Plasminogen DeficiencyHISTORYNCT03797495
Study of Individuals Affected With Hypoplasminogenemia
An observational study of Plasminogen Deficiency, run by Indiana Hemophilia &Thrombosis Center, Inc. at 35 sites in 11 countries.
Trial summary
This is an Investigator initiated retrospective and prospective single cohort study. The study will utilize an international registry and develop a specimen biobank to provide an improved understanding of the natural history of hyposplasminogenemia, to elucidate the heterogeneity of phenotypic expression, identify… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Plasminogen Deficiency
- How many people
- 100 participants
- Where
- 35 sitesin 11 countries
- Study dates
- 18 Dec 2018to 26 Nov 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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: Study population will include males and females affected with hyposplasminogenemia of any age and their first degree family members (siblings and parents).
Who may be able to join
- Signed informed consent and assent as applicable (Appendix 1)
- 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)
- All ages included
- Available clinical history and treatment for at least 1 year prior to entry except for infants < 1 year of age
- Willingness to provide samples for analysis including DNA, plasma etc.
- Willingness to participate in prospective follow-up for up to 3 years
Who cannot join
- Previous organ transplant recipient
- 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
- Refuses to provide informed consent
- Special patient populations, including prisoners or, are deemed medically or cognitively unsuitable for research by their treating physician
- Inability to obtain a blood sample due to poor or limited venous access
Trial locations and contacts
35 sites in 11 countries. Contact the study team or a site near you to ask about taking part.
35 sites in 11 countries
United States13 sites, 11 recruiting
- Recruiting
The University of Alabama (UAB)
Birmingham, Alabama, 35233
Lisa Beatty, RN, 205-638-2394, lbeatty@uabmc.edu
Sharon Davis, sharonddavis@uabmc.edu
- Recruiting
Rush University Medical Center
Chicago, Illinois, 60612
Mindy Simpson, MD, 312-942-3034, Mindy_L_Simpson@rush.edu
Brian Jenkins, 312-563-1883, Brian_H_Jenkins@rush.edu
- Recruiting
Stony Brook University | Stony Brook Medicine
East Setauket, New York, 11733
Nicole Muhlbauer, MD, (631) 444-9016, Nicole.Muhlbauer@stonybrookmedicine.edu
Jessica Mannetta, 516-329-7843, Jessica.Mannetta@stonybrookmedicine.edu
- Recruiting
Cook Children's Medical Center
Forth Worth, Texas, 76104
Heather Urbanek, 682-885-1244, Heather.Urbanek@cookchildrens.org
Marcela Torres, MD, 682 885 4007, marcela.torres@cookchildrens.org
- Completed
The University of Texas Health Science Center at Houston
Houston, Texas, 77030
- Recruiting
Indiana Hemophila @Thrombosis Center
Indianapolis, Indiana, 46260
Neelam Thukral, CCRC, 317-871-0000 ext. 373, nthukral@ihtc.org
Charles Nakar, MD, 317-871-0000, cnakar@ihtc.org
- Recruiting
University of Minnesota, Pediatric Hem/Onc & Cancer Survivorship Program
Minneapolis, Minnesota, 55455
Sharon Hwee, 612.626.3915 ext. 612.626.3915, hweex001@umn.edu
Allison Fullenkamp, 612-625-6125, fulle631@umn.edu
- Completed
Vanderbilt Children's Hematology-Oncology
Nashville, Tennessee, 37232
- Recruiting
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213
Frederico Xavier, MD, 412-209-7411, fxavier@hmc.psu.edu
Debbie Vehec, 412-209-7564, dvehec@vitalant.org
- Recruiting
University of North Carolina at Chapel Hill
Raleigh, North Carolina, 27599
Soham Nanavati, BSPH, 919-843-1822, Soham_Nanavati@med.unc.edu
Brett Phillips, MA, Brett_phillips@med.unc.edu
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105-3901
Gavin Roach, MD, 206-884-1018, Gavin.Roach@seattlechildrens.org
Shana Tang, 206-884-1268, shana.tang@seattlechildrens.org
- Recruiting
SUNY Upstate Medical University, Pediatric Hematology/Oncology
Syracuse, New York, 13210
Karen Bilynsky, RN, 315-464-7601, bilynskk@upstate.edu
Andrea Dvorak, MD, 315 464 5294, DvorakA@upstate.edu
- Recruiting
Wake Forest University
Winston-Salem, North Carolina, 27157
Madeline G Hicks, 336-713-3553, mghicks@wakehealth.edu
Joia Johnson, 336-713-3553, joijohns@wakehealth.edu
Turkey (Türkiye)6 sites, 4 recruiting
- Recruiting
Uludag University, Medical Faculty Sebahattin Gazioğlu, Pediatric Hematology and Oncology Hospital
Bursa, Bursa, 16059
Adalet Meral Güneş, MD, 90 555 316 26 50, adaletm@uludag.edu.tr
Melike Sezgin Evim, MD, 90 555 316 26 50, cmmt63@gmail.com
- Recruiting
Istanbul Üniversitesi Onkoloji Enstitüsü
Istanbul, 34093
Basak Koc, MD, 90 5326003027, s_basakkoc@hotmail.com
Basak Koc, MD
- Active, not recruiting
Istanbul University Cerrahpsasa, Cerrahpsasa Medical Faculty Pediatric Hematology and Oncology Department
Istanbul, 34098
- Active, not recruiting
Dokuz Eylul University pediatric Pulmonology, Allergy and Clinical Immunology
Izmir, Balçova, 35330
- Recruiting
Ondokuz Mayıs Üniversitesi Tıp Fakültesi Pediatrik Hematoloji Bölümü
Samsun, Samsun, 55200
Canan Albayrak, MD, 00905362192407, calbayrak@omu.edu.tr
Dilek Zorlu Kaya, MSc, 90 539 897 31 45, dilek.zorlukaya@medex-smo.com
- Recruiting
Yuzuncu Yil University Faculty of Medicine Department of Ophthalmology
Van, 65040
Muhammed Batur, MD, 904 322 150 473 ext. 6030, muhammedbatur@gmail.com
Muhammed Batur, MD
Canada4 sites, 4 recruiting
- Recruiting
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5
Valerie Arsenault, MD, (514) 345-4931 ext. 3757, valerie.arsenault.med@ssss.gouv.qc.ca
Bianka Courcelle, 514-345-4931 ext. 3757, bianka.courcelle.hsj@ssss.gouv.qc.ca
- Recruiting
CHU de Québec Université Laval
Québec, Quebec, A0121
Marie-Claude Pelland-Marcotte, MD, 14185254444 ext. 79029, marie-claude.pelland-marcotte.1@ulaval.ca
Stéphanie Cyr, 418 525-4444 ext. 40121, stephanie.cyr@crchudequebec.ulaval.ca
- Recruiting
University of Saskatchewan
Saskatoon, SK S7N 0W8
Sarah Tehseen, MBBS, MSc. FRCPC, 639 998 3972, Sarah.Tehseen@saskhealthauthority.ca
Rashmi Nagaraj, 306-978-8306, rashmi.nagaraj@usask.ca
- Recruiting
Windsor Regional Hospital
Windsor, Ontario, N8W 1L9
Erica Figgins, MSc, 519-254-5577 ext. 52300, Erica.Figgins@wrh.on.ca
Batul Khumushi, CCRP, 519 254 5577 ext. 52899, Batul.Khumushi@wrh.on.ca
United Kingdom3 sites, 3 recruiting
- Recruiting
Cardiff and Vale UHB
Cardiff, Wales, CF14 4XW
Deepan Gosrani, MD, 02921 846085, Deepan.Gosrani@wales.nhs.uk
Dawid Grzech, 02921 846085 ext. 43536, Dawid.Grzech@wales.nhs.uk
- Recruiting
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3HR
Mary Mathias, MD, 2078298846, Mary.Mathias@gosh.nhs.uk
Kerry Gilroy, 0207 405 9200 ext. 38504, Kerry.gilroy@gosh.nhs.uk
- Recruiting
Royal Free Hospital, Royal Free London NHS Foundation Trust
London
Amy O'Reilly, 0207 794 0500, amy.oreilly6@nhs.net
Joanna Calvert, 020 3758 2000, joanna.calvert@nhs.net
Australia2 sites, 2 recruiting
- Recruiting
Murdoch Children's Research Institute, The Royal Children's Hospital
Melbourne, Victoria, 3052
Paul Monagle, MD, 61 3 9936-6330, paul.monagle@rch.org.au
Jodi Hislop, RN, 61 3 9936-6330, jodi.hislop@rch.org.au
- Recruiting
Children's Health Queensland Hospital and Health Service
South Brisbane, QLD 4101
Ruby Gander, 61 7 3068 4142, ruby.gander@health.qld.gov.au
Trang Le, 61 7 3069 7206, trang.le2@health.qld.gov.au
Italy2 sites, 2 recruiting
- Recruiting
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center,
Milan, 20122
Marzia Menegatti, PhD, +39 - 02/50320727, marzia.menegatti@guest.unimi.it
Flora Peyvandi, MD,PhD
- Recruiting
University Hospital of Padova
Padua, 35100
Maria Teresa Sartori, MD, 3334530450, mtsart@unipd.it
C. Dalla Porta, MD, 39 049 821 1111, cesare.dallaporta@gmail.com
Argentina1 site, 1 recruiting
- Recruiting
Hospital Britanico Buenos Aires
Buenos Aires, C1280
Jhon A Avila, MD, 549 114 187 9723, jaavilar@gmail.com
Jose M Ceresetto, MD, 541 143 041 081, jceresetto@intramed.net
Austria1 site, 1 recruiting
- Recruiting
Medical University of Innsbruck, University Clinic for Pediatrics and Adolescent Medicine
Innsbruck, A-6020
Lukas Hackl, MD, +43-512-504-83144, lukas-hackl@tirol-kliniken.at
Lukas Hackl, MD
Greece1 site, 1 recruiting
- Recruiting
Alexandra Hospital, Athens, Hematology Department
Athens, 11528
Vasiliki Gkalea, MD, 0030 (690) 682-2189, vasiliki.gkalea@gmail.com
Charis Matsouka, 302132162165, charis.matsouka@gmail.com
Israel1 site, 1 recruiting
- Recruiting
Safra's Children Hospital, Sheba Medical Center
Tel Aviv
Tami Barazani-Brutman, MD, 972 522427949, Tami.BarazaniBrutman@sheba.health.gov.il
Sarina Levy, MD, 972 547299251, Sarina.Levy@sheba.health.gov.il
Thailand1 site, 1 recruiting
- Recruiting
Faculty of Medicine, Chiang Mai University
Chiang Mai, 50200
Rungrote Natesirinilkul, MD, 66 2 201 1749, rungrote.n@cmu.ac.th
Rungrote Natesirinilkul, MD
About this study
Official title: Hypoplasminogenemia: An International RetroSpecTive and PrOspective CohoRt StudY (HISTORY)
This is an Investigator initiated retrospective and prospective single cohort study. The study will utilize an international registry and develop a specimen biobank to provide an improved understanding of the natural history of hyposplasminogenemia, to elucidate the heterogeneity of phenotypic expression, identify markers to predict disease course, and inform improved therapeutic modalities
Read the detailed description
The aims of this study are to:
- Define PLGD natural history in a large cohort of individuals with hypoplasminogenemia and their first-degree family members.
- Identify factors that correlate with disease expression and severity.
- 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
- Plasminogen Deficiency
- Medical subject headings
- Congenital Plasminogen Deficiency
What is being measured
The primary outcome is the main question the study is designed to answer.
Define the natural history of plasminogen deficiency
- Recruit 100 subjects with hypoplasminogenemia and their first-degree family members
- Collect up to 1 year retrospective and 3 year prospective data on symptoms, treatment and interventions
Measured: 2 years
Identify factors that contribute to or correlate with disease expression and severity
- Perform centralized plasminogen activity and antigen analyses
- Perform centralized genetic analysis to identify changes in the plasminogen gene
- Perform centralized analysis of polymorphisms that affect plasminogen activity levels and impact fibrinolysis
- Perform local urine analysis
- Collect samples to explore the interaction of altered plasminogen proteins with bacterial strains
Measured: 5 years
Create a specimen biobank
Bank plasma, serum and DNA on consenting enrolled subjects
Measured: 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2019
- Study start18 Dec 2018
- Primary completion29 Jun 2029 (estimated)
- Study completion26 Nov 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Plasminogen DeficiencyStudies of the same condition, open and under way.
- 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
- Cohort
- Time perspective
- Other
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Indiana Hemophilia &Thrombosis Center, Inc.
- Sponsor type
- University, hospital or other
- Collaborators
- Fondazione Angelo Bianchi Bonomi
- Responsible party
- Principal investigator, Amy D Shapiro, MD, Indiana Hemophilia &Thrombosis Center, Inc.
- Investigators
- Amy D Shapiro, MD, Principal investigator, Indiana Hemophilia &Thrombosis Center, Inc.Flora Peyvandi, MD, PhD, Principal investigator, Univeristy of Milan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03797495
- Sponsor's protocol ID
- HISTORY
- Acronym
- HISTORY
- Status last verified
- Mar 2026
- First submitted
- 28 Dec 2018
- Last updated
- 4 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 32001536Tait 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.
PubMed 1455395Schuster 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.
PubMed 17900274Ma 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.
PubMed 25208887Celkan T. Plasminogen deficiency. J Thromb Thrombolysis. 2017 Jan;43(1):132-138. doi: 10.1007/s11239-016-1416-6.
PubMed 27629020Shapiro 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.
PubMed 29321155Sang 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.
PubMed 40403316
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hypoplasminogenemia: An International RetroSpecTive and PrOspective CohoRt StudY (HISTORY)
- ClinicalTrials.gov ID
- NCT03797495
- Acronym / sponsor ID
- HISTORY / HISTORY
- Conditions
- Plasminogen Deficiency
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Indiana Hemophilia &Thrombosis Center, Inc.University, hospital or other
- Collaborators
- Fondazione Angelo Bianchi Bonomi
- Study start
- 18 Dec 2018 (actual)
- Primary completion
- 29 Jun 2029 (estimated)
- Study completion
- 26 Nov 2029 (estimated)
- First posted
- 9 Jan 2019 (actual)
- 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.
This is an Investigator initiated retrospective and prospective single cohort study. The study will utilize an international registry and develop a specimen biobank to provide an improved understanding of the natural history of hyposplasminogenemia, to elucidate the heterogeneity of phenotypic expression, identify markers to predict disease course, and inform improved therapeutic modalities
Detailed description
The aims of this study are to:
- Define PLGD natural history in a large cohort of individuals with hypoplasminogenemia and their first-degree family members.
- Identify factors that correlate with disease expression and severity.
- 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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Study population will include males and females affected with hyposplasminogenemia of any age and their first degree family members (siblings and parents).
Key inclusion criteria (6 listed)
- Signed informed consent and assent as applicable (Appendix 1)
- 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)
- All ages included
- Available clinical history and treatment for at least 1 year prior to entry except for infants < 1 year of age
- Willingness to provide samples for analysis including DNA, plasma etc.
Key exclusion criteria (5 listed)
- Previous organ transplant recipient
- 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
- Refuses to provide informed consent
- Special patient populations, including prisoners or, are deemed medically or cognitively unsuitable for research by their treating physician
- Inability to obtain a blood sample due to poor or limited venous access
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Define the natural history of plasminogen deficiency
Time frame: 2 years
1. Recruit 100 subjects with hypoplasminogenemia and their first-degree family members 2. Collect up to 1 year retrospective and 3 year prospective data on symptoms, treatment and interventions
Identify factors that contribute to or correlate with disease expression and severity
Time frame: 5 years
1. Perform centralized plasminogen activity and antigen analyses 2. Perform centralized genetic analysis to identify changes in the plasminogen gene 3. Perform centralized analysis of polymorphisms that affect plasminogen activity levels and impact fibrinolysis 4. Perform local urine analysis 5. Collect samples to explore the interaction of altered plasminogen proteins with bacterial strains
Create a specimen biobank
Time frame: 15 years
Bank plasma, serum and DNA on consenting enrolled subjects
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2026.
- Background: PubMed 32001536Shapiro 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.
- Results publication: PubMed 1455395Tait 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.
- Results publication: PubMed 17900274Schuster 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.
- Results publication: PubMed 25208887Ma 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.
- Results publication: PubMed 27629020Celkan T. Plasminogen deficiency. J Thromb Thrombolysis. 2017 Jan;43(1):132-138. doi: 10.1007/s11239-016-1416-6.
- Results publication: PubMed 29321155Shapiro 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.
- Derived from this study: PubMed 40403316Sang 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.
- Individual participant dataSharing planned: Undecided
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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.
Amy D Shapiro, MD principal investigator
Indiana Hemophilia &Thrombosis Center, Inc.
Flora Peyvandi, MD, PhD principal investigator
Univeristy of Milan
35 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Plasminogen Deficiency, run by Indiana Hemophilia &Thrombosis Center, Inc. at 35 sites in 11 countries.
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 6 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 35 sites in United States, Turkey (Türkiye), Canada, United Kingdom, Australia and 6 more countries. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Amy D Shapiro, MD (317-871-0000, ashapiro@ihtc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Define the natural history of plasminogen deficiency (time frame: 2 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03797495, which also lists 7 linked publications.
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 4 Jun 2026.