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

NCT05647213 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: iPSC-CL-001
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

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

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

Keywords

univentricular; heart disease; iPSC; induced pluripotent stem cell

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  12 months

Secondary Outcomes (5)

• Long term safety

  2 years

• Cardiac High Sensitivity Troponin T

  1 month

• NT-pro-BNP

  3 months

• Tumor marker levels

  Three months from date of treatment and every 12 months after treatment, assessed up to 15 years

• Panel Reactive Antibody (PRA) levels

  12 months

Study Arms (2)

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.

Biological: iPSC-CL

Control

NO INTERVENTION

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

Interventions

iPSC-CL BIOLOGICAL

Autologous IPSCL

Treated

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• HeartWorks, Inc.: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55901, United States

RECRUITING

MeSH Terms

Conditions

Univentricular Heart; Heart Defects, Congenital; Heart Diseases

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Timothy J Nelson, M.D., Ph.D.

  HeartWorks, Inc.

  STUDY DIRECTOR

Central Study Contacts

Adam Armstrong

CONTACT

1-(507) 577-1764; adam@webuildhearts.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 30, 2022
• First Posted: December 12, 2022
• Study Start: February 3, 2023
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): February 1, 2029
• Last Updated: March 13, 2026

Record last verified: 2025-06

Locations

US (1)