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Efficacy and Safety of Human Plasma-derived C1-esterase Inhibitor as add-on to Standard of Care for the Treatment of Refractory Antibody Mediated Rejection (AMR) in Adult Renal Transplant Recipients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03221842
Recruitment Status : Terminated (The study was terminated early due to futility of enrolment and not for safety reasons.)
First Posted : July 19, 2017
Results First Posted : January 18, 2022
Last Update Posted : July 29, 2022
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Sequential Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Antibody-mediated Rejection
Interventions Drug: C1-esterase inhibitor
Drug: Placebo
Enrollment 63
Recruitment Details  
Pre-assignment Details Only 13 participants that completed Period 1 were eligible to continue to Period 2. Eligibility based on the amendment in place at the time the subject completed Period 1.
Arm/Group Title C1-INH Placebo
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C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Excipients of C1-INH plus albumin

Placebo: Excipients of C1-INH plus albumin

Period Title: Period 1 (Open-label)
Started 63 0
Completed 53 0
Not Completed 10 0
Reason Not Completed
Adverse Event             4             0
Physician Decision             2             0
Lost to Follow-up             1             0
Terminated by sponsor             1             0
Other             2             0
Period Title: Period 2 (Randomized, Blinded)
Started 7 6
Completed 6 5
Not Completed 1 1
Reason Not Completed
Death             0             1
Lack of Efficacy             1             0
Arm/Group Title C1-INH
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C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Overall Number of Baseline Participants 63
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 63 participants
43.3  (13.83)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 63 participants
>=18 and <65 years 57
>=65 years 6
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 63 participants
Female
27
  42.9%
Male
36
  57.1%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 63 participants
Hispanic or Latino
9
  14.3%
Not Hispanic or Latino
52
  82.5%
Unknown or Not Reported
2
   3.2%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 63 participants
American Indian or Alaska Native
0
   0.0%
Asian
5
   7.9%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
12
  19.0%
White
39
  61.9%
More than one race
1
   1.6%
Unknown or Not Reported
6
   9.5%
1.Primary Outcome
Title Percent of Participants With Loss-of-response During Treatment Period 2 (TP2)
Hide Description

Loss of response is defined as 1 of the following, whichever occurs first:

  • Decline in Estimated Glomerular Filtration Rate (eGFR), or
  • Allograft failure, or
  • Subject death by any cause.
Time Frame Up to 38 weeks
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Hide Analysis Population Description
Modified Intent-to-Treat Analysis Set (mITT) - All subjects randomized under the original protocol and under all protocol amendments were included in this population except the subjects randomized prior to Amendment 3 who did not satisfy the responder criteria updated in Amendment 3.
Arm/Group Title C1-INH Placebo
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Excipients of C1-INH plus albumin

Placebo: Excipients of C1-INH plus albumin

Overall Number of Participants Analyzed 6 5
Measure Type: Number
Unit of Measure: percentage of participants
33.3 40.0
2.Secondary Outcome
Title Number of Participants With All-cause Allograft Failure During TP2
Hide Description

Allograft failure is defined as 1 of the following:

  • Allograft nephrectomy, institution of permanent dialysis, or return to the transplant waitlist for renal transplant, whichever occurs first, OR
  • Subject death by any cause
Time Frame Up to 38 weeks
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Hide Analysis Population Description
mITT
Arm/Group Title C1-INH Placebo
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Excipients of C1-INH plus albumin

Placebo: Excipients of C1-INH plus albumin

Overall Number of Participants Analyzed 6 5
Measure Type: Number
Unit of Measure: number of participants
0 1
3.Secondary Outcome
Title Percent of Participants With All-cause Allograft Failure During TP2
Hide Description [Not Specified]
Time Frame Up to 38 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
mITT
Arm/Group Title C1-INH Placebo
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Excipients of C1-INH plus albumin

Placebo: Excipients of C1-INH plus albumin

Overall Number of Participants Analyzed 6 5
Measure Type: Number
Unit of Measure: percentage of participants
0 20
4.Secondary Outcome
Title Absolute Change From Baseline in Estimated Glomerular Filtration Rate at End of Treatment Period 1 (TP1)
Hide Description [Not Specified]
Time Frame Baseline and 13 weeks
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Hide Analysis Population Description
The Run-in Safety (RiS) analysis set comprised all subjects who received at least 1 dose of C1-INH during TP1.
Arm/Group Title C1-INH
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Overall Number of Participants Analyzed 63
Median (Full Range)
Unit of Measure: mL/min/1.73m^2
-0.75
(-17.0 to 7.5)
5.Secondary Outcome
Title Absolute Change From Baseline in Estimated Glomerular Filtration Rate at End of TP2
Hide Description [Not Specified]
Time Frame Baseline and 38 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
mITT
Arm/Group Title C1-INH Placebo
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Excipients of C1-INH plus albumin

Placebo: Excipients of C1-INH plus albumin

Overall Number of Participants Analyzed 6 4
Mean (Standard Deviation)
Unit of Measure: mL/min/1.73m^2
7.75  (8.454) 15.25  (10.444)
6.Secondary Outcome
Title The Rate of Change of eGFR During TP2 as Defined by the Slope of the Mean Regression of eGFR Over Time at End of TP2
Hide Description The Sponsor terminated the study due to futility of enrolment. Because of the study termination, limited efficacy results are presented in this report.
Time Frame Up to 38 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The Sponsor terminated the study due to futility of enrolment. Because of the study termination, limited efficacy results are presented in this report. Due to study termination, data was not collected for this endpoint.
Arm/Group Title C1-INH Placebo
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Excipients of C1-INH plus albumin

Placebo: Excipients of C1-INH plus albumin

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Time to All-cause Allograft Failure Through the Follow up Period
Hide Description The Sponsor terminated the study due to futility of enrolment. Because of the study termination, limited efficacy results are presented in this report.
Time Frame Up to approximately 208 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The Sponsor terminated the study due to futility of enrolment. Because of the study termination, limited efficacy results are presented in this report. Due to study termination, data was not collected for this endpoint.
Arm/Group Title C1-INH Placebo
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Excipients of C1-INH plus albumin

Placebo: Excipients of C1-INH plus albumin

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
8.Secondary Outcome
Title Number of Responders at the End-of-TP1
Hide Description Responders were defined as subjects whose End-of-TP1 eGFR was ≥ 90% of baseline eGFR and ≥ 20 mL/min/1.73 m2.
Time Frame Up to 13 weeks
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Hide Analysis Population Description
RiS
Arm/Group Title C1-INH
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C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Overall Number of Participants Analyzed 63
Measure Type: Number
Unit of Measure: participants
33
9.Secondary Outcome
Title Percent of Responders at the End-of-TP1
Hide Description Responders were defined as subjects whose End-of-TP1 eGFR was ≥ 90% of baseline eGFR and ≥ 20 mL/min/1.73 m2.
Time Frame Up to 13 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
RiS
Arm/Group Title C1-INH
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Overall Number of Participants Analyzed 63
Measure Type: Number
Unit of Measure: percentage of responders
52.4
10.Secondary Outcome
Title Proportion of Subjects Surviving Through the Follow-up Period
Hide Description The Sponsor terminated the study due to futility of enrolment. Because of the study termination, limited efficacy results are presented in this report.
Time Frame Up to approximately 208 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The Sponsor terminated the study due to futility of enrolment. Because of the study termination, limited efficacy results are presented in this report. Due to study termination, data was not collected for this endpoint.
Arm/Group Title C1-INH Placebo
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Excipients of C1-INH plus albumin

Placebo: Excipients of C1-INH plus albumin

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
11.Secondary Outcome
Title Percent of Participants With Any Adverse Event (AE) Assessed as Related to Investigational Product
Hide Description [Not Specified]
Time Frame Up to approximately 42 weeks after the time of first investigational product administration
Hide Outcome Measure Data
Hide Analysis Population Description
RiS and RWS [The Randomized Withdrawal Safety (RWS) analysis set included all subjects in the ITT analysis set who received at least 1 dose of the investigational product after randomization during TP2]
Arm/Group Title C1-INH (Period 1) C1-INH (Period 2) Placebo
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Excipients of C1-INH plus albumin

Placebo: Excipients of C1-INH plus albumin

Overall Number of Participants Analyzed 63 7 6
Measure Type: Number
Unit of Measure: percentage of participants
20.6 0 16.7
12.Secondary Outcome
Title Mean Pre-dose C1-esterase Inhibitor Functional Activity
Hide Description C1-esterase Inhibitor may play a role in the prevention of antibody-mediated rejection (AMR) following kidney transplant. Low levels of C1 esterase inhibitor concentration and its functional activity may lead to AMR. Patients with AMR may go on to lose their kidney transplant and have other associated health risks. Levels of C1-esterase inhibitor functional activity in plasma is described as a percent.
Time Frame Up to 13 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic analysis set (PK) comprised all subjects who consented to provide rich PK sampling, who received ≥ 1 dose of C1-INH, and who had ≥ 1 measurable level of C1-INH functional activity. (some participants had missing data) or C1-INH antigen concentration.
Arm/Group Title C1-INH
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: percent functional activity
week 2 Number Analyzed 14 participants
168.86  (64.059)
week 11 Number Analyzed 13 participants
179.32  (58.551)
13.Secondary Outcome
Title Area Under the Plasma Concentration Time Curve (AUC0-t) for C1-INH Functional Activity
Hide Description C1-esterase Inhibitor may play a role in the prevention of antibody-mediated rejection (AMR) following kidney transplant. Low levels of C1 esterase inhibitor concentration and its functional activity may lead to AMR. Patients with AMR may go on to lose their kidney transplant and have other associated health risks. Levels of C1-esterase inhibitor functional activity is described as a percent.
Time Frame Up to 72 hours after post-dose on Day 10 and on Day 77 of Period 1
Hide Outcome Measure Data
Hide Analysis Population Description
PK (some participants had missing data)
Arm/Group Title C1-INH
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Overall Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: hours*percent functional activity
Day 10 Number Analyzed 10 participants
15229.84819  (4149.16754)
Day 77 Number Analyzed 9 participants
12742.20883  (4445.31881)
14.Secondary Outcome
Title Time to Maximum Plasma Concentration (Tmax) for C1-INH Functional Activity
Hide Description [Not Specified]
Time Frame Up to 72 hours after post-dose on Day 10 and on Day 77 of Period 1
Hide Outcome Measure Data
Hide Analysis Population Description
PK (some participants had missing data)
Arm/Group Title C1-INH
Hide Arm/Group Description:

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Overall Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: hours
Day 10 Number Analyzed 14 participants
9.9298  (16.3288)
Day 77 Number Analyzed 10 participants
37.9251  (22.8543)
Time Frame Up to 42 weeks per participant
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title C1-INH (Period 1) C1-INH (Period 2) Placebo (Period 2)
Hide Arm/Group Description

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

C1-esterase inhibitor (CSL842)

C1-esterase inhibitor: C1-esterase inhibitor is a human plasma-derived lyophilised powder for reconstitution administered at a dose of 60 IU/kg

Excipients of C1-INH plus albumin

Placebo: Excipients of C1-INH plus albumin

All-Cause Mortality
C1-INH (Period 1) C1-INH (Period 2) Placebo (Period 2)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/63 (0.00%)      0/7 (0.00%)      1/6 (16.67%)    
Hide Serious Adverse Events
C1-INH (Period 1) C1-INH (Period 2) Placebo (Period 2)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   25/63 (39.68%)      1/7 (14.29%)      3/6 (50.00%)    
Blood and lymphatic system disorders       
Anaemia  1  2/63 (3.17%)  2 0/7 (0.00%)  0 0/6 (0.00%)  0
Gastrointestinal disorders       
Vomiting  1  3/63 (4.76%)  3 0/7 (0.00%)  0 0/6 (0.00%)  0
Nausea  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
General disorders       
Pyrexia  1  2/63 (3.17%)  2 0/7 (0.00%)  0 0/6 (0.00%)  0
Hypothermia  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Unevaluable event  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Hepatobiliary disorders       
Cholecystitis acute  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Immune system disorders       
Transplant rejection  1  1/63 (1.59%)  1 0/7 (0.00%)  0 1/6 (16.67%)  1
Infections and infestations       
Gastroenteritis  1  3/63 (4.76%)  3 0/7 (0.00%)  0 0/6 (0.00%)  0
Pneumonia  1  2/63 (3.17%)  2 0/7 (0.00%)  0 0/6 (0.00%)  0
Appendicitis  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Bacteraemia  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Bacterial pyelonephritis  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Erysipelas  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Pneumocystis jirovecii pneumonia  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Pseudomonal sepsis  1  0/63 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1
Rhinovirus infection  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Upper respiratory tract infection  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Urethritis  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Wound infection pseudomonas  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Investigations       
Blood creatine increased  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Anogenital warts  1  0/63 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1
Renal and urinary disorders       
Acute kidney injury  1  2/63 (3.17%)  2 1/7 (14.29%)  1 0/6 (0.00%)  0
Nephrotic syndrome  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Perinephric collection  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Pyelocaliectasis  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Renal failure  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
Urinary tract obstruction  1  1/63 (1.59%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0
1
Term from vocabulary, MedDRA 23.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
C1-INH (Period 1) C1-INH (Period 2) Placebo (Period 2)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   46/63 (73.02%)      4/7 (57.14%)      5/6 (83.33%)    
Blood and lymphatic system disorders       
Anaemia  1  8/63 (12.70%)  9 0/7 (0.00%)  0 0/6 (0.00%)  0
Leukopenia  1  5/63 (7.94%)  7 0/7 (0.00%)  0 0/6 (0.00%)  0
Cardiac disorders       
Palpitations  1  1/63 (1.59%)  1 0/7 (0.00%)  0 1/6 (16.67%)  1
Gastrointestinal disorders       
Abdominal pain upper  1  0/63 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0
Anal pruritus  1  0/63 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1
Diarrhoea  1  11/63 (17.46%)  23 0/7 (0.00%)  0 0/6 (0.00%)  0
Nausea  1  9/63 (14.29%)  12 0/7 (0.00%)  0 1/6 (16.67%)  1
Vomiting  1  11/63 (17.46%)  13 0/7 (0.00%)  0 0/6 (0.00%)  0
General disorders       
Asthenia  1  4/63 (6.35%)  4 1/7 (14.29%)  1 0/6 (0.00%)  0
Discomfort  1  0/63 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0
Oedema  1  8/63 (12.70%)  9 1/7 (14.29%)  1 0/6 (0.00%)  0
Pain  1  2/63 (3.17%)  4 0/7 (0.00%)  0 1/6 (16.67%)  1
Pyrexia  1  5/63 (7.94%)  5 0/7 (0.00%)  0 0/6 (0.00%)  0
Hepatobiliary disorders       
Hepatocellular injury  1  5/63 (7.94%)  5 0/7 (0.00%)  0 0/6 (0.00%)  0
Infections and infestations       
Body tinea  1  0/63 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1
Conjunctivitis viral  1  0/63 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0
Cytomegalovirus infection  1  3/63 (4.76%)  3 1/7 (14.29%)  2 0/6 (0.00%)  0
Staphylococcal infection  1  0/63 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1
Trichomoniasis  1  0/63 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1
Urinary tract infection  1  2/63 (3.17%)  2 0/7 (0.00%)  0 1/6 (16.67%)  1
Injury, poisoning and procedural complications       
Procedural pain  1  1/63 (1.59%)  1 0/7 (0.00%)  0 1/6 (16.67%)  1
Metabolism and nutrition disorders       
Folate deficiency  1  1/63 (1.59%)  1 0/7 (0.00%)  0 1/6 (16.67%)  1
Hyperkalaemia  1  3/63 (4.76%)  3 1/7 (14.29%)  1 1/6 (16.67%)  1
Hyperphosphataemia  1  0/63 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0
Hypokalaemia  1  4/63 (6.35%)  4 0/7 (0.00%)  0 0/6 (0.00%)  0
Metabolic acidosis  1  5/63 (7.94%)  6 1/7 (14.29%)  1 0/6 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Refractory cytopenia with unilineage dysplasia  1  0/63 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0
Nervous system disorders       
Burning sensation  1  0/63 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1
Headache  1  18/63 (28.57%)  32 0/7 (0.00%)  0 0/6 (0.00%)  0
Renal and urinary disorders       
Acute kidney injury  1  6/63 (9.52%)  6 0/7 (0.00%)  0 0/6 (0.00%)  0
Focal segmental glomerulosclerosis  1  0/63 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Asthma  1  0/63 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1
Cough  1  4/63 (6.35%)  4 1/7 (14.29%)  1 0/6 (0.00%)  0
Dyspnoea  1  5/63 (7.94%)  6 0/7 (0.00%)  0 0/6 (0.00%)  0
Epistaxis  1  2/63 (3.17%)  10 0/7 (0.00%)  0 1/6 (16.67%)  1
Skin and subcutaneous tissue disorders       
Alopecia  1  2/63 (3.17%)  2 0/7 (0.00%)  0 1/6 (16.67%)  1
Dermatitis papillaris capillitii  1  0/63 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1
Vascular disorders       
Hypertension  1  6/63 (9.52%)  7 1/7 (14.29%)  1 0/6 (0.00%)  0
1
Term from vocabulary, MedDRA 23.0
Indicates events were collected by systematic assessment
The Sponsor terminated the study due to futility of enrolment. Because of the study termination, there are limitations in interpreting analyses and efficacy results based on small numbers of subjects. No subject reached the 48-month follow-up endpoint.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
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Name/Title: Study Director
Organization: CSL Behring
Phone: 610-878-4000
EMail: clinicaltrials@cslbehring.com
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Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT03221842    
Other Study ID Numbers: CSL842_3001
2017-000348-17 ( EudraCT Number )
First Submitted: July 17, 2017
First Posted: July 19, 2017
Results First Submitted: November 18, 2021
Results First Posted: January 18, 2022
Last Update Posted: July 29, 2022