Phase 3
N=161
Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks
Hereditary Angioedema
Bottom Line
View on ClinicalTrials.gov: NCT04739059 ↗Enrolled (actual)
161
Serious AEs
8.7%
Results posted
Jun 2026
Primary outcome: Primary: Number of Participants With Treatment-emergent Adverse Events (TEAE) — 146 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- CSL312 (Biological)
- Age
- Pediatric, Adult, Older Adult · 12+ 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) |
146 | — |
| PRIMARY Percentage of Participants With TEAE |
91.8 | — |
| PRIMARY Number of TEAE |
1196 | — |
| PRIMARY TEAE Rates Per Injection |
0.20 | — |
| PRIMARY TEAE Rates Per Participant Year |
2.26 | — |
| SECONDARY The Time-normalized Number (Per Month) of Hereditary Angioedema (HAE) Attacks During the Run-in Period and Treatment Period |
2.85; 0.02 | — |
| SECONDARY The Time-normalized Number (Per Year) of HAE Attacks During Treatment Period |
0.25 | — |
| SECONDARY Percentage Reduction in the Attack Rate During the Treatment Period Compared to the Run-in Period |
95.41 | — |
| SECONDARY Number of Participants With Percentage Reduction (of >=50%, >=70%, >=90%, and 100%) in HAE Attacks |
158; 155; 139; 76 | — |
| SECONDARY The Time-normalized Number (Per Month) of HAE Attacks Requiring On-demand Treatment |
0.00 | — |
| SECONDARY The Time-normalized Number (Per Year) of HAE Attacks Requiring On-demand Treatment |
0.00 | — |
| SECONDARY The Time-normalized Number (Per Month) of Moderate and/or Severe HAE Attacks |
0.00 | — |
| SECONDARY The Time-normalized Number (Per Year) of Moderate and/or Severe HAE Attacks |
0.00 | — |
| SECONDARY Number of Participants Rating Their Response to Therapy as Good or Excellent |
136; 130; 108 | — |
| SECONDARY Percentage of Participants Rating Their Response to Therapy as Good or Excellent |
93.8; 97.7; 94.7 | — |
| SECONDARY Number of Participants Experiencing Serious Adverse Events (SAE), Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation |
14; 0; 24; 4 | — |
| SECONDARY Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation |
8.7; 0; 14.9; 2.5 | — |
| SECONDARY Number of Participants Experiencing TEAE by Severity |
121; 111; 20; 1 | — |
| SECONDARY Percentage of Participants Experiencing TEAE by Severity |
75.2; 68.9; 12.4; 0.6 | — |
| SECONDARY Number of Participants Experiencing Adverse Events of Special Interest (AESI) |
0; 0; 0 | — |
| SECONDARY Percentage of Participants Experiencing AESI |
0; 0; 0 | — |
| SECONDARY Number of Participants With Laboratory Findings Reported as TEAE |
6; 4; 2; 2; 2; 2 | — |
| SECONDARY Percentage of Participants With Laboratory Findings Reported as TEAE |
3.7; 2.5; 1.2; 1.2; 1.2; 1.2 | — |
| SECONDARY Number of Participants With Normal C1-esterase Inhibitor (nC1-INH) Experiencing TEAE |
2 | — |
| SECONDARY Percentage of Participants With nC1-INH Experiencing TEAE |
100 | — |
| SECONDARY Number of Participants With Anti-CSL312 Antibodies |
1; 1; 4; 0; 0; 2 | — |
| SECONDARY Percentage of Participants With Anti-CSL312 Antibodies |
0.6; 0.6; 2.5; 0; 0; 1.2 | — |
Summary
This phase 3b study will evaluate long-term safety and efficacy of CSL312 (also known as garadacimab) when administered subcutaneously (SC)
Eligibility Criteria
Inclusion Criteria
- Males and females aged ≥ 12 years
- Diagnosed with clinically confirmed C1-INH HAE
- Experienced ≥ 3 HAE attacks during the 3 months before Screening
- Participated in the Run-in Period for at least 1 month (CSL312-naïve subjects only)
- Experienced at least an average of 1 HAE attack per month during the Run-in Period
Exclusion Criteria
- Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema or recurrent angioedema associated with urticaria
- Use of C1-INH products, androgens, antifibrinolytics or other small molecule medications for routine prophylaxis against HAE attacks at least 2 weeks before the first day of the Run-in Period
- Use of monoclonal antibodies such as lanadelumab (Takhzyro®) 3 months before the first day of the Run-in Period.
- Female subjects use estrogen-containing oral contraceptives or hormone replacement therapy within 4 weeks prior to screening
- Female or male subjects who are fertile and sexually active not using or not willing to use an acceptable method of contraception to avoid pregnancy during the study and for 30 days after receipt of the last dose of CSL312
- Pregnant, breastfeeding, or not willing to cease breastfeeding
Data sourced from ClinicalTrials.gov (NCT04739059). 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.