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| ID | Type | Description | Link |
|---|---|---|---|
| BMT CTN Protocol 1705 | Other Identifier | Blood and Marrow Transplant Clinical Trials Network (BMT CTN) |
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| Name | Class |
|---|---|
| Blood and Marrow Transplant Clinical Trials Network | NETWORK |
| National Institutes of Health (NIH) | NIH |
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
Not provided
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Study CSL964_5001 will investigate the efficacy of AAT with corticosteroids compared with corticosteroids alone as first line therapy for patients with high-risk acute GVHD
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AAT | Experimental | Alpha-1 antitrypsin (AAT) is a lyophilized powder for intravenous administration |
|
| Placebo | Placebo Comparator | Albumin solution administered intravenously |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Alpha-1 antitrypsin (AAT) | Biological | AAT is a lyophilized product for intravenous administration |
|
| Measure | Description | Time Frame |
|---|---|---|
| Overall Response Rate (ORR) to Acute Graft-versus-Host Disease (GVHD) Treatment | The overall response is defined as having a CR or PR at Day 28, along with: being alive, free of any next-line GVHD therapy, and free of escalation of prednisone-equivalent steroid dose to 2.5 milligrams per kilogram (mg/kg)/day or more. The percentage of participants with CR or PR is reported here. Wilson score confidence intervals (CIs) are reported here as a measure of dispersion. The CR was defined as a score of 0 for the GVHD staging in all evaluable organs. The PR was defined as an improvement in one or more organs involved with GVHD symptoms without progression in others. Acute GVHD was graded and assessed for response based on Harris (Mount Sinai Acute GVHD International Consortium [MAGIC]) criteria (stage 0, 1, 2, 3, 4) for skin, liver, upper gastrointestinal (GI) tract, and lower GI tract. Participants who had an escalation of prednisone-equivalent steroid dose to 2.5 mg/kg/day or higher were classified as non-responders (NR). | At Day 28 |
| Measure | Description | Time Frame |
|---|---|---|
| Duration of Response (DOR) | The DOR was defined as time from the Day 28 response (CR or PR) until the first of the following events occurs: progression of acute GVHD, death, any next-line GVHD therapy, or escalation of prednisone-equivalent steroids to greater than or equal to (>=) 2.5 mg/kg/day. Wald CIs are reported here as a measure of dispersion. | Up to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| DOR - Supplementary Analysis | The DOR was defined as time from the Day 28 response (CR or PR) until the first of the following events occurs: death, any next-line GVHD therapy, or escalation of prednisone-equivalent steroids to >= 2.5 mg/kg/day. Wald CIs are reported here as a measure of dispersion. | Up to 12 months |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | CSL Behring | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University | Stanford | California | 94305 | United States | ||
| University of Florida |
CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
Proposed research should seek to answer a previously unanswered important medical or scientific question.
Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.
If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
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A total of 136 participants were randomized in this study.
This study was conducted at 26 investigative sites in the United States of America.
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| ID | Title | Description |
|---|---|---|
| FG000 | Alpha-1 Antitrypsin (AAT) | Participants received AAT twice weekly through Day 28. Responding participants (Complete Response/Partial Response [CR/PR]) continued to receive a once weekly dose of AAT through Day 56. |
| FG001 | Placebo |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 22, 2023 | Jun 9, 2025 |
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| National Cancer Institute (NCI) |
| NIH |
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| Placebo | Drug | Albumin solution administered intravenously |
|
| Number of Participants With Non-relapse Mortality (NRM) Event | An event of NRM was death without prior evidence of relapse/progression of the primary disease, where relapse/progression was treated as a competing risk. Cumulative incidence of NRM at 6 and 12 months post-randomization is reported here for this outcome measure. | At 6 and 12 months |
| Number of Overall and Progression-free Survival Events | An event for overall survival (OS) was death from any cause, while an event for progression-free survival (PFS) was death from any cause or relapse/progression of the primary disease. | At 6 and 12 months |
| Number of Participants With GVHD-free Survival | GVHD-free survival was defined as participants being alive, free of acute or chronic GVHD, and free of any next-line GVHD therapy or escalation of steroids to >=2.5 mg/kg/day prednisone or equivalent for treatment of GVHD. | At Day 56 |
| Percentage of Participants With Response | The percentage of participants with CR, PR (including subset with very good partial response [VGPR]), and treatment failure (TF). The designation of TF consisted of participants with NR, mixed response (MR) or progression. The initiation of additional systemic (next-line) GVHD therapies, escalation of prednisone equivalent steroid dose to >= 2.5 mg/kg/day, or death from any cause prior to the assessment timepoint was also considered a TF. Simultaneous Goodman CIs are provided for category proportions for each arm at each assessment time. | At Day 7, 14, 21, 28, 56, and 86 |
| Percentage of Participants With Response Allowing for Approved Next-line Therapy | The percentage of participants with CR, PR (including subset with VGPR), and TF allowing for treatment with next-line therapy. The designation of TF consisted of participants with NR, MR, or progression. The escalation of prednisone-equivalent steroid dose to >= 2.5 mg/kg/day or death from any cause prior to the assessment timepoint was also considered a TF. Simultaneous Goodman CIs are provided for category proportions for each arm at each assessment time. | At Day 7, 14, 21, 28, 56, and 86 |
| Incidence of Grade 2 to 3 Systemic Infections | The cumulative incidence of Grade 2 to 3 systemic infections. Grade 2 to 3 systemic infections were defined according to the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Manual of Procedures. | Up to 90 days (including 30 days after last dose of study drug) |
| Percentage of Participants With Grade 3 to 5 Treatment-emergent Adverse Events (TEAEs) | The incidence of Grade 3 to 5 TEAEs (per Common Terminology Criteria for Adverse Events [CTCAE] Version 5.0). Wilson score CIs are reported here as a measure of dispersion. | Up to 30 days after the last dose of study drug |
| Cumulative Incidence of Chronic GVHD | Chronic GVHD was defined per National Institutes of Health (NIH) Consensus Criteria. Diagnosis of chronic GVHD of any severity (mild, moderate, or severe) was considered an event for this outcome measure, where death before cGVHD was treated as a competing risk. | At 6 and 12 months |
| Cumulative Incidence of Disease Relapse/ Progression of Primary Disease | The cumulative incidence of relapse/progression of the primary disease, with death prior to relapse/progression treated as a competing risk. Death without prior relapse/progression was treated as a competing risk for relapse/progression. | At 6 months and 12 months |
| Gainesville |
| Florida |
| 32608 |
| United States |
| Moffitt Cancer Center | Tampa | Florida | 33612 | United States |
| Emory University | Atlanta | Georgia | 30322-1059 | United States |
| Northside Hospital | Atlanta | Georgia | 30342 | United States |
| IU Hospital | Indianapolis | Indiana | 46202 | United States |
| Univ. of Kansas Cancer Center | Westwood | Kansas | 66205 | United States |
| Johns Hopkins Hospital | Baltimore | Maryland | 21287 | United States |
| Dana Farber Cancer Institute | Boston | Massachusetts | 02215 | United States |
| U-M Medical Center | Ann Arbor | Michigan | 48109-0312 | United States |
| Karmanos Cancer Center | Detroit | Michigan | 48201 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| MSKCC | New York | New York | 10065 | United States |
| Levine Cancer Center | Charlotte | North Carolina | 28204 | United States |
| Duke | Durham | North Carolina | 27705 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44124 | United States |
| Ohio State Univ.Medical Center | Columbus | Ohio | 43210 | United States |
| Vanderbilt-Ingram Cancer Center | Nashville | Tennessee | 37232 | United States |
| Center for Gene Therapy | Houston | Texas | 77030 | United States |
| MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
| Virginia Commonwealth Univ. | Richmond | Virginia | 23298 | United States |
| Fred Hutchinson Clinic | Seattle | Washington | 98109 | United States |
| University of Wisconsin | Madison | Wisconsin | 53705 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
Participants received albumin solution administered intravenously (IV) to match the investigational medicinal product (IMP) twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of matching placebo through Day 56.
| Treated |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Analysis was performed on the intent-to-treat (ITT) population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Alpha-1 Antitrypsin (AAT) | Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56. |
| BG001 | Placebo | Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| 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 | Overall Response Rate (ORR) to Acute Graft-versus-Host Disease (GVHD) Treatment | The overall response is defined as having a CR or PR at Day 28, along with: being alive, free of any next-line GVHD therapy, and free of escalation of prednisone-equivalent steroid dose to 2.5 milligrams per kilogram (mg/kg)/day or more. The percentage of participants with CR or PR is reported here. Wilson score confidence intervals (CIs) are reported here as a measure of dispersion. The CR was defined as a score of 0 for the GVHD staging in all evaluable organs. The PR was defined as an improvement in one or more organs involved with GVHD symptoms without progression in others. Acute GVHD was graded and assessed for response based on Harris (Mount Sinai Acute GVHD International Consortium [MAGIC]) criteria (stage 0, 1, 2, 3, 4) for skin, liver, upper gastrointestinal (GI) tract, and lower GI tract. Participants who had an escalation of prednisone-equivalent steroid dose to 2.5 mg/kg/day or higher were classified as non-responders (NR). | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Posted | Number | 95% Confidence Interval | percentage of participants | At Day 28 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Duration of Response (DOR) | The DOR was defined as time from the Day 28 response (CR or PR) until the first of the following events occurs: progression of acute GVHD, death, any next-line GVHD therapy, or escalation of prednisone-equivalent steroids to greater than or equal to (>=) 2.5 mg/kg/day. Wald CIs are reported here as a measure of dispersion. | Analysis was performed on the ITT population participants with a response as defined in the measure description of the primary outcome measure. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Posted | Median | 95% Confidence Interval | days | Up to 12 months |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Non-relapse Mortality (NRM) Event | An event of NRM was death without prior evidence of relapse/progression of the primary disease, where relapse/progression was treated as a competing risk. Cumulative incidence of NRM at 6 and 12 months post-randomization is reported here for this outcome measure. | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Posted | Count of Participants | Participants | At 6 and 12 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Overall and Progression-free Survival Events | An event for overall survival (OS) was death from any cause, while an event for progression-free survival (PFS) was death from any cause or relapse/progression of the primary disease. | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Posted | Number | events | At 6 and 12 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With GVHD-free Survival | GVHD-free survival was defined as participants being alive, free of acute or chronic GVHD, and free of any next-line GVHD therapy or escalation of steroids to >=2.5 mg/kg/day prednisone or equivalent for treatment of GVHD. | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Posted | Count of Participants | Participants | At Day 56 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Response | The percentage of participants with CR, PR (including subset with very good partial response [VGPR]), and treatment failure (TF). The designation of TF consisted of participants with NR, mixed response (MR) or progression. The initiation of additional systemic (next-line) GVHD therapies, escalation of prednisone equivalent steroid dose to >= 2.5 mg/kg/day, or death from any cause prior to the assessment timepoint was also considered a TF. Simultaneous Goodman CIs are provided for category proportions for each arm at each assessment time. | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. Here, the 'number analyzed' for Day 86 includes responses for only those participants who remained on maintenance treatment after Day 28. | Posted | Number | 95% Confidence Interval | percentage of participants | At Day 7, 14, 21, 28, 56, and 86 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Response Allowing for Approved Next-line Therapy | The percentage of participants with CR, PR (including subset with VGPR), and TF allowing for treatment with next-line therapy. The designation of TF consisted of participants with NR, MR, or progression. The escalation of prednisone-equivalent steroid dose to >= 2.5 mg/kg/day or death from any cause prior to the assessment timepoint was also considered a TF. Simultaneous Goodman CIs are provided for category proportions for each arm at each assessment time. | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. Here, the 'number analyzed' for Day 86 includes responses for only those participants who remained on maintenance treatment after Day 28. | Posted | Number | 95% Confidence Interval | percentage of participants | At Day 7, 14, 21, 28, 56, and 86 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Incidence of Grade 2 to 3 Systemic Infections | The cumulative incidence of Grade 2 to 3 systemic infections. Grade 2 to 3 systemic infections were defined according to the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Manual of Procedures. | Analysis was performed on the safety population. The safety population included all randomized participants who received at least one dose of study treatment. Participants were classified according to the treatment received. | Posted | Number | events | Up to 90 days (including 30 days after last dose of study drug) |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Grade 3 to 5 Treatment-emergent Adverse Events (TEAEs) | The incidence of Grade 3 to 5 TEAEs (per Common Terminology Criteria for Adverse Events [CTCAE] Version 5.0). Wilson score CIs are reported here as a measure of dispersion. | Analysis was performed on the safety population. The safety population included all randomized participants who received at least one dose of study treatment. Participants were classified according to the treatment received. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to 30 days after the last dose of study drug |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Cumulative Incidence of Chronic GVHD | Chronic GVHD was defined per National Institutes of Health (NIH) Consensus Criteria. Diagnosis of chronic GVHD of any severity (mild, moderate, or severe) was considered an event for this outcome measure, where death before cGVHD was treated as a competing risk. | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Posted | Count of Participants | Participants | At 6 and 12 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Cumulative Incidence of Disease Relapse/ Progression of Primary Disease | The cumulative incidence of relapse/progression of the primary disease, with death prior to relapse/progression treated as a competing risk. Death without prior relapse/progression was treated as a competing risk for relapse/progression. | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Posted | Count of Participants | Participants | At 6 months and 12 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | DOR - Supplementary Analysis | The DOR was defined as time from the Day 28 response (CR or PR) until the first of the following events occurs: death, any next-line GVHD therapy, or escalation of prednisone-equivalent steroids to >= 2.5 mg/kg/day. Wald CIs are reported here as a measure of dispersion. | Analysis was performed on the ITT population participants with a response as defined in the measure description of the primary outcome measure. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Posted | Median | 95% Confidence Interval | days | Up to 12 months |
|
|
From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
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 | Alpha-1 Antitrypsin (AAT) | Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56. | 21 | 65 | 11 | 63 | 37 | 63 |
| EG001 | Placebo | Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56. | 23 | 71 | 23 | 69 | 42 | 69 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA version 27.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA version 27.0 | Systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA version 27.0 | Systematic Assessment |
| |
| Vascular access complication | Injury, poisoning and procedural complications | MedDRA version 27.0 | Systematic Assessment |
| |
| Intracranial mass | Nervous system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Toxic encephalopathy | Nervous system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Depressed level of consciousness | Nervous system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Metabolic encephalopathy | Nervous system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Small intestinal haemorrhage | Gastrointestinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Diabetic ketoacidosis | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Failure to thrive | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Thrombotic microangiopathy | Blood and lymphatic system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Haemolytic uraemic syndrome | Blood and lymphatic system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Methaemoglobinaemia | Blood and lymphatic system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Atrial tachycardia | Cardiac disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Myocardial ischaemia | Cardiac disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Sinus bradycardia | Cardiac disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Embolism | Vascular disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hepatic failure | Hepatobiliary disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hallucination | Psychiatric disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA version 27.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hyperphosphataemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypermagnesaemia | Metabolism and nutrition disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA version 27.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA version 27.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA version 27.0 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA version 27.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA version 27.0 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA version 27.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA version 27.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA version 27.0 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA version 27.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Study Disclosure Manager | CSL Behring | 1- 610-878-4697 | clinicaltrials@cslbehring.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 8, 2024 | Jul 30, 2025 | SAP_002.pdf |
Not provided
| ID | Term |
|---|---|
| D006086 | Graft vs Host Disease |
| ID | Term |
|---|---|
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D000515 | alpha 1-Antitrypsin |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D015843 | Serpins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000209 | Acute-Phase Proteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000510 | Alpha-Globulins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
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 |
|
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| Participants |
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| Participants |
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