Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Overall Response (OR) |
OR is defined as complete response (CR) or partial response (PR) at Day 28 relative to aGvHD status at baseline. CR is defined as resolution of aGvHD in all involved organs. PR is defined as improvement in 1 stage in 1 or more organs involved with aGvHD symptoms without progression in others. Number of participants with OR will be reported. |
Day 28 |
|
| Primary |
Overall Survival |
Overall survival is defined as the time from randomization to the date of death due to any cause. |
Up to Month 24 |
|
| Secondary |
Freedom from Treatment Failure (FFTF) |
FFTF is defined as death, relapse or progression of the underlying disease, or addition or change to any further systemic immunosuppressive aGvHD therapy. Number of participants with FFTF will be reported. |
Up to 6 months |
|
| Secondary |
Acute Graft-versus-host Disease (aGvHD) Response |
Number of participants with aGvHD response will be reported. aGvHD response will be categorized as OR (CR + PR), CR, PR, and NR. NR is defined as the absence of CR or PR. |
Days 28, 60, 100 and 180 |
|
| Secondary |
Change from Baseline in aGvHD Grades |
aGvHD grades: Grade 0- no organ involvement (ie, Stage 0 skin, Stage 0 liver, and Stage 0 GI); Grade I-Stage 1 - 2 skin without liver/GI involvement; Grade II- Stage 3 skin and / or Stage 1 liver and / or Stage 1 GI; Grade III- Stage 2 - 3 liver and / or Stage 2 - 3 GI; Grade IV- Stage 4 skin and / or Stage 4 liver and/or Stage 4 GI. |
Baseline and Days 8, 15, 22, 28, 60, 100 and 180 |
|
| Secondary |
Time to Response |
Time to response is defined as the time from the date of the first treatment administration to the date of response. |
Up to Month 24 |
|
| Secondary |
Duration of Response |
Duration is calculated from time from the first OR (CR or PR) until the time point of no aGvHD response in comparison to baseline. |
Up to Month 24 |
|
| Secondary |
Best Overall Response (OR) |
Best OR is defined as the achievement of an OR at any time point up to and including Day 28. Number of participants with best OR will be reported. |
Up to Day 28 |
|
| Secondary |
Cumulative Dose of Steroids for SR-aGvHD per Kilogram (kg) of Body Weight |
The cumulative dose of steroids given for SR-aGvHD per kg of body weight from baseline until Day 60 and until Visit Month 24 will be analyzed. |
Up to Day 60 and Month 24 |
|
| Secondary |
Number of Participants with Chronic Graft-versus-host Disease (cGvHD) |
Number of participants with cGvHD will be reported. |
Day 60 to Month 24 |
|
| Secondary |
Time to Chronic Graft-versus-host Disease (cGvHD) |
Time to cGvHD is defined as the time between the last day of haematopoietic stem cell transplantation (HSCT) to the first episode of cGvHD. |
Day 60 to Month 24 |
|
| Secondary |
Number of Participants with Graft Failure (GF) |
Number of participants with GF will be reported. |
Up to Month 24 |
|
| Secondary |
Number of Participants with Relapse or Progression in Participants with Underlying Malignant Disease |
Number of participants with relapse or progression in participants with underlying malignant disease will be reported. |
Up to Month 24 |
|
| Secondary |
Time to Relapse or Progression in Participants with Underlying Malignant Disease |
Time to relapse or progression in participants with underlying malignant disease will be reported. |
Up to Month 24 |
|
| Secondary |
Event-free survival (EFS) |
EFS is defined as the time from the date of randomization to the date of the event. An event is defined as GF, relapse or progression of the underlying disease, or death due to any cause. |
Up to Month 24 |
|
| Secondary |
Non-relapse Mortality (NRM) |
NRM is defined as the time from the date of randomisation to the date of the event. An event is defined as death without previous relapse or progression of the underlying disease. |
Up to Month 24 |
|
| Secondary |
Number of Participants with Adverse Events (AEs) and Adverse Reactions (ARs) |
|
Until Day 60 or until 30 days after last administration of trial treatment, whichever is later (Up to Month 24) |
|
| Secondary |
Number of Participants with Adverse Events (AEs) and Adverse Reactions (ARs) by Severity |
Severity will be graded based on Common Terminology Criteria for Adverse Events (CTCAE) v5.0: Grade 1- Mild; Grade 2- Moderate; Grade 3- Severe; Grade 4- Life-threatening consequences; urgent intervention indicated; Grade 4- Death related to the AE. |
Until Day 60 or until 30 days after last administration of trial treatment, whichever is later (Up to Month 24) |
|
| Secondary |
Change from Baseline in Performance score based on Karnofsky scale (recipient age >= 16 years) |
The Karnofsky performance score (KPS), which is reported on an ordinal scale from 0 to 100, provides a rough measure of the participant's well-being, including their ability to conduct activities of daily living and functional capacity. Higher score indicates normal, no complaints and no evidence of disease. |
Baseline, Days 8, 15, 22, 28, 60 and 100 |
|
| Secondary |
Change from Baseline in Performance score based on Lansky Scale |
A Lansky score (recipient age greater than or equal to [>=] 1 years and less than [<] 16 years) will be recorded pre-treatment and measured serially at regular intervals after treatment. The score is a standard performance score that measures overall function of the child with a scale range from 0 to 100. Higher score indicates full activeness. |
Baseline, Days 8, 15, 22, 28, 60 and 100 |
|
| Secondary |
Change from Baseline in EuroQol-5D-5L (EQ-5D-5L): Health Status Index (HSI) |
EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L descriptive system and EQ VAS. EQ-5D-5L descriptive system comprises of 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each has 5 levels of perceived problems (1-no problem, 2-slight problems, 3-moderate problems, 4-severe problems, 5-extreme problems). Participant selects answer for each of 5 dimensions considering response that best matches his/her health "TODAY". Responses were used to generate a HSI. HSI ranges from 0 (dead) to 1.00 (full health). |
Baseline, Days 28, 60, 100 and 180 |
|
| Secondary |
Change from Baseline in EuroQol-5D-5L (EQ-5D-5L): Visual Analogue Scale (VAS) |
EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L descriptive system and EQ VAS. The EQ VAS self-rating records the respondent's own assessment of his or her overall health status at the time of completion, on a scale of 0 (the worst health you can imagine) to 100 (the best health you can imagine). |
Baseline, Days 28, 60, 100 and 180 |
|
| Secondary |
Change from Baseline in Functional Assessment of Cancer Therapy-Bone Marrow Transplantation (FACT-BMT) Score |
The FACT-BMT questionnaire was designed to measure the quality of life in subjects undergoing bone marrow (BM) transplantation. It consists of the following categories of assessment: physical well-being, social / family well-being, emotional well-being, functional well-being, and additional miscellaneous concerns that the subject may have concerning their healthcare, persons involved in their life, and other emotions and incapabilities. Score ranges from 0-164, with higher score indicating better quality of life. |
Baseline, Days 28, 60, 100 and 180 |
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