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Prochymal adult human mesenchymal stem cells

Phase 3

Crohn's Disease | Small molecule | Immunology |Mesoblast Limited|Last Updated: Mar 11, 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-BlindCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment108
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00543374Extended Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-Resistant Moderate-to-Severe Crohn's DiseasePHASE3 COMPLETED 98Sep 17, 2007Apr 28, 2011Mar 11, 202658 United States, Canada
NCT00294112Prochymal™ Adult Human Mesenchymal Stem Cells for Treatment of Moderate-to-severe Crohn's DiseasePHASE2 COMPLETED 10Mar 13, 2006Jul 21, 2006Apr 2, 20204 United States
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Study Endpoints
Primary Endpoints
Duration of clinical benefit (Crohn's disease activity index)
6 months
Re-induction of clinical benefit (Crohn's disease activity index)
6 months
Number of Participants with Reduction in Crohn's Disease Activity Index (CDAI) of at Least 100 Points
28 days

The CDAI is a composite index of 8 disease variables (stool frequency, severity of abdominal pain, degree of general well-being, presence or absence of extra-intestinal manifestations or fistula, use or non-use of antidiarrheal agents, presence or absence of an abdominal mass, hematocrit, and body weight). Scores range from approximately 0 to 600, with a higher score indicating more disease activity.

Secondary Endpoints
Improvement in quality of life (Inflammatory Bowel Disease Questionnaire [IBDQ] instrument)
6 months
Number of Participants with Reduction in CDAI of at Least 70 points
28 days
Improvement as Assessed by the Inflammatory Bowel Disease Questionnaire (IBDQ)
28 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PlaceboPLACEBO_COMPARATORPlacebo
PROCHYMAL Low doseACTIVE_COMPARATORLow dose (total of 600 million cells).
PROCHYMAL High doseACTIVE_COMPARATORHigh dose (total of 1200 cells).
High doseEXPERIMENTALHigh dose (8 million cells per kg of body weight)
Low doseEXPERIMENTALLow dose: 2 million cells per kg body weight
Interventions
NameTypeDescription
PlaceboDRUG -
PROCHYMAL adult human mesenchymal stem cellsDRUGintravenous infusion four times over two weeks; possibly repeated once
Prochymal™ adult human mesenchymal stem cellsDRUGCells in plasmalyte and containing dimethylsulfoxide
adult human mesenchymal stem cellsDRUGtwo infusions, one week apart, each comprising adult human mesenchymal stem cells
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites58

Inclusion Criteria: * Participation in Osiris Protocol 603, with drop of at least 100 points in CDAI at Day 28 Exclusion Criteria: * Substance abuse * Failure to receive full dose of all interventions in Protocol 603

Countries:United StatesCanada
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Recent Changes (Last 90 Days)
MEDIUMMay 21, 2026NCT00543374TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT00543374TRIAL_REMOVED: changed