Actively Recruiting
Safety and Feasibility of Autologous Induced Pluripotent Stem Cells of Cardiac Lineage in Subjects With Congenital Heart Disease
Led by HeartWorks, Inc. · Updated on 2026-03-13
50
Participants Needed
1
Research Sites
74 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and feasibility of lab-grown heart cells derived from stem cells for people with congenital heart disease, specifically those with univentricular heart conditions and advanced heart failure. This Phase 1 study aims to answer whether these autologous induced pluripotent stem cells of cardiac lineage (iPSC-CL) can be safely delivered to humans and if the trial's conduct is practical. The study is sponsored by HeartWorks, Inc. and focuses on participants aged 18 to 40 years with severe heart failure and limited treatment options. Participants in the treated group will receive one dose of the investigational iPSC-CL product, with three escalating dose levels assigned based on product availability and treatment timing. Those who enroll but do not receive the product will serve as controls and receive no intervention. The investigational product is personalized, created from the participant's own cells, and the study includes a comparison between treated and untreated subjects to assess differences. During the study, participants will undergo testing and monitoring before and after receiving the product, including assessments of heart function and safety markers such as cardiac troponin levels and tumor markers. They must agree to lifelong follow-up to track long-term safety and outcomes. Primary outcomes include short-term safety over 3 months and feasibility over 12 months, with additional monitoring extending up to 15 years. Overall, participant involvement includes ongoing evaluations, laboratory tests, and adherence checks throughout the study period.
CONDITIONS
Brief Title
Autologous Induced Pluripotent Stem Cells of Cardiac Lineage for Congenital Heart Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to 40 years old
- Able to understand and provide informed consent
- Diagnosis of univentricular congenital heart disease
- End-stage systolic heart failure classified as NYHA Class IV with ejection fraction below 40%
- Prognosis of 1 to 1.5 years survival at time of skin biopsy
- Currently listed for heart transplant with long expected wait, denied transplant, or on/planning mechanical support as destination therapy
- Maximized all guideline-directed therapy for at least 3 months before enrollment
- Adequate social support to comply with study tests and long-term requirements
You will not qualify if you...
- No available autologous iPSC-CL product (treatment arm only)
- History of symptomatic cardiac arrhythmia requiring defibrillation or medication escalation
- Heart failure with preserved ejection fraction
- Heart failure caused by other conditions like amyloidosis or valvular disease
- QTc interval greater than 500 ms
- Stage III or higher chronic kidney disease
- History of liver cirrhosis or coronary artery disease
- Uncontrolled diabetes mellitus
- Any history of cancer
- Contraindications to amiodarone use up to 3 months (treatment arm only)
- Contraindications for cardiac monitor insertion or LifeVest defibrillator placement
- Positive tests for HIV, Hepatitis B, Hepatitis C, or Syphilis
- Obesity with BMI over 30
- Current alcohol or drug abuse preventing transplant eligibility
- Active infection needing ongoing treatment
- Contraindication to anesthesia
- Other medical problems that increase risk or interfere with study participation
- Inability or unwillingness to consent or follow protocol
- History of non-compliance
- Unable to have around-the-clock support for first 3 weeks after product administration
- Uncontrolled depression
- Denied heart transplant due to social factors
- Participation in other cardiac interventional trials
- Previous heart transplant
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
screening and enrollment visit
Duration - Single treatment event
Participants receive one dose of the investigational autologous induced pluripotent stem cells of cardiac lineage.
1 treatment visit (in-person)
Duration - Up to 15 years
Participants are monitored for safety and feasibility after treatment, including assessments up to 15 years for long-term safety and tumor marker levels.
Regular follow-up visits including assessments at 1 month, 3 months, 12 months, and annually thereafter
Trial Site Locations
Total: 1 location
1
Mayo Clinic
Rochester, Minnesota, United States, 55901
Actively Recruiting
Research Team
A
Adam Armstrong
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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