Univentricular HeartPhase 1NCT05647213
Autologous Induced Pluripotent Stem Cells of Cardiac Lineage for Congenital Heart Disease
A Phase 1 study testing iPSC-CL in people with Univentricular Heart and Congenital Heart Disease, run by HeartWorks, Inc. at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 40 years
- Conditions the study is for
- Univentricular Heart, Congenital Heart Disease, Heart Failure NYHA Class III
- What is being tested?
- iPSC-CL
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Feb 2023to Feb 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 40 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55901
Karen Miller, HLHS@mayo.edu
Lori Riess, HLHS@mayo.edu
About this study
Official title: Safety and Feasibility of Autologous Induced Pluripotent Stem Cells of Cardiac Lineage in Subjects With Congenital Heart Disease
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.
- Conditions
- Univentricular Heart, Congenital Heart Disease, Heart Failure NYHA Class III, Heart Failure NYHA Class IV
- Being tested
- iPSC-CL
- Keywords
- univentricular, heart disease, iPSC, induced pluripotent stem cell
- Medical subject headings
- Univentricular Heart, Heart Defects, Congenital, Heart Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
iPSC-CLBiological
Autologous IPSCL
| Study group | Role | What they receive |
|---|---|---|
| Treated | Experimental | 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. Receives: Biological: iPSC-CL |
| Control | No intervention | Subjects who enroll but do not receive Investigational Product will be placed in the control arm. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Short term safety
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.
Measured: 3 months
Feasibility
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.
Measured: 12 months
Secondary outcomes (5)
Long term safety
Long term safety measured as new or worsening serious adverse events for two years after iPSC-CL delivery as compared to the control arm.
Measured: 2 years
Cardiac High Sensitivity Troponin T
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.
Measured: 1 month
NT-pro-BNP
Change from baseline in NT-pro-BNP levels at 1 and 3 months post iPSC-CL delivery as compared to the control arm.
Measured: 3 months
Tumor marker levels
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.
Measured: Three months from date of treatment and every 12 months after treatment, assessed up to 15 years
Panel Reactive Antibody (PRA) levels
Change from baseline in Panel Reactive Antibody (PRA) levels at 3 and 12 months post-iPSC-CL delivery as compared to the control arm.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2022
- Study start3 Feb 2023
- Primary completionSep 2027 (estimated)
- Study completionFeb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Univentricular HeartStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- HeartWorks, Inc.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Timothy J Nelson, M.D., Ph.D., Study director, HeartWorks, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05647213
- Sponsor's protocol ID
- iPSC-CL-001
- Status last verified
- Jun 2025
- First submitted
- 30 Nov 2022
- Last updated
- 13 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Feasibility of Autologous Induced Pluripotent Stem Cells of Cardiac Lineage in Subjects With Congenital Heart Disease
- ClinicalTrials.gov ID
- NCT05647213
- Acronym / sponsor ID
- iPSC-CL-001
- Conditions
- Univentricular Heart, Congenital Heart Disease, Heart Failure NYHA Class III, Heart Failure NYHA Class IV
- Keywords
- univentricular, heart disease, iPSC, induced pluripotent stem cell
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- HeartWorks, Inc.University, hospital or other
- Study start
- 3 Feb 2023 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Feb 2029 (estimated)
- First posted
- 12 Dec 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treated | Experimental | Biological: iPSC-CLSubjects 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. |
| Control | No intervention | –Subjects who enroll but do not receive Investigational Product will be placed in the control arm. |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (12 listed)
- 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%.
Key exclusion criteria (27 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| iPSC-CLAutologous IPSCL | Biological | Treated |
Primary outcome
Short term safety
Time frame: 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
Time frame: 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.
5 secondary outcomes
Long term safety
Time frame: 2 years
Cardiac High Sensitivity Troponin T
Time frame: 1 month
NT-pro-BNP
Time frame: 3 months
Tumor marker levels
Time frame: Three months from date of treatment and every 12 months after treatment, assessed up to 15 years
Panel Reactive Antibody (PRA) levels
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Timothy J Nelson, M.D., Ph.D. study director
HeartWorks, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing iPSC-CL in people with Univentricular Heart and Congenital Heart Disease, run by HeartWorks, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Adam Armstrong (1-(507) 577-1764, adam@webuildhearts.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Short term safety (time frame: 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Mar 2026.