Using lab-grown heart cells to treat congenital heart disease
Safety and Feasibility of Autologous Induced Pluripotent Stem Cells of Cardiac Lineage in Subjects With Congenital Heart Disease
This study is testing if using heart cells made from patients' own skin can safely help people with congenital heart disease feel better.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 18 Years to 40 Years |
| Sex | All |
| Sponsor | HeartWorks, Inc. Academic / other |
| Locations | 1 site (Rochester, Minnesota) |
| Trial ID | NCT05647213 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety of autologous induced pluripotent stem cells (iPSCs) derived from patients' own skin cells for treating individuals with congenital heart disease. Participants will undergo a skin punch biopsy to create the stem cells, which will then be administered to assess their safety and feasibility. The study will involve monitoring participants before and after the treatment, comparing outcomes between those who receive the investigational product and those who do not. The trial focuses on patients with univentricular heart disease and severe heart failure.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 to 40 with univentricular congenital heart disease and end-stage heart failure classified as NYHA Class IV.
Not a fit: Patients who do not have univentricular congenital heart disease or are outside the age range of 18 to 40 years may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could provide a novel treatment option for patients with severe congenital heart disease, potentially improving their heart function and quality of life.
How similar studies have performed: While the use of iPSCs in cardiac applications is a growing field, this specific approach for congenital heart disease is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Individuals may be considered eligible for enrollment for Part I of this study (Skin Punch Biopsy) if in the best judgment of the Principal Investigator they will meet eligibility criteria outlined below at the time it is determined acceptable investigational product is available for administration (approximately 9 months post skin punch biopsy). Inclusion and exclusion criteria apply to both the treatment and control arms of the study unless otherwise specified. Inclusion Criteria Individuals who meet all the following criteria are eligible for enrollment as study participants: * 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 Criteria Individuals who meet any of the following criteria are not eligible for enrollment as study participants: * 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.
Where this trial is running
Rochester, Minnesota
- Mayo Clinic — Rochester, Minnesota, United States (Recruiting)
Study contacts
- Study coordinator: Adam Armstrong
- Email: adam@webuildhearts.org
- Phone: 1-(507) 577-1764
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.