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NCT02467387

A Study to Assess the Effect of Intravenous Dose of (aMBMC) to Subjects With Non-ischemic Heart Failure

Completed Phase 2 Results posted Last updated 7 January 2020
What this trial tests

Phase 2 trial testing Allogeneic Mesenchymal Bone Marrow Cells (aMBMC) in Non-Ischemic Heart Failure in 23 participants. Completed in 11 May 2017.

Timeline
1 June 2014
Primary endpoint
11 May 2017
11 May 2017

Quick facts

Lead sponsorCardioCell LLC
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designcrossover
Maskingsingle
Primary purposetreatment
Enrollment23
Start date1 June 2014
Primary completion11 May 2017
Estimated completion11 May 2017
Sites5 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

CardioCell LLC — full company profile →

Who can join

18 and older, any sex, with Non-Ischemic Heart Failure. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Safety Will be Evaluated by Number of AE Primary · Total AEs and SAEs within 450 days post-infusion

As identified in the SAP, the safety analysis was the primary objective and was evaluated by the number of AEs

Adverse Events
GroupValue95% CI
Experimental38
Placebo35
Serious Adverse Events
GroupValue95% CI
Experimental1
Placebo1
Cell related adverse events
GroupValue95% CI
Experimental1
Placebo0
All-cause hospitalization
GroupValue95% CI
Experimental0
Placebo0
All-cause death
GroupValue95% CI
Experimental0
Placebo0
Change in LVEF From Baseline to Day 90 Post-initial Infusion. Secondary · Baseline to Day 90

The secondary efficacy endpoint was the change in LVEF from baseline to Day 90 post-initial infusion. Participants with data available at each time point.

Baseline (%)
GroupValue95% CI
Experimental: Human (itMSC)34.3± 7.91
Placebo:Lactated Ringer's Solution (LRS)34.5± 7.49
90 days (%)
GroupValue95% CI
Experimental: Human (itMSC)34.1± 9.7
Placebo:Lactated Ringer's Solution (LRS)36.7± 5.42

Adverse events — posted to ClinicalTrials.gov

Time frame: 450 days. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Experimental
Serious: 1/22 (5%)
Deaths: 0/22
Placebo
Serious: 1/20 (5%)
Deaths: 0/20

Serious adverse events (1 terms)

ReactionSystemExperimentalPlacebo
atrial fibrillationCardiac disorders
Other adverse events (7 terms — click to expand)

ReactionSystemExperimentalPlacebo
General Disorders and administration site condition: chest pain, fatigue, pyrexiaGeneral disorders
Dizziness, headacheNervous system disorders
Upper Respiratory Tract, NasopharyngitisInfections and infestations
Fluid Overload, DehydrationMetabolism and nutrition disorders
Venous Pressure Jugular IncreasedInvestigations
Oropharyngeal PainRespiratory, thoracic and mediastinal disorders
Muscle SpasmsMusculoskeletal and connective tissue disorders

Most-reported serious reactions: atrial fibrillation.

Data from ClinicalTrials.gov NCT02467387 adverse events section.

Sponsor's own description

A phase IIa study to assess the safety and preliminary efficacy of intravenous dose of ischemia-tolerant Allogeneic Mesenchymal Bone Marrow Cells in subjects with non-ischemic heart failure.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Challenges and advances in clinical applications of mesenchymal stromal cells.
    Zhou T, Yuan Z, Weng J, Pei D, et al · · 2021 · cited 489× · PMID 33579329 · DOI 10.1186/s13045-021-01037-x
  2. Trends in mesenchymal stem cell clinical trials 2004-2018: Is efficacy optimal in a narrow dose range?
    Kabat M, Bobkov I, Kumar S, Grumet M. · · 2020 · cited 337× · PMID 31804767 · DOI 10.1002/sctm.19-0202
  3. The therapeutic potential of mesenchymal stem cells for cardiovascular diseases.
    Guo Y, Yu Y, Hu S, Chen Y, et al · · 2020 · cited 207× · PMID 32393744 · DOI 10.1038/s41419-020-2542-9
  4. Intravenous Allogeneic Mesenchymal Stem Cells for Nonischemic Cardiomyopathy: Safety and Efficacy Results of a Phase II-A Randomized Trial.
    Butler J, Epstein SE, Greene SJ, Quyyumi AA, et al · · 2017 · cited 129× · PMID 27856497 · DOI 10.1161/circresaha.116.309717
  5. Overcoming the Roadblocks to Cardiac Cell Therapy Using Tissue Engineering.
    Yanamandala M, Zhu W, Garry DJ, Kamp TJ, et al · · 2017 · cited 83× · PMID 28774384 · DOI 10.1016/j.jacc.2017.06.012
  6. Pharmacokinetic characteristics of mesenchymal stem cells in translational challenges.
    Shan Y, Zhang M, Tao E, Wang J, et al · · 2024 · cited 47× · PMID 39271680 · DOI 10.1038/s41392-024-01936-8
  7. Empowering Adult Stem Cells for Myocardial Regeneration V2.0: Success in Small Steps.
    Broughton KM, Sussman MA. · · 2016 · cited 46× · PMID 26941423 · DOI 10.1161/circresaha.115.305227
  8. The immune system in cardiovascular diseases: from basic mechanisms to therapeutic implications.
    Wang X, Chen L, Wei J, Zheng H, et al · · 2025 · cited 39× · PMID 40404619 · DOI 10.1038/s41392-025-02220-z

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