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This is a study comparing the defibrotide prophylaxis arm vs standard of care arm for the prevention of aGvHD.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Defibrotide Prophylaxis | Experimental | Standard of Care Immunoprophylaxis + Defibrotide |
|
| Standard of Care | Active Comparator | Standard of Care Immunoprophylaxis Alone |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Defibrotide | Drug | 6.25 mg/kg via 2-hour IV infusion every 6 hours |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Cumulative Incidence Percentage of Grade B to D Acute Graft Versus Host Disease (aGvHD) by Day +100 Post-Hematopoietic Stem Cell Transplant (HSCT) | Cumulative Incidence Percentage of Grade B to D aGvHD was defined using the International Bone Marrow Transplant Registry (IBMTR) Severity Index. Grade B is defined as Skin stage = 2 or Liver stage = 1 to 2 or GI stage = 1 to 2. Grade C is defined as Skin stage = 3 or Liver stage = 3 or GI stage = 3. Grade D is defined as a Skin stage = 4 or Liver stage = 4 or GI stage = 4. | HSCT Day (Day +0 post-HSCT) through Day +100 post-HSCT |
| Measure | Description | Time Frame |
|---|---|---|
| Cumulative Incidence Percentage of Grade B to D aGvHD by Day +180 Post-HSCT | Cumulative Incidence Percentage of Grade B to D aGvHD was defined using the IBMTR Severity Index. Grade B is defined as Skin stage = 2 or Liver stage = 1 to 2 or GI stage = 1 to 2. Grade C is defined as Skin stage = 3 or Liver stage = 3 or GI stage = 3. Grade D is defined as a Skin stage = 4 or Liver stage = 4 or GI stage = 4. |
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Inclusion Criteria:
Participant must be ≥1 year of age at screening and undergoing allogeneic Hematopoietic Stem Cell Transplant (HSCT).
Participant must be diagnosed with acute leukemia in morphologic complete remission (CR1 or CR2) or with Myelodysplastic syndrome (MDS) with no circulating blasts and with less than 5% blasts in the bone marrow
Participant must have planned to receive either a myeloablative or reduced-intensity conditioning regimen and have an unrelated donor who is human leukocyte antigen (HLA) matched or single-allele mismatched
Participant must receive the following medical regimen as part of standard of care immunoprophylaxis for GvHD in either study arm at doses and regimen determined by local institutional guidelines, physician preference, and participant need:
Methotrexate (MTX) or Mycophenolate mofetil (MMF) + calcineurin inhibitor (Cyclosporine A [CSA] or Tacrolimus [TAC]) +/- Anti-thymocyte globulin (ATG) (ATG use is limited to 30% of participants).
Graft must be a CD3+ T-cell replete peripheral blood stem cell (PBSC) graft or non-manipulated bone marrow (BM) graft.
Adult participants must be able to understand and sign a written informed consent. For pediatric participants, the parent/legal guardian or representative must be able to understand and sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
Exclusion Criteria:
Participant has had a prior autologous or allogeneic HSCT.
Participant is using or plans to use an investigational agent for the prevention of GvHD.
Participant is receiving or plans to receive other investigational therapy and/or is enrolled or plans to enroll in a separate clinical study.
Participant, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
Participant has a psychiatric illness that would prevent the participant or legal guardian or representative from giving informed consent and/or assent.
Participant has a serious active disease or co-morbid medical condition, as judged by the investigator, which would interfere with the conduct of this study.
Participant is pregnant or lactating and does not agree to stop breastfeeding.
Any other condition that would cause a risk to the participant if he/she participated in the trial.
Participant has a known history of hypersensitivity to defibrotide or any of the excipients.
Participant had acute bleeding that is clinically significant within 24 hours before the start of study treatment, defined as either of the following:
Participant used any medication that increases the risk of bleeding within 24 hours before the start of study treatment, including, but not limited to, systemic heparin, low molecular weight heparin, heparin analogs, alteplase, streptokinase, urokinase, antithrombin III, oral anticoagulants including warfarin, and other agents that increase the risk of bleeding. Participants may have received heparin or other anticoagulants for routine central venous line management and intermittent dialysis or ultrafiltration. Fibrinolytic instillation for central venous line occlusion was also permitted. Note: Heparin used to keep catheters open was allowed (up to 100 U/kg/day).
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| USC Norris Cancer Center | Los Angeles | California | 90033 | United States | ||
| Mattel Children's Hospital UCLA |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36519326 | Derived | Hudspeth M, Mori S, Nachbaur D, Perez-Simon JA, Stolzel F, Riches M, Wu W, Zhang P, Agarwal S, Yakoub-Agha I. A phase II, prospective, randomized, open-label study of defibrotide added to standard-of-care prophylaxis for the prevention of acute graft-versus-host disease after allogeneic hematopoietic cell transplantation. Haematologica. 2023 Apr 1;108(4):1026-1038. doi: 10.3324/haematol.2022.281471. |
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Informed Consent and/or assent was obtained from participants, parents/legal guardians or representatives.
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| ID | Title | Description |
|---|---|---|
| FG000 | Defibrotide Prophylaxis | Participants were administered Defibrotide Prophylaxis solution intravenously by study personnel at a dose of 25 mg/kg/day (6.25 mg/kg via 2-hour IV infusion every 6 hours) and Standard of Care immunoprophylaxis according to local institutional guidelines, physician preference, and patient need. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 19, 2018 | May 12, 2021 |
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| Standard of Care |
| Drug |
Administered according to local institutional guidelines, physician preference, and patient need. |
|
| HSCT Day (Day +0 post-HSCT) through Day +180 post-HSCT |
| Kaplan-Meier Estimate of Grade B to D aGvHD-free Survival by Days +100 and +180 Post-HSCT | Grade B to D aGvHD was defined using the IBMTR Severity Index. Grade B is defined as Skin stage = 2 or Liver stage = 1 to 2 or GI stage = 1 to 2. Grade C is defined as Skin stage = 3 or Liver stage = 3 or GI stage = 3. Grade D is defined as a Skin stage = 4 or Liver stage = 4 or GI stage = 4. | HSCT Day (Day +0 post-HSCT) through Days +100 and +180 post-HSCT |
| Cumulative Incidence Percentage of Grade C to D aGvHD by Days +100 and +180 Post-HSCT | Cumulative Incidence Percentage of Grade C to D aGvHD was defined using the IBMTR Severity Index. Grade C is defined as Skin stage = 3 or Liver stage = 3 or GI stage = 3. Grade D is defined as a Skin stage = 4 or Liver stage = 4 or GI stage = 4. | HSCT Day (Day +0 post-HSCT) through Days +100 and +180 post-HSCT |
| Cumulative Incidence Percentage of Disease Relapse by Days +100 and +180 Post-HSCT | Disease relapse was defined by either morphological evidence of acute leukemia or Myelodysplastic syndrome (MDS) consistent with pre-transplant features, documented or not by biopsy. The event was defined as an increase in size of prior sites of disease or evidence of new sites of disease, documented or not by biopsy. Disease relapse was diagnosed when there was morphological or clinical evidence of the following: reappearance of leukemia blast cells in the peripheral blood, or >5% blasts in the bone marrow (BM), not attributable to another cause (eg, BM regeneration), or the appearance of previous or new dysplastic changes (MDS specific) within the BM, with or without falling donor chimerism, or the development of extramedullary leukemia or leukemic cells in the cerebral spinal fluid, or institution of therapy to treat relapsed disease, including donor lymphocyte infusion. | HSCT Day (Day +0 post-HSCT) through Days +100 and +180 post-HSCT |
| Cumulative Incidence Percentage of Systemic Steroids for the Treatment of aGvHD +180 Days Post-HSCT | For each treatment arm, the cumulative incidence rate of systemic steroid use for the treatment of aGvHD by Day +180 post-HSCT will be estimated using the cumulative incidence competing risk estimator. The calculation of the cumulative incidence rates and the stratified Gray's test was carried out using the LIFETEST procedure in SAS version 9.4. If the participant experienced a competing risk event, then the initiation date was used to calculate the time-to-event variable. | HSCT Day (Day +0 post-HSCT) through Day +180 post-HSCT |
| Change From Baseline to Days +100 and +180 Post-HSCT in the Physical Wellbeing Subscale as Measured by the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT physical wellbeing subscale scores range from a minimum of 0 to a maximum of 28, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Baseline through Days +100 and +180 post-HSCT |
| Change From Baseline to Days +100 and +180 Post-HSCT in the Social/Family Wellbeing Subscale as Measured by the FACT-BMT Questionnaire Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT social/family wellbeing subscale scores range from a minimum of 0 to a maximum of 28, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Baseline through Days +100 and +180 post-HSCT |
| Change From Baseline to Days +100 and +180 Post-HSCT in the Emotional Wellbeing Subscale as Measured by the FACT-BMT Questionnaire Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT emotional wellbeing subscale scores range from a minimum of 0 to a maximum of 24, with higher scores indicating better quality of life. | Baseline through Days +100 and +180 post-HSCT |
| Change From Baseline to Days +100 and +180 Post-HSCT in the Functional Wellbeing Subscale as Measured by the FACT-BMT Questionnaire Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT functional wellbeing subscale scores range from a minimum of 0 to a maximum of 28, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Baseline through Days +100 and +180 post HSCT |
| Change From Baseline to Days +100 and +180 Post-HSCT in the Bone Marrow Transplantation Subscale (BMTS) as Measured by the FACT-BMT Questionnaire Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT-BMT subscale scores range from a minimum of 0 to a maximum of 40, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Baseline through Days +100 and +180 post-HSCT |
| Change From Baseline to Days +100 and +180 Post-HSCT in the General (FACT-G) Questionnaire Score | The FACT-General (FACT-G) is a core component of the FACT-BMT, and includes 4 of the 5 subscales included in the FACT-BMT total score (FACT physical wellbeing score, FACT social/family wellbeing score, FACT emotional wellbeing score, the FACT functional wellbeing score). In line with this similarity, results of the FACT-G exhibited the same pattern as described for the FACT-BMT total score. The FACT-G scores range from a minimParticipants were age ≥16 years at baseline. | Baseline through Days +100 and +180 post-HSCT |
| Change From Baseline to Days +100 and +180 Post-HSCT in the FACT-BMT Total Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT-BMT total score range is from a minimum of 0 to a maximum of 148, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Baseline through Days +100 and +180 post-HSCT |
| Change From Baseline to Days +100 and +180 Post-HSCT in the Functional Assessment of Cancer Therapy-Bone Marrow Transplant-Trial Outcomes Index (FACT-BMT-TOI) | The FACT-BMT-TOI is defined as the sum of the FACT physical wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. Scores range from a minimum of 0 to a maximum of 96, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Baseline through Days +100 and +180 post-HSCT |
| Change From Baseline to Days +100 and +180 Post-HSCT in Participant Reported Outcomes Measured Based on the EQ-5D-5L Index Value for Health States | The 5-Level EuroQol-5D health questionnaire (EQ-5D-5L) index value, which is country-specific, was only performed for participants in the US. The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Change from the baseline assessment to Days +100 and +180 post-HSCT in the index value was assessed. The index value total range is from a minimum value of -1 to a maximum value of +1. A higher EQ-5D-5L index value represents better quality of life (QoL), thus a positive change in the index value represents improved QoL. | Baseline through Days +100 and +180 post-HSCT |
| Change From Baseline to Days +100 and +180 Post-HSCT in Participant Reported Outcomes as Measured Based on the EQ Visual Analog Scale (EQ VAS) | The EQ VAS score at baseline and each of the post-HSCT assessments was summarized and presented using descriptive statistics. A higher EQ VAS score represents better QoL. For each of the post-HSCT assessments, change between baseline and that assessment in the EQ VAS score was calculated similarly to the EQ-5D-5L index value for a specific participant and was summarized and presented using descriptive statistics. The score ranges from a minimum of 0 and a maximum of 100. A negative change in the score indicates a decrease in quality of life. | Baseline through Days +100 and +180 post-HSCT |
| Change From Baseline to Day +100 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Mobility Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +100 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Baseline through Day +100 post-HSCT |
| Change From Baseline to Day +180 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Mobility Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +180 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Baseline through Day +180 post-HSCT |
| Change From Baseline to Day +100 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Pain/Discomfort Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Days +100 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Baseline through Day +100 post-HSCT |
| Change From Baseline to Day +180 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Pain/Discomfort Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +180 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Baseline through Day +180 post-HSCT |
| Change From Baseline to Day +100 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Self-Care Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +100 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Baseline through Day +100 post-HSCT |
| Change From Baseline to Day +180 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Self-Care Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +180 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Baseline through Day +180 post-HSCT |
| Change From Baseline to Day +100 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Usual Activities Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +100 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Baseline through Day +100 post-HSCT |
| Change From Baseline to Day +180 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Usual Activities Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +180 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Baseline through Day +180 post-HSCT |
| Change From Baseline to Day +100 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Anxiety/Depression Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +100 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Baseline through Day +100 post-HSCT |
| Change From Baseline to Day +180 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Anxiety/Depression Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +180 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Baseline through Day +180 post-HSCT |
| Los Angeles |
| California |
| 90095 |
| United States |
| Stanford University | Palo Alto | California | 94304 | United States |
| University of California, San Francisco Medical Center | San Francisco | California | 94143 | United States |
| Mayo Clinic Jacksonville - PPDS | Jacksonville | Florida | 32224 | United States |
| Blood & Marrow Transplant Center | Orlando | Florida | 32804 | United States |
| Emory University Hospital | Atlanta | Georgia | 30322 | United States |
| Northwestern Memorial Hospital | Chicago | Illinois | 60611 | United States |
| University of Kansas Medical Center | Westwood | Kansas | 66205 | United States |
| James Graham Brown Cancer Center | Louisville | Kentucky | 40202 | United States |
| Ochsner Clinic Foundation | New Orleans | Louisiana | 70121 | United States |
| University of Nebraska Medical Center | Omaha | Nebraska | 68198 | United States |
| Montefiore Einstein Cancer Center | The Bronx | New York | 10467 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27514 | United States |
| Penn State Milton S Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| MUSC-Hollings Cancer Center | Charleston | South Carolina | 29425 | United States |
| VA Puget Sound Health Care System | Seattle | Washington | 98108 | United States |
| West Virginia University Hospital | Morgantown | West Virginia | 26506 | United States |
| Universitätsklinikum Innsbruck | Innsbruck | 6020 | Austria |
| Ordensklinikum Linz, Krankenhaus der Elisabethinen GmbH | Linz | 4020 | Austria |
| Cliniques Universitaires Saint-Luc | Brussels | 1200 | Belgium |
| UZ Gent | Ghent | 9000 | Belgium |
| UZ Leuven | Leuven | 3000 | Belgium |
| Specialized Hospital for Active Treatment of Haematological Diseases - Sofia | Sofia | 1756 | Bulgaria |
| Hôpital Maisonneuve-Rosemont | Montreal | H1T 2M4 | Canada |
| Sainte Justine Hospital | Montreal | H3T 1C5 | Canada |
| McGill University Health Center | Montreal | H4A 3J1 | Canada |
| Klinichki Bolnicki Centar Zagreb | Zagreb | 10000 | Croatia |
| Hospital d Instructions des Armees Percy | Clamart | 92141 | France |
| CHRU Lille | Lille | 59037 | France |
| Institut Universitaire du Cancer de Toulouse - Oncopole | Toulouse | 31059 | France |
| Institut Gustave Roussy | Villejuif | 94805 | France |
| Helios Klinikum Berlin Buch | Berlin | 13125 | Germany |
| Medizinische Universitätsklinik Knappschaftskrankenhaus | Bochum | 44892 | Germany |
| Klinikum Frankfurt (Oder) GmbH | Brandenburg | 15236 | Germany |
| Uniklinik Köln | Cologne | 50937 | Germany |
| Universitätsklinikum Carl Gustav Carus an der TU Dresden | Dresden | 01307 | Germany |
| University Medicine Göttingen Germany | Göttingen | 37075 | Germany |
| Universitaetsklinikum Halle (Saale) | Halle | 06120 | Germany |
| Universitatsklinikum Leipzig | Leipzig | 04103 | Germany |
| Klinikum Mannheim Universitätsklinikum gGmbH | Mannheim | 68167 | Germany |
| Klinikum rechts der Isar der Technischen Universität München | München | 81675 | Germany |
| Klinikum der Universitat Regensburg | Regensburg | 93053 | Germany |
| Attikon University General Hospital | Athens | 12462 | Greece |
| University General Hospital of Patras | Pátrai | 26500 | Greece |
| ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda Ca' Granda | Milan | 20162 | Italy |
| Azienda Ospedaliero Universitaria di Parma | Parma | 41236 | Italy |
| Ospedale Pediatrico Bambino Gesù | Roma | 00165 | Italy |
| Centrum Onkologii im. Marii Sklodowskiej-Curie | Warsaw | 00-001 | Poland |
| Dolnoslaskie Centrum Transplantacji Komorkowych z Krajowym Bankiem Dawcow Szpiku | Wroclaw | 53-439 | Poland |
| Instituto Português de Oncologia de Lisboa Francisco Gentil, E.P.E. | Lisbon | 1099 | Portugal |
| Hospital Universitario Marques de Valdecilla | A Coruña | 15006 | Spain |
| Hospital de Gran Canaria Doctor Negrin | Las Palmas de Gran Canaria | 35010 | Spain |
| Hospital Universitario Puerta de Hierro - Majadahonda | Madrid | 28222 | Spain |
| Complejo Asistencial Universitario de Salamanca - H. Clinico | Salamanca | 37007 | Spain |
| Hospital Universitario Virgen del Rocio | Seville | 41013 | Spain |
| Hospital Clinico Universitario de Valencia | Valencia | 41013 | Spain |
| Birmingham Heartlands Hospital | Birmingham | B9 5SS | United Kingdom |
| St James University Hospital | Leeds | LS9 7TF | United Kingdom |
| St. James University Hospital | Leeds | LS9 7TF | United Kingdom |
| Leicester Royal Infirmary | Leicester | LE1 5WW | United Kingdom |
| Manchester Royal Infirmary | Manchester | LE1 5WW | United Kingdom |
| Standard of Care Immunoprophylaxis |
Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Baseline characteristics were assessed using the Intent-to-Treat (ITT) Analysis Set.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Defibrotide Prophylaxis | Participants were administered Defibrotide Prophylaxis solution intravenously by study personnel at a dose of 25 mg/kg/day (6.25 mg/kg via 2-hour IV infusion every 6 hours) and Standard of Care immunoprophylaxis according to local institutional guidelines, physician preference, and patient need. |
| BG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Cumulative Incidence Percentage of Grade B to D Acute Graft Versus Host Disease (aGvHD) by Day +100 Post-Hematopoietic Stem Cell Transplant (HSCT) | Cumulative Incidence Percentage of Grade B to D aGvHD was defined using the International Bone Marrow Transplant Registry (IBMTR) Severity Index. Grade B is defined as Skin stage = 2 or Liver stage = 1 to 2 or GI stage = 1 to 2. Grade C is defined as Skin stage = 3 or Liver stage = 3 or GI stage = 3. Grade D is defined as a Skin stage = 4 or Liver stage = 4 or GI stage = 4. | Cumulative Incidence Rate of Grade B to D Acute Graft Versus Host Disease (aGvHD) was assessed using the Intent-to-Treat Analysis Set. | Posted | Number | cumulative incidence percentage | HSCT Day (Day +0 post-HSCT) through Day +100 post-HSCT |
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| Secondary | Cumulative Incidence Percentage of Grade B to D aGvHD by Day +180 Post-HSCT | Cumulative Incidence Percentage of Grade B to D aGvHD was defined using the IBMTR Severity Index. Grade B is defined as Skin stage = 2 or Liver stage = 1 to 2 or GI stage = 1 to 2. Grade C is defined as Skin stage = 3 or Liver stage = 3 or GI stage = 3. Grade D is defined as a Skin stage = 4 or Liver stage = 4 or GI stage = 4. | Cumulative Incidence Rate of Grade B to D Acute Graft Versus Host Disease (aGvHD) was assessed using the Intent-to-Treat Analysis Set. | Posted | Number | cumulative incidence percentage | HSCT Day (Day +0 post-HSCT) through Day +180 post-HSCT |
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| Secondary | Kaplan-Meier Estimate of Grade B to D aGvHD-free Survival by Days +100 and +180 Post-HSCT | Grade B to D aGvHD was defined using the IBMTR Severity Index. Grade B is defined as Skin stage = 2 or Liver stage = 1 to 2 or GI stage = 1 to 2. Grade C is defined as Skin stage = 3 or Liver stage = 3 or GI stage = 3. Grade D is defined as a Skin stage = 4 or Liver stage = 4 or GI stage = 4. | Kaplan-Meier Estimate of Grade B to D aGvHD-free Survival was assessed using the Intent-to-Treat Analysis Set. | Posted | Number | percentage of participants | HSCT Day (Day +0 post-HSCT) through Days +100 and +180 post-HSCT |
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| Secondary | Cumulative Incidence Percentage of Grade C to D aGvHD by Days +100 and +180 Post-HSCT | Cumulative Incidence Percentage of Grade C to D aGvHD was defined using the IBMTR Severity Index. Grade C is defined as Skin stage = 3 or Liver stage = 3 or GI stage = 3. Grade D is defined as a Skin stage = 4 or Liver stage = 4 or GI stage = 4. | Cumulative Incidence Rate of Grade C to D Acute Graft Versus Host Disease (aGvHD) was assessed using the Intent-to-Treat Analysis Set. | Posted | Number | cumulative incidence percentage | HSCT Day (Day +0 post-HSCT) through Days +100 and +180 post-HSCT |
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| Secondary | Cumulative Incidence Percentage of Disease Relapse by Days +100 and +180 Post-HSCT | Disease relapse was defined by either morphological evidence of acute leukemia or Myelodysplastic syndrome (MDS) consistent with pre-transplant features, documented or not by biopsy. The event was defined as an increase in size of prior sites of disease or evidence of new sites of disease, documented or not by biopsy. Disease relapse was diagnosed when there was morphological or clinical evidence of the following: reappearance of leukemia blast cells in the peripheral blood, or >5% blasts in the bone marrow (BM), not attributable to another cause (eg, BM regeneration), or the appearance of previous or new dysplastic changes (MDS specific) within the BM, with or without falling donor chimerism, or the development of extramedullary leukemia or leukemic cells in the cerebral spinal fluid, or institution of therapy to treat relapsed disease, including donor lymphocyte infusion. | Cumulative Incidence Rate of Disease Relapse was assessed using the Intent-to-Treat Analysis Set. | Posted | Number | cumulative incidence percentage | HSCT Day (Day +0 post-HSCT) through Days +100 and +180 post-HSCT |
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| Secondary | Cumulative Incidence Percentage of Systemic Steroids for the Treatment of aGvHD +180 Days Post-HSCT | For each treatment arm, the cumulative incidence rate of systemic steroid use for the treatment of aGvHD by Day +180 post-HSCT will be estimated using the cumulative incidence competing risk estimator. The calculation of the cumulative incidence rates and the stratified Gray's test was carried out using the LIFETEST procedure in SAS version 9.4. If the participant experienced a competing risk event, then the initiation date was used to calculate the time-to-event variable. | Cumulative Incidence Rate of Systemic Steroids for the Treatment of aGvHD was assessed using the Intent-to-Treat Analysis Set. | Posted | Number | cumulative incidence percentage | HSCT Day (Day +0 post-HSCT) through Day +180 post-HSCT |
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| Secondary | Change From Baseline to Days +100 and +180 Post-HSCT in the Physical Wellbeing Subscale as Measured by the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT physical wellbeing subscale scores range from a minimum of 0 to a maximum of 28, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Only participants with age >=16 years at baseline in the Safety Analysis Set were used in this analysis. | Posted | Mean | Standard Deviation | unit on a scale | Baseline through Days +100 and +180 post-HSCT |
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| Secondary | Change From Baseline to Days +100 and +180 Post-HSCT in the Social/Family Wellbeing Subscale as Measured by the FACT-BMT Questionnaire Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT social/family wellbeing subscale scores range from a minimum of 0 to a maximum of 28, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Only participants with age >=16 years at baseline in the Safety Analysis Set were used in this analysis. | Posted | Mean | Standard Deviation | unit on a scale | Baseline through Days +100 and +180 post-HSCT |
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| Secondary | Change From Baseline to Days +100 and +180 Post-HSCT in the Emotional Wellbeing Subscale as Measured by the FACT-BMT Questionnaire Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT emotional wellbeing subscale scores range from a minimum of 0 to a maximum of 24, with higher scores indicating better quality of life. | Only participants with age >=16 years at baseline in the Safety Analysis Set were used in this analysis. | Posted | Mean | Standard Deviation | unit on a scale | Baseline through Days +100 and +180 post-HSCT |
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| Secondary | Change From Baseline to Days +100 and +180 Post-HSCT in the Functional Wellbeing Subscale as Measured by the FACT-BMT Questionnaire Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT functional wellbeing subscale scores range from a minimum of 0 to a maximum of 28, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Only participants with age >=16 years at baseline in the Safety Analysis Set were used in this analysis. | Posted | Mean | Standard Deviation | unit on a scale | Baseline through Days +100 and +180 post HSCT |
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| Secondary | Change From Baseline to Days +100 and +180 Post-HSCT in the Bone Marrow Transplantation Subscale (BMTS) as Measured by the FACT-BMT Questionnaire Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT-BMT subscale scores range from a minimum of 0 to a maximum of 40, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Only participants with age >=16 years at baseline in the Safety Analysis Set were used in this analysis. | Posted | Mean | Standard Deviation | unit on a scale | Baseline through Days +100 and +180 post-HSCT |
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| Secondary | Change From Baseline to Days +100 and +180 Post-HSCT in the General (FACT-G) Questionnaire Score | The FACT-General (FACT-G) is a core component of the FACT-BMT, and includes 4 of the 5 subscales included in the FACT-BMT total score (FACT physical wellbeing score, FACT social/family wellbeing score, FACT emotional wellbeing score, the FACT functional wellbeing score). In line with this similarity, results of the FACT-G exhibited the same pattern as described for the FACT-BMT total score. The FACT-G scores range from a minimParticipants were age ≥16 years at baseline. | Only participants with age >=16 years at baseline in the Safety Analysis Set were used in this analysis. | Posted | Mean | Standard Deviation | unit on a scale | Baseline through Days +100 and +180 post-HSCT |
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| Secondary | Change From Baseline to Days +100 and +180 Post-HSCT in the FACT-BMT Total Score | The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) total score is the sum of the FACT physical wellbeing score, the FACT social/family wellbeing score, the FACT emotional wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. The FACT-BMT total score range is from a minimum of 0 to a maximum of 148, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Only participants with age >=16 years at baseline in the Safety Analysis Set were used in this analysis. | Posted | Mean | Standard Deviation | unit on a scale | Baseline through Days +100 and +180 post-HSCT |
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| Secondary | Change From Baseline to Days +100 and +180 Post-HSCT in the Functional Assessment of Cancer Therapy-Bone Marrow Transplant-Trial Outcomes Index (FACT-BMT-TOI) | The FACT-BMT-TOI is defined as the sum of the FACT physical wellbeing score, the FACT functional wellbeing score, and the FACT-BMT subscale. Scores range from a minimum of 0 to a maximum of 96, with higher scores indicating better quality of life. A negative change from baseline indicates a decrease in score. | Only participants with age >=16 years at baseline in the Safety Analysis Set were used in this analysis. | Posted | Mean | Standard Deviation | unit on a scale | Baseline through Days +100 and +180 post-HSCT |
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| Secondary | Change From Baseline to Days +100 and +180 Post-HSCT in Participant Reported Outcomes Measured Based on the EQ-5D-5L Index Value for Health States | The 5-Level EuroQol-5D health questionnaire (EQ-5D-5L) index value, which is country-specific, was only performed for participants in the US. The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Change from the baseline assessment to Days +100 and +180 post-HSCT in the index value was assessed. The index value total range is from a minimum value of -1 to a maximum value of +1. A higher EQ-5D-5L index value represents better quality of life (QoL), thus a positive change in the index value represents improved QoL. | Only participants with age >=16 years at baseline in the Safety Analysis Set were used in this analysis. | Posted | Mean | Standard Deviation | unit on a scale | Baseline through Days +100 and +180 post-HSCT |
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| Secondary | Change From Baseline to Days +100 and +180 Post-HSCT in Participant Reported Outcomes as Measured Based on the EQ Visual Analog Scale (EQ VAS) | The EQ VAS score at baseline and each of the post-HSCT assessments was summarized and presented using descriptive statistics. A higher EQ VAS score represents better QoL. For each of the post-HSCT assessments, change between baseline and that assessment in the EQ VAS score was calculated similarly to the EQ-5D-5L index value for a specific participant and was summarized and presented using descriptive statistics. The score ranges from a minimum of 0 and a maximum of 100. A negative change in the score indicates a decrease in quality of life. | Only participants with age >=16 years at baseline in the Safety Analysis Set were used in this analysis. | Posted | Mean | Standard Deviation | unit on a scale | Baseline through Days +100 and +180 post-HSCT |
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| Secondary | Change From Baseline to Day +100 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Mobility Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +100 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Only participants with age >=16 years at baseline in the Safety Analysis Set who were on the study at the time of the scheduled post-baseline assessment were included in the analysis. | Posted | Count of Participants | Participants | Baseline through Day +100 post-HSCT |
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| Secondary | Change From Baseline to Day +180 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Mobility Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +180 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Only participants with age >=16 years at baseline in the Safety Analysis Set who were on the study at the time of the scheduled post-baseline assessment were included in the analysis. | Posted | Count of Participants | Participants | Baseline through Day +180 post-HSCT |
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| Secondary | Change From Baseline to Day +100 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Pain/Discomfort Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Days +100 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Only participants with age >=16 years at baseline in the Safety Analysis Set who were on the study at the time of the scheduled post-baseline assessment were included in the analysis. | Posted | Count of Participants | Participants | Baseline through Day +100 post-HSCT |
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| Secondary | Change From Baseline to Day +180 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Pain/Discomfort Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +180 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Only participants with age >=16 years at baseline in the Safety Analysis Set who were on the study at the time of the scheduled post-baseline assessment were included in the analysis. | Posted | Count of Participants | Participants | Baseline through Day +180 post-HSCT |
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| Secondary | Change From Baseline to Day +100 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Self-Care Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +100 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Only participants with age >=16 years at baseline in the Safety Analysis Set who were on the study at the time of the scheduled post-baseline assessment were included in the analysis. | Posted | Count of Participants | Participants | Baseline through Day +100 post-HSCT |
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| Secondary | Change From Baseline to Day +180 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Self-Care Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +180 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Only participants with age >=16 years at baseline in the Safety Analysis Set who were on the study at the time of the scheduled post-baseline assessment were included in the analysis. | Posted | Count of Participants | Participants | Baseline through Day +180 post-HSCT |
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| Secondary | Change From Baseline to Day +100 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Usual Activities Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +100 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Only participants with age >=16 years at baseline in the Safety Analysis Set who were on the study at the time of the scheduled post-baseline assessment were included in the analysis. | Posted | Count of Participants | Participants | Baseline through Day +100 post-HSCT |
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| Secondary | Change From Baseline to Day +180 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Usual Activities Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +180 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Only participants with age >=16 years at baseline in the Safety Analysis Set who were on the study at the time of the scheduled post-baseline assessment were included in the analysis. | Posted | Count of Participants | Participants | Baseline through Day +180 post-HSCT |
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| Secondary | Change From Baseline to Day +100 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Anxiety/Depression Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +100 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Only participants with age >=16 years at baseline in the Safety Analysis Set who were on the study at the time of the scheduled post-baseline assessment were included in the analysis. | Posted | Count of Participants | Participants | Baseline through Day +100 post-HSCT |
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| Secondary | Change From Baseline to Day +180 Post-HSCT in 5-Level European Quality of Life (EQ-5D-5L) Anxiety/Depression Dimension for Participants With Age >=16 Years | The EQ-5D-5L is a standardized instrument for use as a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each of the 5 dimensions based on the descriptive system of the EQ-5D-5L, the numbers of participants for all categories (the 5 levels of reported problems) were assessed. For Day +180 Post-HSCT change between baseline in each dimension will be categorized as follows for a specific participant: Condition improved, if the reported level of problem is lower at that assessment than at baseline; Condition unchanged, if the reported level of problem remains the same; Condition deteriorated, if the reported level of problem is higher at that assessment than at baseline; Unknown, if the reported level of problem is not completed or missing either at baseline or at that assessment. | Only participants with age >=16 years at baseline in the Safety Analysis Set who were on the study at the time of the scheduled post-baseline assessment were included in the analysis. | Posted | Count of Participants | Participants | Baseline through Day +180 post-HSCT |
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All Adverse Events (AEs) and Serious Adverse Events (SAEs) were reported from the time of signed informed consent form (ICF) and were recorded up to Day +63 post-HSCT. SAEs considered by the investigator to be related to study drug or study procedures were reported up to Day +180 post-HSCT.
Treatment-emergent adverse events (TEAEs) were defined as events that occurred after randomization into this study. A TEAE is defined as any event with a start date on or after baseline through the end of the study, or any ongoing event that worsens in severity after baseline through the end of the study. Adverse events were assessed using the Safety Analysis Set.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Defibrotide Prophylaxis | Participants were administered Defibrotide Prophylaxis solution intravenously by study personnel at a dose of 25 mg/kg/day (6.25 mg/kg via 2-hour IV infusion every 6 hours) and Standard of Care immunoprophylaxis according to local institutional guidelines, physician preference, and patient need. | 10 | 74 | 31 | 74 | 74 | 74 |
| EG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone in a 1:1 ratio according to local institutional guidelines, physician preference, and patient need. | 9 | 70 | 31 | 70 | 70 | 70 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Obstructive shock | Vascular disorders | MedDRA 21.1 | Systematic Assessment |
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| Shock | Vascular disorders | MedDRA 21.1 | Systematic Assessment |
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| Venoocclusive disease | Vascular disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Acute lymphocytic leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 21.1 | Systematic Assessment |
| |
| Acute myeloid leukaemia recurrent | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 21.1 | Systematic Assessment |
| |
| Epstein-Barr virus associated lymphoproliferative disorder | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 21.1 | Systematic Assessment |
| |
| Acute graft versus host disease | Immune system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Acute graft versus host disease in skin | Immune system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Graft versus host disease | Immune system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Graft versus host disease in liver | Immune system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Acute graft versus host disease in intestine | Immune system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| General physical health deterioration | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Mucosal haemorrhage | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Mucosal inflammation | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Toxicity to various agents | Injury, poisoning and procedural complications | MedDRA 21.1 | Systematic Assessment |
| |
| Transfusion reaction | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Blood potassium decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Ejection fraction decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Atrial flutter | Cardiac disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Cardiac tamponade | Cardiac disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Cardio-respiratory arrest | Cardiac disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Cardiogenic shock | Cardiac disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Stress cardiomyopathy | Cardiac disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Supraventricular tachycardia | Cardiac disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Lung disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pharyngeal inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Eosinophilia | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Evans syndrome | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Thrombotic microangiopathy | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypertensive encephalopathy | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Acute abdomen | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Intestinal haemorrhage | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Oesophagitis | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Venoocclusive liver disease | Hepatobiliary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Cystitis haemorrhagic | Renal and urinary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Rash vesicular | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Toxic skin eruption | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Failure to thrive | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypovolaemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Arthritis bacterial | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Bacterial sepsis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Cystitis viral | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Cytomegalovirus viraemia | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Epstein-Barr viraemia | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Epstein-Barr virus infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Fungaemia | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Serratia infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Urinary tract infection bacterial | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Vascular device infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Flushing | Vascular disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Graft versus host disease in skin | Immune system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Mucosal inflammation | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 21.1 | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA 21.1 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 21.1 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 21.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Blood magnesium decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Epstein-Barr virus test positive | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hiccups | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Restless legs syndrome | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Eye swelling | Eye disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Odynophagia | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Oesophagitis | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Cystitis haemorrhagic | Renal and urinary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pruritus generalised | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Fluid overload | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Fluid retention | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypernatraemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypophagia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Malnutrition | Metabolism and nutrition disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
|
The sponsor can review trial results communications prior to public release and can embargo such communications for a period of at least 60 days from the time submitted to sponsor for review. If requested by sponsor, the PI will withhold publication for up to an additional 30 days. Furthermore, the first publication of study results must be a joint publication of all study sites unless a joint manuscript has not been submitted for publication within 12 months of completion of the study.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Director, Disclosure & Transparency | Jazz Pharmaceuticals | 215-870-9177 | ClinicalTrialDisclosure@JazzPharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 22, 2020 | May 12, 2021 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D006086 | Graft vs Host Disease |
| ID | Term |
|---|---|
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C036901 | defibrotide |
| D059039 | Standard of Care |
| ID | Term |
|---|---|
| D019984 | Quality Indicators, Health Care |
| D011787 | Quality of Health Care |
| D006298 | Health Services Administration |
| D017530 | Health Care Quality, Access, and Evaluation |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Counts |
|---|
| Participants |
|
|
| Participants |
|
|
| Participants |
|
|
Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
|
|
|
|
| OG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|
| OG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|
| OG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|
| OG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|
| OG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|
| OG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|
| OG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|
| OG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|
| OG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|
| OG001 | Standard of Care Immunoprophylaxis | Participants were administered Standard of Care immunoprophylaxis alone according to local institutional guidelines, physician preference, and patient need. |
|
|