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. Author manuscript; available in PMC: 2014 Aug 11.
Published in final edited form as: Curr Cardiol Rep. 2013 Jun;15(6):369. doi: 10.1007/s11886-013-0369-z

Table 1.

Summary of Stem Cell Clinical Trials.

Study Design Objectives Endpoints Findings Comments
The REPAIR-AMI Trial Phase III, randomized,
double-blind, placebo-controlled trial

Enrollment: 204
patients		 To determine the
efficacy of infusing
BMMCs into the infarct
vessel (after successful
reperfusion therapy) in
improving ventricular
contractile function.		 Primary Endpoint:
Change in global left
ventricular function in
quantitative LV
angiography

Secondary Endpoints:
Several including
improvement of
regional wall motion in
infarct area, reduction
of LVESV, major
adverse cardiac events,
etc.		 Intracoronary
administration of
BMMCs improved
recovery of left
ventricular contractile
function in patients
with acute MI.		 1 yr. follow-up revealed
BMMC-treated patients
had improved event-free survival as
compared to the
placebo; further large
scale studies are
warranted to
determine effect of
BMMC treatment on
morbidity and
mortality.
The TIME Randomized
Trial 		Randomized, 2×2
factorial, double-blind,
placebo-controlled trial

Enrollment: 120
patients		 To determine the effect
of intracoronary
autologous BMMC
delivery after STEMI on
recovery of global and
regional LV function; to
determine whether
timing of BMMC
delivery, 3 days vs. 7
days after reperfusion,
influences the effect.		 Primary Endpoints:
Change in global (LVEF)
and regional (wall motion) LV function in
infarct and border
zones at 6 months

Secondary Endpoints:
Major adverse
cardiovascular events,
changes in LV volumes,
and infarct size		 No significant effect on
recovery of global or
regional LV function
compared with placebo
after administration of
intracoronary BMMCs
at either 3 days or 7
days after the event.		 While the TIME and
LateTIME trials both did
not find BMMCs
effective in improving
LV function post-STEMI,
long-term follow-up
and new composite
endpoints may be
warranted to
determine whether
there is a role for
BMMCs after AMI.
The LateTIME
Randomized Trial 		Randomized, double-blind, placebo-controlled trial

Enrollment: 87 patients		 To determine the effect
of intracoronary
delivery of autologous
BMMCs on global and
regional LV function
when delivered 2 to 3
weeks after first AMI.		 Primary Endpoints:
Changes in global LVEF
and regional (wall motion) LV function in
the infarct and border
zone at 6 months

Secondary Endpoints:
Changes in LV volumes
and infarct size		 Intracoronary infusion
of autologous BMMCs
vs. placebo infusion, 2
to 3 weeks after PCI,
did not improve global
or regional function at 6
months.
FOCUS-HF Phase I, randomized,
single-blind study

Enrollment: 30 patients		 To determine the safety
and efficacy of the
transendocardial
delivery of ABMMNCs
in no-option patients
with chronic HF.		 Primary Endpoint:
Safety: SAEs

Secondary Endpoint:
Efficacy: MVO2, SPECT,
and 2-dimensional
echocardiography, and
QOL assessment		 ABMMNC therapy is
safe. It improves
symptoms, QOL, and
possibly perfusion in
patients with chronic
HF.		 The small sample size
must betaken into
account when
considering the findings
on safety and efficacy.
The FOCUS-CCTRN trial Phase II, randomized
double-blind, placebo-controlled trial

Enrollment: 153
patients		 To determine the effect
of administration of
BMMCs through
transendocardial
injections on LVESV, or
MVO2in patients with
CAD or LV dysfunction,
and limiting HF or
angina.		 Primary Endpoints:
Changes in LVESV,
maximal oxygen
consumption, and,
reversibility on SPECT		 Transendocardial
injection of autologous
BMMCs (compared
with placebo) did not
improve LVESV, MVO2
or reversibility on
SPECT.
The FOCUS Study Phase II, randomized,
double-blind, placebo-controlled trial

Estimated Enrollment:
92 patients		 To determine the safety
and efficacy of
intramyocardial
injection of autologous
BMMCs under
electromechanical
guidance for patients
with chronic ischemic
heart disease and LV
dysfunction.		 Primary Endpoints:
Change in maximal
oxygen consumption,
LV end systolic volume
(LVESV), and in
reversible defect size

Secondary Endpoints:
Regional wall motion,
regional blood flow
improvement, regional
wall motion, and
clinical improvements,
including change in
anginal score, incidence
of a major adverse
cardiac event, and
reduction in fixed
perfusion defect(s)		 Pending Results from this study
may help clarify the
discrepancy between
findings from the
FOCUS-HF and FOCUS-CCTRN trials.
NOGA-DCM Phase II, randomized,
single-blind, placebo-controlled trial

Estimated Enrollment:
60 patients		 To determine the safety
and efficacy of
intramyocardial stem
cell therapy in patients
with non-ischemic
dilated
cardiomyopathy; to
compare clinical effects
of intracoronary and
intramyocardial stem
cell delivery.		 Primary Endpoints:
Changes in LV ejection
fraction and dimensions

Secondary Endpoints:
Changes in exercise
capacity, and changes
in NT-proBNP levels		 Pending Studies such as these
will help elucidate the
role of stem cells in
treating DCM.
Progenitor Cell Therapy
in Dilative
Cardiomyopathy 		Phase I/II, randomized,
open label trial

Estimated Enrollment:
30 patients		 To determine the effect
of transplanting bone
marrow-derived
progenitor cells on
recovery of LV function
in patients with non-
ischemic dilatative
cardiomyopathy.		 Primary Endpoints:
LV function (EF at 3 months)		 Pending
Study of Intravenous
Adult Human
Mesenchymal Stem
Cells after Acute
Myocardial Infarction 		Phase I, randomized,
double blind, placebo
controlled, dose
escalating trial

Enrollment: 53 patients		 To determine the safety
and efficacy of
intravenous allogeneic
MSCs in patients with
AMI.		 Primary Endpoints:
Safety: TE-SAEs within 6
months

Efficacy: LV volumes
and EF		 Intravenous allogeneic
MSC treatment is safe
in patients with AMI.
Findings show
provisional efficacy.
The POSEIDON
randomized trial 		Phase I/II, randomized,
open label comparison
of allogeneic and
autologous MSCs

Enrollment: 30 patients		 To determine whether
allogeneic MSCs are as
safe and effective as
autologous MSCs in
patients with LV
dysfunction due to ICM.		 Primary Endpoints:
Safety: 30 day post
catheterization
incidence of predefined
TE-SAEs

Efficacy: 6-minute walk
test, exercise peak VO2
MLHFQ, NYHAC, LV
volumes, EF, early
enhancement defect
(EED; infarct size), and
sphericity index		 Low rates of TE-SAEs.

In aggregate, MSC
injection favorably
affected patient
functional capacity,
quality of life, and
ventricular remodeling.		 Allogeneic MSCs have
been found to be
beneficial in treating
ICM and should be
explored for treating
DCM. A larger number
of patients must be
studied in following
trials.
The POSEIDON-DCM
Study 		Phase I/II, randomized,
open label, pilot study

Estimated Enrollment:
36 patients		 To comparative the
safety and efficacy of
transendocardial
injection of autologous
MSCs vs. allogeneic
MSCs in patients with
non-ischemic dilated
cardiomyopathy.		 Primary Endpoints:
Incidence of anyTE-SAEs

Secondary Endpoints:
Changes in regional LV
function		 Pending This study will help
elucidate the role of
MSCs in treating DCM.
A larger number of
patients is warranted in
future trials.
Intramuscular Injection
of MSCs for Treatment
of Children with
Idiopathic Dilated
Cardiomyopathy 		Phase I/II, randomized,
open label trial

Estimated Enrollment:
30 patients		 To determine the
effects of intramuscular
injection of umbilical
cord MSCs on the
ventricular function of
children with IDCM.		 Primary Endpoints:
Echocardiography

Secondary Endpoints:
24h HOLTER, level of
serum BNP,TNI,HGF,
LIF and G/M-CSF; the
expression level of c-kit,CD31,CD133 on
peripheral blood
mononuclear cells		 Pending This is the first pediatric
trial investigating the
role of umbilical cord
MSCs in treating IDCM.
It will provide insight on
the role of MSCs in
treating IDCM.
MAGIC Randomized, placebo-controlled, double-blind study

Enrollment: 97 patients		 To determine the safety
and efficacy of skeletal
myoblast
transplantation in
patients with LV
dysfunction, MI, and
indication for coronary
surgery.		 Primary Endpoints:
Efficacy: Changes in
global and regional LV
function at 6 months

Safety: A composite
index of major cardiac
adverse events and
ventricular arrhythmias		 Myoblast injections
combined with
coronary surgery in
patients with depressed
LV function did not
improve
echocardiographic
heart function.		 In this trial, there was
an increase in number
of early postoperative
arrhythmic events after
myoblast
transplantation.
Skeletal myoblasts have
had minimal success in
treating ICM.
CADUCEUS Phase I, randomized,
open label trial

Estimated Enrollment:
31		 To determine the safety
and efficacy of
intracoronary delivery
of cardiosphere-derived
stem cells in patients
with ischemic LV
dysfunction and a
recent myocardial
infarction.		 Primary Endpoints:
Proportion of patients
who died due to v-tach, v-fib, or sudden
unexpected death at 6
months, or had MI after
cell infusion, new
cardiac tumor
formation on MRI, or a
major adverse cardiac
event.		 Pending Findings from this study
will help assess the role
of CSCs in treating MI.
They should be
considered for treating
DCM.

ABMMNC, Autologous Bone Marrow Mononuclear Cell; BMMC, Bone Marrow Mononuclear Cell; CAD, Coronary Artery Disease; DCM, Dilated Cardiomyopathy; EF, Ejection Fraction; IDCM, Idiopathic Dilated Cardiomyopathy; HF, Heart Failure; ICM, Ischemic Cardiomyopathy; LV, Left Ventricular; LVEF, Left Ventricular Ejection Fraction; LVESV, Left Ventricular End-Systolic Volume; MVO2, Maximal Oxygen Consumption; NYHAC, New York Heart Association Class; QOL, Quality of Life; MI, Myocardial Infarction; MLHFQ, Minnesota Living with Heart Failure Questionnaire; MSC, Mesenchymal Stem Cell; SAE, Serious Adverse Event; SPECT, Single Photon Emission Computed Tomography; STEMI, ST-Elevated Myocardial Infarction; TE-SAE, Treatment-Emergent Serious Adverse Events

Event Free Survival: death, recurrence of MI, revascularization, or rehospitalization for heart failure