Graft vs Host DiseaseNCT00759018
Prochymal Expanded Access Treatment for Pediatric Patients Who Have Failed Steroids for Acute GVHD
A study testing remestemcel-L in people with Graft vs Host Disease and Graft-Versus-Host Disease, run by Mesoblast, Inc. at 27 sites in 2 countries.
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. Read more about this study
- Who is it for?
- Ages 2 months to 17 years
- Conditions the study is for
- Graft vs Host Disease, Graft-Versus-Host Disease
- What is being tested?
- remestemcel-L
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 27 sitesin 2 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 months to 17 years
- All sexes
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
27 sites in 2 countries.
27 sites in 2 countries
United States24 sites
Medical University of South Carolina
Charleston, South Carolina, 29425
Levine Children's Hospital
Charlotte, North Carolina, 28232
Childrens Memorial
Chicago, Illinois, 60614
Wayne State University/Childrens' Hospital
Detroit, Michigan, 48201
Duke University Medical Center
Durham, North Carolina, 27710
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Texas Childrens Hospital
Houston, Texas, 77030
James Witcomb Riley Hosptial for Children
Indianapolis, Indiana, 46202
Nemours Children's Clinic
Jacksonville, Florida, 32207
Children's Mercy Hospital
Kansas City, Missouri, 64108
University of Miami
Miami, Florida, 33136
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232
LSU Health Science Center/Children's Hospital
New Orleans, Louisiana, 70118
Columbia Medical Center
New York, New York, 10032
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
Children's Hospital of Orange County
Orange, California, 92868
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Doernbecher Children's Hospital (OHSU)
Portland, Oregon, 97239
Massey Cancer Center
Richmond, Virginia, 23298
Methodist Childrens Hospital of South Texas
San Antonio, Texas, 78229
UCSF
San Francisco, California, 94143
Washington University School of Medicine
St Louis, Missouri, 63110
Children's National
Washington D.C., District of Columbia, 20010
Canada3 sites
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
Montreal Children's Hospital
Montreal, Quebec, H3H1P3
CHU Sainte-Justine
Montreal, Quebec, H3T1C5
About this study
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
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.
Read the 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.
- Conditions
- Graft vs Host Disease, Graft-Versus-Host Disease
- Being tested
- remestemcel-L
- Keywords
- GVHD, Prochymal, Graft vs Host Disease
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
remestemcel-LBiological
Also called Prochymal®, Ex-vivo Cultured Adult Human Mesenchymal Stem Cells
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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2008 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Graft vs Host DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Mesoblast, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mahboob Rahman, Study director, Mesoblast, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00759018
- Sponsor's protocol ID
- 275
- Status last verified
- Mar 2020
- First submitted
- 23 Sep 2008
- Last updated
- 9 Mar 2020
- Results posted
- No
Publications
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.
PubMed 32044400
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00759018
- Acronym / sponsor ID
- 275
- Conditions
- Graft vs Host Disease, Graft-Versus-Host Disease
- Keywords
- GVHD, Prochymal, Graft vs Host Disease
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Mesoblast, Inc.Industry (pharma, biotech, devices)
- First posted
- 25 Sep 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 2 months to 17 years
- Sex
- All
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| remestemcel-LAlso: Prochymal®, Ex-vivo Cultured Adult Human Mesenchymal Stem CellsPatients 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. | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2020.
- Derived from this study: PubMed 32044400Kurtzberg 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.
Related trials for Graft vs Host Disease
Active, not recruitingPhase 2
De-escalated Cyclophosphamide (PTCy) and Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis
Graft-versus-host Disease
RecruitingEarly Phase 1
HMCT/CT2401: Abatacept GVHD Prophylaxis Following Omidubicel HCT
Hematologic Malignancy
RecruitingObservational study
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
Graft-Versus-Host Disease, Graft-versus-leukemia
RecruitingPhase 1/2
The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
Peripheral Blood Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation
RecruitingPhase 1/2
Phase I/II Study of Pacritinib, A JAK2/IRAK1/CSF1R Inhibitor, in Refractory Chronic Graft-Versus-Host Disease (cGVHD) After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Graft vs Host Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mahboob Rahman study director
Mesoblast, Inc.
27 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing remestemcel-L in people with Graft vs Host Disease and Graft-Versus-Host Disease, run by Mesoblast, Inc. at 27 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 9 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00759018, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2020.