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

NCT ID: NCT05647213

Last Updated: 2025-06-25

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-02-03
• Study Completion Date: 2029-02-28

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.

Conditions

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treated

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.

Group Type: EXPERIMENTAL

iPSC-CL

Intervention Type: BIOLOGICAL

Autologous IPSCL

Control

Subjects who enroll but do not receive Investigational Product will be placed in the control arm.

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

iPSC-CL

Autologous IPSCL

Intervention Type: BIOLOGICAL

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 40 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

HeartWorks, Inc.

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Timothy J Nelson, M.D., Ph.D.

Role: STUDY_DIRECTOR

HeartWorks, Inc.

Locations

Mayo Clinic

Rochester, Minnesota, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Adam Armstrong

Role: CONTACT

adam@webuildhearts.org

1-(507) 577-1764

Facility Contacts

Karen Miller

Role: primary

HLHS@mayo.edu

Lori Riess

Role: backup

HLHS@mayo.edu

Other Identifiers

iPSC-CL-001

Identifier Type: -

Identifier Source: org_study_id