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Prochymal - 2 Million cells/kg

Phase 2

Graft Vs Host Disease | Small molecule | Other |Mesoblast Limited|Last Updated: Jan 31, 2022

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
RandomizedACTIVE_CONTROLLEDBiomarker
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00136903Safety and Efficacy Study of Adult Human Mesenchymal Stem Cells to Treat Acute Graft Versus Host Disease (GVHD)PHASE2 COMPLETED 32Apr 27, 2005Jul 14, 2008Jan 31, 20229 United States
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Study Endpoints
Primary Endpoints
Protocol 260 - Response by Day 28, also called Overall Response (OR). OR includes complete response (CR) and partial response (PR)
28 Days
Protocol 261-The incidence rate of different adverse events among participants treated with either dose of Prochymal® in the preceding study (Protocol No. 260).
2 Years
Secondary Endpoints
Protocol 260 - Partial Response or Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at Day 1,
28 Days
Protocol 260 - Time to best response of GVHD
28 Days
Protocol 260 - Time to improvement of GVHD in one or more organs
28 Days
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Prochymal® - 2 Million cells/kgACTIVE_COMPARATORParticipants will receive Prochymal® consisting of 2 million hMSCs/kg actual body weight, intravenously (IV) on Days 1 and 4 along with daily standard of care which includes methylprednisolone 2 milligrams (mg)/kg IV or prednisone 2.5 mg/kg orally. Participants will also continue cyclosporine, tacrolimus, and/or mycophenolate mofetil (MMF) at full therapeutic doses.
Prochymal® - 8 Million cells/kgACTIVE_COMPARATORParticipants will receive Prochymal® consisting of 8 million hMSCs/kg actual body weight IV on Days 1 and 4 along with daily standard of care which includes methylprednisolone 2 mg/kg IV or prednisone 2.5 mg/kg orally. Participants will also continue cyclosporine, tacrolimus, and/or mycophenolate mofetil (MMF) at full therapeutic doses.
Interventions
NameTypeDescription
Prochymal® - 2 Million cells/kgDRUG2 million hMSCs/kg actual body weight, IV on study Days 1 and 4
Prochymal®- 8 Million cells/kgDRUG8 million hMSCs/kg actual body weight IV on study Days 1 and 4
MethylprednisoloneDRUGMethylprednisolone 2 mg/kg administered intravenously.
PrednisoneDRUGPrednisone 2.5 mg/kg administered orally.
CyclosporineDRUGAdministered as prescribed by the caregiver.
TacrolimusDRUGAdministered as prescribed by the caregiver.
Mycophenolate MofetilDRUGAdministered as prescribed by the caregiver.
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites9

Protocol 260 Inclusion Criteria: * Participant must be 18 to 70 years of age inclusive. * If female and of child-bearing age, participant must be non-pregnant, not breast feeding, and use adequate contraception. Males must use adequate contraception. * Participant must have newly diagnosed, Grade I...

Countries:United States
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