Trial Review

Did you know?

The ANZCTR now automatically displays published trial results and simplifies the addition of trial documents such as unpublished protocols and statistical analysis plans.

These enhancements will offer a more comprehensive view of trials, regardless of whether their results are positive, negative, or inconclusive.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03797495




Registration number
NCT03797495
Ethics application status
Date submitted
28/12/2018
Date registered
9/01/2019
Date last updated
1/11/2024

Titles & IDs
Public title
Study of Individuals Affected With Hypoplasminogenemia
Scientific title
Hypoplasminogenemia: An International RetroSpecTive and PrOspective CohoRt StudY (HISTORY)
Secondary ID [1] 0 0
HISTORY
Universal Trial Number (UTN)
Trial acronym
HISTORY
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Plasminogen Deficiency 0 0
Condition category
Condition code

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Define the natural history of plasminogen deficiency
Timepoint [1] 0 0
2 years
Primary outcome [2] 0 0
Identify factors that contribute to or correlate with disease expression and severity
Timepoint [2] 0 0
5 years
Primary outcome [3] 0 0
Create a specimen biobank
Timepoint [3] 0 0
15 years

Eligibility
Key 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
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
18/12/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
19/04/2027
Actual
Sample size
Target
100
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute, The Royal Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Illinois
Country [2] 0 0
United States of America
State/province [2] 0 0
Indiana
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
United States of America
State/province [5] 0 0
Tennessee
Country [6] 0 0
United States of America
State/province [6] 0 0
Texas
Country [7] 0 0
United States of America
State/province [7] 0 0
Washington
Country [8] 0 0
Argentina
State/province [8] 0 0
Buenos Aires
Country [9] 0 0
Austria
State/province [9] 0 0
Innsbruck
Country [10] 0 0
Canada
State/province [10] 0 0
Quebec
Country [11] 0 0
Canada
State/province [11] 0 0
Québec
Country [12] 0 0
Canada
State/province [12] 0 0
Saskatoon
Country [13] 0 0
Greece
State/province [13] 0 0
Athens
Country [14] 0 0
Italy
State/province [14] 0 0
Milano
Country [15] 0 0
Italy
State/province [15] 0 0
Padua
Country [16] 0 0
Thailand
State/province [16] 0 0
Chiang Mai
Country [17] 0 0
Turkey
State/province [17] 0 0
Balçova
Country [18] 0 0
Turkey
State/province [18] 0 0
Istanbul
Country [19] 0 0
Turkey
State/province [19] 0 0
Van

Funding & Sponsors
Primary sponsor type
Other
Name
Indiana Hemophilia &Thrombosis Center, Inc.
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Fondazione Angelo Bianchi Bonomi
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief 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 markers to predict disease course, and inform improved therapeutic modalities
Trial website
https://clinicaltrials.gov/study/NCT03797495
Trial related presentations / 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.
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.
Public notes

Contacts
Principal investigator
Name 0 0
Amy D Shapiro, MD
Address 0 0
Indiana Hemophilia &Thrombosis Center, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Amy D Shapiro, MD
Address 0 0
Country 0 0
Phone 0 0
317-871-0000
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03797495