CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks

CSL Behring64 enrolled

Overview

This is a multicenter, double-blind, randomized, placebo-controlled, parallel-arm study to investigate the efficacy and safety of subcutaneous administration of CSL312 (garadacimab) in the prophylactic treatment of hereditary angioedema.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

QUADRUPLE

Enrollment

Conditions

Hereditary Angioedema

Interventions

CSL312BIOLOGICAL

Fully human immunoglobulin G subclass 4/lambda recombinant monoclonal antibody

PlaceboDRUG

Buffer without active ingredient

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male or female ≥ 12 years of age; diagnosed with clinically confirmed C1-INH hereditary angioedema; experience ≥ 3 attacks during the 3 months before screening. Note: For subjects taking any prophylactic HAE therapy during the 3 months before Screening, ≥ 3 HAE attacks may be documented over 3 consecutive months before commencing the prophylactic therapy. Exclusion Criteria: * Concomitant diagnosis of another form of angioedema such as idiopathic or acquired angioedema, recurrent angioedema associated with urticarial or hereditary angioedema type 3

Locations (28)

Outcomes

Primary Outcomes

Time-Normalized Number of Hereditary Angioedema (HAE) Attacks Per Month During Treatment Period

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

Secondary Outcomes

Percentage Change in the Time-normalized Number of HAE Attacks Per Month During the Treatment Period Compared to the Run-in Period

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

Time-Normalized Number of HAE Attacks Per Month Requiring On-Demand Treatment

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

Time-Normalized Number of Moderate or Severe HAE Attacks Per Month

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

Time-normalized Number of HAE Attacks Per Month in the First 3-months and Second 3-months of Treatment Period

Data from ClinicalTrials.gov

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Clinical Research Center of Alabama

Birmingham, Alabama, United States

Medical Research of Arizona

Scottsdale, Arizona, United States

Raffi Tachdjian MD, Inc.

Santa Monica, California, United States

Allergy and Asthma Clinical Research

Walnut Creek, California, United States

Institute of Asthma and Allergy

Chevy Chase, Maryland, United States

Bernstein Clinical Research Center LLC

Cincinnati, Ohio, United States

Pennsylvania State University

Hershey, Pennsylvania, United States

AARA Research Center

Dallas, Texas, United States

University of Alberta - Research Transition Facility

Edmonton, Alberta, Canada

Ottawa Allergy Research Corp

Ottawa, Ontario, Canada

...and 18 more locations

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

Relative Difference in Means in the Time-Normalized Number of HAE Attacks Per Month Between CSL312 to Placebo

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

Percentage of Participants With a Response to Subject's Global Assessment of Response to Therapy (SGART)

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

Number of Participants With at Least One Adverse Event (AE), Serious Adverse Event (SAE), and AEs of Special Interest (AESI)

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)

Number of Participants With CSL312-induced Anti-CSL312 Antibodies

Time frame: Up to 8 months

Number of Participants With Clinically Significant Abnormalities in Laboratory Assessments Reported as Treatment Emergent Adverse Events (TEAEs)

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)

Percentage of Participants With at Least One AE, SAE, and AESI

Time frame: From first dose of study drug up to 3 months after the last injection (approximately 8 months)

Percentage of Participants With CSL312-induced Anti-CSL312 Antibodies

Time frame: Up to 6 months

Percentage of Participants With Clinically Significant Abnormalities in Laboratory Assessments Reported as TEAEs

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)