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CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks

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: NCT04656418
Recruitment Status : Completed
First Posted : December 7, 2020
Results First Posted : June 29, 2023
Last Update Posted : June 29, 2023
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Prevention
Condition Hereditary Angioedema
Interventions Biological: CSL312
Drug: Placebo
Enrollment 64
Recruitment Details Participants were enrolled at study centers in Canada, Germany, Hungary, Israel, Japan, Netherlands, and the United States from 27 January 2021 to 07 June 2022.
Pre-assignment Details A total of 80 participants were screened, of which 64 participants were randomized and received the loading dose in the treatment period.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description Participants received a CSL312 loading dose of 400 mg as two 200 mg subcutaneous (SC) injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6. Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Period Title: Overall Study
Started 39 25
Intention-to-treat (ITT) Analysis Set [1] 39 25
Safety Analysis Set [2] 39 25
Completed 38 22
Not Completed 1 3
Reason Not Completed
Withdrawal by Subject             1             3
[1]
ITT analysis set included all the randomized participants who provided written informed consent and underwent study screening procedures.
[2]
Safety analysis set included all the randomized participants who provided written informed consent, underwent study screening procedures and received at least 1 dose of the investigational product.
Arm/Group Title CSL312 Placebo Total
Hide Arm/Group Description Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6. Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6. Total of all reporting groups
Overall Number of Baseline Participants 39 25 64
Hide Baseline Analysis Population Description
ITT analysis set included all the randomized participants who provided written informed consent and underwent study screening procedures.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 39 participants 25 participants 64 participants
43.3  (17.45) 37.8  (12.80) 41.2  (15.92)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 39 participants 25 participants 64 participants
Female
24
  61.5%
14
  56.0%
38
  59.4%
Male
15
  38.5%
11
  44.0%
26
  40.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 39 participants 25 participants 64 participants
Hispanic or Latino
1
   2.6%
2
   8.0%
3
   4.7%
Not Hispanic or Latino
37
  94.9%
23
  92.0%
60
  93.8%
Unknown or Not Reported
1
   2.6%
0
   0.0%
1
   1.6%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Race Number Analyzed 39 participants 25 participants 64 participants
Asian
4
  10.3%
2
   8.0%
6
   9.4%
Black or African American
0
   0.0%
1
   4.0%
1
   1.6%
Native Hawaiian or Other Pacific Islander
1
   2.6%
0
   0.0%
1
   1.6%
White
33
  84.6%
22
  88.0%
55
  85.9%
Other
1
   2.6%
0
   0.0%
1
   1.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
 Number Analyzed 39 participants 25 participants 64 participants
Canada 5 3 8
Netherlands 2 1 3
Hungary 2 0 2
United States 12 9 21
Japan 4 2 6
Israel 7 2 9
Germany 7 8 15
1.Primary Outcome
Title Time-Normalized Number of Hereditary Angioedema (HAE) Attacks Per Month During Treatment Period
Hide Description Time-normalized number of HAE attacks per month during treatment was calculated per participant as: [number of HAE attacks / length of participant treatment in days] * 30.4375.
Time Frame First injection up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set included all the randomized participants who provided written informed consent and underwent study screening procedures. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 24
Mean (Standard Deviation)
Unit of Measure: number of HAE attacks per month
0.27  (0.683) 2.01  (1.341)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection CSL312, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Compared the time-normalized number of HAE attacks in the active and placebo arms by using a two-sided Wilcoxon test (Hierarchical Testing H01) at alpha = 5%.
Method Two-sided Wilcoxon test
Comments [Not Specified]
2.Secondary Outcome
Title Percentage Change in the Time-normalized Number of HAE Attacks Per Month During the Treatment Period Compared to the Run-in Period
Hide Description

Percentage change in the time-normalized number of HAE attacks was calculated within a participant as:

100 * [1 - (time-normalized number of HAE attacks per month during treatment period / time-normalized number of HAE attacks per month during run-in period)]. Time-normalized number of HAE attacks per month during treatment period was calculated per participant as: [number of HAE attacks / length of participant treatment in days] * 30.4375.
Time Frame 6 months, first 3-months and second 3-months of treatment period
Hide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set included all the randomized participants who provided written informed consent and underwent study screening procedures. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at the specified time point.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 24
Mean (Standard Deviation)
Unit of Measure: percentage change in HAE attacks/month
6 Months of Treatment Number Analyzed 39 participants 24 participants
90.67  (22.433) 20.21  (42.661)
First 3-months of Treatment Number Analyzed 39 participants 24 participants
91.10  (21.255) 18.89  (53.837)
Second 3-months of Treatment Number Analyzed 39 participants 22 participants
90.12  (25.624) 29.87  (55.529)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection CSL312, Placebo
Comments 6 Months of treatment
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Compared the time-normalized number of HAE attacks in the active and placebo arms by using a two-sided Wilcoxon test.
Method Two-sided Wilcoxon test
Comments [Not Specified]
3.Secondary Outcome
Title Time-Normalized Number of HAE Attacks Per Month Requiring On-Demand Treatment
Hide Description Time-normalized number of HAE attacks per month requiring on-demand treatment was calculated per participant as: [number of HAE attacks requiring on-demand treatment / length of participant in days] * 30.4375.
Time Frame 6 months, first 3-months and second 3-months of treatment period
Hide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set included all the randomized participants who provided written informed consent and underwent study screening procedures. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at the specified time point.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 24
Overall Number of Units Analyzed
Type of Units Analyzed: Total number of HAE attacks
 63 264
Mean (Standard Deviation)
Unit of Measure: number of HAE attacks per month
6 Months of Treatment Number Analyzed 63 Total number of HAE attacks 264 Total number of HAE attacks
0.23  (0.663) 1.86  (1.412)
First 3-months of Treatment Number Analyzed 31 Total number of HAE attacks 144 Total number of HAE attacks
0.24  (0.748) 1.76  (1.378)
Second 3-months of Treatment Number Analyzed 32 Total number of HAE attacks 120 Total number of HAE attacks [1] 
0.23  (0.610) 1.80  (1.626)
[1]
22 participants
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection CSL312, Placebo
Comments 6 Months of treatment
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Tested the differences between the active and placebo arms using a two-sided Wilcoxon test at alpha = 5%.
Method Two-sided Wilcoxon test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection CSL312, Placebo
Comments First 3-months of treatment
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Tested the differences between the active and placebo arms using a two-sided Wilcoxon test at alpha = 5%.
Method Two-sided Wilcoxon test
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection CSL312, Placebo
Comments Second 3-months of treatment
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Tested the differences between the active and placebo arms using a two-sided Wilcoxon test at alpha = 5%.
Method Two-sided Wilcoxon test
Comments [Not Specified]
4.Secondary Outcome
Title Time-Normalized Number of Moderate or Severe HAE Attacks Per Month
Hide Description Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: [number of moderate or severe HAE attacks / length of participant treatment in days] * 30.4375.
Time Frame 6 months, first 3-months and second 3-months of treatment period
Hide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set included all the randomized participants who provided written informed consent and underwent study screening procedures. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at the specified time point.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 24
Overall Number of Units Analyzed
Type of Units Analyzed: Total number of HAE attacks
 63 264
Mean (Standard Deviation)
Unit of Measure: number of HAE attacks per month
6 Months of Treatment Number Analyzed 63 Total number of HAE attacks 264 Total number of HAE attacks
0.13  (0.296) 1.35  (1.166)
First 3-months of Treatment Number Analyzed 63 Total number of HAE attacks 264 Total number of HAE attacks
0.12  (0.305) 1.25  (1.091)
Second 3-months of Treatment Number Analyzed 63 Total number of HAE attacks 264 Total number of HAE attacks [1] 
0.13  (0.320) 1.24  (1.296)
[1]
22 participants
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection CSL312, Placebo
Comments 6 Months of treatment
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Tested the differences between the active and placebo arms using a two-sided Wilcoxon test at alpha = 5%.
Method Two-sided Wilcoxon test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection CSL312, Placebo
Comments First 3-months of treatment
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Tested the differences between the active and placebo arms using a two-sided Wilcoxon test at alpha = 5%.
Method Two-sided Wilcoxon test
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection CSL312, Placebo
Comments Second 3-months of treatment
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Tested the differences between the active and placebo arms using a two-sided Wilcoxon test at alpha = 5%.
Method Two-sided Wilcoxon test
Comments [Not Specified]
5.Secondary Outcome
Title Time-normalized Number of HAE Attacks Per Month in the First 3-months and Second 3-months of Treatment Period
Hide Description Time-normalized number of HAE attacks per month during treatment was calculated per participant as: [number of HAE attacks / length of participant treatment in days] * 30.4375.
Time Frame First 3-months and second 3-months of treatment period
Hide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set included all the randomized participants who provided written informed consent and underwent study screening procedures. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at the specified time point.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 24
Mean (Standard Deviation)
Unit of Measure: number of HAE attacks per month
First 3-months of Treatment Number Analyzed 39 participants 24 participants
0.26  (0.749) 1.97  (1.287)
Second 3-months of Treatment Number Analyzed 39 participants 22 participants
0.28  (0.652) 1.86  (1.603)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection CSL312, Placebo
Comments First 3-months of treatment
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Tested the differences between the active and placebo arms using a two-sided Wilcoxon test at alpha = 5%.
Method Two-sided Wilcoxon test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection CSL312, Placebo
Comments Second 3-months of treatment
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Tested the differences between the active and placebo arms using a two-sided Wilcoxon test at alpha = 5%.
Method Two-sided Wilcoxon test
Comments [Not Specified]
6.Secondary Outcome
Title Relative Difference in Means in the Time-Normalized Number of HAE Attacks Per Month Between CSL312 to Placebo
Hide Description Relative difference in means in the time-normalized number of HAE attacks per month CSL312 to Placebo was calculated as: 100 * [(mean time-normalized number of HAE attacks for CSL312 - mean time-normalized number of HAE attacks for placebo) / mean time-normalized number of HAE attacks for placebo]. Time-normalized number of HAE attacks per month during treatment was calculated per participant as: [number of HAE attacks / length of participant treatment in days] * 30.4375.
Time Frame 6 months, first 3-months and second 3-months of treatment period
Hide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set included all randomized participants who provided written informed consent and underwent study screening procedures. 'Overall number of participants analyzed' indicates number of participants with data available for outcome measure (OM) analysis. 'Number analyzed' indicates number of participants with data available for analysis at specified time point. As pre-specified in protocol and SAP, data for this OM was reported for participants of CSL312 and Placebo Comparison group.
Arm/Group Title CSL312 and Placebo Comparison Group
Hide Arm/Group Description:

CSL312: Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.

Placebo: Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 63
Mean (95% Confidence Interval)
Unit of Measure: number of HAE attacks per month
6 Months of Treatment Number Analyzed 63 participants
-86.51
(-95.68 to -57.84)
First 3-months of Treatment Number Analyzed 63 participants
-86.64
(-95.87 to -56.76)
Second 3-months of Treatment Number Analyzed 61 participants
-85.01
(-95.62 to -48.74)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection CSL312 and Placebo Comparison Group
Comments [Not Specified]
Type of Statistical Test Other
Comments Test for differences
Statistical Test of Hypothesis P-Value <0.001
Comments Compared the time-normalized number of HAE attacks in the active and placebo arms by using a two-sided Wilcoxon test (Hierarchical testing H02) at alpha = 5%.
Method Two-sided Wilcoxon test
Comments [Not Specified]
7.Secondary Outcome
Title Percentage of Participants With a Response to Subject's Global Assessment of Response to Therapy (SGART)
Hide Description SGART is a self-assessment by the participant and measures the subject's overall treatment response to the investigational product using the following ratings: 0 (none: worse or no response at all, not acceptable), 1 (poor: very little response, not acceptable), 2 (fair: some response, acceptable but could be better), 3 (good: good response, acceptable), and 4 (excellent: excellent response, as good as can be imagined).
Time Frame Up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set included all the randomized participants who provided written informed consent and underwent study screening procedures. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 38 24
Measure Type: Number
Unit of Measure: percentage of participants
None 2.6 41.7
Poor 7.9 16.7
Fair 7.9 8.3
Good 15.8 20.8
Excellent 65.8 12.5
8.Secondary Outcome
Title Number of Participants With at Least One Adverse Event (AE), Serious Adverse Event (SAE), and AEs of Special Interest (AESI)
Hide Description AE is any untoward medical occurrence in a participant administered with an investigational product which does not necessarily have a causal relationship with treatment, can be any unfavorable and unintended sign, symptom, or disease temporally associated with use of an investigational product, whether or not considered related to product. SAE is any untoward medical occurrence that results in death, is life-threatening, requires in-patient hospitalization or prolongation of existing hospitalization, is a congenital anomaly or birth defect, or is a medically significant event. An AESI is an AE of scientific and medical concern specific to sponsor's product or program, for which ongoing monitoring and rapid communication by investigator to sponsor is appropriate.
Time Frame From first dose of study drug up to 3 months after the last injection (approximately 8 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all the randomized participants who provided written informed consent, underwent study screening procedures and received at least 1 dose of the investigational product.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 25
Measure Type: Count of Participants
Unit of Measure: Participants
AE
25
  64.1%
15
  60.0%
SAE
1
   2.6%
0
   0.0%
AESI
0
   0.0%
0
   0.0%
9.Secondary Outcome
Title Number of Participants With CSL312-induced Anti-CSL312 Antibodies
Hide Description [Not Specified]
Time Frame Up to 8 months
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all the randomized participants who provided written informed consent, underwent study screening procedures and received at least 1 dose of the investigational product.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 25
Measure Type: Count of Participants
Unit of Measure: Participants
1
   2.6%
0
   0.0%
10.Secondary Outcome
Title Number of Participants With Clinically Significant Abnormalities in Laboratory Assessments Reported as Treatment Emergent Adverse Events (TEAEs)
Hide Description Laboratory assessments included: Hematology, biochemistry, urinalysis, and coagulation parameters.
Time Frame From first dose of study drug up to 3 months after the last injection (approximately 8 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all the randomized participants who provided written informed consent, underwent study screening procedures and received at least 1 dose of the investigational product.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 25
Measure Type: Count of Participants
Unit of Measure: Participants
1
   2.6%
2
   8.0%
11.Secondary Outcome
Title Percentage of Participants With at Least One AE, SAE, and AESI
Hide Description AE is any untoward medical occurrence in a participant administered with an investigational product which does not necessarily have a causal relationship with treatment, can be any unfavorable and unintended sign, symptom, or disease temporally associated with use of an investigational product, whether or not considered related to product. SAE is any untoward medical occurrence that results in death, is life-threatening, requires in-patient hospitalization or prolongation of existing hospitalization, is a congenital anomaly or birth defect, or is a medically significant event. An AESI is an AE of scientific and medical concern specific to sponsor's product or program, for which ongoing monitoring and rapid communication by investigator to sponsor is appropriate.
Time Frame From first dose of study drug up to 3 months after the last injection (approximately 8 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all the randomized participants who provided written informed consent, underwent study screening procedures and received at least 1 dose of the investigational product. The percentage of participants are rounded off to the single decimal point.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 25
Measure Type: Number
Unit of Measure: percentage of participants
AE 64.1 60.0
SAE 2.6 0
AESI 0 0
12.Secondary Outcome
Title Percentage of Participants With CSL312-induced Anti-CSL312 Antibodies
Hide Description [Not Specified]
Time Frame Up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all the randomized participants who provided written informed consent, underwent study screening procedures and received at least 1 dose of the investigational product. The percentage of participants are rounded off to the single decimal point.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 25
Measure Type: Number
Unit of Measure: percentage of participants
2.6 0
13.Secondary Outcome
Title Percentage of Participants With Clinically Significant Abnormalities in Laboratory Assessments Reported as TEAEs
Hide Description Laboratory assessments included: Hematology, biochemistry, urinalysis, and coagulation parameters.
Time Frame From first dose of study drug up to 3 months after the last injection (approximately 8 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all the randomized participants who provided written informed consent, underwent study screening procedures and received at least 1 dose of the investigational product. The percentage of participants are rounded off to the single decimal point.
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description:
Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6.
Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
Overall Number of Participants Analyzed 39 25
Measure Type: Number
Unit of Measure: percentage of participants
2.6 8.0
Time Frame From first dose of study drug up to 3 months after the last injection (approximately 8 months)
Adverse Event Reporting Description Safety analysis set included all the randomized participants who provided written informed consent, underwent study screening procedures and received at least 1 dose of the investigational product.
 
Arm/Group Title CSL312 Placebo
Hide Arm/Group Description Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg SC injections, once monthly from Months 2 to 6. Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.
All-Cause Mortality
CSL312 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/39 (0.00%)   0/25 (0.00%) 
Hide Serious Adverse Events
CSL312 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   1/39 (2.56%)   0/25 (0.00%) 
Congenital, familial and genetic disorders     
Hereditary angioedema † 1  1/39 (2.56%)  0/25 (0.00%) 
1
Term from vocabulary, MedDRA 25.0
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
CSL312 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   17/39 (43.59%)   12/25 (48.00%) 
Eye disorders     
Visual impairment † 1  2/39 (5.13%)  0/25 (0.00%) 
Gastrointestinal disorders     
Diarrhoea † 1  2/39 (5.13%)  1/25 (4.00%) 
Abdominal pain † 1  2/39 (5.13%)  0/25 (0.00%) 
Nausea † 1  0/39 (0.00%)  2/25 (8.00%) 
General disorders     
Fatigue † 1  0/39 (0.00%)  3/25 (12.00%) 
Injection site erythema † 1  1/39 (2.56%)  2/25 (8.00%) 
Pyrexia † 1  1/39 (2.56%)  2/25 (8.00%) 
Infections and infestations     
Upper respiratory tract infection † 1  4/39 (10.26%)  2/25 (8.00%) 
Nasopharyngitis † 1  3/39 (7.69%)  1/25 (4.00%) 
COVID-19 † 1  0/39 (0.00%)  3/25 (12.00%) 
Gastrointestinal infection † 1  2/39 (5.13%)  1/25 (4.00%) 
Conjunctivitis † 1  2/39 (5.13%)  0/25 (0.00%) 
Sinusitis † 1  2/39 (5.13%)  0/25 (0.00%) 
Urinary tract infection † 1  2/39 (5.13%)  0/25 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back pain † 1  2/39 (5.13%)  1/25 (4.00%) 
Pain in extremity † 1  0/39 (0.00%)  2/25 (8.00%) 
Nervous system disorders     
Headache † 1  3/39 (7.69%)  4/25 (16.00%) 
Respiratory, thoracic and mediastinal disorders     
Oropharyngeal pain † 1  2/39 (5.13%)  1/25 (4.00%) 
1
Term from vocabulary, MedDRA 25.0
†
Indicates events were collected by systematic assessment
[Not Specified]
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
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: CSL Behring
Phone: 610-878-4000
EMail: clinicaltrials@cslbehring.com
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT04656418    
Other Study ID Numbers: CSL312_3001
2020-000570-25 ( EudraCT Number )
First Submitted: December 1, 2020
First Posted: December 7, 2020
Results First Submitted: June 7, 2023
Results First Posted: June 29, 2023
Last Update Posted: June 29, 2023