Stem Cell Therapy for Dilated Cardiomyopathy
(DCMII Trial)
Recruiting in Palo Alto (17 mi)
+2 other locations
Overseen byJoshua Hare, MD
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: Joshua M Hare
Must be taking: Beta blockers, ACE inhibitors
Disqualifiers: Coronary artery disease, Valvular heart disease, Liver dysfunction, others
Prior Safety Data
Trial Summary
What is the purpose of this trial?
The purpose of this study is to evaluate the safety and effectiveness of an experimental drug called human allogeneic mesenchymal stem cell therapy.
Will I have to stop taking my current medications?
The trial requires participants to be on a stable regimen of certain heart medications, like beta blockers and ACE inhibitors, for at least 30 days before the procedure. If you're already taking these medications, you won't need to stop them, but you should discuss any other medications with the trial team.
What data supports the effectiveness of the treatment Allogeneic Human Mesenchymal Stem Cells (hMSCs) for Dilated Cardiomyopathy?
Is stem cell therapy safe for humans?
How is the stem cell treatment for dilated cardiomyopathy different from other treatments?
This treatment uses allogeneic human mesenchymal stem cells (hMSCs) which are derived from donors, making it less invasive than treatments requiring the patient's own cells. It is administered intravenously, which avoids the need for cardiac catheterization, and it has shown potential to reduce inflammation and improve heart function in dilated cardiomyopathy.12456
Eligibility Criteria
This trial is for adults aged 18-80 with non-ischemic dilated cardiomyopathy (NIDCM) and a left ventricular ejection fraction ≤45%. Participants must be on stable heart medication for at least 30 days. Exclusions include drug/alcohol abuse, certain types of cardiomyopathy, recent organ/cell transplant rejection, coronary artery disease history, known LV thrombus or aneurysm, severe allergies to specific antibiotics or DMSO.Inclusion Criteria
I am between 18 and 80 years old.
I am eligible for a heart catheterization procedure.
I am on a stable heart medication regimen for non-ischemic cardiomyopathy.
See 2 more
Exclusion Criteria
I have had a transplant rejection in the past.
You have had problems with drugs or alcohol in the past 9 months.
I need a procedure to improve blood flow to my heart.
See 9 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive either the experimental human allogeneic mesenchymal stem cell therapy or placebo based on their genotype group
12 months
Follow-up
Participants are monitored for safety and effectiveness after treatment, including assessments of cardiac function and exercise tolerance
12 months
Treatment Details
Interventions
- Allogeneic Human Mesenchymal Stem Cells (hMSCs) (Mesenchymal Stem Cell Therapy)
- Placebo (Other)
Trial OverviewThe study tests the safety and effectiveness of allogeneic human mesenchymal stem cells (hMSCs) versus placebo in treating NIDCM. Patients will receive either hMSCs or placebo through transendocardial injection during cardiac catheterization.
Participant Groups
6Treatment groups
Experimental Treatment
Placebo Group
Group I: Genotype C administered with hMSC GroupExperimental Treatment1 Intervention
Participants whose blood genotyping resulted with Genotype C (pathogenic or likely pathogenic variants) and randomized to the treatment group will receive the hMSC intervention.
Group II: Genotype B administered with hMSC GroupExperimental Treatment1 Intervention
Participants whose blood genotyping resulted with Genotype B (variants of uncertain significance) and randomized to the treatment group will receive the hMSC intervention.
Group III: Genotype A administered with hMSC GroupExperimental Treatment1 Intervention
Participants whose blood genotyping resulted with Genotype A (absence of any variant/ presence of benign variant) and randomized to the treatment group will receive the hMSC intervention.
Group IV: Genotype A administered with placebo GroupPlacebo Group1 Intervention
Participants whose blood genotyping resulted with Genotype A (absence of any variant/ presence of benign variant) and randomized to the placebo group will receive the placebo intervention.
Group V: Genotype C administered with placebo GroupPlacebo Group1 Intervention
Participants whose blood genotyping resulted with Genotype C (pathogenic or likely pathogenic variants) and randomized to the placebo group will receive the placebo intervention.
Group VI: Genotype B administered with placebo GroupPlacebo Group1 Intervention
Participants whose blood genotyping resulted with Genotype B (variants of uncertain significance) and randomized to the placebo group will receive the placebo intervention.
Allogeneic Human Mesenchymal Stem Cells (hMSCs) is already approved in European Union, Japan, India, Japan for the following indications:
🇪🇺 Approved in European Union as Alofisel for:
- Complex perianal fistulas in patients with Crohn’s disease
🇯🇵 Approved in Japan as Stemirac for:
- Treatment of spinal cord injury
🇮🇳 Approved in India as Stempeucel for:
- Critical Limb Ischemia
🇯🇵 Approved in Japan as TEMCELL for:
- Acute graft-versus-host disease
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Texas Heart InstituteHouston, TX
Stanford UniversityStanford, CA
University of Miami Miller School of MedicineMiami, FL
Loading ...
Who Is Running the Clinical Trial?
Joshua M HareLead Sponsor
United States Department of DefenseCollaborator
The University of Texas Health Science Center, HoustonCollaborator
References
Placental mesenchymal and cord blood stem cell therapy for dilated cardiomyopathy. [2019]Regenerative treatment of dilated, non-ischaemic cardiomyopathy represents a significant unmet clinical need. Intracoronary administration of autologous bone marrow stem cells has demonstrated positive results in treatment of post-infarct and chronic ischaemic patients. Limitations of this procedure include: invasiveness of bone marrow extraction and cardiac catheterization, and dependence on stem cell populations that are aged and possibly senescent. Here, the use of intravenously administered allogeneic placental matrix derived mesenchymal stem cells for treatment of dilated cardiomyopathy is discussed. Safety of this cell population has already been established in completed Phase I and II trials; however, to date, clinical implementation for dilated cardiomyopathy has not been reported. Preclinical studies have demonstrated that mesenchymal stem cells: (i) inhibit myocardial inflammation; (ii) inhibit cardiomyocyte apoptosis; (iii) stimulate angiogenesis; and (iv) display therapeutic activity in models of dilated cardiomyopathy. Clinical studies have demonstrated the ability of mesenchymal stem cells to inhibit post-infarct remodelling, as well as potently block inflammatory processes in graft versus host and Crohn disease. Presented here is case report of a patient with dilated cardiomyopathy treated with intravenous allogeneic mesenchymal stem cells and expanded umbilical cord blood CD34 cells who underwent a profound clinical improvement.
Randomized Comparison of Allogeneic Versus Autologous Mesenchymal Stem Cells for Nonischemic Dilated Cardiomyopathy: POSEIDON-DCM Trial. [2022]Although human mesenchymal stem cells (hMSCs) have been tested in ischemic cardiomyopathy, few studies exist in chronic nonischemic dilated cardiomyopathy (NIDCM).
Mesenchymal stem cell therapy for cardiac repair. [2022]Stem cell therapy for repair of damaged cardiac tissue is an attractive option to improve the health of the growing number of heart failure patients. Mesenchymal stem cells (MSCs) possess unique properties that may make them a better option for cardiac repair than other cell types. Unlike other adult stem cells, they appear to escape allorecognition by the immune system and they have immune-modulating properties, thus making it possible to consider them for use as an allogeneic cell therapy product. There is a large and growing body of preclinical and early clinical experience with MSC therapy that shows great promise in realizing the potential of stem cell therapy to effect repair of damaged cardiac tissue. This review discusses the mechanism of action of MSC therapy and summarizes the current literature in the field.
Intracoronary administration of autologous mesenchymal stem cells in a critically ill patient with dilated cardiomyopathy. [2016]Relatively high prevalence of dilated cardiomyopathy in children, unfavorable response to traditional drug therapy, and limitations in heart transplantation call for new therapeutic options. Stem cell therapy can be promising in children suffering from this disease. The presented case documents that intracoronary injection of autologous bone marrow-derived mesenchymal stem cells in a boy with progressive dilated cardiomyopathy is feasible and safe. Furthermore, it may positively influence functional class, quality of life, and echocardiographic indices of cardiac function.
A Randomized Comparative Study on the Efficacy of Intracoronary Infusion of Autologous Bone Marrow Mononuclear Cells and Mesenchymal Stem Cells in Patients With Dilated Cardiomyopathy. [2017]Stem cell therapy has shown therapeutic benefit in dilated cardiomyopathy (DCM), but doubt remains about the most appropriate stem cell subpopulation. The current study compared the efficacy of intracoronary administration of bone marrow mononuclear cells (BMMC) or mesenchymal stem cells (BMSC) in patients with DCM.Fifty-three patients with DCM and reduced ( 0.05). There were no significant differences between the transplantation groups during follow-up (P > 0.05). There were no differences in MACE among the 3 groups (P = 0.817).Intracoronary bone marrow stem cell transplantation in DCM is safe and effective, while BMSC and BMMC infusion possess comparable effectiveness.
Comparison of allogeneic vs autologous bone marrow–derived mesenchymal stem cells delivered by transendocardial injection in patients with ischemic cardiomyopathy: the POSEIDON randomized trial. [2023]Mesenchymal stem cells (MSCs) are under evaluation as a therapy for ischemic cardiomyopathy (ICM). Both autologous and allogeneic MSC therapies are possible; however, their safety and efficacy have not been compared.
A randomized, double-blind, placebo-controlled, dose-escalation study of intravenous adult human mesenchymal stem cells (prochymal) after acute myocardial infarction. [2022]Our aim was to investigate the safety and efficacy of intravenous allogeneic human mesenchymal stem cells (hMSCs) in patients with myocardial infarction (MI).
Mesenchymal stem cells and cardiac repair: principles and practice. [2021]Mesenchymal stem cells (MSCs) are cluster of differentiation 34 (CD34)-CD45-negative nonhematopoietic progenitors derived typically from the stromal fraction of the bone marrow. These stem cells display multipotent properties with a demonstrable differentiation capacity along multiple mesodermal lineages. In the setting of myocardial injury, preclinical studies indicate benefit of both autologous and allogeneic transplantation in line with a recognized immunotolerant profile. Initial clinical experience supports the value of mesenchymal stem-cell-based therapy in ischemic cardiomyopathy. Experience is however limited to naïve mesenchymal stem cells, with efforts underway to identify optimal means of enhancing the cardiogenic potential of transplanted cells through guided cardiopoiesis with the ultimate aim of achieving standardized therapy of the ischemic myocardium.