Grade B Acute Graft Versus Host Disease, Grade C Acute Graft Versus Host Disease, Grade D Acute Graft Versus Host Disease
Conditions
Keywords
GVHD
Brief summary
Ongoing safety assessment follow-up to Protocol MSB-GVHD001 (NCT02336230) of remestemcel-L treatment in pediatric participants with acute graft versus host disease (aGVHD), following allogeneic hematopoietic stem cell transplant (HSCT), that have failed to respond to treatment with systemic corticosteroid therapy.
Detailed description
This is a safety follow-up study through 180 days of remestemcel-L treatment in participants who took part in MSB-GVHD001. This study will also explore duration of response over time. Participants who took part in in MSB-GVHD001 and received at least one dose of remestemcel-L as outlined in that protocol will be evaluated at baseline (Day 100) and at Days 120, 140, 160 and 180 for safety endpoints. Participants who took part in Protocol MSB-GVHD001 and received the first 8 doses of remestemcel-L as outlined in that protocol will be evaluated at baseline (Day 100) and at Days 120, 140, 160 and 180 after initial remestemcel-L infusion for evidence of duration of response over time.
Interventions
BIOLOGICALRemestemcel-L
No intervention was given in Study MSB-GVHD002 (NCT02652130). It was a safety follow-up trial of remestemcel-L-treated participants from Study MSB-GVHD001.
Sponsors
Quintiles, Inc.
Mesoblast, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Months to 17 Years
Healthy volunteers
No
Inclusion criteria
* Participants must have participated in MSB-GVHD001 and have received at least one infusion of remestemcel-L. * Participant or participant's authorized representative must be capable of providing written informed consent. Assent, if applicable, must also be collected when required by the Institutional Review Board (IRB)/Ethics Committee (EC). * Female participants of childbearing potential (≥ 10 years of age) must use a medically accepted method of contraception and must agree to continue use of this method for the duration of the study and for the follow-up time period. Acceptable methods of contraception include abstinence, barrier method with spermicide, intrauterine device (IUD), or steroidal contraceptive (oral, transdermal, implanted, and injected) in conjunction with a barrier method. * The participant must be willing and able to comply with study procedures, remain at the clinic as required during the study period, and return to the clinic for the follow-up evaluation as specified in this protocol.
Exclusion criteria
* The investigator believes it to be in the best interest of the participant not to participate in the safety follow-up study. * Participant has participated or is currently participating in any autologous or allogeneic stem cell or gene therapy study for the treatment of aGVHD. Participants participating in investigative protocols aimed at modification of the transplant graft (such as T cell depletion) or aimed at modification of the conditioning regimen will be allowed in the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival Rate Through Day 180 | From Baseline Day 1 in the Study MSB-GVHD001 up to Day 180 in Study MSB-GVHD002 (180 days) | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival Rate at Day 180 for Participants Who Had Overall Response (OR) at Day 28 of Study MSB-GVHD001 | From Baseline (Day 1) in the Study MSB-GVHD001 up to Day 180 in the Study MSB-GVHD002 (180 days) | 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. |
Countries
United States
Participant flow
Recruitment details
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.
Participants by arm
| Arm | Count |
|---|---|
| Remestemcel-L All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001. | 32 |
| Total | 32 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 1 |
Baseline characteristics
| Characteristic | Remestemcel-L |
|---|---|
| Age, Continuous | 7.8 years STANDARD_DEVIATION 5.24 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 12 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 20 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race/Ethnicity, Customized Race American Indian or Alaska Native | 1 Participants |
| Race/Ethnicity, Customized Race Asian | 2 Participants |
| Race/Ethnicity, Customized Race Black or African American | 3 Participants |
| Race/Ethnicity, Customized Race Other | 6 Participants |
| Race/Ethnicity, Customized Race White | 20 Participants |
| Sex: Female, Male Female | 11 Participants |
| Sex: Female, Male Male | 21 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 32 |
| other Total, other adverse events | 0 / 32 |
| serious Total, serious adverse events | 15 / 32 |
Outcome results
Primary
Overall Survival Rate Through Day 180
The overall survival rate is defined as the percentage of participants alive at the given time point. OS is defined as the time to death from the start of drug therapy.
Time frame: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Remestemcel-L | Overall Survival Rate Through Day 180 | 68.5 percentage of participants |
Secondary
Overall Survival Rate at Day 180 for Participants Who Had Overall Response (OR) at Day 28 of Study MSB-GVHD001
The overall survival rate is defined as the percentage of participants alive at the given time point. OS is defined as the time to death from the start of drug therapy.
Time frame: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Remestemcel-L | 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 |