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| Name | Class |
|---|---|
| Quintiles, Inc. | INDUSTRY |
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Ongoing safety assessment follow-up to Protocol MSB-GVHD001 (NCT02336230) of remestemcel-L treatment in pediatric participants with acute graft versus host disease (aGVHD), following allogeneic hematopoietic stem cell transplant (HSCT), that have failed to respond to treatment with systemic corticosteroid therapy.
This is a safety follow-up study through 180 days of remestemcel-L treatment in participants who took part in MSB-GVHD001. This study will also explore duration of response over time. Participants who took part in in MSB-GVHD001 and received at least one dose of remestemcel-L as outlined in that protocol will be evaluated at baseline (Day 100) and at Days 120, 140, 160 and 180 for safety endpoints. Participants who took part in Protocol MSB-GVHD001 and received the first 8 doses of remestemcel-L as outlined in that protocol will be evaluated at baseline (Day 100) and at Days 120, 140, 160 and 180 after initial remestemcel-L infusion for evidence of duration of response over time.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Safety population | Experimental | All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Remestemcel-L | Biological | No intervention was given in Study MSB-GVHD002 (NCT02652130). It was a safety follow-up trial of remestemcel-L-treated participants from Study MSB-GVHD001. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival Rate Through Day 180 | The overall survival rate is defined as the percentage of participants alive at the given time point. OS is defined as the time to death from the start of drug therapy. | From Baseline Day 1 in the Study MSB-GVHD001 up to Day 180 in Study MSB-GVHD002 (180 days) |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival Rate at Day 180 for Participants Who Had Overall Response (OR) at Day 28 of Study MSB-GVHD001 | The overall survival rate is defined as the percentage of participants alive at the given time point. OS is defined as the time to death from the start of drug therapy. | From Baseline (Day 1) in the Study MSB-GVHD001 up to Day 180 in the Study MSB-GVHD002 (180 days) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Christopher James | Mesoblast, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States | ||
| CHOC Children's Hospital of Orange County |
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54 participants enrolled in Study MSB-GVHD001 [NCT02336230], received >=1 dose of remestemcel-L; evaluated up to Day100. These participants were followed-up for OS, GVHD activity up to Day180 (end of Study MSB-GVHD002 [NCT02652130]). 32 of 54 participants were enrolled in Study MSB-GVHD002; evaluated at Baseline (Day 100) and at Days120, 140, 160 and 180.
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| ID | Title | Description |
|---|---|---|
| FG000 | Remestemcel-L | All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 10, 2018 |
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| Orange |
| California |
| 92868 |
| United States |
| UCSF Benioff Children's Hospital | San Francisco | California | 94143 | United States |
| Children's Hospital Colorado Center for Cancer/Blood Disorders | Aurora | Colorado | 80045 | United States |
| Alfred I. duPont Hospital for Children of the Nemours Foundation | Wilmington | Delaware | 19803 | United States |
| Miami Children's Research Institute | Miami | Florida | 33155 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Children's Hospital of Michigan | Detroit | Michigan | 48201 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Memorial Sloan Kettering Cancer Center | New York | New York | 10174 | United States |
| The Children's Hospital at Montefiore | New York | New York | 10467 | United States |
| Duke University Medical Center | Durham | North Carolina | 27705 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Texas Transplant Institute | San Antonio | Texas | 78229 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23284 | United States |
| Fred Hutchinson Cancer Center | Seattle | Washington | 98109 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| COMPLETED |
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| NOT COMPLETED |
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Safety population included all participants who signed the informed consent form and received at least one dose of study treatment (complete or partial) in the study MSB-GVHD001 and who signed the informed consent form for the study MSB-GVHD002 were followed across both the studies MSB-GVHD001 and MSB-GVHD002.
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| ID | Title | Description |
|---|---|---|
| BG000 | Remestemcel-L | All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Overall Survival Rate Through Day 180 | The overall survival rate is defined as the percentage of participants alive at the given time point. OS is defined as the time to death from the start of drug therapy. | Safety population included all participants who signed the informed consent form and received at least one dose of study treatment (complete or partial) in the study MSB-GVHD001 and who signed the informed consent form for the study MSB-GVHD002 were followed across both the studies MSB-GVHD001 and MSB-GVHD002. | Posted | Number | percentage of participants | From Baseline Day 1 in the Study MSB-GVHD001 up to Day 180 in Study MSB-GVHD002 (180 days) |
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| Secondary | Overall Survival Rate at Day 180 for Participants Who Had Overall Response (OR) at Day 28 of Study MSB-GVHD001 | The overall survival rate is defined as the percentage of participants alive at the given time point. OS is defined as the time to death from the start of drug therapy. | Duration of Response population included all participants who participated in Study MSB-GVHD001 and showed OR or very good partial response (VGPR) to remestemcel-L at Day 28 in Study MSB-GVHD001. The OS was evaluated across both Studies MSB-GVHD001 and MSB-GVHD002. | Posted | Number | percentage of participants | From Baseline (Day 1) in the Study MSB-GVHD001 up to Day 180 in the Study MSB-GVHD002 (180 days) |
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Baseline (Day 100) up to Day 180 in Study MSB-GVHD002
Safety population included all participants who signed the informed consent form and received at least one dose of study treatment (complete or partial) in Study MSB-GVHD001 and who signed the informed consent form for Study MSB-GVHD002. As no treatment was administered in this study, only serious adverse events are reported. Any non-serious treatment-emergent adverse events that occurred within 30 days of last treatment at the 5% threshold will be reported in Study MSB-GVHD001 [NCT02336230].
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Remestemcel-L | All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001. | 1 | 32 | 15 | 32 | 0 | 32 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (20.0) | Systematic Assessment |
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| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (20.0) | Systematic Assessment |
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| Pneumatosis intestinalis | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
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| Haematochezia | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA (20.0) | Systematic Assessment |
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| Cholecystitis | Hepatobiliary disorders | MedDRA (20.0) | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
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| Septic shock | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
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| Bacteraemia | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
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| Bronchopulmonary aspergillosis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
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| Enterococcal infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
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| Nocardiosis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
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| Osteomyelitis acute | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
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| Pneumonia pneumococcal | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
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| Pseudomonas infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
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| Vulval abscess | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
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| Laceration | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
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| Weight decreased | Investigations | MedDRA (20.0) | Systematic Assessment |
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| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (20.0) | Systematic Assessment |
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| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA (20.0) | Systematic Assessment |
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| Hyponatraemia | Metabolism and nutrition disorders | MedDRA (20.0) | Systematic Assessment |
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| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
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| Acute lymphocytic leukaemia recurrent | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.0) | Systematic Assessment |
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| Post transplant lymphoproliferative disorder | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.0) | Systematic Assessment |
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| Eczema | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
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Publications (abstracts, posters or presentations) must be presented to the Publication Steering Committee for review prior to submission or public display and are not allowed prior to the publication of the primary manuscript, or eighteen (18) months from the conclusion of the Study. PI shall provide Sponsor a copy of any proposed public disclosure at least 30 days prior to submission. Sponsor may ask PI to delay the disclosure for a maximum of 60 days to file proprietary protection.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Christopher James, VP, Head of Clinical Operations | Mesoblast, Inc. | 212-880-2060 | 7925 | Christopher.James@Mesoblast.com |
| Dec 30, 2021 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| C000711674 | remestemcel-l |
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| Unknown or Not Reported |
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| Asian |
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| American Indian or Alaska Native |
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| Other |
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