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

Recruitment Status
COMPLETED
(See Contacts and Locations)Verified December 2025 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
Last Update Posted
January 25, 2026
Last Verified
December 2025

ClinicalTrials.gov processed this data on January 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 subjects with treatment emergent adverse events (TEAEs)
  2. Percent of subjects with TEAEs
  3. Number of TEAEs
  4. TEAE rates per injection
  5. TEAE rates per subject year
  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 and per year
  2. Time-normalized number of HAE attacks treated with on-demand treatment per month and per year
  3. Time-normalized number of moderate and / or severe HAE attacks per month and per year
  4. Percentage reduction in the time-normalized number of HAE attacks
  5. The number of subjects experiencing at least ? 50%, ? 70%, ? 90%, or equal to 100% (attack-free) reduction in the time-normalized number of HAE attacks
  6. Number of subjects with serious adverse events (SAEs)
  7. Percent of subjects with SAEs
  8. Number of subjects experiencing death
  9. Percent of subjects experiencing death
  10. Number of subjects with related TEAEs
  11. Percent of subjects with related TEAEs
  12. Number of subjects with TEAEs leading to study discontinuation
  13. Percent of subjects with TEAEs leading to study discontinuation
  14. Number of subjects with TEAEs by severity
  15. Percent of subjects with TEAEs by severity
  16. Number of subjects with Anti-CSL312 antibodies
  17. Percent of subjects with Anti-CSL312 antibodies
  18. Number of subjects with adverse events of special interest (AESIs)
  19. Percent of subjects with AESIs
  20. FXIIa-mediated kallikrein activity
    Blood samples will be collected on the same day as CSL312 administration for assessment of FXIIa-mediated kallikrein activity
  21. Number of subjects with laboratory findings reported as AEs
  22. Percent of subjects with laboratory findings reported as AEs

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