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Biguanide

Metformin for Cardiac Fibrosis

Phase 4

Waitlist Available

Led By Magdalena Lewandowska, 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

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

Study 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.Check my eligibility

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.See study design

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.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

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

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 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Indiana Hemophilia &Thrombosis Center, Inc.Lead Sponsor

5 Previous Clinical Trials

671 Total Patients Enrolled

Magdalena Lewandowska, MDPrincipal InvestigatorIndiana Hemophilia and Thrombosis Center, Inc

Sweta Gupta, MDPrincipal InvestigatorIndiana Hemophilia and Thrombosis Center, Inc

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has the Metformin Treatment Group been given regulatory clearance by the FDA?

"Metformin Treatment Group earned a score of 3, as this phase 4 trial has been approved by regulatory authorities and proven to be safe."

Answered by AI

Is recruitment still open for participation in this experiment?

"Correct. According to data hosted on clinicaltrials.gov, this medical trail is presently recruiting participants after being initially posted in October of 2022 and subsequently revised in February of 2023."

Answered by AI

What goals is this medical experiment attempting to achieve?

"The primary metric for assessing the efficacy of this clinical trial, measured annually over a 5-year span, is the number of individuals homozygous for PAI-1 deficiency that show either stable or improved transforming growth factor (TGF-β1) readings. Secondary outcomes include changes in ejection fraction as evaluated by transthoracic echocardiogram scans, any shifts in cardiac symptoms using both New york Heart Association and Kansas City Cardiomyopathy Questionnaire scales, and fluctuations of N-terminal prohormone beta natriuretic peptide levels."

Answered by AI

Is participation in this study limited to those of a certain age?

"Qualified candidates for this study must fall between ages 18 and 65. For minors, there are 71 clinical trials available while those over age 65 have access to 360 different medical studies."

Answered by AI

Who is eligible to take part in this clinical trial?

"To be accepted into this study, applicants must have a deficiency in plasminogen activator inhibitor-1 and fall between the ages of 18 to 65. At present, there is space for around 15 candidates."

Answered by AI

What is the aggregate size of the sample population engaged in this trial?

"Affirmative. According to records on clinicaltrials.gov, this trial is currently accepting applications and began recruiting participants since October 10th 2022. The last update was February 6th 2023 with the aim of collecting 15 patients at one medical centre."

Answered by AI

Recent research and studies

Journal of the American College of CardiologyJournal

Development and Evaluation of the Kansas City Cardiomyopathy Questionnaire: A New Health Status Measure for Heart Failure

Green, C.Patrick, Charles B. Porter, Dennis R. Bresnahan, and John A. Spertus. 2000. “Development and Evaluation of the Kansas City Cardiomyopathy Questionnaire: A New Health Status Measure for Heart Failure”. Journal of the American College of Cardiology. Elsevier BV. doi:10.1016/s0735-1097(00)00531-3.

Diabetes CareJournal

Use of Metformin in the Setting of Mild-to-moderate Renal Insufficiency

Lipska, Kasia J., Clifford J. Bailey, and Silvio E. Inzucchi. 2011. “Use of Metformin in the Setting of Mild-to-moderate Renal Insufficiency”. Diabetes Care. American Diabetes Association. doi:10.2337/dc10-2361.

JamaJournal

Metformin in Patients with Type 2 Diabetes and Kidney Disease

Inzucchi, Silvio E., Kasia J. Lipska, Helen Mayo, Clifford J. Bailey, and Darren K. McGuire. 2014. “Metformin in Patients with Type 2 Diabetes and Kidney Disease”. Jama. American Medical Association (AMA). doi:10.1001/jama.2014.15298.

clinicaltrials.govStudy