Registry for Vascular Anomalies Associated With Coagulopathy | NCT00576888

Studies
Registry for Vascular Anomalies Associated With Coagulopathy

NCT00576888

Sponsor

Medical College of Wisconsin

Status

Completed

Last Update Posted

Sep 16, 2019Actual

Enrollment

30Actual

Phase

Not provided

Conditions

Multifocal Lymphangioendotheliomatosis With Thrombocytopenia
Cutaneovisceral Angiomatosis With Thrombocytopenia
Vascular Anomaly With Thrombocytopenia
Hemangiomas

Interventions

no intervention

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Sep 2019

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 2007

Primary Completion Date

Dec 2018Actual

Completion Date

Apr 5, 2019Actual

First Submitted Date

Dec 18, 2007

First Submission Date that Met QC Criteria

Dec 18, 2007

First Posted Date

Dec 19, 2007Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 12, 2019

Last Update Posted Date

Sep 16, 2019Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Beth A Drolet, MD, Professor of Dermatology and Pediatrics, Medical College of WisconsinPrincipal Investigator

Lead Sponsor

Medical College of WisconsinOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief 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

Detailed 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.

Conditions Module

Conditions

Multifocal Lymphangioendotheliomatosis With Thrombocytopenia
Cutaneovisceral Angiomatosis With Thrombocytopenia
Vascular Anomaly With Thrombocytopenia
Hemangiomas

Keywords

Multifocal lymphangioendoltheliomatosis with thrombocytopenia
Cutaneovisceral angiomatosis with thrombocytopenia
Thrombocytopenia
Hemangiomas
Hemangiomatosis
Lymphatic malformation
Kasabach-Merritt
Blue-rubber bleb nevus
Angiomatosis

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

30Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Vascular Anomaly with Coagulopathy		All patients diagnosed with Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT) or with a vascular anomaly with coagulopathy	Other: no intervention

Interventions

Name	Type	Description	Arm Group Labels	Other Names
no intervention	Other	no intervention - observational only	Vascular Anomaly with Coagulopathy

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Subjects with a vascular anomaly with coagulopathy

Exclusion Criteria:

Subjects without a vascular anomaly with coagulopathy

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

All patients diagnosed with Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT) or with a vascular anomaly with coagulopathy

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Beth Drolet, MD	Medical College of Wisconsin	Principal Investigator
Ulrich Broeckel, MD	Medical College of Wisconsin	Principal Investigator
Howard Jacob, PhD	Medical College of Wisconsin	Principal Investigator
Michael Kelly, MD, PhD	Medical College of Wisconsin	Principal Investigator
Richard Noel, MD, PhD	Medical College of Wisconsin	Principal Investigator
Paula North, MD, PhD	Medical College of Wisconsin	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Medical College of Wisconsin		Milwaukee	Wisconsin	53226	United States

Condition Browse Module

MeSH Terms

ID	Term
D006391	Hemangioma
D013921	Thrombocytopenia
D044148	Lymphatic Abnormalities
C536240	Blue rubber bleb nevus syndrome
D000798	Angiomatosis

Ancestor Terms

ID	Term
D009383	Neoplasms, Vascular Tissue
D009370	Neoplasms by Histologic Type
D009369	Neoplasms
D001791	Blood Platelet Disorders

Browse Leaves

Not provided

Browse Branches

Not provided