Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
100 participants
OBSERVATIONAL
2018-12-18
2027-04-19
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Biological, Genetic and Environmental Involved in the Complications of Sickle Cell Disease
NCT04205123
The Genetics and Functional Basis of Inherited Platelet, White Blood Cell, Red Blood Cell, and Blood Clotting Disorders.
NCT00230165
Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia
NCT01496963
Bone Marrow for Hemoglobinopathy Research
NCT00669305
Identification of Acute Intermittent Porphyria Modifying Genes
NCT05502133
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
1. Define PLGD natural history in a large cohort of individuals with hypoplasminogenemia and their first-degree family members.
2. Identify factors that correlate with disease expression and severity.
3. Create a specimen biobank for further studies, available to other researchers.
The project will be international in scope with two collaborating centers that have created and will collect the subject data and samples. In North/Central/South America, the Indiana Hemophilia \& Thrombosis Center (IHTC) will serve as the primary site while University of Milan will serve as the center for all other sites. The database is housed at the University of Milan, Italy.
Study population will include males and females affected with hyposplasminogenemia of any age. Both one-year retrospective and three-year prospective data will be collected on an international cohort of 100 affected individuals and their first degree family members (parents, siblings; total estimated study population \~500).
Study sample analysis, except for urine analyses, will be centralized and performed in Italy; the plasminogen antibody analysis will be batched for analysis, and the urine analyses will be performed locally. A sample biorepository will be created and ultimately housed in Italy. The study will provide testing for plasminogen activity and antigen, plasminogen genetic analysis, polymorphisms in genes that impact plasminogen expression and fibrinolysis, and global hemostatic assays. In addition, stored samples will be used for further testing and analyses to potentially include whole genome sequencing to further identify plasminogen genetic mutations as needed and to investigate other genetic modifiers of disease expression. An exploratory aim includes investigating the potential relationship with streptococcal strains and altered plasminogen products.
The study period will be 3 years for each enrolled subject. In-person visits will be conducted and samples for analysis will be collected at baseline and at end of study. Interval follow-up will be performed every 6 months by telephone. data will be collected at unscheduled visits that are performed for clinical need at the treating physician's discretion.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
COHORT
OTHER
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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
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
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Fondazione Angelo Bianchi Bonomi
OTHER
Indiana Hemophilia &Thrombosis Center, Inc.
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Amy D Shapiro, MD
Medical Director
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Amy D Shapiro, MD
Role: PRINCIPAL_INVESTIGATOR
Indiana Hemophilia &Thrombosis Center, Inc.
Flora Peyvandi, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Univeristy of Milan
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
The University of Alabama (UAB)
Birmingham, Alabama, United States
Rush University Medical Center
Chicago, Illinois, United States
Indiana Hemophila @Thrombosis Center
Indianapolis, Indiana, United States
University of Minnesota, Pediatric Hem/Onc & Cancer Survivorship Program
Minneapolis, Minnesota, United States
Stony Brook University | Stony Brook Medicine
East Setauket, New York, United States
SUNY Upstate Medical University, Pediatric Hematology/Oncology
Syracuse, New York, United States
Wake Forest University
Winston-Salem, North Carolina, United States
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States
Vanderbilt Children's Hematology-Oncology
Nashville, Tennessee, United States
Cook Children's Medical Center
Forth Worth, Texas, United States
The University of Texas Health Science Center at Houston
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Hospital Britanico Buenos Aires
Buenos Aires, , Argentina
Murdoch Children's Research Institute, The Royal Children's Hospital
Melbourne, Victoria, Australia
Children's Health Queensland Hospital and Health Service
South Brisbane, , Australia
Medical University of Innsbruck, University Clinic for Pediatrics and Adolescent Medicine
Innsbruck, , Austria
Windsor Regional Hospital
Windsor, Ontario, Canada
CHU Sainte-Justine
Montreal, Quebec, Canada
CHU de Québec Université Laval
Québec, Quebec, Canada
University of Saskatchewan
Saskatoon, , Canada
Alexandra Hospital, Athens, Hematology Department
Athens, , Greece
Safra's Children Hospital, Sheba Medical Center
Tel Aviv, , Israel
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center,
Milan, , Italy
University Hospital of Padova
Padua, , Italy
Faculty of Medicine, Chiang Mai University
Chiang Mai, , Thailand
Dokuz Eylul University pediatric Pulmonology, Allergy and Clinical Immunology
Izmir, Balçova, Turkey (Türkiye)
Istanbul Üniversitesi Onkoloji Enstitüsü
Istanbul, , Turkey (Türkiye)
Istanbul University Cerrahpsasa, Cerrahpsasa Medical Faculty Pediatric Hematology and Oncology Department
Istanbul, , Turkey (Türkiye)
Yuzuncu Yil University Faculty of Medicine Department of Ophthalmology
Van, , Turkey (Türkiye)
Royal Free Hospital, Royal Free London NHS Foundation Trust
London, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
References
Explore related publications, articles, or registry entries linked to this study.
Tait RC, Walker ID, Conkie JA, Islam SI, McCall F, Mitchell R, Davidson JF. Plasminogen levels in healthy volunteers--influence of age, sex, smoking and oral contraceptives. Thromb Haemost. 1992 Nov 10;68(5):506-10.
Schuster V, Hugle B, Tefs K. Plasminogen deficiency. J Thromb Haemost. 2007 Dec;5(12):2315-22. doi: 10.1111/j.1538-7836.2007.02776.x. Epub 2007 Sep 26.
Ma Q, Ozel AB, Ramdas S, McGee B, Khoriaty R, Siemieniak D, Li HD, Guan Y, Brody LC, Mills JL, Molloy AM, Ginsburg D, Li JZ, Desch KC. Genetic variants in PLG, LPA, and SIGLEC 14 as well as smoking contribute to plasma plasminogen levels. Blood. 2014 Nov 13;124(20):3155-64. doi: 10.1182/blood-2014-03-560086. Epub 2014 Sep 10.
Celkan T. Plasminogen deficiency. J Thromb Thrombolysis. 2017 Jan;43(1):132-138. doi: 10.1007/s11239-016-1416-6.
Shapiro AD, Nakar C, Parker JM, Albert GR, Moran JE, Thibaudeau K, Thukral N, Hardesty BM, Laurin P, Sandset PM. Plasminogen replacement therapy for the treatment of children and adults with congenital plasminogen deficiency. Blood. 2018 Mar 22;131(12):1301-1310. doi: 10.1182/blood-2017-09-806729. Epub 2018 Jan 10.
Shapiro AD, Menegatti M, Palla R, Boscarino M, Roberson C, Lanzi P, Bowen J, Nakar C, Janson IA, Peyvandi F. An international registry of patients with plasminogen deficiency (HISTORY). Haematologica. 2020 Mar;105(3):554-561. doi: 10.3324/haematol.2019.241158. Epub 2020 Jan 30.
Sang Y, Menegatti M, Brody JA, Wiggins KL, Cooley BC, Kapfer KN, Kangro K, de Laat B, Peyvandi F, Flick MJ, Smith NL, Shapiro AD, Wolberg AS. Plasminogen activation and plasmin activity are not necessary to prevent venous thrombosis/thromboembolism. Blood. 2025 Sep 11;146(11):1346-1358. doi: 10.1182/blood.2025028680.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
HISTORY
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.