Safety and Efficacy of Prochymal for the Salvage of Treatment-Refractory Acute GVHD Participants

A Phase II Open Label Study to Evaluate the Safety and Efficacy of Prochymal (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) Infusion for the Salvage of Treatment-Refractory Acute GVHD Patients

This study is designed to evaluate the safety and efficacy of Prochymal® (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) in participants experiencing treatment-refractory acute GVHD, Grades III-IV, that is refractory to standard first-line therapies and at least one second-line therapy.

Study Overview

• Status: Completed
• Conditions: Graft Versus Host Disease
• Intervention / Treatment: Drug: Prochymal

Detailed Description

Allogeneic HSCT is used in the treatment of a variety of hematological, myeloproliferative and lymphoproliferative disorders, and malignancies involving solid tumors. Participants receiving HSCT can develop a life-threatening condition called GVHD. GVHD occurs when donor T cells from the donor bone marrow recognize host cells as "foreign" and initiate an inflammatory immunological response. The standard of care for treatment of acute GVHD consists of intravenous delivery of methylprednisolone starting on Day 1 and continuation of either cyclosporine or tacrolimus. This regimen of steroids and immunosuppressive drugs may relieve symptoms of GVHD, but some participants are refractory to current standard of care treatment. For treatment-refractory participants with grades III-IV GVHD mortality is approximately 80%. A therapy that could effectively suppress the immunological response from GVHD and help repair the damaged tissue could significantly decrease the mortality rate from this disease.

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Durham, North Carolina, United States, 27708
      • Duke University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 70 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participants must be 6 months to 70 years of age inclusive.
• If female and of childbearing age, participants must be non-pregnant, not breast-feeding, and use adequate contraception. Male participants must use adequate contraception.
• Participants must have Grade III-IV acute GVHD that has failed to respond to standard first and at least one second-line therapy. Biopsy for confirmation of both skin and gastrointestinal GVHD is not mandatory, but is recommended when feasible. Enrollment should not be delayed awaiting biopsy results.
• Participants must have minimal renal function as defined by: Calculated creatinine clearance (CrCl) of > 30 milliliters/minute (mL/min) using the Cockcroft-Gault equation.
• Participant must provide written informed consent and authorization for use and disclosure of protected health information (PHI).

Exclusion Criteria:

• Participant has uncontrolled alcohol or substance abuse within 6 months of treatment.
• Participant has any underlying or current medical or psychiatric condition that, in the opinion of the Investigator, would interfere with the evaluation of the participant (e.g., uncontrolled infection, right heart failure, pulmonary hypertension, etc.).
• Participant has a clinically significant, unstable arrhythmia.
• Participant has a known allergy to bovine or porcine products.
• Participant is unwilling to sign consent form for the long-term follow-up study, protocol 271.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PROCHYMAL™	Drug: Prochymal; Intravenous infusion of ex-vivo cultured adult human mesenchymal stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Response by 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.	Day 28

Secondary Outcome Measures

Outcome Measure	Time Frame
Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at day 1	Day 1
Best stage of each involved organ by Day 28	Day 28
Time to improvement or resolution of GVHD in one or more organs	Up to approximately 12 months
Adverse events	Up to approximately 12 months
Infusional toxicity	Up to approximately 12 months
Overall relapse of underlying disease	Up to approximately 12 months
Overall survival	Up to approximately 12 months
Formation of ectopic tissue foci	Up to approximately 12 months
Incidence of infection	Up to approximately 12 months

Collaborators and Investigators

• Sponsor: Mesoblast, Inc.
• Investigators: Study Director: Christopher James, PA, Mesoblast, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): November 18, 2005
• Primary Completion (Actual): April 25, 2006
• Study Completion (Actual): February 8, 2007

Study Registration Dates

• First Submitted: January 31, 2006
• First Submitted That Met QC Criteria: January 31, 2006
• First Posted (Estimated): February 1, 2006

Study Record Updates

• Last Update Posted (Estimated): October 30, 2025
• Last Update Submitted That Met QC Criteria: October 29, 2025
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Keywords: GVHD; Graft vs Host Disease; Graft versus Host Disease
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease; Anti-Infective Agents; Antiviral Agents; Anti-Inflammatory Agents; remestemcel-l
• Other Study ID Numbers: Osiris 270-271

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No