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Autologous iPSC-CL for Congenital Heart Disease

This study is testing a new treatment called autologous iPSC-CL (lab-grown heart cells made from your own stem cells) for people with congenital heart disease (heart problems present at birth) and severe heart failure (NYHA Class III or IV). The main goals are to see if this treatment is safe and if the study itself can be successfully carried out. You might be eligible if you are between 18 and 40 years old. Researchers plan to enroll 50 participants. The study will look at safety after 3 months and feasibility after 12 months. Participants will receive the investigational product and agree to lifelong follow-up.

Study design
This is an interventional study with a planned enrollment of 50 participants. It aims to compare treated and untreated subjects from the same group.
What's involved
You will agree to testing and monitoring before and after receiving the investigational product. You will also agree to lifelong follow-up.
Compensation
Not stated in the trial record.
Follow-up
Participants will agree to lifelong follow-up after receiving the treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05647213

Autologous Induced Pluripotent Stem Cells of Cardiac Lineage for Congenital Heart Disease

Recruiting
PHASE1Ages 18–40InterventionalTreatment
HeartWorks, Inc.
~50 participants
Updated 2026-03-13 on ClinicalTrials.gov
What's tested:iPSC-CL

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Short term safety
Measured over 3 months
+1 more outcome measured

Conditions

Univentricular Heart
Congenital Heart Disease
Heart Failure NYHA Class III
Heart Failure NYHA Class IV

Explore related trials

Common Ventricle DisorderCongenital Heart DiseaseHeart Failure

Where it's being run

1 sites across 1 states
Minnesota1

Study leadership

  • Timothy J Nelson, M.D., Ph.D. · STUDY_DIRECTOR · HeartWorks, Inc.

Who to contact

Adam Armstrong
[email protected]
1-(507) 577-1764
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Age 18 to 40 years old
Subject must be able to understand and provide informed consent.
Univentricular congenital heart disease.
End-stage systolic heart failure, defined as Class IV according to New York Heart Association (NYHA) with abnormal visually estimated ejection fraction below 40%.
Prognosis of 1 to 1.5 years survival at time of skin biopsy.
The patient falls into one of the following categories:
Currently listed for heart transplantation at an accredited program in the US but has an expected waiting time for a suitable organ that is likely longer than anticipated life-expectancy.
Has been denied access to a heart transplantation at an accredited US institution.
Is currently on or planning to be on mechanical support as destination therapy.
All guideline directed therapy available to the subject has been maximized, for a minimum of 3 months prior to enrollment.
Adequate social support system that facilitates subject participation in all study required tests and procedures and supports the subject's ability to comply with long-term study requirements.

Exclusion

No available autologous iPSC-CL as defined by the manufacturer's release criteria. (This applies to Part II of the study and applies to the treatment arm only.)
History of symptomatic episodes of cardiac arrythmia requiring cardiac defibrillation or escalation of medications.
Heart failure with preserved ejection fraction.
Heart failure due to co-morbid conditions (e.g., amyloidosis, valvular heart disease, refractory anemia).
QTc greater than 500 ms.
Stage III or higher chronic kidney disease.
History of liver cirrhosis.
History of coronary artery disease.
Uncontrolled diabetes mellitus.
Any history of cancer.
Contraindication for use of amiodarone for up to 3 months (treatment arm only).
Contraindication for insertion of Insertable Cardiac Monitor.
Contraindication for placement of LifeVest cardioverter defibrillator.
Positive serology testing for HIV, Hepatitis B, Hepatitis C or Syphilis.
Obesity with BMI greater than 30.
Current alcohol or drug abuse precluding heart transplantation.
Active infection requiring ongoing treatment.
Contraindication to anesthesia.
Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality of the data obtained from the study.
Inability or unwillingness of a participant to give written informed consent or comply with study protocol.
History of non-compliance.
Inability to be accompanied around the clock for any part of the first 3 weeks post product administration.
Uncontrolled depression.
Denied heart transplant due to social determinants.
Current participation in another cardiac interventional clinical trial that could confound the results of this study.
Previous heart transplant.

What this trial measures

  • Short term safety3 months

    The primary safety endpoint is short term safety defined as the rate of new or worsening serious adverse events (SAE) from any System Organ Class (SOC) within 3 months of the iPSC-CL delivery as compared to the control arm.

  • Feasibility12 months

    The primary feasibility endpoint is the percentage of individuals with collected skin cells that meet all iPSC-CL release criteria and the percentage of individuals that have cells delivered.