019
• Source: Medical College of Wisconsin
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

PURPOSE The purpose of this study is to learn more about multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT). MLT is a rare vascular disorder characterized by multiple congenital skin and visceral lesions, profound thrombocytopenia, and gastrointestinal bleeding. The skin lesions may appear red, brown or blue, often misdiagnosed as hemangiomas. The gastrointestinal tract, liver, and lungs are the most common internal organs involved. The severe thrombocytopenia (low platelets) is believed to be the result of platelet trapping within the skin and visceral vascular lesions. Severe and chronic gastrointestinal bleeding is common during infancy and early childhood. Although a relatively newly described entity, MLT was likely previously reported as hemangiomas, blue rubber bleb nevus syndrome, diffuse hemangiomatosis, Kasabach-Merritt phenomenon, and hereditary hemorrhagic telangiectasia. The term cutaneovisceral angiomatosis with thrombocytopenia is also a term used for this same disease. This study is a longitudinal cohort study of MLT to collect detailed clinical data on the distribution of disease, disease severity, and complications. This data will be used to create diagnostic criteria and an evaluation protocol for infants with this disease

Description:

After informed consent is obtained a detailed question will be mailed to participating patients and families. This questionnaire will also be available electronically through an educational website. Data collected will include photographs of skin lesions, video images of gastrointestinal lesions, demographic data, clinical information, therapeutic interventions, glass slides of tissue biopsies, and collection of DNA. Enrollment will be patient family driven and modeled after several successful registries of rare diseases.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: April 5, 2019
• Est. primary completion date: December 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Subjects with a vascular anomaly with coagulopathy

Exclusion Criteria:

• Subjects without a vascular anomaly with coagulopathy

Related Conditions & MeSH terms

• Congenital Abnormalities
• Cutaneovisceral Angiomatosis With Thrombocytopenia
• Hemangioma
• Hemangiomas
• Multifocal Lymphangioendotheliomatosis With Thrombocytopenia
• Thrombocytopenia
• Vascular Anomaly With Thrombocytopenia
• Vascular Malformations

Intervention

Other:
• no intervention
no intervention - observational only

Locations

Country	Name	City	State
United States	Medical College of Wisconsin	Milwaukee	Wisconsin

Sponsors (1)

Lead Sponsor	Collaborator
Medical College of Wisconsin

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of patients with genetic mutations, copy number variations and/or expression analysis	Expand knowledge on consensus diagnostic criteria, atypical presentations and long term outcomes of patients with vascular anomalies	After DNA collected and batches are sent for analysis