Table 3 Open in new tab Ongoing clinical...

Trials

Phase

Placebo- controlled; randomized; double-blind

Follow-up

Cell type, dose, and treatment groups

Delivery method

Participants

Endpoint

STEM VAD¹¹⁷

IIa

Yes; yes; yes

12 months

Three doses of allo BM-MSCs, 1.5 × 10⁶ cells/kg^a given 1 month apart

HF (ischaemic or non-ischaemic) requiring LVAD implantation and deemed stable on LVAD

Primary: uncontrolled infection, all-cause mortality. Secondary: RV systolic function, admission for RV failure, 6MWT, reduction in NK cells, change in NT-proBNP

CardiAMP-HF¹¹⁸ Raval et al.

III

No (sham controlled); yes; yes

12 months

250

200 × 10⁶ auto BM-MNCs

LVEF 20–40% secondary to remote MI

Primary: 6MWT; secondary: survival, MACE, MLHFQ

SCIENCE¹¹⁹

Yes; yes; yes

12 months

133

100 × 10⁶ adipose-derived allo MSCs vs. placebo

Ischaemic cardiomyopathy, EF ≤45% on echo, CT or MRI

Primary: change in LVESV; Secondary: serious adverse events

Allogenic Stem Cell Therapy in Heart Failure (CSCC_ASCII)¹²⁰

Yes; yes; yes

12 months

100 × 10⁶ adipose-derived allo MSCs vs. placebo, 2:1 randomization

Ischaemic cardiomyopathy, EF ≤45%

Primary: change in LVESV; secondary: incidence of treatment-emergent adverse events, LVEF, KCCQ, Seattle Angina Questionnaire, 6MWT

REPEAT¹²¹

II/III

No; yes; no

5 years

Autologous BM-MNCs, single dose vs. two doses 4 months apart

Ischaemic cardiomyopathy, EF ≤45%

Primary: mortality at 2 years; Secondary: morbidity efficacy endpoints (cardiac and CV mortality, HF hospitalization, ischaemic CE, CR, cardiac transplantation, VAD, ST, ICD, NYHA status, MLHFQ) and safety endpoints (BE, HE, LTA, new malignancies)

Table 3

Open in new tab

Ongoing clinical trials in ischaemic or non-ischaemic cardiomyopathy

Trials	Phase	Placebo- controlled; randomized; double-blind	Follow-up	n	Cell type, dose, and treatment groups	Delivery method	Participants	Endpoint
STEM VAD¹¹⁷	IIa	Yes; yes; yes	12 months	30	Three doses of allo BM-MSCs, 1.5 × 10⁶ cells/kg^a given 1 month apart	IV	HF (ischaemic or non-ischaemic) requiring LVAD implantation and deemed stable on LVAD	Primary: uncontrolled infection, all-cause mortality. Secondary: RV systolic function, admission for RV failure, 6MWT, reduction in NK cells, change in NT-proBNP
CardiAMP-HF¹¹⁸ Raval et al.	III	No (sham controlled); yes; yes	12 months	250	200 × 10⁶ auto BM-MNCs	TO	LVEF 20–40% secondary to remote MI	Primary: 6MWT; secondary: survival, MACE, MLHFQ
SCIENCE¹¹⁹	II	Yes; yes; yes	12 months	133	100 × 10⁶ adipose-derived allo MSCs vs. placebo	TO	Ischaemic cardiomyopathy, EF ≤45% on echo, CT or MRI	Primary: change in LVESV; Secondary: serious adverse events
Allogenic Stem Cell Therapy in Heart Failure (CSCC_ASCII)¹²⁰	II	Yes; yes; yes	12 months	81	100 × 10⁶ adipose-derived allo MSCs vs. placebo, 2:1 randomization	TO	Ischaemic cardiomyopathy, EF ≤45%	Primary: change in LVESV; secondary: incidence of treatment-emergent adverse events, LVEF, KCCQ, Seattle Angina Questionnaire, 6MWT
REPEAT¹²¹	II/III	No; yes; no	5 years	81	Autologous BM-MNCs, single dose vs. two doses 4 months apart	IC	Ischaemic cardiomyopathy, EF ≤45%	Primary: mortality at 2 years; Secondary: morbidity efficacy endpoints (cardiac and CV mortality, HF hospitalization, ischaemic CE, CR, cardiac transplantation, VAD, ST, ICD, NYHA status, MLHFQ) and safety endpoints (BE, HE, LTA, new malignancies)

Trials	Phase	Placebo- controlled; randomized; double-blind	Follow-up	n	Cell type, dose, and treatment groups	Delivery method	Participants	Endpoint
STEM VAD¹¹⁷	IIa	Yes; yes; yes	12 months	30	Three doses of allo BM-MSCs, 1.5 × 10⁶ cells/kg^a given 1 month apart	IV	HF (ischaemic or non-ischaemic) requiring LVAD implantation and deemed stable on LVAD	Primary: uncontrolled infection, all-cause mortality. Secondary: RV systolic function, admission for RV failure, 6MWT, reduction in NK cells, change in NT-proBNP
CardiAMP-HF¹¹⁸ Raval et al.	III	No (sham controlled); yes; yes	12 months	250	200 × 10⁶ auto BM-MNCs	TO	LVEF 20–40% secondary to remote MI	Primary: 6MWT; secondary: survival, MACE, MLHFQ
SCIENCE¹¹⁹	II	Yes; yes; yes	12 months	133	100 × 10⁶ adipose-derived allo MSCs vs. placebo	TO	Ischaemic cardiomyopathy, EF ≤45% on echo, CT or MRI	Primary: change in LVESV; Secondary: serious adverse events
Allogenic Stem Cell Therapy in Heart Failure (CSCC_ASCII)¹²⁰	II	Yes; yes; yes	12 months	81	100 × 10⁶ adipose-derived allo MSCs vs. placebo, 2:1 randomization	TO	Ischaemic cardiomyopathy, EF ≤45%	Primary: change in LVESV; secondary: incidence of treatment-emergent adverse events, LVEF, KCCQ, Seattle Angina Questionnaire, 6MWT
REPEAT¹²¹	II/III	No; yes; no	5 years	81	Autologous BM-MNCs, single dose vs. two doses 4 months apart	IC	Ischaemic cardiomyopathy, EF ≤45%	Primary: mortality at 2 years; Secondary: morbidity efficacy endpoints (cardiac and CV mortality, HF hospitalization, ischaemic CE, CR, cardiac transplantation, VAD, ST, ICD, NYHA status, MLHFQ) and safety endpoints (BE, HE, LTA, new malignancies)

6MWT, 6-min walk test; allo, allogeneic; BE, bleeding events; BMMNCs, bone marrow mononuclear cells; BM-MSCs, bone marrow-derived mesenchymal stromal cells; CDCs, cardiosphere-derived cells; CE, cardiac events; CR, coronary revascularization; CT, computed tomography; CV, cardiovascular; Echo, echocardiography; EF, ejection fraction; HE, all in-hospital events during therapy; HF-MACE, heart failure major adverse cardiac events; IC, intracoronary; ICD, implantable cardioverter-defibrillator; IV, intravenous; KCCQ, Kansas City Cardiomyopathy Questionnaire; LTA, life-threatening arrhythmias; LV, left ventricle; LVEDVI, left ventricular end-diastolic volume index; LVEF, left ventricular ejection fraction; LVESVI, left ventricular end-systolic volume index; MACE, major adverse cardiac events; MLHFQ, Minnesota Living with Heart Failure Questionnaire; MPC, Mesenchymal Precursor Cells; n, number of patients; MRI, magnetic resonance imaging; NT-proBNP, N-terminal pro-brain natriuretic peptide; NYHA, New York Heart Association; RV, right ventricle; ST, new synchronization therapy; TCE, time-to-first terminal cardiac event; TE, transendocardial; VAD, assisted device implantation; VO₂ max, maximal oxygen consumption.

Cells were cultured at 5% O₂.

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