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| ID | Type | Description | Link |
|---|---|---|---|
| 2015-000726-11 | EudraCT Number |
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Following a pre-scheduled interim analysis performed by the DMC, it was determined that the study met the pre-specified criteria for futility.
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The main purpose of the study is to evaluate the efficacy of CINRYZE administered with plasmapheresis, plasma exchange, or immune adsorption treatments and sucrose-free immunoglobulin (IVIg) for the treatment of acute antibody-mediated rejection (AMR) of renal allograft in kidney transplant recipients as measured by the proportion of participants with new or worsening transplant glomerulopathy (TG) at 6 months after treatment initiation.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cinryze® | Experimental | Participants will receive 5000 Units of CINRYZE (50 millilitre [mL] of CINRYZE/ 50 mL of normal saline) on Day 1 and 2500 Units of CINRYZE (25 mL of CINRYZE/ 75 mL of normal saline) on Day 3, 5, 7, 9, 11, and 13 respectively. |
|
| Placebo | Placebo Comparator | Participants will receive 7 doses of matched placebo over 13 days of treatment. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cinryze® | Biological | Participants will receive 5000 Units of CINRYZE (50 millilitre [mL] of CINRYZE/ 50 mL of normal saline) on Day 1 and 2500 Units of CINRYZE (25 mL of CINRYZE/ 75 mL of normal saline) on Day 3, 5, 7, 9, 11, and 13 respectively. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With New or Worsening Transplant Glomerulopathy (TG) at Month 6 Post-Treatment | New or worsening TG at month 6 by the standard score was defined as an increase in one or more between qualifying biopsy and 6-month biopsy. New or worsening TG was measured by Banff 2013 criteria (standard score) using allograft glomerulopathy (Cg0-Cg3): Cg0- No GBM double contours by light microscopy (LM) or electron microscopy (EM); Cg1- no GBM double contours by LM but GBM double contours in at least 3 glomerular capillaries by EM; Cg2- Double contours affecting 26 to 50% of peripheral capillary loops in the most affected of nonsclerotic glomeruli; Cg3- Double contours affecting more than 50% of peripheral capillary loops in the most affected of nonsclerotic glomeruli with a score range of 0 (no allograft glomerulopathy) and 3 (severe glomerulopathy). Percentage of participants with new or worsening TG at Month 6 post-treatment was reported. | Month 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With All-Cause Graft Failure at Month 48 | Graft failure was determined as the presence of one or more of the following criteria: institution of permanent dialysis (defined as dialysis treatment more than [>] 30 days), current transplant nephrectomy, and/or a clinical determination of cessation of kidney graft function and estimated glomerular filtration rate (eGFR) less than or equal to (<=) 15 milliliter (mL)/minute (min)/1.73 meter (m)^2. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Shire | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kidney Transplant Research Office at UCLA | Los Angeles | California | 90024 | United States | ||
| Keck School of Medicine at USC |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35332298 | Derived | Karpman D, Bekassy Z, Grunenwald A, Roumenina LT. A role for complement blockade in kidney transplantation. Cell Mol Immunol. 2022 Jul;19(7):755-757. doi: 10.1038/s41423-022-00854-5. Epub 2022 Mar 24. No abstract available. |
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Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
A total of 39 participants were randomized and received treatment.
The study was conducted at 49 sites between 20 May 2016 (first participant first visit) and 31 May 2019 (last participant last visit).
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants received a total of seven doses with an initial 100 mL intravenous (IV) infusion containing 5000 units of placebo (100 mL 0.9 percent [%] sodium chloride) on day 1 followed by 2500 units of placebo (100 mL 0.9% sodium chloride) on Days 3, 5, 7, 9, 11, and 13. |
| 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 | Nov 22, 2017 | May 29, 2020 |
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| Placebo | Drug | Participants will receive 7 doses of matched placebo over 13 days of treatment. |
|
| Month 48 |
| Change From Baseline in Renal Function up to Month 48 | Renal function was measured as glomerular filtration rate calculated by the modification of diet in renal disease (eGFRMDRD). | Baseline, up to Month 48 |
| Change From Baseline With Pre-Antibody-Mediated Rejection (AMR) in Renal Function up to Month 48 | Renal function was measured as glomerular filtration rate calculated by the modification of diet in renal disease (eGFRMDRD). Pre-AMR baseline was the highest eGFRMDRD value obtained following the kidney transplant and within 30 days prior to the qualifying AMR episode. | Pre-AMR Baseline, up to Month 48 |
| Number of Participants With Proteinuria Levels at Month 48 | Proteinuria included spot urine protein, urine creatinine, and urine protein/urine creatinine ratio. | Month 48 |
| Change From Pre-Antibody-Mediated Rejection (AMR) Baseline in Histopathology Per Banff Criteria at Month 6 | Histopathological diagnosis of acute rejection was measured by Banff 2013 criteria: Glomerulitis score (g0-g3), allograft glomerulopathy (Cg0-cg3), Tubulitis score (T0-T3), Intimal arteritis score (V0-V3), peritubular capillaritis (PTC) (ptc0-ptc3) and Interstitial Inflammation score (i0-i3). The histopathology was a composite of the sub-scores. Each of the sub-scores or histopathology score ranges from 0 ( no histopathology) to 3 (more severe histopathology). | Pre-AMR Baseline, Month 6 |
| Number of Participants With All-Cause Graft Failure at Month 6 | Graft failure was determined by the presence of one or more of the following criteria: institution of permanent dialysis (defined as dialysis treatment more than [>] 30 days), current transplant nephrectomy, and/or a clinical determination of cessation of kidney graft function and estimated glomerular filtration rate (eGFR) less than or equal to (<=) 15 milliliter (mL)/minute (min)/1.73 meter (m)^2. | Month 6 |
| Number of Participants With Graft Failure Due to Antibody-Mediated Rejection (AMR) Episodes at Month 48 | Graft failure was determined by the presence of one or more of the following criteria: institution of permanent dialysis (defined as dialysis treatment more than [>] 30 days), current transplant nephrectomy, and/or a clinical determination of cessation of kidney graft function and estimated glomerular filtration rate (eGFR) less than or equal to (<=) 15 milliliter (mL)/minute (min)/1.73 meter (m)^2. | Month 48 |
| Time to All-Cause Graft Failure up to Month 48 | Graft failure was determined by the presence of one or more of the following criteria: institution of permanent dialysis (defined as dialysis treatment more than [>] 30 days), current transplant nephrectomy, and/or a clinical determination of cessation of kidney graft function and estimated glomerular filtration rate (eGFR) less than or equal to (<=) 15 milliliter (mL)/minute (min)/1.73 meter (m)^2. Time to all-cause graft failure in months was calculated as (Date of graft failure - Date of first dose + 1)/30.25. | Up to Month 48 |
| Time to Graft Failure Due to Antibody-Mediated Rejection (AMR) Episodes up to Month 48 | Graft failure was determined by the presence of one or more of the following criteria: institution of permanent dialysis (defined as dialysis treatment > 30 days), current transplant nephrectomy, and/or a clinical determination of cessation of kidney graft function and eGFR <=15 mL/ min/1.73m^2. Time to graft failure due to AMR episodes in months was calculated as (Date of graft failure due to AMR - Date of first dose + 1)/30.25. | Up to Month 48 |
| Number of Participants With Resolution of the Qualifying Antibody-Mediated Rejection (AMR) Episodes at Month 48 | Number of participants with resolution of the qualifying AMR episodes at Month 48. | Month 48 |
| Time to Resolution of Qualifying Antibody-Mediated Rejection (AMR) Episodes up to Month 48 | Time to resolution of qualifying AMR episodes was calculated as (Date of qualifying AMR resolution - Date of first dose + 1)/30.25. Participants who didn't had resolution of qualifying AMR episodes and still on-study were censored at the date of last visit; Participants who had completed the study without resolution of qualifying AMR were censored at the date of study completion; participants who discontinued from the study without resolution of qualifying AMR were censored at the date of early discontinuation. | Up to Month 48 |
| Number of Participants Who Were Alive at Month 36 | Number of participants who were alive at Month 36 (study terminated instead of Month 48) were reported. | Month 36 |
| Time to All-Cause Mortality up to Month 48 | Time to all-cause mortality was calculated as (Date of discontinuation due to death - Date of first dose + 1)/30.25. Participants who are alive and still on-study were censored at the date of last visit; Participants who had completed the study were censored at the date of study completion; Participants who discontinued from the study but not due to death were censored at the date of early discontinuation. | Up to Month 48 |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) was any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurred in a participant participating in a clinical study with the sponsor's product, regardless of causal relationship. TEAEs were defined as events that started or worsened on or after the date of the first dose of investigational product, but no later than 30 days following the last dose of investigational product, within a treatment period. | From start of study drug administration up to study termination (Month 36) |
| Los Angeles |
| California |
| 90033 |
| United States |
| University of California San Francisco | San Francisco | California | 94110 | United States |
| University Of Colorado School Of Medicine | Aurora | Colorado | 80045 | United States |
| MedStar Georgetown University Hospital | Washington D.C. | District of Columbia | 20057 | United States |
| Jackson Memorial Hospital | Miami | Florida | 33136 | United States |
| Florida Hospital Transplant Institute | Orlando | Florida | 32804 | United States |
| Piedmont Hospital | Atlanta | Georgia | 30309 | United States |
| Northwestern Memorial Hospital | Chicago | Illinois | 60611 | United States |
| University of Maryland Medical Center | Baltimore | Maryland | 21201 | United States |
| Johns Hopkins University | Baltimore | Maryland | 21287 | United States |
| Brigham and Womens Hospital | Boston | Massachusetts | 02115 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55414 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Saint Barnabas Medical Center | Livingston | New Jersey | 07039 | United States |
| Mount Sinai Hospital | New York | New York | 10029 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| New York Presbyterian Hospital - Weill-Cornell | New York | New York | 10065 | United States |
| NYU Longone Medical Center | White Plains | New York | 10605 | United States |
| University of Cincinnati | Cincinnati | Ohio | 45220 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Ohio State University Medical Center | Columbus | Ohio | 43210 | United States |
| INTEGRIS Nazih Zuhdi Transplant Institute | Oklahoma City | Oklahoma | 73112 | United States |
| University of Pittsburgh Medical Center | Monroeville | Pennsylvania | 15146 | United States |
| University of Pennsylvania Health System | Philadelphia | Pennsylvania | 19104 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37204 | United States |
| Baylor All Saints Medical Center | Fort Worth | Texas | 76104 | United States |
| Houston Methodist Hospital | Houston | Texas | 77030 | United States |
| University of Utah Health Sciences Center | Salt Lake City | Utah | 84132 | United States |
| Providence Health Care Research Institute | Vancouver | British Columbia | V6Z 2K5 | Canada |
| Royal Victoria Hospital | Montreal | Quebec | H4A 3J1 | Canada |
| Hotel Dieu | Nantes | Loire-Atlantique | 44093 | France |
| Hopital Henri Mondor | Créteil | Val-De-Marne | 94010 | France |
| CHU Michallon | Grenoble | 38043 | France |
| Centre Hospitalier Universitaire de Bicêtre | Le Kremlin-Bicêtre | 94275 | France |
| Hôpital Saint Louis | Paris | 75010 | France |
| Groupe Hospitalier Necker Enfants Malades | Paris | 75015 | France |
| Hôpital de Rangueil | Toulouse | 31059 | France |
| Universität Heidelberg | Heidelberg | Baden-Wurttemberg | 69120 | Germany |
| Universitätsklinikum Frankfurt | Steinbach | Hesse | 61449 | Germany |
| Universitätsklinikum Hamburg Eppendorf | Hamburg | 20246 | Germany |
| Universitair Medisch Centrum Groningen | Groningen | 9713 GZ | Netherlands |
| Erasmus MC | Rotterdam | 3015 CE | Netherlands |
| Hospital Universitario Germans Trias i Pujol | Badalona | Barcelona | 08916 | Spain |
| Hospital Universitari de Bellvitge | Barcelona | 8907 | Spain |
| Hospital Clinico San Carlos | Madrid | 28040 | Spain |
| Hospital Universitario 12 de Octubre | Madrid | 28041 | Spain |
| CINRYZE |
Participants received a total of seven doses with an initial 100 mL intravenous (IV) infusion containing 5000 units of CINRYZE on Day 1 followed by 2500 units of CINRYZE in 100 mL of IV infusion on Day 3, 5, 7, 9, 11, and 13. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Full analysis set (FAS) consisted of all participants who had taken at least 1 dose of investigational product.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants received a total of seven doses with an initial 100 mL intravenous (IV) infusion containing 5000 units of placebo (100 mL 0.9% sodium chloride) on day 1 followed by 2500 units of placebo (100 mL 0.9% sodium chloride) on Days 3, 5, 7, 9, 11, and 13. |
| BG001 | CINRYZE | Participants received a total of seven doses with an initial 100 mL intravenous (IV) infusion containing 5000 units of CINRYZE on Day 1 followed by 2500 units of CINRYZE in 100 mL of IV infusion on Day 3, 5, 7, 9, 11, and 13. |
| 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/Ethnicity, Customized | 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 | Percentage of Participants With New or Worsening Transplant Glomerulopathy (TG) at Month 6 Post-Treatment | New or worsening TG at month 6 by the standard score was defined as an increase in one or more between qualifying biopsy and 6-month biopsy. New or worsening TG was measured by Banff 2013 criteria (standard score) using allograft glomerulopathy (Cg0-Cg3): Cg0- No GBM double contours by light microscopy (LM) or electron microscopy (EM); Cg1- no GBM double contours by LM but GBM double contours in at least 3 glomerular capillaries by EM; Cg2- Double contours affecting 26 to 50% of peripheral capillary loops in the most affected of nonsclerotic glomeruli; Cg3- Double contours affecting more than 50% of peripheral capillary loops in the most affected of nonsclerotic glomeruli with a score range of 0 (no allograft glomerulopathy) and 3 (severe glomerulopathy). Percentage of participants with new or worsening TG at Month 6 post-treatment was reported. | FAS consisted of all participants who had taken at least 1 dose of investigational product. | Posted | Number | Percentage of participants | Month 6 |
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| Secondary | Number of Participants With All-Cause Graft Failure at Month 48 | Graft failure was determined as the presence of one or more of the following criteria: institution of permanent dialysis (defined as dialysis treatment more than [>] 30 days), current transplant nephrectomy, and/or a clinical determination of cessation of kidney graft function and estimated glomerular filtration rate (eGFR) less than or equal to (<=) 15 milliliter (mL)/minute (min)/1.73 meter (m)^2. | This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. Hence, data for this outcome measure was not collected as planned, analyzed and reported. | Posted | Month 48 |
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| Secondary | Change From Baseline in Renal Function up to Month 48 | Renal function was measured as glomerular filtration rate calculated by the modification of diet in renal disease (eGFRMDRD). | This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. Hence, data for this outcome measure was not collected as planned, analyzed and reported. | Posted | Baseline, up to Month 48 |
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| Secondary | Change From Baseline With Pre-Antibody-Mediated Rejection (AMR) in Renal Function up to Month 48 | Renal function was measured as glomerular filtration rate calculated by the modification of diet in renal disease (eGFRMDRD). Pre-AMR baseline was the highest eGFRMDRD value obtained following the kidney transplant and within 30 days prior to the qualifying AMR episode. | This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. Hence, data for this outcome measure was not collected as planned, analyzed and reported. | Posted | Pre-AMR Baseline, up to Month 48 |
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| Secondary | Number of Participants With Proteinuria Levels at Month 48 | Proteinuria included spot urine protein, urine creatinine, and urine protein/urine creatinine ratio. | This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. Hence, data for this outcome measure was not collected as planned, analyzed and reported. | Posted | Month 48 |
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| Secondary | Change From Pre-Antibody-Mediated Rejection (AMR) Baseline in Histopathology Per Banff Criteria at Month 6 | Histopathological diagnosis of acute rejection was measured by Banff 2013 criteria: Glomerulitis score (g0-g3), allograft glomerulopathy (Cg0-cg3), Tubulitis score (T0-T3), Intimal arteritis score (V0-V3), peritubular capillaritis (PTC) (ptc0-ptc3) and Interstitial Inflammation score (i0-i3). The histopathology was a composite of the sub-scores. Each of the sub-scores or histopathology score ranges from 0 ( no histopathology) to 3 (more severe histopathology). | This study was prematurely terminated at Month 36 due to futility issues. The data of this secondary outcome measure was not collected as planned and the secondary analysis was not performed, due to early study termination. | Posted | Pre-AMR Baseline, Month 6 |
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| Secondary | Number of Participants With All-Cause Graft Failure at Month 6 | Graft failure was determined by the presence of one or more of the following criteria: institution of permanent dialysis (defined as dialysis treatment more than [>] 30 days), current transplant nephrectomy, and/or a clinical determination of cessation of kidney graft function and estimated glomerular filtration rate (eGFR) less than or equal to (<=) 15 milliliter (mL)/minute (min)/1.73 meter (m)^2. | This study was prematurely terminated at Month 36 due to futility issues. The data of this secondary outcome measure was not collected as planned and the secondary analysis was not performed, due to early study termination. | Posted | Month 6 |
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| Secondary | Number of Participants With Graft Failure Due to Antibody-Mediated Rejection (AMR) Episodes at Month 48 | Graft failure was determined by the presence of one or more of the following criteria: institution of permanent dialysis (defined as dialysis treatment more than [>] 30 days), current transplant nephrectomy, and/or a clinical determination of cessation of kidney graft function and estimated glomerular filtration rate (eGFR) less than or equal to (<=) 15 milliliter (mL)/minute (min)/1.73 meter (m)^2. | This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. Hence, data for this outcome measure was not collected as planned, analyzed and reported. | Posted | Month 48 |
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| Secondary | Time to All-Cause Graft Failure up to Month 48 | Graft failure was determined by the presence of one or more of the following criteria: institution of permanent dialysis (defined as dialysis treatment more than [>] 30 days), current transplant nephrectomy, and/or a clinical determination of cessation of kidney graft function and estimated glomerular filtration rate (eGFR) less than or equal to (<=) 15 milliliter (mL)/minute (min)/1.73 meter (m)^2. Time to all-cause graft failure in months was calculated as (Date of graft failure - Date of first dose + 1)/30.25. | This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. Hence, data for this outcome measure was not collected as planned, analyzed and reported. | Posted | Up to Month 48 |
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| Secondary | Time to Graft Failure Due to Antibody-Mediated Rejection (AMR) Episodes up to Month 48 | Graft failure was determined by the presence of one or more of the following criteria: institution of permanent dialysis (defined as dialysis treatment > 30 days), current transplant nephrectomy, and/or a clinical determination of cessation of kidney graft function and eGFR <=15 mL/ min/1.73m^2. Time to graft failure due to AMR episodes in months was calculated as (Date of graft failure due to AMR - Date of first dose + 1)/30.25. | This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. Hence, data for this outcome measure was not collected as planned, analyzed and reported. | Posted | Up to Month 48 |
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| Secondary | Number of Participants With Resolution of the Qualifying Antibody-Mediated Rejection (AMR) Episodes at Month 48 | Number of participants with resolution of the qualifying AMR episodes at Month 48. | This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. Hence, data for this outcome measure was not collected as planned, analyzed and reported. | Posted | Month 48 |
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| Secondary | Time to Resolution of Qualifying Antibody-Mediated Rejection (AMR) Episodes up to Month 48 | Time to resolution of qualifying AMR episodes was calculated as (Date of qualifying AMR resolution - Date of first dose + 1)/30.25. Participants who didn't had resolution of qualifying AMR episodes and still on-study were censored at the date of last visit; Participants who had completed the study without resolution of qualifying AMR were censored at the date of study completion; participants who discontinued from the study without resolution of qualifying AMR were censored at the date of early discontinuation. | This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. Hence, data for this outcome measure was not collected as planned, analyzed and reported. | Posted | Up to Month 48 |
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| Secondary | Number of Participants Who Were Alive at Month 36 | Number of participants who were alive at Month 36 (study terminated instead of Month 48) were reported. | Safety analysis set consisted of all participants who had taken at least 1 dose of investigational product. This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. | Posted | Count of Participants | Participants | Month 36 |
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| Secondary | Time to All-Cause Mortality up to Month 48 | Time to all-cause mortality was calculated as (Date of discontinuation due to death - Date of first dose + 1)/30.25. Participants who are alive and still on-study were censored at the date of last visit; Participants who had completed the study were censored at the date of study completion; Participants who discontinued from the study but not due to death were censored at the date of early discontinuation. | This study was prematurely terminated at Month 36 due to futility issue. This outcome measure was planned to analyze at Month 48. Hence, data for this outcome measure was not collected as planned, analyzed and reported. | Posted | Up to Month 48 |
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| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) was any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurred in a participant participating in a clinical study with the sponsor's product, regardless of causal relationship. TEAEs were defined as events that started or worsened on or after the date of the first dose of investigational product, but no later than 30 days following the last dose of investigational product, within a treatment period. | Safety analysis set consisted of all participants who had taken at least 1 dose of investigational product. | Posted | Count of Participants | Participants | From start of study drug administration up to study termination (Month 36) |
|
From start of study drug administration up to study termination (Month 36)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Participants received a total of seven doses with an initial 100 mL intravenous (IV) infusion containing 5000 units of placebo (100 mL 0.9% sodium chloride) on day 1 followed by 2500 units of placebo (100 mL 0.9% sodium chloride) on Days 3, 5, 7, 9, 11, and 13. | 0 | 19 | 6 | 19 | 15 | 19 |
| EG001 | CINRYZE | Participants received a total of seven doses with an initial 100 mL intravenous (IV) infusion containing 5000 units of CINRYZE on Day 1 followed by 2500 units of CINRYZE in 100 mL of IV infusion on Day 3, 5, 7, 9, 11, and 13. | 0 | 20 | 3 | 20 | 16 | 20 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Catheter site thrombosis | General disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Anaphylactic shock | Immune system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Klebsiella sepsis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Arteriovenous fistula thrombosis | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Lymphocele | Vascular disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Oesophagitis | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Tooth disorder | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Malaise | General disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Nodule | General disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| BK virus infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Catheter site infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Peritonitis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Urinary tract infection bacterial | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Arteriovenous fistula site haematoma | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Citrate toxicity | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Complications of transplanted kidney | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Toxicity to various agents | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Vascular access complication | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Blood bicarbonate decreased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Blood bicarbonate increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Blood glucose increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Blood magnesium decreased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Blood phosphorus decreased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Blood potassium increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Blood urea increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Blood uric acid increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Glomerular filtration rate decreased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Neutrophil count increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Chronic kidney disease | Renal and urinary disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Diabetes mellitus | Metabolism and nutrition disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Folate deficiency | Metabolism and nutrition disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Dry throat | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Sinus disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
|
This study was prematurely terminated at Month 36 due to futility issue.
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@takeda.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 27, 2019 | May 29, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| C469952 | SERPING1 protein, human |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| American Indian or Alaska Native |
|
| Asian/Non-Japanese |
|
| Asian/Japanese |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| Latino |
|
| Unknown |
|
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