CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

Recruitment Status
COMPLETED - HAS RESULTS
(See Contacts and Locations)Verified March 2026 by CSL Behring
Sponsor
CSL Behring
Information Provided by (Responsible Party)
CSL Behring
Clinicaltrials.gov Identifier
NCT05819775
Other Study ID Numbers:
CSL312_3003
First Submitted
April 3, 2023
First Posted
April 18, 2023
Results First Posted
May 5, 2026
Last Update Posted
June 1, 2026
Last Verified
March 2026

ClinicalTrials.gov processed this data on May 2026. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

Condition or DiseaseIntervention/Treatment
Hereditary Angioedema (HAE)
Biological: CSL312

Study Design

Study TypeInterventional
Actual Enrollment22 participants
Design AllocationN/A
Interventional ModelSingle Group Assignment
MaskingNone (Open Label)
Primary PurposeTreatment
Official TitleA Phase 3 Open-label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema in Pediatric Subjects 2 to 11 Years of Age
Study Start DateMay 29, 2023
Actual Primary Completion DateNovember 18, 2025
Actual Study Completion DateNovember 18, 2025

Groups and Cohorts

Group/CohortIntervention/Treatment
CSL312
Ages 2-5 years and 6-11 years will have specific subcutaneous dosing schedules
Biological: CSL312
Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody administered subcutaneously (SC)

Outcome Measures

Primary Outcome Measures
  1. Number of Participants With Treatment Emergent Adverse Events (TEAE)
  2. Percentage of Participants With TEAE
    The percentage of participants was rounded to one place of decimal.
  3. Number of TEAE
  4. TEAE Rates Per Injection
    The TEAE rate per injection was calculated as the number of TEAE/ number of injections. The number of injections was defined as the total injections a participant received during the Safety Evaluation Period under the dosing regimen to which the TEAE was assigned.
  5. TEAE Rates Per Participant-Year
    The TEAE rate per participant year was calculated as number of TEAEs/ participant years. Participant-years of exposure were calculated as the sum of each participant's exposure duration (in years) under the specified dosing regimen or overall. For the time assigned to a dosing regimen, each study day was counted under the corresponding regimen.
  6. Maximum Concentration (Cmax) of CSL312 at Steady-state
  7. Trough Concentration (Ctrough) of CSL312 at Steady-state
  8. Time to Maximum Concentration (Tmax) of CSL312 at Steady-State
Secondary Outcome Measures
  1. Time-normalized Number of HAE Attacks Per Month
    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.
  2. Time-normalized Number of HAE Attacks Per Year
    Time-normalized number of HAE attacks per year during treatment was calculated per participant as: \[Number of HAE attacks / Length of participant treatment in days\] \* 365.25.
  3. Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Month
    The time-normalized number of HAE attacks per month treated with on-demand treatment were calculated as follows: \[(Number of HAE attacks treated with on - demand treatment during treatment period)/ Length of participant treatment in days\] ∗ 30.4375.
  4. Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Year
    The time-normalized number of HAE attacks per year treated with on-demand treatment were calculated as follows: \[(Number of HAE attacks treated with on - demand treatment during treatment period)/ Length of participant treatment in days\] ∗ 365.25.
  5. Time-normalized Number of Moderate and/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.
  6. Time-normalized Number of Moderate and/or Severe HAE Attacks Per Year
    Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: \[number of moderate or severe HAE attacks / length of participant treatment in days\] \* 365.25.
  7. Percentage Reduction in the Time-normalized Number of HAE Attacks
    The percentage reduction in the time-normalized number of HAE attacks was calculated within a participant as follows: 100\*\[ 1 - (Time-normalized number of HAE attacks per month during treatment period/Time-normalized number of HAE attacks per month from historical data)\].
  8. Number of Participants Experiencing at Least Greater Than or Equal to (>=) 50 Percent (%), >= 70%, >= 90%, or Equal to 100% (Attack-free) Reduction in the Time-normalized Number of HAE Attacks
    A participant was classified as a responder if the percentage reduction in the time-normalized number of HAE attacks under treatment compared to the time-normalized number of HAE attacks documented in the medical records was \>= 50%. Percent Reduction = 100 \* \[1 - (time-normalized number of HAE attacks during corresponding time window / time-normalized number of HAE attacks based on historical data)\]. Here number of participants experiencing at least \>= 50%, \>= 70%, \>= 90%, or equal to 100% (Attack-free) reduction in the time-normalized number of HAE attacks are reported. The number of responders at each reduction category have been reported.
  9. Number of Participants Experiencing Serious Adverse Events (SAE), Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
  10. Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
    The percentage of participants was rounded to one decimal place.
  11. Number of Participants With TEAE by Severity
    Severity of AE was assessed by the investigator and categorized as mild, moderate and severe where: Mild: AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. Moderate: AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention.
  12. Percentage of Participants With TEAE by Severity
    Severity of AE was assessed by the investigator and categorized as mild, moderate and severe where: Mild: AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. Moderate: AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention. The percentage of participants was rounded to one place of decimal.
  13. Number of Participants With Anti-CSL312 Antibodies
  14. Percentage of Participants With Anti-CSL312 Antibodies
    The percentage of participants was rounded to one place of decimal.
  15. Number of Participants With Adverse Events of Special Interest (AESI)
    AESI included severe hypersensitivity including anaphylaxis. The AESI reported have been identified by investigators and suggestive events were independently identified for further review with a Standardized MedDRA Query (SMQ).
  16. Percentage of Participants With AESI
    AESI included severe hypersensitivity including anaphylaxis. The AESI reported have been identified by investigators and suggestive events were independently identified for further review with an SMQ. The percentage of participants was rounded to one place of decimal.
  17. FXIIa-mediated Kallikrein Activity
  18. Percent of Baseline FXIIa-mediated Kallikrein Activity
    Percent of Baseline at Visit \[i\] = 100 \* (actual value at Visit \[i\] / Baseline value), where Baseline is defined as the most recent, non-missing value before the first IP administration (including unscheduled visits). Here unit of measure is Percent (%) of FXIIa-mediated Kallikrein Activity.
  19. Number of Participants With Laboratory Findings Reported as AE
  20. Percentage of Participants With Laboratory Findings Reported as AE
    The participant data were rounded to one decimal place.

Eligibility Criteria

Ages Eligible for Study(Child)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
Inclusion Criteria
1. Male or female 2. Aged 2 to 11 years, inclusive, with body weight ≥ 10th percentile based on age 3. Diagnosed with clinically confirmed C1-INH HAE 4. Experienced ≥ 2 HAE attacks during the 6 months before Screening
Exclusion Criteria
1. Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type 3 2. Use of C1-INH products, androgens, antifibrinolytics, approved or future approved medications, or other small molecule medications for routine prophylaxis against HAE attacks within a minimum of 2 weeks before the Treatment Period 3. Participation in another interventional clinical study during the 30 days before the Treatment Period or within 5 half-lives of the final dose of the investigational product administered during the previous interventional study, whichever is longer 4. Having laboratory clinical abnormalities assessed as clinically significant by the investigator in results of hematology or chemistry assessments performed during Screening 5. Currently receiving a therapy not permitted during the study 6. Being pregnant or breastfeeding.

Contacts and Locations

Sponsors and CollaboratorsCSL Behring
Locations
Research Solutions of Arizona | Litchfield Park Arizona, United States, 85340Medical Research of Arizona | Scottsdale Arizona, United States, 85251Donald S. Levy M.D. | Orange California, United States, 92868Raffi Tachdjian MD, Inc. | Santa Monica California, United States, 90404Bernstein Clinical Research | Cincinnati Ohio, United States, 45236PennState Health Milton S. Hershey Medical Center | Hershey Pennsylvania, United States, 17033AARA Research Center | Dallas Texas, United States, 75231Campbelltown Hospital, Western Sydney University | Campbelltown , Australia, NSW 2560Ottawa Allergy Research Corp | Ottawa , Canada, K1H1E4HZRM Hämophilie Zentrum Rhein Main GmbH | Frankfurt am Main Hesse, Germany, 60596Charité - Universitätsmedizin Berlin | Berlin , Germany, 12203Universitätsklinikum Frankfurt | Frankfurt am Main , Germany, 60590Barzilai University Medical Center | Ashkelon , Israel, 7830604
Investigators
Study Director: Study Director, CSL Behring
Study Documents (Full Text)
Documents provided by CSL Behring: Study Protocol  January 12, 2025Documents provided by CSL Behring: Statistical Analysis Plan  March 4, 2025