Review Article

Cell Therapies in Cardiomyopathy: Current Status of Clinical Trials

Table 1

Published stem-like cell clinical trials for cardiac myopathy.

Study name (sponsor)NCT IDNumber of patients (treatment/controls)Delivery routeCell dose (×106)Follow-up interval (months)OutcomesAdverse effect

Skeletal myoblasts
MAGIC (Genzyme)NCT0010212867/30IMLow dose: 400
High dose: 800
6Neutral for LVEF
Reduced LVESV (−9.5%)
Reduced LVEDV from high dose (−13.6%)
Low dose: 4 with ventricular arrhythmias and 5 deaths
High dose: 5 with arrhythmias and 4 deaths
CAUSMIC (Mytogen)NCT0062631412/11IM30, 100, 300, 60012Improved LVEF (12.4%)
Reduced NYHA
6 with ventricular arrhythmias out of 12 patients
SEISMIC (Bioheart)NCT0037581726/14IM150–8006Neutral for LVEF
Reduced for NYHA
Increased 6 MWD
12 with ventricular arrhythmias out of 26 patients and 1 death
Bone marrow mononuclear cells
Janssens et al. (Universitaire Ziekenhuizen Leuven)NCT0026431633/34IC3044Neutral for LVEF
Reduced infarct size (−28%)
No major adverse events
REPAIR-AMI (A. M. Zeiher)NCT00279175102/102IC20024Improved LVEF (2%)
Reduced LVESV (−1%)
No major adverse events
Cardio 133 (German Heart Institute)NCT0046277430/30IM56Reduced LVEF (−2.1%)
Neutral LVESV
Neutral LVEDV
No major adverse events
FINCELL (University of Oulu)NCT0036332440/40IC3606Improved LVEF (5%)
Reduced LVESV (−5.7%)
Reduced LVEDV (−1.3%)
No major adverse events
MiHeart-AMI (Ministry of Health, Brazil)NCT00350766150/150IC10012No study resultsNo study results
BOOST (Hannover Medical School)NCT0022453630/30IC20006, 18, 60Larger scar: improved LVEF (7.6%)
Smaller scar: reduced LVEF (−6.6%)
Increased LVESV (42%)
Increased LVEDV (29%)
No major adverse events
DanCell-CHF (Odense University Hospital)NCT0023541732/0IC1st: 650
2nd: 900
4, 8, 12Neutral for LVEF
Improved LV filling
No major adverse events
MYSTAR (Medical University of Vienna)NCT0038498240/20IC/IM2003Early group: Improved LVEF (3.5%)
Late group: Improved LVEF (3.9%)
No major adverse events
BALANCE (University of Düsseldorf)Reference [46]62/62IC603, 12, 60Improved LVEF (10%)
Reduced LVESV (−4.0%)
Reduced infarct size (−8.2%)
No major adverse events
REGENT (Silesian School of Medicine)NCT00316381160/40IC2006Neutral for LVEFNo major adverse events
STAR-heart (University of Düsseldorf)Reference [68]191/200IC663, 12, 60Improved LVEF (6.2%)
Reduced LVESV (−12.9%)
Reduced LVEDV (−5.1%)
No major adverse events
AMRS (Emory University)NCT0031333928/22IC5, 10, 156Improved LVEF (2.1%)
Reduced infarct size (−5.5%)
No major adverse events
TOPCARE-AMI (Johann Wolfgang Goethe University Hospital)NCT0028982229/30IC5.560Improved LVEF (11.6%)
Reduced LVESV (−16.3%)
Reduced LVEDV (−49.5%)
No major adverse events
Hu et al. (China National Center for Cardiovascular Diseases)NCT0039581131/29IC10–7006Improved LVEF (5.7%)
Reduced LVESV (−14.8%)
Reduced LVEDV (−10.7%)
No major adverse events
BONAMI (Nantes University Hospital)NCT0020070752/49IC10012Neutral for LVEF
Neutral for infarct size
1 death in cell treatment group after 1 month
PERFECT (Miltenyi Biotec GmbH)NCT0095027441/40IM0.5–56No study resultsNo study results
TIME (The University of Texas Health Science Center, Houston)NCT0068402143/24IC1506Neutral for LVEFNo major adverse events
ASTAMI (Oslo University Hospital)NCT0019982350/50IC803, 6, 12, 36Neutral for LVEF
Reduced infarct size (−25%)
No major adverse events
SCAMI (University of Ulm)NCT0066922728/14IC3246, 12, 24, 366 months: improved LVEF (4.1%)
12 months: improved LVEF (3.8%)
24 months: improved LVEF (4.5%)
36 months: reduced LVEF (5.6%)
No major adverse events
METHOD (Cardiocentro Ticino)NCT0166613236/18IC/IM50–5006, 12Neutral for LVEFNo major adverse events
SWISS-AMI (University of Zurich)NCT00355186128/64IC119–1534, 6, 12Neutral for LVEF
Reduced LVESV (−9.4%)
Reduced LVEDV (−8.4%)
Reduced infarct size (−4.67%)
No major adverse events
TAC-HFT (University of Miami)NCT0076806619/10IM100, 20012Neutral for infarct sizeNo major adverse events
Helsinki BMMC (Helsinki University)NCT0041841820/19IC84012Neutral for LVEF
Reduced infarct size (13.1%)
No major adverse events
MultiStem (Athersys, Inc.)NCT0067722219/6IC20, 50, 100420 M cells: Improved LVEF (3.9%)
50 M cells: Improved LVEF (13.5%)
100 M cells: Improved LVEF (10.9%)
No major adverse events
Hu et al. (Second Affiliated Hospital, School of Medicine, Zhejiang University)NCT0123418122/14IC1006, 12Neutral for LVEFNo major adverse events
Ge et al. (Fudan University)NCT0242535879/25IC5001224 after PCI: improved LVEF (4.5%)
3−7 days after PCI: improved LVEF (3.5%)
3−30 days after PCI: improved LVEF (1.3%)
No major adverse events
TECAM (TECAM Group)NCT0098417889/31IC512Improved LVEF (4%)
Neutral for LVESV
Neutral LVEDV
No major adverse events
REGENERATE-AMI (Barts & The London NHS Trust)NCT0076545355/45IC59.83, 12Improved LVEF (2.2%)
Reduced infarct size (−33.3%)
No major adverse events
      
Mesenchymal stem cells
Prochymal (Mesoblast International Sàrl)NCT0011445239/21IV30–3003, 6, 12Improved LVEF (6.7%)No major adverse events
POSEIDON-Pilot (University of Miami)NCT0108799615/16 (autologous/allogeneic)IM20, 100, 20013Neutral for LVEF
20 M cells: Improved LVEF
Reduced infarct size (−33.21%)
Allogeneic: Reduced LVEDV
No major adverse events
PROMETHEUS (Joshua M Hare)NCT005879906/3IM20, 2003, 6, 18Improved LVEF (9.5%)
Reduced infarct size (−47.5%)
No major adverse events
TAC-HFT (University of Miami)NCT0076806619/10IM100, 20012Neutral for LVEF
Reduced infarct size (−18.9%)
Increased 6 MWD
No major adverse events
SEED-MSC (Yonsei University)NCT0139210530/28IC726Improved LVEF (5.9%)No major adverse events
Stempeucel (Stempeutics Research Pvt. Ltd.)NCT0088372710/10IV20024Improved LVEF (2%)No major adverse events
WJ-MSC-AMI (Navy General Hospital, Beijing)NCT0129132958/57IC618Improved LVEF (5%)
Reduced LVESV (−22.6%)
Reduced LVEDV (−9.5%)
No major adverse events
      
Adipose derived mesenchymal stem cells
PRECISE (Cytori Therapeutics)NCT0042686821/6IM426, 12, 18Neutral for LVEF
Reduced infarct size (−12.8%)
No major adverse events
CSCC_ASC (Jkastrup, Denmark)NCT0238772310IM1006No study resultsNo study results
ATHENA (Cytori Therapeutics)NCT0155602214/14IM406, 12No study resultsNo study results
      
Cardiac stem cells
SCIPIO (University of Louisville)NCT0047446116/7IC14, 12Improved LVEF (12.3%)
Reduced infarct size (−30.2% )
No major adverse events
CADUCEUS (Cedars-Sinai Medical Center)NCT0089336017/8IC12.5–256, 12Neutral for LVEF
Neutral for LVESV
Neutral for LVEDV
Reduced infarct size (−12.3%)
4 patients had serious adverse events

IC, intracoronary infusion; IM, intramyocardial injection; IV, intravenous infusion; LV, left ventricular; LVEDV, LV end-diastolic volume; LVEF, LV ejection fraction; LVESV, LV end-systolic volume; MWD, minute walk distance; NYHA, New York Heart Association; PCI, percutaneous coronary intervention.