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

Sponsor
Mesoblast, Inc. (Industry)
Overall Status
Completed
CT.gov ID
NCT00284986
Collaborator
(none)
11
Enrollment
1
Location
1
Arm
14.7
Actual Duration (Months)
0.7
Patients Per Site Per Month

Study Details

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.

Condition or Disease Intervention/Treatment Phase
Phase 2

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.

Study Design

Study Type:
Interventional
Actual Enrollment :
11 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
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
Actual Study Start Date :
Nov 18, 2005
Actual Primary Completion Date :
Apr 25, 2006
Actual Study Completion Date :
Feb 8, 2007

Arms and Interventions

Arm Intervention/Treatment
Experimental: Prochymal®

Drug: Prochymal®
Intravenous infusion of ex-vivo cultured adult human mesenchymal stem cells
Other Names:
  • Remestemcel-L

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Response by Day 28 [Day 28]

    Secondary Outcome Measures

    1. Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at day 1 [Day 1]

    2. Best stage of each involved organ by Day 28 [Day 28]

    3. Time to improvement or resolution of GVHD in one or more organs [Up to approximately 12 months]

    4. Adverse events [Up to approximately 12 months]

    5. Infusional toxicity [Up to approximately 12 months]

    6. Overall relapse of underlying disease [Up to approximately 12 months]

    7. Overall survival [Up to approximately 12 months]

    8. Formation of ectopic tissue foci [Up to approximately 12 months]

    9. Incidence of infection [Up to approximately 12 months]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Months to 70 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Duke University Durham North Carolina United States 27708

    Sponsors and Collaborators

    • Mesoblast, Inc.

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Mesoblast, Inc.
    ClinicalTrials.gov Identifier:
    NCT00284986
    Other Study ID Numbers:
    • Osiris 270-271
    First Posted:
    Feb 1, 2006
    Last Update Posted:
    Jan 31, 2022
    Last Verified:
    Jan 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Mesoblast, Inc.
    GVHD
    Graft vs Host Disease
    Graft versus Host Disease
    Additional relevant MeSH terms:
    Graft vs Host Disease
    Remestemcel-l

    Study Results

    No Results Posted as of Jan 31, 2022