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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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment136
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04476901Administration of Allogeneic-MSC in Patients With Non-Ischemic Dilated CardiomyopathyPHASE2 RECRUITING 136May 7, 2021Apr 1, 2027Mar 24, 20264 United States
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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 — 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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