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

Phase 1

Recruiting

• Conditions: Univentricular Heart; Heart Failure NYHA Class IV; Congenital Heart Disease; Heart Failure NYHA Class III
• Interventions: Biological: iPSC-CL

• Registration Number: NCT05647213
• Lead Sponsor: HeartWorks, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-11-30
• Study First Submitted QC: 2022-12-09
• Study First Posted: 2022-12-12
• Study Start: 2023-02-03
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-17
• Last Update Posted: 2025-03-19
• Primary Completion: 2026-02
• Study Completion: 2029-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Treated	iPSC-CL	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.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States