Study of Individuals Affected With Hypoplasminogenemia (HISTORY)
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| ClinicalTrials.gov Identifier: NCT03797495 |
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Recruitment Status :
Recruiting
First Posted : January 9, 2019
Last Update Posted : May 3, 2022
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| Condition or disease |
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| Plasminogen Deficiency |
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 Type : | Observational |
| Estimated Enrollment : | 100 participants |
| Observational Model: | Cohort |
| Time Perspective: | Other |
| Official Title: | Hypoplasminogenemia: An International RetroSpecTive and PrOspective CohoRt StudY (HISTORY) |
| Actual Study Start Date : | December 18, 2018 |
| Estimated Primary Completion Date : | January 18, 2025 |
| Estimated Study Completion Date : | June 15, 2025 |
- Define the natural history of plasminogen deficiency [ Time Frame: 2 years ]
- 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
- Identify factors that contribute to or correlate with disease expression and severity [ Time Frame: 5 years ]
- 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
- Create a specimen biobank [ Time Frame: 15 years ]Bank plasma, serum and DNA on consenting enrolled subjects
Biospecimen Retention: Samples With DNA
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Probability Sample |
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03797495
| Contact: Amy D Shapiro, MD | 317-871-0000 | ashapiro@ihtc.org | |
| Contact: Charles Nakar, MD | 317-871-0000 | cnakar@ihtc.org |
| United States, Indiana | |
| Indiana Hemophila @Thrombosis Center | Recruiting |
| Indianapolis, Indiana, United States, 46260 | |
| Contact: Neelam Thukral, CCRC 317-871-0000 ext 373 nthukral@ihtc.org | |
| Contact: Charles Nakar, MD 317-871-0000 cnakar@ihtc.org | |
| Principal Investigator: Amy D Shapiro, MD | |
| Sub-Investigator: Charles Nakar, MD | |
| United States, Pennsylvania | |
| Hemophilia Center of Western Pennsylvania | Recruiting |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| Contact: Frederico Xavier, MD 412-209-7411 fxavier@hmc.psu.edu | |
| Contact: Debbie Vehec 412-209-7564 dvehec@vitalant.org | |
| United States, Tennessee | |
| Vanderbilt Children's Hematology-Oncology | Recruiting |
| Nashville, Tennessee, United States, 37232 | |
| Contact: Delia Darst 615-343-7190 delia.h.darst@vumc.org | |
| Contact: Heather McDaniel, MD, MSCI 615 936 1762 heather.mcdaniel@vumc.org | |
| Principal Investigator: Heather McDaniel, MD, MSCI | |
| United States, Texas | |
| Cook Children's Medical Center | Recruiting |
| Fort Worth, Texas, United States, 76104 | |
| Contact: Heather Urbanek 682-885-1244 Heather.Urbanek@cookchildrens.org | |
| Contact: Marcela Torres, MD 682 885 4007 marcela.torres@cookchildrens.org | |
| Principal Investigator: Marcela Torres, MD | |
| The University of Texas Health Science Center at Houston | Recruiting |
| Houston, Texas, United States, 77030 | |
| Contact: Katherine Addy, RN, BSN, MPH 713-500-8352 Katherine.E.Addy@uth.tmc.edu | |
| Contact: Nidra Rodriguez, MD 713 500 8360 Nidra.I.Rodriguez@uth.tmc.edu | |
| Principal Investigator: Nidra Rodriguez, MD | |
| Argentina | |
| Hospital Britanico Buenos Aires | Recruiting |
| Buenos Aires, Argentina, C1280 | |
| Contact: Jhon A Avila, MD 549 114 187 9723 jaavilar@gmail.com | |
| Contact: Jose M Ceresetto, MD 541 143 041 081 jceresetto@intramed.net | |
| Principal Investigator: Jose M Ceresetto, MD | |
| Sub-Investigator: Jhon A Avila, MD | |
| Italy | |
| Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, | Recruiting |
| Milano, Italy, 20122 | |
| Contact: Marzia Menegatti, PhD +39 - 02/50320727 marzia.menegatti@guest.unimi.it | |
| Principal Investigator: Flora Peyvandi, MD,PhD | |
| Thailand | |
| Faculty of Medicine, Chiang Mai University | Recruiting |
| Chiang Mai, Thailand, 50200 | |
| Contact: Rungrote Natesirinilkul, MD 66 2 201 1749 rungrote.n@cmu.ac.th | |
| Principal Investigator: Rungrote Natesirinilkul, MD | |
| Turkey | |
| Istanbul University Cerrahpsasa, Cerrahpsasa Medical Faculty Pediatric Hematology and Oncology Department | Recruiting |
| Istanbul, Turkey, 34098 | |
| Contact: Ayse Gonca Kacar, MD 505 259 1229 goncakacar@gmail.com | |
| Contact: Tulin Celkan, MD 532 57 60723 tirajecelkan@yahoo.com | |
| Principal Investigator: Tulin Celkan, MD | |
| Yuzuncu Yil University Faculty of Medicine Department of Ophthalmology | Recruiting |
| Van, Turkey, 65040 | |
| Contact: Muhammed Batur, MD 904 322 150 473 ext 6030 muhammedbatur@gmail.com | |
| Principal Investigator: Muhammed Batur, MD | |
| Principal Investigator: | Amy D Shapiro, MD | Indiana Hemophilia &Thrombosis Center, Inc. | |
| Principal Investigator: | Flora Peyvandi, MD, PhD | Univeristy of Milan |
Other Publications:
| Responsible Party: | Amy D Shapiro, MD, Medical Director, Indiana Hemophilia &Thrombosis Center, Inc. |
| ClinicalTrials.gov Identifier: | NCT03797495 |
| Other Study ID Numbers: |
HISTORY |
| First Posted: | January 9, 2019 Key Record Dates |
| Last Update Posted: | May 3, 2022 |
| Last Verified: | April 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Undecided |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |