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Lomecel-B medicinal signaling cells

Phase 2

Hypoplastic Left Heart Syndrome | Monoclonal antibody | Other |Longeveron Inc.|Last Updated: Jun 25, 2025

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-BlindNO_TREATMENT_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment40
FDA Designations
FAST_TRACKRMATORPHAN_DRUGRARE_PEDIATRIC_DISEASE
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04925024Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS): A Phase IIb Clinical Trial.PHASE2 ACTIVE NOT_RECRUITING 40Jun 25, 2021Aug 31, 2026Jun 25, 202510 United States
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Study Endpoints
Primary Endpoints
Change in right ventricular ejection fraction (RVEF)
Baseline, 12 Months

Efficacy will be reported as change in right ventricular ejection fraction (RVEF) assessed as a percentage and will be measured via cardiac magnetic resonance (CMR) imaging.

Secondary Endpoints
Change in right ventricular ejection fraction (RVEF)
Baseline, 6 Months
Change in right ventricular mass index at diastole
Baseline, 12 Months
Change in right ventricular end-diastolic volume index (RVEDVI)
Baseline, 12 Months
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Lomecel B GroupEXPERIMENTALParticipants randomized to receive Lomecel-B injections during their Stage II palliation.
No Study Intervention Control GroupNO_INTERVENTIONParticipants randomized to receive no study intervention during their Stage II palliation.
Interventions
NameTypeDescription
Lomecel-B medicinal signaling cellsBIOLOGICALA single administration of Lomecel-B will be performed via 6-10 intramyocardial injections into the right ventricle during the participant's standard of care stage II palliation. Dosing is based on body weight. Each patient will be given 2.5 x 10\^5 cells per kg of body weight. The entire dose of the cells will be roughly 600 microliters.
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Eligibility Criteria
Age RangeN/A — 12 Months
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion: All participants must have HLHS (includes all types) requiring Stage II palliation (Glenn or Hemi-Fontan operation). Exclusion: 1. Requirement for ongoing mechanical circulatory support immediately prior to Stage II palliation within 5 days 2. Need for concomitant surgery for aortic co...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04925024primaryCompletionDate: changed
LOWMay 24, 2026NCT04925024studyFirstPostDate: changed