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

Primary Purpose

Graft vs Host Disease, Graft-Versus-Host Disease

Status
No longer available
Phase
Locations
International
Study Type
Expanded Access
Intervention
remestemcel-L
Sponsored by
Mesoblast, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Graft vs Host Disease focused on measuring GVHD, Prochymal, Graft vs Host Disease

Eligibility Criteria

2 Months - 17 Years (Child)All Sexes

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.

Sites / Locations

  • Phoenix Children's Hospital
  • Children's Hospital of Orange County
  • UCSF
  • Children's National
  • Nemours Children's Clinic
  • University of Miami
  • Childrens Memorial
  • James Witcomb Riley Hosptial for Children
  • LSU Health Science Center/Children's Hospital
  • Wayne State University/Childrens' Hospital
  • Children's Mercy Hospital
  • Washington University School of Medicine
  • Hackensack University Medical Center
  • Columbia Medical Center
  • Memorial Sloan-Kettering Cancer Center
  • Levine Children's Hospital
  • Duke University Medical Center
  • Doernbecher Children's Hospital (OHSU)
  • Medical University of South Carolina
  • Vanderbilt Children's Hospital
  • Texas Childrens Hospital
  • Methodist Childrens Hospital of South Texas
  • Massey Cancer Center
  • Medical College of Wisconsin
  • Alberta Children's Hospital
  • Montreal Children's Hospital
  • CHU Sainte-Justine

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
September 23, 2008
Last Updated
March 5, 2020
Sponsor
Mesoblast, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT00759018
Brief Title
Prochymal Expanded Access Treatment for Pediatric Patients Who Have Failed Steroids for Acute GVHD
Official Title
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
Study Type
Expanded Access

2. Study Status

Record Verification Date
March 2020
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mesoblast, Inc.

4. Oversight

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Graft vs Host Disease, Graft-Versus-Host Disease
Keywords
GVHD, Prochymal, Graft vs Host Disease

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Biological
Intervention Name(s)
remestemcel-L
Other Intervention Name(s)
Prochymal®, Ex-vivo Cultured Adult Human Mesenchymal Stem Cells
Intervention Description
Patients will be treated with Prochymal® twice per week at a dose of 2 x 10^6 hMSC/kg (actual body weight) for each of 4 consecutive weeks. Infusions will be administered at least 3 days apart.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Months
Maximum Age & Unit of Time
17 Years
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mahboob Rahman
Organizational Affiliation
Mesoblast, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
UCSF
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Children's National
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Nemours Children's Clinic
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Childrens Memorial
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States
Facility Name
James Witcomb Riley Hosptial for Children
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
LSU Health Science Center/Children's Hospital
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70118
Country
United States
Facility Name
Wayne State University/Childrens' Hospital
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Hackensack University Medical Center
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601
Country
United States
Facility Name
Columbia Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Memorial Sloan-Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Levine Children's Hospital
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28232
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Doernbecher Children's Hospital (OHSU)
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Vanderbilt Children's Hospital
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Texas Childrens Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Methodist Childrens Hospital of South Texas
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
Massey Cancer Center
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Alberta Children's Hospital
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T3B 6A8
Country
Canada
Facility Name
Montreal Children's Hospital
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3H1P3
Country
Canada
Facility Name
CHU Sainte-Justine
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3T1C5
Country
Canada

12. IPD Sharing Statement

Citations:
PubMed Identifier
32044400
Citation
Kurtzberg J, Prockop S, Chaudhury S, Horn B, Nemecek E, Prasad V, Satwani P, Teira P, Hayes J, Burke E; MSB-275 Study Group. Study 275: Updated Expanded Access Program for Remestemcel-L in Steroid-Refractory Acute Graft-versus-Host Disease in Children. Biol Blood Marrow Transplant. 2020 May;26(5):855-864. doi: 10.1016/j.bbmt.2020.01.026. Epub 2020 Feb 7.
Results Reference
derived

Learn more about this trial

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

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