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

NCT ID: NCT00759018

Last Updated: 2020-03-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NO_LONGER_AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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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

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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.

Conditions

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Graft vs Host Disease Graft-Versus-Host Disease

Interventions

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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.

Intervention Type BIOLOGICAL

Other Intervention Names

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Prochymal® Ex-vivo Cultured Adult Human Mesenchymal Stem Cells

Eligibility Criteria

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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.
Minimum Eligible Age

2 Months

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Mesoblast, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Mahboob Rahman

Role: STUDY_DIRECTOR

Mesoblast, Inc.

Locations

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Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

Children's Hospital of Orange County

Orange, California, United States

Site Status

UCSF

San Francisco, California, United States

Site Status

Children's National

Washington D.C., District of Columbia, United States

Site Status

Nemours Children's Clinic

Jacksonville, Florida, United States

Site Status

University of Miami

Miami, Florida, United States

Site Status

Childrens Memorial

Chicago, Illinois, United States

Site Status

James Witcomb Riley Hosptial for Children

Indianapolis, Indiana, United States

Site Status

LSU Health Science Center/Children's Hospital

New Orleans, Louisiana, United States

Site Status

Wayne State University/Childrens' Hospital

Detroit, Michigan, United States

Site Status

Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status

Columbia Medical Center

New York, New York, United States

Site Status

Memorial Sloan-Kettering Cancer Center

New York, New York, United States

Site Status

Levine Children's Hospital

Charlotte, North Carolina, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Doernbecher Children's Hospital (OHSU)

Portland, Oregon, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Vanderbilt Children's Hospital

Nashville, Tennessee, United States

Site Status

Texas Childrens Hospital

Houston, Texas, United States

Site Status

Methodist Childrens Hospital of South Texas

San Antonio, Texas, United States

Site Status

Massey Cancer Center

Richmond, Virginia, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Alberta Children's Hospital

Calgary, Alberta, Canada

Site Status

Montreal Children's Hospital

Montreal, Quebec, Canada

Site Status

CHU Sainte-Justine

Montreal, Quebec, Canada

Site Status

Countries

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United States Canada

References

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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.

Reference Type DERIVED
PMID: 32044400 (View on PubMed)

Other Identifiers

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275

Identifier Type: -

Identifier Source: org_study_id

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