Phase 2
Completed N=125
Combination of Mesenchymal and C-kit+ Cardiac Stem Cells as Regenerative Therapy for Heart Failure
Ischemic Cardiomyopathy
Source: ClinicalTrials.gov NCT02501811 ↗
Enrolled (actual)
125
Serious AEs
47.2%
Results posted
Apr 2021
Primary outcomePrimary: Change From Baseline in Left Ventricular Ejection Fraction (LVEF) — -0.42; 0.37; 1.05; -0.33 percentage of end diastolic volume — p=0.993
Summary
This is a phase II, randomized, placebo-controlled clinical trial designed to assess feasibility, safety, and effect of autologous bone marrow-derived mesenchymal stem cells (MSCs) and c-kit+ cells both alone and in combination (Combo), compared to placebo (cell-free Plasmalyte-A medium) as well as each other, administered by transendocardial injection in subjects with ischemic cardiomyopathy.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Left Ventricular Ejection Fraction (LVEF) |
-0.42; 0.37; 1.05; -0.33 | 0.993 |
| PRIMARY Change From Baseline in Global Strain (HARP MRI) |
-0.36; 0.48; 0.56; -0.10 | 0.942 |
| PRIMARY Change From Baseline in Regional Strain (HARP MRI) |
-0.50; -0.51; -0.76; -0.91 | 0.733 |
| PRIMARY Change From Baseline in Left Ventricular End Diastolic Volume Index (LVEDVI) |
0.97; 0.21; -1.13; 2.99 | 0.602 |
| PRIMARY Change From Baseline in Left Ventricular End Systolic Volume Index (LVESVI) |
0.98; -0.22; -2.37; 2.23 | 0.659 |
| PRIMARY Change From Baseline in Left Ventricular Sphericity Index |
0.00; -0.00; 0.00; 0.01 | 0.989 |
| PRIMARY Change From Baseline in Scar Size Percent (DEMRI) |
-0.57; -0.70; -1.06; -0.53 | 0.569 |
| PRIMARY Change From Baseline in Scar Tissue Mass (DEMRI) |
-0.87; -1.13; -2.51; -1.31 | 0.992 |
| PRIMARY Change From Baseline in Maximal Oxygen Consumption (Peak VO2) |
-0.89; -0.83; 0.77; 0.13 | 0.157 |
| PRIMARY Change From Baseline in Exercise Tolerance (Six Minute Walk Test) |
19.24; 23.85; 2.36; 0.67 | 0.348 |
| PRIMARY Change From Baseline in Minnesota Living With Heart Failure Questionnaire (MLHFQ) Score |
-15.32; -20.78; -8.65; -5.68 | 0.023 sig |
| PRIMARY Change From Baseline in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) |
-724.46; 52.6; 204.09; 687.25 | 0.110 |
| PRIMARY Change From Baseline in Left Ventricular Ejection Fraction (LVEF)-Trajectory |
0.167; 0.422; 0.633; -0.175 | 0.269 |
| PRIMARY Change From Baseline in Global Strain (HARP MRI)-Trajectory |
0.126; -0.324; 0.276; 0.576 | 0.361 |
| PRIMARY Change From Baseline in Regional Strain (HARP MRI)-Trajectory |
-0.084; -0.385; -0.325; -0.288 | 0.107 |
| PRIMARY Change From Baseline in Left Ventricular End Diastolic Volume Index (LVEDVI)-Trajectory |
1.538; 0.320; 1.796; 1.862 | 0.095 |
| PRIMARY Change From Baseline in Left Ventricular End Systolic Volume Index (LVESVI)-Trajectory |
0.826; -0.394; 0.626; 1.077 | 0.379 |
| PRIMARY Change From Baseline in Left Ventricular Sphericity Index-Trajectory |
0.004; -0.004; 0.008; 0.002 | 0.401 |
| PRIMARY Change From Baseline in Scar Size Percent (DEMRI)-Trajectory |
-0.341; -0.290; -0.427; -0.013 | 0.126 |
| PRIMARY Change From Baseline in Scar Tissue Mass (DEMRI)-Trajectory |
-0.309; -0.266; -0.958; -0.390 | 0.139 |
| PRIMARY Change From Baseline in Maximal Oxygen Consumption (Peak VO2)-Trajectory |
-0.711; 0.632; -0.165; -0.093 | 0.472 |
| PRIMARY Change From Baseline in Exercise Tolerance (Six Minute Walk Test)-Trajectory |
9.996; 11.434; 6.776; -4.248 | 0.025 sig |
| PRIMARY Change From Baseline in Minnesota Living With Heart Failure Questionnaire (MLHFQ) Score-Trajectory |
-9.258; -7.295; 0.776; -2.378 | 0.037 sig |
| PRIMARY Change From Baseline in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)-Trajectory |
74.650; 102.250; 61.680; 185.210 | 0.092 |
| PRIMARY Participants With Major Adverse Cardiac Events (MACE) |
3; 7; 2; 9 | — |
| PRIMARY Participants Experiencing Other Significant Clinical Events |
8; 3; 11; 7 | — |
| PRIMARY Cumulative Days Alive and Out of Hospital for Heart Failure |
472; 448; 438; 427 | — |
Eligibility Criteria
Inclusion Criteria
- Be ≥ 21 and 10%
- Hematologic abnormality evidenced by hematocrit < 25%, white blood cell < 2,500/ul or platelet count < 100,000/ul
- Liver dysfunction evidenced by enzymes (AST and ALT) ˃ 3 times the upper limit of normal (ULN)
- Coagulopathy (INR ≥ 1.3) not due to a reversible cause (e.g., warfarin and/or Factor Xa inhibitors). Subjects who cannot be withdrawn from anticoagulation will be excluded.
- HIV and/or active hepatitis B virus (HBV) or hepatitis C virus (HCV)
- Allergy to radiographic contrast material that cannot adequately be managed by premedication
- Known history of anaphylactic reaction to penicillin or streptomycin
- Received gene or cell-based therapy from any source within the previous 12 months
- History of malignancy within 5 years (i.e., subjects with prior malignancy must be disease free for 5 years), excluding basal cell carcinoma and cervical carcinoma in situ which have been definitively treated
- Condition that limits lifespan to < 1 year
- History of drug abuse (illegal "street" drugs except marijuana, or prescription medications not being used appropriately for a pre-existing medical condition) or alcohol abuse (≥ 5 drinks/day for ˃ 3 months), or documented medical, occupational, or legal problems arising from the use of alcohol or drugs within the past 24 months
- Participation in an investigational therapeutic or device trial within 30 days of consent
- Cognitive or language barriers that prohibit obtaining informed consent or any study elements
- Pregnancy or lactation or plans to become pregnant in the next 12 months
- Any other condition that, in the judgment of the Investigator or Sponsor, would impair enrollment, study product administration, or follow-up
Data sourced from ClinicalTrials.gov (NCT02501811). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.