Study for Plasminogen Deficiency

(HISTORY Trial)

Recruiting at 34 trial locations

Overseen By Charles Nakar, MD

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Indiana Hemophilia &Thrombosis Center, Inc.

Disqualifiers: Organ transplant, Psychiatric disorder, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 6

100 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand hypoplasminogenemia, a condition that affects how the body breaks down blood clots. Researchers seek to learn how the disease manifests in different individuals and find ways to predict its progression. While they will not test new treatments, they will collect information and samples, such as DNA and plasma, to aid future studies. Individuals with type 1 plasminogen deficiency (where plasminogen levels are less than half of normal) or close family members of someone with the condition are well-suited for this trial. As an unphased trial, this study offers participants the chance to contribute to foundational research that could lead to future breakthroughs.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Why are researchers excited about this trial?

Researchers are excited about this trial because it aims to deepen our understanding of hypoplasminogenemia, a rare condition with limited treatment options mostly involving plasminogen replacement therapy. Unlike current approaches that primarily focus on replacing the deficient protein, this trial could uncover new insights into the condition itself, potentially paving the way for innovative treatment strategies. By studying individuals affected by hypoplasminogenemia, scientists hope to identify novel targets for intervention and improve diagnosis and management of this challenging disorder.

Who Is on the Research Team?

Amy D Shapiro, MD

Principal Investigator

Indiana Hemophilia &Thrombosis Center, Inc.

Flora Peyvandi, MD, PhD

Principal Investigator

Univeristy of Milan

Are You a Good Fit for This Trial?

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Retrospective Data Collection

Collect up to 1 year retrospective data on symptoms, treatment, and interventions

1 year

Prospective Data Collection

Collect 3 years prospective data on symptoms, treatment, and interventions

3 years

In-person visits at baseline and end of study, interval follow-up every 6 months by telephone

Follow-up

Participants are monitored for safety and effectiveness after data collection

2 years

What Are the Treatments Tested in This Trial?

Interventions

• No Investigational New Treatment

Who Is Running the Clinical Trial?

Indiana Hemophilia &Thrombosis Center, Inc.

Lead Sponsor

Trials

6

Recruited

690+

Fondazione Angelo Bianchi Bonomi

Collaborator

Trials

3

Recruited

670+