Clinical trial NCT00759018: 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
Overall status: NO_LONGER_AVAILABLE
Conditions: Graft vs Host Disease
Graft-Versus-Host Disease
Keywords: GVHD
Prochymal
Graft vs Host Disease
Lead sponsor: Mesoblast, Ltd.
Sponsor class: INDUSTRY
First posted: 2008-09-25
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD

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.

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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: San Francisco
country: United States
facility: UCSF
state: California
city: Washington
country: United States
facility: Children's National
state: District of Columbia
city: Jacksonville
country: United States
facility: Nemours Children's Clinic
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Chicago
country: United States
facility: Childrens Memorial
state: Illinois
city: Indianapolis
country: United States
facility: James Witcomb Riley Hosptial for Children
state: Indiana
city: New Orleans
country: United States
facility: LSU Health Science Center/Children's Hospital
state: Louisiana
city: Detroit
country: United States
facility: Wayne State University/Childrens' Hospital
state: Michigan
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: New York
country: United States
facility: Columbia Medical Center
state: New York
city: New York
country: United States
facility: Memorial Sloan-Kettering Cancer Center
state: New York
city: Charlotte
country: United States
facility: Levine Children's Hospital
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Portland
country: United States
facility: Doernbecher Children's Hospital (OHSU)
state: Oregon
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt Children's Hospital
state: Tennessee
city: Houston
country: United States
facility: Texas Childrens Hospital
state: Texas
city: San Antonio
country: United States
facility: Methodist Childrens Hospital of South Texas
state: Texas
city: Richmond
country: United States
facility: Massey Cancer Center
state: Virginia
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Calgary
country: Canada
facility: Alberta Children's Hospital
state: Alberta
city: Montreal
country: Canada
facility: Montreal Children's Hospital
state: Quebec
city: Montreal
country: Canada
facility: CHU Sainte-Justine
state: Quebec
Study officials:
affiliation: Mesoblast, Inc.
name: Mahboob Rahman
role: STUDY_DIRECTOR
References:
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.