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allogeneic human mesenchymal stem cells

Phase 2

Non-ischemic Dilated Cardiomyopathy | Monoclonal antibody | Cardiovascular |United Therapeutics Corporation|Last Updated: Mar 24, 2026

Target and mechanism

ModalityMonoclonal antibody

Also known as allogeneic human mesenchymal stem cells (hMSCs)

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment136

FDA Designations

No designations recorded

Clinical trial landscape

allogeneic human mesenchymal stem cells · 1 trial · 1 indication

Phase 2 1
NCT04476901Administration of Allogeneic-MSC in Patients With Non-Ischemic Dilated CardiomyopathyNon-ischemic Dilated Cardiomyopathy
RECRUITING136 Analytics
PHASE2RECRUITING
Administration of Allogeneic-MSC in Patients With Non-Ischemic Dilated Cardiomyopathy
Non-ischemic Dilated CardiomyopathyUnlock trial analytics

Study Endpoints

Primary Endpoints

Change in LVEF
Baseline, 12 months

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

Secondary Endpoints

Change in global ventricular strain
Baseline, 12 months
Change in left regional strain
Baseline, 12 months
Left ventricular function concordance
12 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Genotype A administered with placebo GroupPLACEBO_COMPARATORParticipants 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.
Genotype A administered with hMSC GroupEXPERIMENTALParticipants 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.
Genotype B administered with placebo GroupPLACEBO_COMPARATORParticipants whose blood genotyping resulted with Genotype B (variants of uncertain significance) and randomized to the placebo group will receive the placebo intervention.
Genotype B administered with hMSC GroupEXPERIMENTALParticipants whose blood genotyping resulted with Genotype B (variants of uncertain significance) and randomized to the treatment group will receive the hMSC intervention.
Genotype C administered with placebo GroupPLACEBO_COMPARATORParticipants whose blood genotyping resulted with Genotype C (pathogenic or likely pathogenic variants) and randomized to the placebo group will receive the placebo intervention.
Genotype C administered with hMSC GroupEXPERIMENTALParticipants whose blood genotyping resulted with Genotype C (pathogenic or likely pathogenic variants) and randomized to the treatment group will receive the hMSC intervention.

Interventions

NameTypeDescription
allogeneic human mesenchymal stem cells (hMSCs)BIOLOGICALallo-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).
PlaceboOTHERPlacebo 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)
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Eligibility Criteria

Age Range18 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: In order to be eligible to participate in this study, an individual must meet all of the following criteria: 1. Men and women aged 18 to 80 years (inclusive) at the time of signing the informed consent form. 2. Diagnosis of NIDCM with left ventricular ejection fraction ≤45%. 3....

Countries:United States
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Frequently asked questions about allogeneic human mesenchymal stem cells

What is allogeneic human mesenchymal stem cells used for?

Allogeneic human mesenchymal stem cells (hMSCs) are being studied for the treatment of non-ischemic dilated cardiomyopathy, a condition in which the heart muscle becomes weakened and enlarged. The therapy is currently in Phase 2 clinical development and is being evaluated in a randomized, double-blind, placebo-controlled trial.

What does allogeneic human mesenchymal stem cells target?

Allogeneic human mesenchymal stem cells are a cell-based therapy, not a targeted molecular agent. They are intended to modulate the immune response and promote tissue repair in the heart. The exact mechanism of action in non-ischemic dilated cardiomyopathy is under investigation in clinical trials.

Who makes allogeneic human mesenchymal stem cells?

Allogeneic human mesenchymal stem cells are being developed by United Therapeutics Corporation, a biotechnology company traded on the NASDAQ under the ticker symbol UTHR. The company is sponsoring the ongoing Phase 2 clinical trial of this investigational cell therapy.

What phase is allogeneic human mesenchymal stem cells in?

Allogeneic human mesenchymal stem cells are in Phase 2 clinical development. The therapy is investigational and has not been approved by the FDA. It is currently being evaluated in a Phase 2 trial for non-ischemic dilated cardiomyopathy, with the study actively recruiting participants.

What clinical trials is allogeneic human mesenchymal stem cells in?

Allogeneic human mesenchymal stem cells are being studied in one active Phase 2 clinical trial, identified as NCT04476901. This trial is titled 'Administration of Allogeneic-MSC in Patients With Non-Ischemic Dilated Cardiomyopathy' and is enrolling 136 participants in the United States. The study is randomized, double-blind, and placebo-controlled.

Is allogeneic human mesenchymal stem cells the same as allogeneic human mesenchymal stem cells (hMSCs)?

Yes, allogeneic human mesenchymal stem cells are also known as allogeneic human mesenchymal stem cells (hMSCs). The abbreviation hMSCs is commonly used to refer to this cell therapy in clinical and scientific contexts.