Phase 3
N=64
CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks
Hereditary Angioedema
Bottom Line
View on ClinicalTrials.gov: NCT04656418 ↗Enrolled (actual)
64
Serious AEs
1.6%
Results posted
Jun 2023
Primary outcome: Primary: Time-Normalized Number of Hereditary Angioedema (HAE) Attacks Per Month During Treatment Period — 0.27; 2.01 number of HAE attacks per month — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- CSL312 (Biological); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- CSL Behring
- Primary completion
- Jun 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time-Normalized Number of Hereditary Angioedema (HAE) Attacks Per Month During Treatment Period |
0.27; 2.01 | <0.001 sig |
| SECONDARY Percentage Change in the Time-normalized Number of HAE Attacks Per Month During the Treatment Period Compared to the Run-in Period |
90.67; 20.21; 91.10; 18.89; 90.12; 29.87 | <0.001 sig |
| SECONDARY Time-Normalized Number of HAE Attacks Per Month Requiring On-Demand Treatment |
0.23; 1.86; 0.24; 1.76; 0.23; 1.80 | <0.001 sig |
| SECONDARY Time-Normalized Number of Moderate or Severe HAE Attacks Per Month |
0.13; 1.35; 0.12; 1.25; 0.13; 1.24 | <0.001 sig |
| SECONDARY Time-normalized Number of HAE Attacks Per Month in the First 3-months and Second 3-months of Treatment Period |
0.26; 1.97; 0.28; 1.86 | <0.001 sig |
| SECONDARY Relative Difference in Means in the Time-Normalized Number of HAE Attacks Per Month Between CSL312 to Placebo |
-86.51; -86.64; -85.01 | <0.001 sig |
| SECONDARY Percentage of Participants With a Response to Subject's Global Assessment of Response to Therapy (SGART) |
2.6; 41.7; 7.9; 16.7; 7.9; 8.3 | — |
| SECONDARY Number of Participants With at Least One Adverse Event (AE), Serious Adverse Event (SAE), and AEs of Special Interest (AESI) |
25; 15; 1; 0; 0; 0 | — |
| SECONDARY Number of Participants With CSL312-induced Anti-CSL312 Antibodies |
1; 0 | — |
| SECONDARY Number of Participants With Clinically Significant Abnormalities in Laboratory Assessments Reported as Treatment Emergent Adverse Events (TEAEs) |
1; 2 | — |
| SECONDARY Percentage of Participants With at Least One AE, SAE, and AESI |
64.1; 60.0; 2.6; 0; 0; 0 | — |
| SECONDARY Percentage of Participants With CSL312-induced Anti-CSL312 Antibodies |
2.6; 0 | — |
| SECONDARY Percentage of Participants With Clinically Significant Abnormalities in Laboratory Assessments Reported as TEAEs |
2.6; 8.0 | — |
Summary
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT04656418). 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.