Congenital Heart Disease, Heart Failure NYHA Class III, Heart Failure NYHA Class IV, Univentricular Heart
Conditions
Keywords
univentricular, heart disease, iPSC, induced pluripotent stem cell
Brief summary
The goal of this clinical trial is to test the safety of lab-grown heart cells made from stem cells in subjects with congenital heart disease. The main questions it aims to answer are: * Is this product safe to deliver to humans * Is the conduct of this trial feasible Participants will be asked to: * Agree to testing and monitoring before and after product administration * Receive investigational product * Agree to lifelong follow-up Researchers will compare subjects from the same pool to see if there is a difference between treated and untreated subjects.
Interventions
BIOLOGICALiPSC-CL
Autologous IPSCL
Sponsors
HeartWorks, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 40 Years
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Short term safety | 3 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. |
| Feasibility | 12 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cardiac High Sensitivity Troponin T | 1 month | Change from baseline in Cardiac High Sensitivity Troponin T at 3 hours (±30 min) and 6 hours (±30 min) after iPSC-CL delivery and at 1 month post-surgery as compared to the control arm. |
| Tumor marker levels | Three months from date of treatment and every 12 months after treatment, assessed up to 15 years | Change from baseline in tumor marker levels (PSA (males only), CA 125, CEA, CA 19-9, alpha-fetoprotein (AFP), CA 195, Alpha Subunit HCG) 3 months and annually after iPSC-CL delivery as compared to the control arm. |
| Panel Reactive Antibody (PRA) levels | 12 months | Change from baseline in Panel Reactive Antibody (PRA) levels at 3 and 12 months post-iPSC-CL delivery as compared to the control arm. |
| Long term safety | 2 years | Long term safety measured as new or worsening serious adverse events for two years after iPSC-CL delivery as compared to the control arm. |
| NT-pro-BNP | 3 months | Change from baseline in NT-pro-BNP levels at 1 and 3 months post iPSC-CL delivery as compared to the control arm. |
Countries
United States
Contacts
CONTACTAdam Armstrong
STUDY_DIRECTORTimothy J Nelson, M.D., Ph.D.
HeartWorks, Inc.
Outcome results
None listed