Stem Cell Therapy for Dilated Cardiomyopathy

(DCMII Trial)

Recruiting in Palo Alto (17 mi)

+2 other locations

Overseen by Joshua 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?

Research shows that mesenchymal stem cells (MSCs) can help repair damaged heart tissue by reducing inflammation and promoting new blood vessel growth. Studies have demonstrated their potential to improve heart function in conditions similar to dilated cardiomyopathy. ^{1 2 3 4 5}

Is stem cell therapy safe for humans?

Safety of allogeneic mesenchymal stem cells (hMSCs) has been established in Phase I and II trials, showing they are generally safe in humans, although specific data for dilated cardiomyopathy is limited. ^{1 2 6 7 8}

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. ^{1 2 4 5 6}

Research Team

Joshua Hare, MD

Principal Investigator

University of Miami

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.

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.

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 Overview The 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

Group I: Genotype C administered with hMSC Group Experimental Treatment 1 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 Group Experimental Treatment 1 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 Group Experimental Treatment 1 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 Group Placebo Group 1 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 Group Placebo Group 1 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 Group Placebo Group 1 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 Japan, India, Japan for the following indications:

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 Nearby Select from list below to view details:

Texas Heart Institute Houston, TX

Stanford University Stanford, CA

University of Miami Miller School of Medicine Miami, FL

Who Is Running the Clinical Trial?

Joshua M Hare

Lead Sponsor

Trials

17

Patients Recruited

430+

United States Department of Defense

Collaborator

Trials

940

Patients Recruited

339,000+

The University of Texas Health Science Center, Houston

Collaborator

Trials

974

Patients Recruited

361,000+

Findings from Research

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placental mesenchymal and cord blood stem cell therapy for dilated cardiomyopathy. Ichim, TE., Solano, F., Brenes, R., et al.[2019]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized Comparison of Allogeneic Versus Autologous Mesenchymal Stem Cells for Nonischemic Dilated Cardiomyopathy: POSEIDON-DCM Trial. Hare, JM., DiFede, DL., Rieger, AC., et al.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cell therapy for cardiac repair. Boyle, AJ., McNiece, IK., Hare, JM.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intracoronary administration of autologous mesenchymal stem cells in a critically ill patient with dilated cardiomyopathy. Zeinaloo, A., Zanjani, KS., Bagheri, MM., et al.[2016]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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. Xiao, W., Guo, S., Gao, C., et al.[2017]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of allogeneic vs autologous bone marrow–derived mesenchymal stem cells delivered by transendocardial injection in patients with ischemic cardiomyopathy: the POSEIDON randomized trial. Hare, JM., Fishman, JE., Gerstenblith, G., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized, double-blind, placebo-controlled, dose-escalation study of intravenous adult human mesenchymal stem cells (prochymal) after acute myocardial infarction. Hare, JM., Traverse, JH., Henry, TD., et al.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cells and cardiac repair: principles and practice. Bartunek, J., Behfar, A., Vanderheyden, M., et al.[2021]

References

1. Netherlands pubmed.ncbi.nlm.nih.gov

Placental mesenchymal and cord blood stem cell therapy for dilated cardiomyopathy. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Randomized Comparison of Allogeneic Versus Autologous Mesenchymal Stem Cells for Nonischemic Dilated Cardiomyopathy: POSEIDON-DCM Trial. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cell therapy for cardiac repair. [2022]

4. Denmark pubmed.ncbi.nlm.nih.gov

Intracoronary administration of autologous mesenchymal stem cells in a critically ill patient with dilated cardiomyopathy. [2016]

5. Japan pubmed.ncbi.nlm.nih.gov

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]

6. United States pubmed.ncbi.nlm.nih.gov

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]

7. United States pubmed.ncbi.nlm.nih.gov

A randomized, double-blind, placebo-controlled, dose-escalation study of intravenous adult human mesenchymal stem cells (prochymal) after acute myocardial infarction. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cells and cardiac repair: principles and practice. [2021]