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

Sponsor

Mesoblast, Inc. (Industry)

Overall Status

No longer available

CT.gov ID

NCT00759018

Collaborator

(none)

Locations

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Graft vs Host Disease Graft-Versus-Host Disease	Biological: remestemcel-L

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 Design

Study Type:

Expanded Access

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

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Months to 17 Years

Sexes Eligible for Study:

All

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.

Contacts and Locations

Locations

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