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Biguanide

Metformin for Cardiac Fibrosis

Phase 4

Waitlist Available

Led By Sweta Gupta, MD

Research Sponsored by Indiana Hemophilia &Thrombosis Center, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed homozygosity for mutation in SERPINE-1 for PAI-1 deficiency

Male or female aged 18-65 years

Must not have

Currently prescribed cimetidine, dolutegravir, patiromer, ranolazine, or tafenoquine and no alternate therapy is possible

Ages <18 or >65 years

Timeline

Screening 3 weeks

Treatment Varies

Follow Up approximately monthly (±4 weeks) until maximum tolerated dose for metformin is achieved until 6 months (±4 weeks) and then every 3 months (±4 weeks) for the entire study period for the metformin group

Awards & highlights

Drug Has Already Been Approved

Pivotal Trial

All Individual Drugs Already Approved

No Placebo-Only Group

Approved for 20 Other Conditions

Summary

This trialwill study the effects of metformin on people aged 18-65 with a certain genetic deficiency and/or heart fibrosis over 5 years. Doses of 500-2000 mg/day will be used to assess safety and efficacy.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Metformin may cause stomach upset, diarrhea, nausea and vomiting; it might also affect how your body handles sugar levels leading to low blood sugar events.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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I am between 18 and 65 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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I am younger than 18 or older than 65.

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My kidney function is significantly reduced.

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I do not have two copies of the SERPINE-1 mutation.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ approximately monthly (±4 weeks) until maximum tolerated dose for metformin is achieved until 6 months (±4 weeks) and then every 3 months (±4 weeks) for the entire study period for the metformin group

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~approximately monthly (±4 weeks) until maximum tolerated dose for metformin is achieved until 6 months (±4 weeks) and then every 3 months (±4 weeks) for the entire study period for the metformin group

This trial's timeline: 3 weeks for screening, Varies for treatment, and approximately monthly (±4 weeks) until maximum tolerated dose for metformin is achieved until 6 months (±4 weeks) and then every 3 months (±4 weeks) for the entire study period for the metformin group for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of individuals homozygous for PAI-1 deficiency with stable or improved Transforming growth factor (TGF-β1)

Number of individuals homozygous for PAI-1 deficiency with stable or improved cardiac fibrosis

Secondary study objectives

Number of individuals homozygous for PAI-1 deficiency with additional signs of heart failure assessed by measuring N- terminal prohormone beta natriuretic peptide (NT-pro BNP)

Number of individuals homozygous for PAI-1 deficiency with clinical symptoms of heart failure as measured by the New York Heart Association (NYHA) scale and as needed, the Kansas City Cardiomyopathy Questionnaire (KCCQ-12)

Number of individuals homozygous for PAI-1 deficiency with clinical symptoms of heart failure impacting their health as measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ-12)

+2 more

Awards & Highlights

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Approved for 20 Other Conditions

This treatment demonstrated efficacy for 20 other conditions.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Metformin Treatment GroupExperimental Treatment1 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 GroupActive Control1 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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Metformin

FDA approved

0 / 6Eligibility criteria met