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

Graft vs Host Disease Clinical Trial

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

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT00759018
• Other study ID #: 275
• Status: No longer available

Study information

• Verified date: March 2020
• Source: Mesoblast, Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: No longer available
• Enrollment: 0
• Gender: All
• Age group: 2 Months to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Graft vs Host Disease
• Graft-Versus-Host Disease

Intervention

Biological:
• remestemcel-L
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.

Locations

Country	Name	City	State
Canada	Alberta Children's Hospital	Calgary	Alberta
Canada	CHU Sainte-Justine	Montreal	Quebec
Canada	Montreal Children's Hospital	Montreal	Quebec
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Levine Children's Hospital	Charlotte	North Carolina
United States	Childrens Memorial	Chicago	Illinois
United States	Wayne State University/Childrens' Hospital	Detroit	Michigan
United States	Duke University Medical Center	Durham	North Carolina
United States	Hackensack University Medical Center	Hackensack	New Jersey
United States	Texas Childrens Hospital	Houston	Texas
United States	James Witcomb Riley Hosptial for Children	Indianapolis	Indiana
United States	Nemours Children's Clinic	Jacksonville	Florida
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	University of Miami	Miami	Florida
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	Vanderbilt Children's Hospital	Nashville	Tennessee
United States	LSU Health Science Center/Children's Hospital	New Orleans	Louisiana
United States	Columbia Medical Center	New York	New York
United States	Memorial Sloan-Kettering Cancer Center	New York	New York
United States	Children's Hospital of Orange County	Orange	California
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Doernbecher Children's Hospital (OHSU)	Portland	Oregon
United States	Massey Cancer Center	Richmond	Virginia
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	Methodist Childrens Hospital of South Texas	San Antonio	Texas
United States	UCSF	San Francisco	California
United States	Children's National	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Mesoblast, Inc.

Countries where clinical trial is conducted

United States,  Canada,