Trial Outcomes & Findings for A Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Participants Who Have Failed to Respond to Steroid Treatment for Acute Graft-Versus-Host Disease (aGVHD) (NCT NCT02336230)
NCT ID: NCT02336230
Last Updated: 2022-03-17
Results Overview
ORR was defined as the percentage of participants who had achieved overall response. Overall response was defined as complete response (CR) plus partial response (PR) as per aGVHD response criteria. CR was defined as resolution of aGVHD in all involved organs. PR was defined as organ improvement of at least 1 stage without worsening of any other organ.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
55 participants
Primary outcome timeframe
Day 28
Results posted on
2022-03-17
Participant Flow
Participant milestones
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
Participants were treated with intravenous (IV) remestemcel-L at a dose of 2×10\^6 mesenchymal stromal cells (MSCs)/kilogram (kg) actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
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|---|---|
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Overall Study
STARTED
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55
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Overall Study
Full Analysis Set (FAS)
|
55
|
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Overall Study
Safety Analysis Population
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54
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Overall Study
COMPLETED
|
42
|
|
Overall Study
NOT COMPLETED
|
13
|
Reasons for withdrawal
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
Participants were treated with intravenous (IV) remestemcel-L at a dose of 2×10\^6 mesenchymal stromal cells (MSCs)/kilogram (kg) actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
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|---|---|
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Overall Study
Adverse Event
|
1
|
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Overall Study
Withdrawal of consent
|
1
|
|
Overall Study
Death
|
9
|
|
Overall Study
Dose not received due to shipping delay
|
1
|
|
Overall Study
Discontinuation of MSC infusion
|
1
|
Baseline Characteristics
A Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Participants Who Have Failed to Respond to Steroid Treatment for Acute Graft-Versus-Host Disease (aGVHD)
Baseline characteristics by cohort
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
n=55 Participants
Participants were treated with IV remestemcel-L at a dose of 2×10\^6 MSCs/kg actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
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|---|---|
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Age, Continuous
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7.3 years
STANDARD_DEVIATION 5.45 • n=5 Participants
|
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Sex: Female, Male
Female
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20 Participants
n=5 Participants
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Sex: Female, Male
Male
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35 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Hispanic or Latino
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18 Participants
n=5 Participants
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|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
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36 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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3 Participants
n=5 Participants
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|
Race (NIH/OMB)
Asian
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3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
31 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 28Population: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study.
ORR was defined as the percentage of participants who had achieved overall response. Overall response was defined as complete response (CR) plus partial response (PR) as per aGVHD response criteria. CR was defined as resolution of aGVHD in all involved organs. PR was defined as organ improvement of at least 1 stage without worsening of any other organ.
Outcome measures
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
n=55 Participants
Participants were treated with IV remestemcel-L at a dose of 2×10\^6 MSCs/kg actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
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|---|---|
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Overall Response Rate (ORR) at Day 28 Post Initiation of Therapy
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69.1 percentage of participants
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SECONDARY outcome
Timeframe: Day 100Population: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study.
Overall survival rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy.
Outcome measures
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
n=55 Participants
Participants were treated with IV remestemcel-L at a dose of 2×10\^6 MSCs/kg actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
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|---|---|
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Overall Survival (OS) Rate at Day 100 Post Initiation of Therapy
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74.5 percentage of participants
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SECONDARY outcome
Timeframe: Day 100Population: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Number analyzed is the number of participants with data available for given category.
Overall survival rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy.
Outcome measures
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
n=55 Participants
Participants were treated with IV remestemcel-L at a dose of 2×10\^6 MSCs/kg actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
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|---|---|
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OS Rate at Day 100 Post Initiation of Therapy, Stratified by Responder Status at Day 28
Responders
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86.8 percentage of participants
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OS Rate at Day 100 Post Initiation of Therapy, Stratified by Responder Status at Day 28
Non- Responders
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47.1 percentage of participants
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SECONDARY outcome
Timeframe: Day 100Population: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Number analyzed is the number of participants with data available for given category.
OS rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy. Maximum severity of acute GVHD was assessed by using International Bone Marrow Transplant Registry (IBMTR) index. The severity index was defined as: Grade A (skin Stage 1: extent of rash \<25%); Grade B (skin Stage 2: extent of rash 25 to 50% or liver Stage 1 to 2: total bilirubin 34 to 102 micromoles per liter \[mcmol/L\] or intestinal tract Stage 1 to 2: volume of diarrhea 550 to 1500 milliliters per day \[mL/day\]); Grade C (skin Stage 3: extent of rash \> 50% or liver Stage 3: total bilirubin 103 to 255 mcmol/L or intestinal tract Stage 3: volume of diarrhea \>1500 mL/day); Grade D (skin Stage 4: extent of rash bullae or liver Stage 4: total bilirubin \>255 or intestinal tract Stage 4: volume of diarrhea severe pain and ileus).
Outcome measures
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
n=55 Participants
Participants were treated with IV remestemcel-L at a dose of 2×10\^6 MSCs/kg actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
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|---|---|
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OS Rate at Day 100 Post Initiation of Therapy, Stratified by Baseline aGVHD Grade
Grade B
|
50.0 percentage of participants
Interval 11.81 to 88.19
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OS Rate at Day 100 Post Initiation of Therapy, Stratified by Baseline aGVHD Grade
Grade C
|
82.6 percentage of participants
Interval 61.22 to 95.05
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OS Rate at Day 100 Post Initiation of Therapy, Stratified by Baseline aGVHD Grade
Grade D
|
73.1 percentage of participants
Interval 52.21 to 88.43
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SECONDARY outcome
Timeframe: Day 100Population: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Number analyzed is the number of participants with data available for given category.
OS rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy. The data was summarized for organ involvement: skin only, lower GI only, and multi-organ.
Outcome measures
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
n=55 Participants
Participants were treated with IV remestemcel-L at a dose of 2×10\^6 MSCs/kg actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
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|---|---|
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OS Rate at Day 100 Post Initiation of Therapy, Stratified by Organ Involvement
Skin Only
|
78.6 percentage of participants
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OS Rate at Day 100 Post Initiation of Therapy, Stratified by Organ Involvement
Lower GI Only
|
76.2 percentage of participants
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OS Rate at Day 100 Post Initiation of Therapy, Stratified by Organ Involvement
Multi-organ (Any Combination)
|
70.0 percentage of participants
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SECONDARY outcome
Timeframe: Day 56 and Day 100Population: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study.
OR rate was defined as the percentage of participants who had achieved overall response. Overall response was defined as CR plus PR as per aGVHD response criteria. CR was defined as resolution of aGVHD in all involved organs. PR was defined as organ improvement of at least 1 stage without worsening of any other organ.
Outcome measures
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
n=55 Participants
Participants were treated with IV remestemcel-L at a dose of 2×10\^6 MSCs/kg actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
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|---|---|
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OR Rate at Day 56 and 100 Post Initiation of Therapy
Day 56
|
58.2 percentage of participants
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OR Rate at Day 56 and 100 Post Initiation of Therapy
Day 100
|
69.1 percentage of participants
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Adverse Events
Remestemcel-L 2×10^6 MSCs/kg
Serious events: 35 serious events
Other events: 52 other events
Deaths: 13 deaths
Serious adverse events
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
n=54 participants at risk
Participants were treated with IV remestemcel-L at a dose of 2×10\^6 MSCs/kg actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
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|---|---|
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Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
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3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
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Vascular disorders
Capillary leak syndrome
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1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
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|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
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1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
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Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
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Respiratory, thoracic and mediastinal disorders
Pulmonary mass
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
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|
Blood and lymphatic system disorders
Haemolytic uraemic syndrome
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
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|
Blood and lymphatic system disorders
Febrile neutropenia
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Blood and lymphatic system disorders
Haemolysis
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Blood and lymphatic system disorders
Pancytopenia
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Blood and lymphatic system disorders
Thrombotic microangiopathy
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Cardiac disorders
Cardiac arrest
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Cardiac disorders
Cardiac failure
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Cardiac disorders
Pericardial effusion
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Pneumatosis intestinalis
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Abdominal pain
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Diarrhoea
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Jejunal perforation
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Lower gastrointestinal haemorrhage
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Nausea
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Vomiting
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
General disorders
Pyrexia
|
9.3%
5/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
General disorders
Multiple organ dysfunction syndrome
|
9.3%
5/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
General disorders
Asthenia
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Immune system disorders
Acute graft versus host disease
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Immune system disorders
Graft versus host disease in skin
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Immune system disorders
Graft versus host disease
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Staphylococcal infection
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
BK virus infection
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Escherichia urinary tract infection
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Human herpesvirus 6 infection
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Pneumonia
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Sepsis
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Staphylococcal bacteraemia
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Adenovirus infection
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Cellulitis
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Clostridium difficile infection
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Enterococcal infection
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Epstein-Barr viraemia
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Fungaemia
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Fungal infection
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Gastroenteritis
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Lactobacillus infection
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Rotavirus infection
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Staphylococcal sepsis
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Investigations
Platelet count decreased
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Investigations
White blood cell count decreased
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Dehydration
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hypermetabolism
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute myeloid leukaemia recurrent
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute megakaryocytic leukaemia
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Nervous system disorders
Posterior reversible encephalopathy syndrome
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Nervous system disorders
Somnolence
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Renal and urinary disorders
Acute kidney injury
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Renal and urinary disorders
Cystitis haemorrhagic
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
9.3%
5/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
|
3.7%
2/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Vascular disorders
Hypertensive crisis
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Vascular disorders
Hypovolaemic shock
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Vascular disorders
Peripheral ischaemia
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Vascular disorders
Shock haemorrhagic
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Vascular disorders
Venoocclusive disease
|
1.9%
1/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
Other adverse events
| Measure |
Remestemcel-L 2×10^6 MSCs/kg
n=54 participants at risk
Participants were treated with IV remestemcel-L at a dose of 2×10\^6 MSCs/kg actual body weight at Screening, twice per week, for each of 4 consecutive weeks (initial therapy) given at least 3 days apart and no more than 5 days apart for any infusion. Eligible participants received an additional once per week infusion, for each of 4 consecutive weeks (continued therapy) of remestemcel-L and twice per week infusions, for each of 4 consecutive weeks (aGVHD flare therapy) of remestemcel-L at the same initial therapy dose of 2×10\^6 MSCs/kg actual body weight at Screening.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Blood and lymphatic system disorders
Neutropenia
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Cardiac disorders
Sinus tachycardia
|
9.3%
5/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Cardiac disorders
Pericardial effusion
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Cardiac disorders
Tachycardia
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Endocrine disorders
Adrenal insufficiency
|
9.3%
5/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Endocrine disorders
Cushingoid
|
9.3%
5/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Abdominal pain
|
18.5%
10/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Vomiting
|
16.7%
9/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Diarrhoea
|
11.1%
6/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Abdominal distension
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Haematochezia
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Constipation
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Flatulence
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Gastrointestinal disorders
Nausea
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
General disorders
Pyrexia
|
24.1%
13/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
General disorders
Oedema peripheral
|
13.0%
7/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
General disorders
Generalised oedema
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Immune system disorders
Chronic graft versus host disease
|
11.1%
6/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Immune system disorders
Hypogammaglobulinaemia
|
11.1%
6/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Immune system disorders
Graft versus host disease in skin
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Adenovirus infection
|
18.5%
10/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Epstein-Barr viraemia
|
13.0%
7/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
BK virus infection
|
9.3%
5/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Epstein-Barr virus infection
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Urinary tract infection
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Cytomegalovirus infection
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Oral candidiasis
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Infections and infestations
Pneumonia fungal
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Injury, poisoning and procedural complications
Allergic transfusion reaction
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Investigations
Transaminases increased
|
9.3%
5/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Investigations
Alanine aminotransferase increased
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Investigations
Blood creatinine increased
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Investigations
Electrocardiogram QT prolonged
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
13.0%
7/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
13.0%
7/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
11.1%
6/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
9.3%
5/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hypernatraemia
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Metabolism and nutrition disorders
Malnutrition
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
14.8%
8/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Nervous system disorders
Headache
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Nervous system disorders
Tremor
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Psychiatric disorders
Agitation
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Psychiatric disorders
Anxiety
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Psychiatric disorders
Insomnia
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Renal and urinary disorders
Cystitis haemorrhagic
|
9.3%
5/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Renal and urinary disorders
Acute kidney injury
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Renal and urinary disorders
Dysuria
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.0%
7/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
7.4%
4/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
5.6%
3/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Vascular disorders
Hypertension
|
18.5%
10/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
|
Vascular disorders
Hypotension
|
14.8%
8/54 • From Baseline through 100 days of follow up
All-cause Mortality: FAS included all participants who provided informed consent, were screened, and were found eligible to enter the study. Serious and Other (Non-serious) AEs: Safety Analysis Population included all participants who signed the informed consent form and received at least 1 dose of remestemcel-L.
|
Additional Information
Christopher James, VP Head of Clinical Operations
Mesoblast, Inc.
Phone: 212-880-2060
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER