Clinical trials investigating cell therapy identifierStudy designStudy durationMain inclusion criteriaPrimary outcome measuresSecondary outcome measures
Autologous EPCs transfected with human eNOSNCT03001414Multicentre double-blind crossover RCT versus placebo: phase 2 (SAPPHIRE)24 weeksPAH in WHO FC II–IV on stable PAH-specific therapyChange in 6MWDChange in 6MWD (3, 9 and 12 months) and PVR; number of deaths or clinical worsening of PAH; change in RV function (echocardiography and MRI) and QoL (SF-36)
Allogeneic human cardiosphere-derived stem cellsNCT03145298Phase 1a: open-label; phase 1 (ALPHA)1 yearPAH in WHO FC II–III on stable PAH-specific therapySafety (gas exchange, haemodynamics); arrhythmia; sudden death; mortality and morbidityLong-term safety end-points; TTCW (including death)
Phase 1b: double-blind RCT versus placebo: phase 1 (ALPHA)1 year

EPC: endothelial progenitor cell; eNOS: endothelial nitric oxide synthase; RCT: randomised controlled trial; PAH: pulmonary arterial hypertension; WHO: World Health Organization; FC: functional class; 6MWD: 6-min walk distance; PVR: pulmonary vascular resistance; RV: right ventricle; MRI: magnetic resonance imaging; QoL: quality of life; SF-36: Short Form-36; TTCW: time to clinical worsening.