Trial Outcomes & Findings for Safety Follow-up of Treatment With Remestemcel-L in Pediatric Participants Who Have Failed to Respond to Steroid Treatment for Acute GVHD (NCT NCT02652130)

Last Updated: 2022-03-16

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

32 participants

Primary outcome timeframe

From Baseline Day 1 in the Study MSB-GVHD001 up to Day 180 in Study MSB-GVHD002 (180 days)

Results posted on

2022-03-16

Participant Flow

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.

Participant milestones

Participant milestones
Measure	Remestemcel-L All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001.
Overall Study STARTED	32
Overall Study COMPLETED	31
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Remestemcel-L All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001.
Overall Study Death	1

Baseline Characteristics

Safety Follow-up of Treatment With Remestemcel-L in Pediatric Participants Who Have Failed to Respond to Steroid Treatment for Acute GVHD

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Remestemcel-L n=32 Participants All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001.
Age, Continuous	7.8 years STANDARD_DEVIATION 5.24 • n=99 Participants
Sex: Female, Male Female	11 Participants n=99 Participants
Sex: Female, Male Male	21 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	12 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	20 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Race/Ethnicity, Customized Race · White	20 Participants n=99 Participants
Race/Ethnicity, Customized Race · Black or African American	3 Participants n=99 Participants
Race/Ethnicity, Customized Race · Asian	2 Participants n=99 Participants
Race/Ethnicity, Customized Race · American Indian or Alaska Native	1 Participants n=99 Participants
Race/Ethnicity, Customized Race · Other	6 Participants n=99 Participants

PRIMARY outcome

Timeframe: From Baseline Day 1 in the Study MSB-GVHD001 up to Day 180 in Study MSB-GVHD002 (180 days)

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Remestemcel-L n=54 Participants All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001.
Overall Survival Rate Through Day 180	68.5 percentage of participants

SECONDARY outcome

Timeframe: From Baseline (Day 1) in the Study MSB-GVHD001 up to Day 180 in the Study MSB-GVHD002 (180 days)

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Remestemcel-L n=38 Participants All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001.
Overall Survival Rate at Day 180 for Participants Who Had Overall Response (OR) at Day 28 of Study MSB-GVHD001	78.9 percentage of participants

Adverse Events

Remestemcel-L

Serious events: 15 serious events

Other events: 0 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Remestemcel-L n=32 participants at risk All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001.
Blood and lymphatic system disorders Febrile neutropenia	3.1% 1/32 • 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 \[NCT02336230\].
Blood and lymphatic system disorders Thrombocytopenia	3.1% 1/32 • 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 \[NCT02336230\].
Gastrointestinal disorders Pneumatosis intestinalis	3.1% 1/32 • 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 \[NCT02336230\].
Gastrointestinal disorders Haematochezia	3.1% 1/32 • 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 \[NCT02336230\].
General disorders Oedema peripheral	3.1% 1/32 • 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 \[NCT02336230\].
Hepatobiliary disorders Cholecystitis	3.1% 1/32 • 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 \[NCT02336230\].
Infections and infestations Pneumonia	6.2% 2/32 • 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 \[NCT02336230\].
Infections and infestations Septic shock	6.2% 2/32 • 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 \[NCT02336230\].
Infections and infestations Bacteraemia	3.1% 1/32 • 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 \[NCT02336230\].
Infections and infestations Bronchopulmonary aspergillosis	3.1% 1/32 • 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 \[NCT02336230\].
Infections and infestations Enterococcal infection	3.1% 1/32 • 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 \[NCT02336230\].
Infections and infestations Nocardiosis	3.1% 1/32 • 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 \[NCT02336230\].
Infections and infestations Osteomyelitis acute	3.1% 1/32 • 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 \[NCT02336230\].
Infections and infestations Pneumonia pneumococcal	3.1% 1/32 • 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 \[NCT02336230\].
Infections and infestations Pseudomonas infection	3.1% 1/32 • 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 \[NCT02336230\].
Infections and infestations Vulval abscess	3.1% 1/32 • 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 \[NCT02336230\].
Injury, poisoning and procedural complications Laceration	3.1% 1/32 • 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 \[NCT02336230\].
Investigations Weight decreased	3.1% 1/32 • 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 \[NCT02336230\].
Metabolism and nutrition disorders Hyperglycaemia	3.1% 1/32 • 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 \[NCT02336230\].
Metabolism and nutrition disorders Hypoalbuminaemia	3.1% 1/32 • 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 \[NCT02336230\].
Metabolism and nutrition disorders Hyponatraemia	3.1% 1/32 • 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 \[NCT02336230\].
Musculoskeletal and connective tissue disorders Osteonecrosis	3.1% 1/32 • 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 \[NCT02336230\].
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Acute lymphocytic leukaemia recurrent	3.1% 1/32 • 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 \[NCT02336230\].
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Post transplant lymphoproliferative disorder	3.1% 1/32 • 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 \[NCT02336230\].
Skin and subcutaneous tissue disorders Eczema	3.1% 1/32 • 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 \[NCT02336230\].

Other adverse events

Adverse event data not reported

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