Back to resultsSafety and Efficacy of Prochymal® for the Salvage of Treatment-Refractory Acute GVHD Patients
Primary Purpose
Graft Versus Host Disease
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Prochymal®
Sponsored by
About this trial
This is an interventional treatment trial for Graft Versus Host Disease focused on measuring GVHD, Graft vs Host Disease, Graft versus Host Disease
Eligibility Criteria
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. Participants 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.
Sites / Locations
- Duke University
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Prochymal®
Arm Description
Outcomes
Primary Outcome Measures
Response by Day 28
Secondary Outcome Measures
Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at day 1
Best stage of each involved organ by Day 28
Time to improvement or resolution of GVHD in one or more organs
Adverse events
Infusional toxicity
Overall relapse of underlying disease
Overall survival
Formation of ectopic tissue foci
Incidence of infection
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00284986
Brief Title
Safety and Efficacy of Prochymal® for the Salvage of Treatment-Refractory Acute GVHD Patients
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
January 2022
Overall Recruitment Status
Completed
Study Start Date
November 18, 2005 (Actual)
Primary Completion Date
April 25, 2006 (Actual)
Study Completion Date
February 8, 2007 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mesoblast, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
Detailed Description
Allogeneic hematopoetic stem cell transplantation (HCT) is used in the treatment of a variety of hematological, myeloproliferative and lymphoproliferative disorders, and malignancies involving solid tumors. Patients receiving HCT can develop a life-threatening condition called graft versus host disease (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 patients are refractory to current standard of care treatment. For treatment-refractory patients 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Graft Versus Host Disease
Keywords
GVHD, Graft vs Host Disease, Graft versus Host Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Prochymal®
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Prochymal®
Other Intervention Name(s)
Remestemcel-L
Intervention Description
Intravenous infusion of ex-vivo cultured adult human mesenchymal stem cells
Primary Outcome Measure Information:
Title
Response by Day 28
Time Frame
Day 28
Secondary Outcome Measure Information:
Title
Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at day 1
Time Frame
Day 1
Title
Best stage of each involved organ by Day 28
Time Frame
Day 28
Title
Time to improvement or resolution of GVHD in one or more organs
Time Frame
Up to approximately 12 months
Title
Adverse events
Time Frame
Up to approximately 12 months
Title
Infusional toxicity
Time Frame
Up to approximately 12 months
Title
Overall relapse of underlying disease
Time Frame
Up to approximately 12 months
Title
Overall survival
Time Frame
Up to approximately 12 months
Title
Formation of ectopic tissue foci
Time Frame
Up to approximately 12 months
Title
Incidence of infection
Time Frame
Up to approximately 12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Participants 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Christopher James, PA
Organizational Affiliation
Mesoblast, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Duke University
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27708
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Safety and Efficacy of Prochymal® for the Salvage of Treatment-Refractory Acute GVHD Patients
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