Congenital heart diseaseRandomised controlled trial
Phase I Randomized Study of Cardiac Stem Cells in Patients With Hypoplastic Left Heart Syndrome
- Phase 1 randomised controlled trial
- multicentre
- double-blind
- nonrandomised consecutive cohort (group A)
All caregivers and cores except the surgeon blinded to treatment
Authors' conclusion
Injecting nCPCs into the RV during the stage 2 operation for HLHS patients appears to be safe but does not improve RV function.
Study context
- Population
- Patients with hypoplastic left heart syndrome undergoing stage 2 operation
- Participants
- 9 / 8 / 8
- Comparison
- Autologous nCPC injection into right ventricle (RV) myocardium vs standard of care during stage 2 operation
- nCPCn = 9
- nCPCn = 8
- Standard-of-care (SOC)n = 8reference
- Primary endpoint
- Safety and feasibility; efficacy: RV size and function
- Follow-up
- 1 year
Primary result
As reported in the abstract
Safety, feasibility and RV size/function
- Primary safety and feasibility endpoints met
- Primary efficacy endpoint (changes in RV size or function by CMR and echocardiography) not achieved
Secondary outcomes
| Outcome | nCPC | nCPC | SOC | P value |
|---|---|---|---|---|
| In-hospital length of stay for cardiac and vascular diseases (group B) · 1 year | Not reported in the abstract | Not reported in the abstract | 15 days per 100 person-days | P = 0.035 |
| Death or listing for transplantation (groups A and B combined) · combined nCPC arms of groups A and B · group B alone: numbers insufficient to show significance | Not reported in the abstract | Not reported in the abstract | 3 events | log-rank P = 0.005 |
- In-hospital length of stay for cardiac and vascular diseases (group B): nCPC 2 days per 100 person-days
- Death or listing for transplantation (groups A and B combined): nCPC 0 events
Plasma VEGFC, VEGFD, TNF-α, monocyte chemotactic protein-1 (mechanistic) · postoperative day 5
- Noticeable increases from baseline with nCPC vs SOC, not significant after false discovery rate adjustment
Safety
| Outcome | nCPC | nCPC | SOC | P value |
|---|---|---|---|---|
| MACE rate after stage 2 operation (group B) | Not reported in the abstract | Not reported in the abstract | mean 0.23 per 100 person-days | P = 0.013 |
- MACE rate after stage 2 operation (group B): nCPC mean 0.00 per 100 person-days
Original publication
Phase I Randomized Study of Cardiac Stem Cells in Patients With Hypoplastic Left Heart Syndrome. JACC Heart Fail. Published 19 November 2025. doi:10.1016/j.jchf.2025.102723
Summarised from the published abstract; every number is checked against it