RecruitingOBSERVATIONAL

Study of Individuals Affected With Hypoplasminogenemia

This international study aims to better understand a rare inherited condition called plasminogen deficiency. People with this condition have low levels of plasminogen, a protein important for breaking down blood clots and healing. Researchers will follow up to 100 people with plasminogen deficiency and their close family members over three years. They will collect medical information and blood samples to learn how the condition progresses, why symptoms vary among individuals, and to identify markers that might predict the disease course. The study also plans to create a bank of samples for future research. This information is hoped to lead to improved treatments and a better understanding of this uncommon condition.

At a glance

Status

Recruiting

Sponsor

Indiana Hemophilia &Thrombosis Center, Inc.

Enrolment target

100

Start

18 Dec 2018

Estimated completion

26 Nov 2029

Plasminogen Deficiency

What is this study about?

This study is about a rare health condition called plasminogen deficiency (sometimes called hypoplasminogenemia). People with this condition have lower-than-normal levels of a protein called plasminogen in their blood. Plasminogen is really important because it helps your body break down blood clots and heal properly. When there isn't enough plasminogen, it can lead to various problems, including issues with wound healing.

The main goal of this study is to learn more about how plasminogen deficiency affects people. Because it's a rare condition, doctors don't fully understand why some people have very mild symptoms while others have more serious problems. Researchers want to collect information from a large group of people with this condition and their close family members from all over the world. They hope to understand how the condition develops over time, why different people experience it differently, and if there are any signs that can predict how the condition might progress.

Another important part of this study is to create a 'biobank.' This is like a secure library of blood samples and other materials from participants. These samples will be stored safely and can be used for future research, even after this study is finished. By sharing these samples with other scientists, researchers hope to speed up discoveries and find even better ways to diagnose and treat plasminogen deficiency in the future.

Key takeaways

This study aims to understand a rare condition called plasminogen deficiency.
It will follow people with the condition and their close family for three years.
Researchers will collect health information and samples to learn more.
The goal is to improve understanding and future treatments.
It's an international study, gathering data from around the world.
Participation involves clinic visits and providing blood/urine samples.

Who may be eligible?

To be able to join this study, people must meet certain requirements. You could be eligible if you have been diagnosed with a specific type of plasminogen deficiency (called Type 1 PD) and tests show your plasminogen activity levels are below 50%. Alternatively, if you are a close family member (like a parent, brother, sister, or half-sibling) of someone diagnosed with Type 1 PD, you might also be able to take part.

The study welcomes people of all ages, from infants to older adults. It's important that you have at least one year of your medical history and treatment information available, although this doesn't apply to babies under one year old. You also need to be willing to provide samples, such as blood, and be happy to be followed up by the study team for up to three years.

There are also some reasons why you might not be able to join. For example, if you have had an organ transplant, or if you have a medical or mental health condition that would make it difficult for you to understand the study or follow its requirements. Problems getting blood samples, or being a prisoner, would also mean you couldn't participate.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Have I been diagnosed with Type 1 plasminogen deficiency (plasminogen activity below 50%)?
Am I a parent, brother, sister, or half-sibling of someone with Type 1 plasminogen deficiency?
Do I have at least one year of my medical history available (or am I an infant under 1 year old)?
Am I willing to provide blood samples?
Am I willing to be followed by the study team for up to three years?
Do I fully understand the study and feel able to follow its requirements?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be involved for about three years. You'll have clinic visits where the study team will gather information about your health and medical history. They will also ask for samples, like blood and urine, which will help them understand more about your plasminogen deficiency. These samples will be used for various tests, including checking your plasminogen levels and looking at your genes.

Some of the tests on your samples will be done in Italy, and a collection of all the samples will be stored there securely for future research. For each visit, the study team will explain exactly what will happen. You won't be given any new medication as part of this study; it's focused on understanding the condition and collecting information. You'll have regular follow-up to monitor your health over the three-year period.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit of joining this study is that you would be contributing to a better understanding of plasminogen deficiency, which could lead to improved diagnosis and treatments for people with the condition in the future. You may also receive specific test results related to your plasminogen levels and genetic information. Potential risks are generally low, mainly related to blood draws, such as mild bruising, discomfort, or rarely, infection. There are no new medications or procedures involved in this study. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (35)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

The University of Alabama (UAB)
Verified postcode
Birmingham, United States· Recruiting
Rush University Medical Center
Verified postcode
Chicago, United States· Recruiting
Indiana Hemophila @Thrombosis Center
Verified postcode
Indianapolis, United States· Recruiting
University of Minnesota, Pediatric Hem/Onc & Cancer Survivorship Program
Verified postcode
Minneapolis, United States· Recruiting
Stony Brook University | Stony Brook Medicine
Verified postcode
East Setauket, United States· Recruiting
SUNY Upstate Medical University, Pediatric Hematology/Oncology
Verified postcode
Syracuse, United States· Recruiting
University of North Carolina at Chapel Hill
Verified postcode
Raleigh, United States· Recruiting
Wake Forest University
Verified postcode
Winston-Salem, United States· Recruiting
Hemophilia Center of Western Pennsylvania
Verified postcode
Pittsburgh, United States· Recruiting
Vanderbilt Children's Hematology-Oncology
Verified postcode
Nashville, United States· Completed
Cook Children's Medical Center
Unverified
Forth Worth, United States· Recruiting
The University of Texas Health Science Center at Houston
Verified postcode
Houston, United States· Completed

Common questions

What is plasminogen deficiency?

It's a rare inherited condition where your body doesn't make enough of a protein called plasminogen, which is important for breaking down blood clots and healing.

How long will I be involved in the study?

If you join, you will be part of the study for about three years.

Will I have to take any new medicine?

No, this study is about understanding the condition, not about testing new medications. You will continue with your usual medical care.

Where is the study happening?

This is an international study, with key research centres in the USA and Italy. Data and samples will be collected globally.

Can my family join too?

Yes, close family members (like parents, siblings) of someone with plasminogen deficiency may also be eligible to participate.

How to find out more

Amy D Shapiro, MD

ashapiro@ihtc.org 317-871-0000 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.