Phase 3
N=22
CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema
Hereditary Angioedema (HAE)
Bottom Line
View on ClinicalTrials.gov: NCT05819775 ↗Enrolled (actual)
22
Serious AEs
4.6%
Results posted
Jun 2026
Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAE) — 5; 11 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- CSL312 (Biological)
- Age
- Pediatric · 2+ yrs
- Sex
- All
- Sponsor
- CSL Behring
- Primary completion
- Nov 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAE) |
5; 11 | — |
| PRIMARY Percentage of Participants With TEAE |
83.3; 68.8 | — |
| PRIMARY Number of TEAE |
14; 51 | — |
| PRIMARY TEAE Rates Per Injection |
0.39; 0.27 | — |
| PRIMARY TEAE Rates Per Participant-Year |
2.34; 3.21 | — |
| PRIMARY Maximum Concentration (Cmax) of CSL312 at Steady-state |
30374.0; 29100.0 | — |
| PRIMARY Trough Concentration (Ctrough) of CSL312 at Steady-state |
10494.7; 3147.3; 3478.0; 9645.0; 10003.3; 4907.5 | — |
| PRIMARY Time to Maximum Concentration (Tmax) of CSL312 at Steady-State |
6.939; 6.985 | — |
| SECONDARY Time-normalized Number of HAE Attacks Per Month |
0.000; 0.000 | — |
| SECONDARY Time-normalized Number of HAE Attacks Per Year |
0.000; 0.000 | — |
| SECONDARY Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Month |
0.000; 0.000 | — |
| SECONDARY Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Year |
0.000; 0.000 | — |
| SECONDARY Time-normalized Number of Moderate and/or Severe HAE Attacks Per Month |
0.000; 0.000 | — |
| SECONDARY Time-normalized Number of Moderate and/or Severe HAE Attacks Per Year |
0.000; 0.000 | — |
| SECONDARY Percentage Reduction in the Time-normalized Number of HAE Attacks |
99.297; 93.535 | — |
| SECONDARY 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 |
6; 15; 6; 15; 6; 11 | — |
| SECONDARY Number of Participants Experiencing Serious Adverse Events (SAE), Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation |
0; 1; 0; 0; 1; 3 | — |
| SECONDARY Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation |
0; 6.3; 0; 0; 16.7; 18.8 | — |
| SECONDARY Number of Participants With TEAE by Severity |
4; 9; 3; 5; 0; 2 | — |
| SECONDARY Percentage of Participants With TEAE by Severity |
66.7; 56.3; 50.0; 31.3; 0; 12.5 | — |
| SECONDARY Number of Participants With Anti-CSL312 Antibodies |
0; 0; 0; 2; 0; 2 | — |
| SECONDARY Percentage of Participants With Anti-CSL312 Antibodies |
0; 0; 0; 13.3; 0; 12.5 | — |
| SECONDARY Number of Participants With Adverse Events of Special Interest (AESI) |
0; 0; 1; 3 | — |
| SECONDARY Percentage of Participants With AESI |
0; 0; 16.7; 18.8 | — |
| SECONDARY FXIIa-mediated Kallikrein Activity |
0.2039; 0.2632; 0.2932; 0.1863; 0.1162; 0.1910 | — |
| SECONDARY Percent of Baseline FXIIa-mediated Kallikrein Activity |
122.45; 166.43; 168.12; 128.12; 62.54; 114.56 | — |
| SECONDARY Number of Participants With Laboratory Findings Reported as AE |
0; 1 | — |
| SECONDARY Percentage of Participants With Laboratory Findings Reported as AE |
0; 6.3 | — |
Summary
The purpose of this study is to investigate the safety, PK / PD, and efficacy of SC CSL312 for prophylactic treatment of pediatric subjects with HAE.
Eligibility Criteria
Inclusion Criteria
- Male or female
- Aged 2 to 11 years, inclusive, with body weight ≥ 10th percentile based on age
- Diagnosed with clinically confirmed C1-INH HAE
- Experienced ≥ 2 HAE attacks during the 6 months before Screening
Exclusion Criteria
- Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type 3
- 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
- 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
- Having laboratory clinical abnormalities assessed as clinically significant by the investigator in results of hematology or chemistry assessments performed during Screening
- Currently receiving a therapy not permitted during the study
- Being pregnant or breastfeeding.
Data sourced from ClinicalTrials.gov (NCT05819775). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.