Long-term safety and efficacy of CSL312 (garadacimab) in the prophylactic treatment of hereditary angioedema attacks

An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-003918-12-DE

Enrollment

150

Registered

2020-11-09

Start date

2021-06-08

Completion date

Unknown

Last updated

2024-06-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hereditary angioedema MedDRA version: 23.1 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: Factor XIIa inhibitor monoclonal antibody Product Code: CSL312 garadacimab Pharmaceutical Form: Solution for injection INN or Proposed INN: FXIIa inhibitor monoclonal antibody Current Sp

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: "1. Males and females aged = 12 years 2. Diagnosed with clinically confirmed C1-INH HAE 3. Experienced = 3 HAE attacks during the 3 months before Screening 4. Participated in the Run-in Period for at least 1 month (CSL312-naïve subjects only) 5. Experienced at least an average of 1 HAE attack per month during the Run-in Period" Are the trial subjects under 18? yes Number of subjects for this age range: 20 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 110 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20

Exclusion criteria

Exclusion criteria: "1. Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema or recurrent angioedema associated with urticaria 2. 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 3. Use of monoclonal antibodies such as lanadelumab (Takhzyro®) 3 months before the first day of the Run-in Period. 4. Female subjects use estrogen-containing oral contraceptives or hormone replacement therapy within 4 weeks prior to screening 5. 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 6. Pregnant, breastfeeding, or not willing to cease breastfeeding"

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective of the study is to evaluate the long-term safety of SC administration of CSL312 in the prophylactic treatment of subjects with C1-INH HAE ;Secondary Objective: The secondary objectives of this study are to evaluate the long-term efficacy, safety and patient reported assessment of response to therapy ;Primary end point(s): 1. Number of subjects with treatment emergent adverse events (TEAEs) 2. Percentage of subjects with TEAEs 3. TEAEs rates per injection 4. TEAEs rates per subject year ;Timepoint(s) of evaluation of this end point: 1-4. Up to 45 months	—

Measure

Time frame

Main Objective: The primary objective of the study is to evaluate the long-term safety of SC administration of CSL312 in the prophylactic treatment of subjects with C1-INH HAE ;Secondary Objective: The secondary objectives of this study are to evaluate the long-term efficacy, safety and patient reported assessment of response to therapy ;Primary end point(s): 1. Number of subjects with treatment emergent adverse events (TEAEs) 2. Percentage of subjects with TEAEs 3. TEAEs rates per injection 4. TEAEs rates per subject year ;Timepoint(s) of evaluation of this end point: 1-4. Up to 45 months

—

Secondary

Measure	Time frame
Secondary end point(s): 1. The time-normalized number (per month and year) of Hereditary Angioedema (HAE attacks) for the entire study 2. The percentage reduction and 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 on Treatment compared to Run-in Period 3. The time-normalized number (per month and year) of HAE attacks requiring on-demand treatment in subjects on treatment 4. The time-normalized number (per month and year) of moderate and/or severe HAE attacks in subjects on treatment 5. Number and percentage of subjects rating their response to therapy as good or excellent 6. The number and percentage of subjects experiencingTEAEs with CSL312 7. The number and percentage of subjects experiencing adverse events of special interest (AESIs) with CSL312 8. The number and percentage of subjects experiencing serious adverse events (SAEs), including deaths, with CSL312 9. The number and percentage of subjects experiencing CSL312 induced anti-CSL312 antibodies with CSL312 ;Timepoint(s) of evaluation of this end point: 1.-5. = Up to 43 months 6.-9. = Up to 45 months	—

Measure

Time frame

Secondary end point(s): 1. The time-normalized number (per month and year) of Hereditary Angioedema (HAE attacks) for the entire study 2. The percentage reduction and 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 on Treatment compared to Run-in Period 3. The time-normalized number (per month and year) of HAE attacks requiring on-demand treatment in subjects on treatment 4. The time-normalized number (per month and year) of moderate and/or severe HAE attacks in subjects on treatment 5. Number and percentage of subjects rating their response to therapy as good or excellent 6. The number and percentage of subjects experiencingTEAEs with CSL312 7. The number and percentage of subjects experiencing adverse events of special interest (AESIs) with CSL312 8. The number and percentage of subjects experiencing serious adverse events (SAEs), including deaths, with CSL312 9. The number and percentage of subjects experiencing CSL312 induced anti-CSL312 antibodies with CSL312 ;Timepoint(s) of evaluation of this end point: 1.-5. = Up to 43 months 6.-9. = Up to 45 months

—

Countries

Australia, Canada, Czechia, Czech Republic, Germany, Hong Kong, Hungary, Israel, Italy, Japan, Netherlands, New Zealand, Russian Federation, Spain, Taiwan, United Kingdom, United States

Contacts

Public ContactTrial Registration Coordinator

CSL Behring LLC

clinicaltrials@cslbehring.com+16108784000

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026