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Stem Cell Therapy

iPSC-CL for Congenital Heart Disease

Phase 1

Recruiting

Research Sponsored by HeartWorks, Inc.

Eligibility Criteria Checklist

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

Must have

Subject must be able to understand and provide informed consent

Univentricular congenital heart disease

Must not have

Any history of cancer

Obesity with BMI greater than 30

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial aims to test if lab-grown heart cells, made from stem cells, are safe and feasible to use to treat people with congenital heart disease. Participants must agree to testing and monitoring before and after product administration, and lifelong follow-up.

Who is the study for?

This trial is for adults aged 18-40 with single ventricle congenital heart disease, class IV heart failure, and an ejection fraction below 40%. They must have maximized all treatments, be ineligible or waiting for a heart transplant, possibly on mechanical support, and able to consent. Exclusions include substance abuse, active infections, uncontrolled depression or other conditions that could risk safety or compliance.

What is being tested?

The trial tests the safety of lab-grown heart cells (iPSC-CL) in those with congenital heart disease. Participants will receive these cells and undergo monitoring before and after treatment to assess safety and feasibility. The study includes lifelong follow-up comparing treated subjects against untreated ones from the same pool.

What are the potential side effects?

Potential side effects are not explicitly listed but may include risks associated with stem cell therapy such as immune reactions, infection risks due to immunosuppression if used, potential for abnormal tissue growth or arrhythmias given the cardiac nature of the cells.

Eligibility Criteria

Inclusion Criteria

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

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I understand the study and can give my consent.

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I have a heart condition where I was born with only one ventricle.

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I am on the US heart transplant list but may not receive a heart in time.

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I have been on the best standard treatment for my condition for at least 3 months.

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I am between 18 and 40 years old.

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I have severe heart failure with poor heart pumping function.

Exclusion Criteria

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

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I have had cancer in the past.

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My BMI is over 30, indicating obesity.

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I cannot have anesthesia due to health reasons.

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My heart pumps well but still fails.

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I am currently being treated for an infection.

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My kidney disease is at stage III or more advanced.

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I have had heart rhythm problems that needed treatment.

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I have a history of liver cirrhosis.

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My diabetes is not well-managed.

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I have a history of heart artery disease.

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My heart failure is caused by another health condition like amyloidosis.

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I cannot have someone with me 24/7 for the first 3 weeks after treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Feasibility

Short term safety

Secondary study objectives

Cardiac High Sensitivity Troponin T

Long term safety

NT-pro-BNP

+2 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: TreatedExperimental Treatment1 Intervention

Subjects in Treated arm will receive one dose of Investigational Product. Within this arm are three dose levels. Dose level selection will be determined by product availability subjects have available product and when they can be treated. Dose levels will escalate in order of treatment date.

Group II: ControlActive Control1 Intervention

Subjects who enroll but do not receive Investigational Product will be placed in the control arm.

0 / 18Eligibility criteria met