Metformin for Cardiac Fibrosis

Recruiting in Palo Alto (17 mi)

Overseen by Sweta Gupta, MD, MS, MBBS

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Waitlist Available

Sponsor: Indiana Hemophilia &Thrombosis Center, Inc.

No Placebo Group

Prior Safety Data

Approved in 6 Jurisdictions

Trial Summary

What is the purpose of this trial?

This study will evaluate the efficacy and safety of metformin, in patients 18-65 years of age with homozygous plasminogen activator inhibitor-1 (PAI-1) deficiency, with or without cardiac fibrosis, for a period of 60 months. The starting dose of metformin will be 500 mg up to a maximum dose of 2000 mg for a period of 5 years with the aim to assess the safety and efficacy of metformin on prevention/stabilization or regression of cardiac fibrosis in a Treated population vs. a Comparison population.

Research Team

Magdalena Lewandowska, MD

Principal Investigator

Indiana Hemophilia and Thrombosis Center, Inc

Sweta Gupta, MD, MS, MBBS

Principal Investigator

Indiana Hemophilia and Thrombosis Center, Inc

Amy D Shapiro, MD

Principal Investigator

Indiana Hemophilia and Thrombosis Center, Inc

Eligibility Criteria

Adults aged 18-65 with a confirmed genetic mutation for PAI-1 deficiency can join this trial. They must be willing to take metformin daily or just be observed without the drug, and able to follow study rules. People with kidney issues, allergies to metformin, alcohol use problems, or taking certain drugs can't participate.

Inclusion Criteria

Have read, understood and be able to provide written informed consent

I have a confirmed genetic mutation related to PAI-1 deficiency.

I am between 18 and 65 years old.

Exclusion Criteria

Inability to understand and provide written informed consent

Unwillingness to avoid alcohol

I am taking cimetidine, dolutegravir, patiromer, ranolazine, or tafenoquine and cannot switch medications.

Treatment Details

Interventions

• Metformin (Biguanide)

Trial Overview The trial is testing if extended-release metformin tablets (500 mg up to 2000 mg) over five years can prevent or reduce heart scarring in patients with PAI-1 deficiency. Participants will either receive metformin treatment or no drug for observation.

Participant Groups

Group I: Metformin Treatment Group Experimental Treatment 1 Intervention

Subjects with PAI-1 deficiency with or without cardiac fibrosis, receiving daily treatment with metformin for a daily range of 500-2000mg

Group II: Observation Group Active Control 1 Intervention

Subjects with PAI-1 deficiency with or without cardiac fibrosis, not receiving treatment with metformin Subjects are allowed to switch between the two groups

Metformin is already approved in Canada, Japan, China, Switzerland for the following indications:

Approved in Canada as Glucophage for:

• Type 2 diabetes

Approved in Japan as Glucophage for:

• Type 2 diabetes

Approved in China as Glucophage for:

• Type 2 diabetes

Approved in Switzerland as Glucophage for:

• Type 2 diabetes

Who Is Running the Clinical Trial?

Indiana Hemophilia &Thrombosis Center, Inc.

Lead Sponsor

Trials

6

Recruited

690+