Multifocal Lymphangioendotheliomatosis With ThrombocytopeniaVACNCT00576888
Registry for Vascular Anomalies Associated With Coagulopathy
An observational study of Multifocal Lymphangioendotheliomatosis With Thrombocytopenia and Cutaneovisceral Angiomatosis With Thrombocytopenia, run by Medical College of Wisconsin at 1 site in United States.
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Multifocal Lymphangioendotheliomatosis With Thrombocytopenia, Cutaneovisceral Angiomatosis With Thrombocytopenia, Vascular Anomaly With Thrombocytopenia
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2007to 5 Apr 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All patients diagnosed with Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT) or with a vascular anomaly with coagulopathy
Who may be able to join
- Subjects with a vascular anomaly with coagulopathy
Who cannot join
- Subjects without a vascular anomaly with coagulopathy
Trial locations and contacts
1 site in 1 country.
United States1 site
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
About this study
Official title: International Registry for Vascular Anomalies Associated With Coagulopathy
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
Read the 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
- Multifocal Lymphangioendotheliomatosis With Thrombocytopenia, Cutaneovisceral Angiomatosis With Thrombocytopenia, Vascular Anomaly With Thrombocytopenia, Hemangiomas
- Being tested
- no intervention
- Keywords
- Multifocal lymphangioendoltheliomatosis with thrombocytopenia, Cutaneovisceral angiomatosis with thrombocytopenia, Thrombocytopenia, Hemangiomas, Hemangiomatosis, Lymphatic malformation, Kasabach-Merritt, Blue-rubber bleb nevus, Angiomatosis
- Medical subject headings
- Hemangioma, Thrombocytopenia, Lymphatic Abnormalities, Blue rubber bleb nevus syndrome, Angiomatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
no interventionOther
no intervention - observational only
| Study group | Role | What they receive |
|---|---|---|
| Vascular Anomaly with Coagulopathy | All patients diagnosed with Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT) or with a vascular anomaly with coagulopathy Receives: Other: no intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: After DNA collected and batches are sent for analysis
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2007 (estimated)
- Study startNov 2007
- Primary completionDec 2018
- Study completion5 Apr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multifocal Lymphangioendotheliomatosis With ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Medical College of WisconsinThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical College of Wisconsin
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Beth A Drolet, MD, Medical College of Wisconsin
- Investigators
- Beth Drolet, MD, Principal investigator, Medical College of WisconsinUlrich Broeckel, MD, Principal investigator, Medical College of WisconsinHoward Jacob, PhD, Principal investigator, Medical College of WisconsinMichael Kelly, MD, PhD, Principal investigator, Medical College of WisconsinRichard Noel, MD, PhD, Principal investigator, Medical College of WisconsinPaula North, MD, PhD, Principal investigator, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00576888
- Sponsor's protocol ID
- CHW07/226
- Acronym
- VAC
- Status last verified
- Sep 2019
- First submitted
- 18 Dec 2007
- Last updated
- 16 Sep 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- International Registry for Vascular Anomalies Associated With Coagulopathy
- ClinicalTrials.gov ID
- NCT00576888
- Acronym / sponsor ID
- VAC / CHW07/226
- 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
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Medical College of WisconsinUniversity, hospital or other
- Study start
- Nov 2007
- Primary completion
- Dec 2018 (actual)
- Study completion
- 5 Apr 2019 (actual)
- First posted
- 19 Dec 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Other
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vascular Anomaly with Coagulopathy | – | Other: no interventionAll patients diagnosed with Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT) or with a vascular anomaly with coagulopathy |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All patients diagnosed with Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT) or with a vascular anomaly with coagulopathy
Key inclusion criteria (1 listed)
- Subjects with a vascular anomaly with coagulopathy
Key exclusion criteria (1 listed)
- Subjects without a vascular anomaly with coagulopathy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| no interventionno intervention - observational only | Other | Vascular Anomaly with Coagulopathy |
Primary outcome
Number of patients with genetic mutations, copy number variations and/or expression analysis
Time frame: After DNA collected and batches are sent for analysis
Expand knowledge on consensus diagnostic criteria, atypical presentations and long term outcomes of patients with vascular anomalies
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2019.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Beth Drolet, MD principal investigator
Medical College of Wisconsin
Ulrich Broeckel, MD principal investigator
Medical College of Wisconsin
Howard Jacob, PhD principal investigator
Medical College of Wisconsin
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Multifocal Lymphangioendotheliomatosis With Thrombocytopenia and Cutaneovisceral Angiomatosis With Thrombocytopenia, run by Medical College of Wisconsin at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients with genetic mutations, copy number variations and/or expression analysis (time frame: After DNA collected and batches are sent for analysis). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2019.