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Prochymal

Phase 3

Graft Versus Host Disease | Small molecule | Immunology |Mesoblast Limited|Last Updated: Mar 11, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials3
Total Enrollment463

FDA Designations

No designations recorded

Clinical trial landscape

Prochymal · 8 trials · 10 indications

Phase 3 4Phase 2 4
NCT01233960Evaluation of PROCHYMAL® for Treatment-refractory Moderate-to-severe Crohn's DiseaseCrohn's Disease
COMPLETED73 Analytics
NCT00562497Efficacy and Safety of Prochymal® Infusion in Combination With Corticosteroids for the Treatment of Newly Diagnosed Acute Graft Versus Host Disease (GVHD)Graft Versus Host Disease
COMPLETED192 Analytics
NCT00482092Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-resistant Moderate-to-severe Crohn's DiseaseCrohn's Disease
COMPLETED330 Analytics
NCT00366145Efficacy and Safety of Adult Human Mesenchymal Stem Cells to Treat Steroid Refractory Acute Graft Versus Host Disease (GVHD)Graft Versus Host Disease
COMPLETED260 Analytics
PHASE3COMPLETED
Evaluation of PROCHYMAL® for Treatment-refractory Moderate-to-severe Crohn's Disease
Crohn's DiseaseUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Prochymal® Infusion in Combination With Corticosteroids for the Treatment of Newly Diagnosed Acute Graft Versus Host Disease (GVHD)
Graft Versus Host DiseaseUnlock trial analytics
PHASE3COMPLETED
Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-resistant Moderate-to-severe Crohn's Disease
Crohn's DiseaseUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Adult Human Mesenchymal Stem Cells to Treat Steroid Refractory Acute Graft Versus Host Disease (GVHD)
Graft Versus Host DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Disease remission
180 Days after first infusion in Protocol 603

Crohn's Disease Activity Index (CDAI) at or below 150 and increase in IBDQ

Percentage of Participants with Treatment Success
90 Days

Treatment was considered a success if all of the following conditions were met: Achieved induction of a complete response (CR) within 28 days after first infusion; CR followed by 28 days maintenance of a clinically meaningful response defined as the response that did not require an increase in corticosteroid dose (methylprednisolone doses \>2 milligram/kilogram/day \[mg/kg/d\] or prednisone doses \>2.5 mg/kg/d) for more than 7 consecutive days; Did not require second line/escalation therapy through Study Day 56; and survived 90 study days.

Disease remission (CDAI at or below 150)
28 days
Percentage of Participants achieving Complete Response of Greater Than or Equal to 28 Days Duration
up to 100 Days post first infusion

A complete response was defined as complete resolution of all clinical signs of Graft versus host disease (GVHD)- that had to be maintained for at least 28 consecutive days (durable complete response \[DCR\]) within 100 days post first infusion.

Change from Baseline in Cardiac Magnetic Resonance (CMR) imaging-determined End Systolic Volume (ESV) at Month 3
Baseline, Month 3
C-peptide area under the concentration curve (AUC) response (MMTT)
1 year
Number of Participants with Adverse Events (AEs)
Up to 2 Years
Response by Day 28
Day 28

Responses included complete response (CR), partial response (PR), failure to respond. CR is defined as a complete resolution of graft-versus-host disease (GVHD). PR is defined as improvement in at least one organ by at least one full stage in the absence of progression in any other organ, or resolution of GVHD in at least one organ with a need for additional treatment because of abnormalities persisting in another organ. Failure to respond is defined as progression of GVHD.

Secondary Endpoints

Disease Improvement
180 Days after first infusion in Protocol 603
Improvement in Quality of Life (IBDQ)
180 Days after first infusion in Protocol 603
Number of Adverse events as a measure of safety
180 Days after first infusion in Protocol 603
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Prochymal®EXPERIMENTALInfusions of Prochymal on days 42-45, 84-87, and 126-129 after first infusion in Protocol 603. Each infusion of PROCHYMAL (remestemcel-L) will contain 200 million cells.
PlaceboPLACEBO_COMPARATORParticipants will receive 6 infusions of placebo-matching Prochymal® intravenously (IV) during the first 4 weeks of the study. The first infusion will be administered within 72 hours of the start of systemic corticosteroid therapy. Participants will receive 4 infusions during the first 2 weeks (twice weekly at least 3 days apart), followed by 2 infusions administered once weekly over the subsequent 2 weeks up to Day 28.
Prochymal® 2x10^6 hMSC/kgACTIVE_COMPARATORParticipants will receive 6 infusions of Prochymal® 2x10\^6 human mesenchymal stem cells (hMSC)/kg IV during the first 4 weeks of the study. The first infusion will be administered within 72 hours of the start of systemic corticosteroid therapy. Participants will receive 4 infusions during the first 2 weeks (twice weekly at least 3 days apart), followed by 2 infusions administered once weekly over the subsequent 2 weeks up to Day 28.
Prochymal® - Low doseACTIVE_COMPARATORParticipants will receive a total dose of Prochymal® 600 x 10\^6 cells, IV infusion, on four days, once daily.
Prochymal® - High doseACTIVE_COMPARATORParticipants will receive a total dose of Prochymal® 1200 x 10\^6 cells, IV infusion, on four days, once daily.
ProchymalEXPERIMENTALPROCHYMAL®
Prochymal™EXPERIMENTALParticipants received Prochymal™ a total of 400×10\^6 cells, intravenous (IV) infusions on Days 0, 30, 60, and 90.

Interventions

NameTypeDescription
Prochymal®DRUGPROCHYMAL will be administered IV in a total volume of 300 ml (200 million cells) at a rate of 4-6 ml/minute. Treatments will be administered on Days 42-45, Days 84-87, and Days 126-129 following first infusion in Protocol 603.
PlaceboOTHERPlacebo-matching Prochymal® intravenous infusion.
CorticosteroidOTHERAdministration will be intravenously as prescribed by the caregiver.
Standard of Care for GVHDDRUGInstitutionally defined standard of care (e.g., maintenance of steroid treatment and the addition of a second-line therapy)
Prochymal™DRUGIV infusion of ex- vivo cultured adult human mesenchymal stem cells.
ProchymalDRUGIntravenous infusion of ex-vivo cultured adult human mesenchymal stem cells
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Eligibility Criteria

Age Range18 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites20

Inclusion Criteria: * Participant must have qualified for, enrolled in, and provided written informed consent form (ICF) and authorization for use and disclosure of protected health information (PHI) for Protocol 603 after the August 3, 2010. * Participant successfully completed all screening asses...

Countries:United StatesAustraliaNew ZealandCanadaItalySwitzerlandUnited Kingdom
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Competitive Landscape -Graft-versus-Host Disease 27 trials (matched to "Graft Versus Host Disease")

Recent Changes (Last 90 Days)

MEDIUMMay 21, 2026NCT00690066TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT00683722TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT01233960TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT00877903TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT00482092TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT00690066TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT00683722TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT00877903TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT01233960TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT00482092TRIAL_REMOVED: changed

Frequently asked questions about Prochymal

What is PROCHYMAL used for?

PROCHYMAL is an investigational stem cell therapy being studied for Crohn's Disease, Graft Versus Host Disease, Type 1 Diabetes Mellitus, Myocardial Infarction, Pulmonary Disease, and Chronic Obstructive Pulmonary Disease. It is developed by Mesoblast Limited (MESO) and is currently in Phase 2 clinical development.

What does PROCHYMAL target?

PROCHYMAL is a small molecule therapeutic that targets inflammatory and immune-mediated pathways. It is being evaluated for its potential to modulate the immune response in conditions like Crohn's Disease and Graft Versus Host Disease. The specific molecular target has not been disclosed.

Who makes PROCHYMAL?

PROCHYMAL is developed by Mesoblast Limited, a biopharmaceutical company traded under the ticker MESO. Mesoblast is conducting clinical trials to evaluate the safety and efficacy of PROCHYMAL across multiple indications, including Crohn's Disease and Type 1 Diabetes Mellitus.

What phase is PROCHYMAL in?

PROCHYMAL is in Phase 2 clinical development. It is an investigational therapy and has not been approved by regulatory authorities. Mesoblast has completed several clinical trials, including Phase 2 and Phase 3 studies, but the drug remains under investigation.

What clinical trials is PROCHYMAL in?

PROCHYMAL has been studied in several completed clinical trials, including NCT00482092 for Crohn's Disease, NCT00690066 for Type 1 Diabetes Mellitus, and NCT00877903 for Myocardial Infarction. These trials were randomized, double-blind, and placebo-controlled, with a total enrollment of 463 participants.

Is PROCHYMAL the same as remestemcel-L?

PROCHYMAL is also known as remestemcel-L, a name used in some clinical contexts. It is an investigational stem cell therapy developed by Mesoblast Limited. The drug has been evaluated in clinical trials for various conditions, including Crohn's Disease and Myocardial Infarction.