Understanding Hypoplasminogenemia and Its Effects

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to investigate the natural history of hypoplasminogenemia by collecting data from a large cohort of affected individuals and their first-degree family members. It will utilize both retrospective and prospective data collection methods, focusing on identifying factors that correlate with disease expression and severity. Additionally, a specimen biobank will be established to facilitate further research on this condition. The study will be conducted internationally, with primary data collection at the Indiana Hemophilia & Thrombosis Center and collaboration with the University of Milan.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Signed informed consent and assent as applicable (Appendix 1)
2. A. Males or females with type 1 PD diagnosed locally with plasminogen activity levels \<50% OR B. First degree family members of a person diagnosed with type 1 PD (includes parents, siblings, half-siblings)
3. All ages included
4. Available clinical history and treatment for at least 1 year prior to entry except for infants \< 1 year of age
5. Willingness to provide samples for analysis including DNA, plasma etc.
6. Willingness to participate in prospective follow-up for up to 3 years

Exclusion Criteria:

1. Previous organ transplant recipient
2. Any psychiatric disorder, other mental disorder, or any other medical disorder that impairs the subject's ability to give informed consent or to comply with the requirements of the study protocol
3. Refuses to provide informed consent
4. Special patient populations, including prisoners or, are deemed medically or cognitively unsuitable for research by their treating physician
5. Inability to obtain a blood sample due to poor or limited venous access

Where it's running

• The University of Alabama (UAB)
  Birmingham, Alabama, United States Recruiting
• Rush University Medical Center
  Chicago, Illinois, United States Recruiting
• Indiana Hemophila @Thrombosis Center
  Indianapolis, Indiana, United States Recruiting
• University of Minnesota, Pediatric Hem/Onc & Cancer Survivorship Program
  Minneapolis, Minnesota, United States Recruiting
• Stony Brook University | Stony Brook Medicine
  East Setauket, New York, United States Recruiting
• SUNY Upstate Medical University, Pediatric Hematology/Oncology
  Syracuse, New York, United States Recruiting

Study contacts

• Principal investigator: Amy D Shapiro, MD — Indiana Hemophilia &Thrombosis Center, Inc.
• Study coordinator: Amy D Shapiro, MD
• Email: ashapiro@ihtc.org
• Phone: 317-871-0000

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Using Hemlibra to treat mild hemophilia A

  This study is testing if Hemlibra can safely help men with mild hemophilia A manage their condition and improve their joint health and quality of life.

  Recruiting Phase 4 Indiana Hemophilia &Thrombosis Center, Inc. · Indianapolis, Indiana · NCT04567511

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.