NCT04476901

Brief Summary

The purpose of this study is to evaluate the safety and effectiveness of an experimental drug called human allogeneic mesenchymal stem cell therapy.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
136

participants targeted

Target at P75+ for phase_2

Timeline
11mo left

Started May 2021

Longer than P75 for phase_2

Geographic Reach
1 country

4 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress85%
May 2021Apr 2027

First Submitted

Initial submission to the registry

July 15, 2020

Completed
5 days until next milestone

First Posted

Study publicly available on registry

July 20, 2020

Completed
10 months until next milestone

Study Start

First participant enrolled

May 7, 2021

Completed
5.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2027

Last Updated

March 24, 2026

Status Verified

March 1, 2026

Enrollment Period

5.9 years

First QC Date

July 15, 2020

Last Update Submit

March 20, 2026

Conditions

Non-ischemic Dilated Cardiomyopathy

Keywords

Allogeneic mesenchymal stem cellsBone marrow-derived mesenchymal stem cells

Outcome Measures

Primary Outcomes (1)

  • Change in LVEF

    Change in Left Ventricular Ejection Fraction (LVEF) as assessed via cardiac Magnetic Resonance Imaging (MRI)

    Baseline, 12 months

Secondary Outcomes (14)

  • Change in global ventricular strain

    Baseline, 12 months

  • Change in left regional strain

    Baseline, 12 months

  • Left ventricular function concordance

    12 months

  • Change in LVEDVI

    Baseline, 12 months

  • Change in LVESVI

    Baseline, 12 months

  • +9 more secondary outcomes

Study Arms (6)

Genotype A administered with placebo Group

PLACEBO COMPARATOR

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.

Other: Placebo

Genotype A administered with hMSC Group

EXPERIMENTAL

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.

Biological: allogeneic human mesenchymal stem cells (hMSCs)

Genotype B administered with placebo Group

PLACEBO COMPARATOR

Participants whose blood genotyping resulted with Genotype B (variants of uncertain significance) and randomized to the placebo group will receive the placebo intervention.

Other: Placebo

Genotype B administered with hMSC Group

EXPERIMENTAL

Participants whose blood genotyping resulted with Genotype B (variants of uncertain significance) and randomized to the treatment group will receive the hMSC intervention.

Biological: allogeneic human mesenchymal stem cells (hMSCs)

Genotype C administered with placebo Group

PLACEBO COMPARATOR

Participants whose blood genotyping resulted with Genotype C (pathogenic or likely pathogenic variants) and randomized to the placebo group will receive the placebo intervention.

Other: Placebo

Genotype C administered with hMSC Group

EXPERIMENTAL

Participants whose blood genotyping resulted with Genotype C (pathogenic or likely pathogenic variants) and randomized to the treatment group will receive the hMSC intervention.

Biological: allogeneic human mesenchymal stem cells (hMSCs)

Interventions

PlaceboOTHER

Placebo will be administered as injections of plasmalyte A supplemented with 1% of 25% human serum albumin (HSA). 0.5 ml/ injection x 10 injections or an intravenous placebo infusion of Cell-free PlasmaLyte-A medium supplemented with 1% of 25% human serum albumin (HSA)

Genotype A administered with placebo GroupGenotype B administered with placebo GroupGenotype C administered with placebo Group
allogeneic human mesenchymal stem cells (hMSCs)BIOLOGICAL

allo-hMSCs, 16-20 million cells/ml delivered at a dose of 0.5 ml/ injection x 10 injections for a total of 80-100 million allo-hMSCs or a single administration of intravenous allogeneic hMSCs (100 million).

Genotype A administered with hMSC GroupGenotype B administered with hMSC GroupGenotype C administered with hMSC Group

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • In order to be eligible to participate in this study, an individual must meet all of the following criteria:
  • Men and women aged 18 to 80 years (inclusive) at the time of signing the informed consent form.
  • Diagnosis of NIDCM with left ventricular ejection fraction ≤45%.
  • Appropriate guideline-directed optimal medical therapy for non-ischemic cardiomyopathy. At a minimum, subjects must be on beta blockers and angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) or Angiotensin Receptor Neprilysin Inhibitors (ARNI) or have appropriate medical indication precluding use of one or both of these agents. Subjects must be on a stable regimen for at least 30 days prior to the procedure. Dose titration is allowed.
  • Be a candidate for cardiac catheterization\*
  • Be willing to undergo DNA test.

You may not qualify if:

  • An individual who meets any of the following criteria will be excluded from participation in this study:
  • Be eligible for or require standard-of-care surgical or percutaneous intervention for the treatment of non-ischemic dilated cardiomyopathy
  • Clinical manifestation of coronary artery disease (CAD) (e.g., chest pain and concomitant clinical findings such as electrocardiogram changes suggestive of coronary ischemia, myocardial infarction) or evidence of endocardial or transmural scar on cardiac MRI suggestive of undiagnosed CAD or history of percutaneous coronary intervention (PCI) or coronary artery bypass surgery (CABG). Be indicated for or require coronary artery revascularization
  • Documented presence of epicardial stenosis of 70% or greater in one or more major epicardial coronary arteries
  • Valvular heart disease including 1) aortic valve prosthesis, mechanical mitral valve, and mitral valve clip; 2) severe aortic valve insufficiency/regurgitation within 12 months of consent\*
  • Aortic stenosis with valve area ≤ 1.5cm2\*
  • Cardiomyopathy due to acute Post-partum (within 6 months), Non-compaction\*, or Hypertrophic\* cardiomyopathy
  • Cardiomyopathy due to known toxin (e.g amyloid) Note: anthracycline induced cardiomyopathy will be allowed
  • QTc interval \> 550 ms on baseline electrocardiogram (ECG) (note: QTc interval is the interval between the start of the Q wave and the end of the T wave in the heart's electrical cycle)
  • Automated Implantable Cardioverter Defibrillator (AICD) appropriate firing or anti tachycardia pacing for ventricular tachycardia or ventricular fibrillation within 30 days prior to consent
  • Have an estimated baseline glomerular filtration rate below the clinical site's institutional cutoff
  • A hematologic abnormality during baseline testing as evidenced by hemoglobin \< 9 g/dl; hematocrit \< 30%; absolute neutrophil count \< 2,000 or total WBC count more than 2 times upper limit of normal; or platelet values \< 100,000/ul
  • Have liver dysfunction, as evidenced by enzymes Aspartate Transaminase Enzyme (AST) and Alanine Aminotransferase Enzyme (ALT) greater than three times the ULN
  • Have a bleeding diathesis or coagulopathy (International Normalised Ratio (INR) \> 1.5), cannot be withdrawn from anticoagulation therapy, or will refuse blood transfusions
  • Be a solid organ transplant recipient. This does not include prior cell based therapy (\>12 months prior to enrollment), bone, skin, ligament, tendon or corneal grafting.
  • +25 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Stanford University

Stanford, California, 94304, United States

RECRUITING

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

RECRUITING

University of Louisville

Louisville, Kentucky, 40202, United States

RECRUITING

Texas Heart Institute

Houston, Texas, 77030, United States

RECRUITING

Related Links

Study Officials

  • Joshua Hare, MD

    University of Miami

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Shelly L Sayre, MPH

CONTACT

713-500-9529Shelly.L.Sayre@uth.tmc.edu

Lina Caceres

CONTACT

305-243-5399lvc25@med.miami.edu

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

July 15, 2020

First Posted

July 20, 2020

Study Start

May 7, 2021

Primary Completion (Estimated)

April 1, 2027

Study Completion (Estimated)

April 1, 2027

Last Updated

March 24, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

Locations

US(4)