Prochymal Expanded Access Treatment for Pediatric Patients Who Have Failed Steroids for Acute GVHD

March 5, 2020 updated by: Mesoblast, Inc.

Expanded Access of Prochymal (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) Infusion for the Treatment of Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD

This protocol allows for the treatment of pediatric patients, male and female, between the ages of 2 months and 17 years. Patients must have failed steroid treatment for Grade B-D acute GVHD.

Study Overview

Status

No longer available

Conditions

Intervention / Treatment

Detailed Description

For the treatment of pediatric patients who have failed to respond to steroid treatment for acute graft-versus-host disease (GVHD). Failing steroid treatment for acute GVHD is defined as any Grade B-D (IBMTR grading) of acute GVHD that is not improving after at least 3 days of methylprednisolone (≥ 1 mg/kg/day) or equivalent.

Patients will be treated with Prochymal twice per week at a dose of 2 x 10^6 hematopoietic stem cells (hMSC)/kg (actual body weight) for each of 4 consecutive weeks. Infusions will be administered at least 3 days apart.

Study Type

Expanded Access

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3B 6A8
        • Alberta Children's Hospital
    • Quebec
      • Montreal, Quebec, Canada, H3T1C5
        • CHU Sainte-Justine
      • Montreal, Quebec, Canada, H3H1P3
        • Montreal Children's Hospital
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Phoenix Children's Hospital
    • California
      • Orange, California, United States, 92868
        • Children's Hospital of Orange County
      • San Francisco, California, United States, 94143
        • UCSF
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National
    • Florida
      • Jacksonville, Florida, United States, 32207
        • Nemours Children's Clinic
      • Miami, Florida, United States, 33136
        • University of Miami
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Childrens Memorial
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • James Witcomb Riley Hosptial for Children
    • Louisiana
      • New Orleans, Louisiana, United States, 70118
        • LSU Health Science Center/Children's Hospital
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Wayne State University/Childrens' Hospital
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Children's Mercy Hospital
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack University Medical Center
    • New York
      • New York, New York, United States, 10065
        • Memorial Sloan-Kettering Cancer Center
      • New York, New York, United States, 10032
        • Columbia Medical Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28232
        • Levine Children's Hospital
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Doernbecher Children's Hospital (OHSU)
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt Children's Hospital
    • Texas
      • Houston, Texas, United States, 77030
        • Texas Childrens Hospital
      • San Antonio, Texas, United States, 78229
        • Methodist Childrens Hospital of South Texas
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Massey Cancer Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 months to 17 years (Child)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

Inclusion criteria:

  • Patients must sign an informed consent form (ICF) before any protocol-related procedures, including any pre-treatment procedures, are performed.
  • Since patients being treated with Prochymal® under this protocol are under 18, a parental signature of informed consent will be required.
  • Patients must be 2 months to 17 years of age, inclusive.
  • Patients must have failed to respond to steroid treatment for Grades B-D acute GVHD • Failure to respond to steroid treatment for acute GVHD is defined as any Grade B-D acute GVHD that is not improving after at least 3 days of methylprednisolone (≥1 milligram per kilogram per day [mg/kg/day]) or equivalent.

Exclusion criteria:

  • Patient must not have a known allergy to bovine or porcine products.
  • Patient must not have received a transplant for a solid tumor disease.
  • Patients must not have evidence of a pulmonary infiltrate or diffuse alveolar hemorrhage and must be unlikely to require more than 2 liters (L) of oxygen via face mask or an estimated fraction of inspired oxygen (FiO2) of 28% via other delivery methods in order to sustain an O2 saturation of 92% during the next 3 days.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mesoblast, Inc.

Investigators

  • Study Director: Mahboob Rahman, Mesoblast, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

September 23, 2008

First Submitted That Met QC Criteria

September 23, 2008

First Posted (Estimate)

September 25, 2008

Study Record Updates

Last Update Posted (Actual)

March 9, 2020

Last Update Submitted That Met QC Criteria

March 5, 2020

Last Verified

March 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 275

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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