Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab

Phase 4

Recruiting

• Conditions: Hereditary Angioedema
• Interventions: Biological: Garadacimab

• Registration Number: NCT06806657
• Lead Sponsor: CSL Behring

Brief Summary

This study is designed to evaluate the safety after switching to garadacimab from another prophylactic hereditary angioedema (HAE) treatment (marketed kallikrein \[KK\] inhibitor or plasma-derived C1-esterase inhibitor \[pdC1INH\]prophylactic) when administered once monthly for approximately 3 months in participants aged greater than or equal to (\>=) 12 years with HAE.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-02-03
• Study First Submitted QC: 2025-02-03
• Study First Posted: 2025-02-04
• Study Start: 2025-03-19
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-19
• Last Update Posted: 2025-05-20
• Primary Completion: 2026-06-15
• Study Completion: 2026-06-15

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

• Aged >= 12 years at the time of providing written informed consent / assent.

• Have a history of response to on-demand HAE treatment for the treatment of acute HAE attacks.

• Documented laboratory diagnosis in medical records of C1-esterase inhibitor hereditary angioedema (HAE-C1INH) type 1 or type 2:

  • Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling episodes without accompanying urticaria),
  • C1-esterase inhibitor (C1INH) antigen concentration or functional activity less than (<) 50% of normal as documented in the participant's medical record, or
  • C4-antigen concentration below the lower limit of the reference range as documented in the participant's medical record.

For HAE-nC1INH: Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling episodes without accompanying urticaria); an HAE-associated FXII gene mutation (eg, FXII point mutation Thr328Lys or Thr328Arg, or deletion of 72 base pairs [c.971_1018 + 24del72], or duplication of 18 base pairs [c.892-909dup]), as documented in the participant's medical record, OR an HAE-associated plasminogen gene mutation (PLG) gene mutation (eg, PLG point mutation Lys330Glu), as documented in the participant's medical record; C1INH antigen concentration or functional activity 70 to 120% of the normal level, as documented in the participant's medical record.

• Use of lanadelumab, berotralstat, or pdC1INH for the prophylactic treatment of HAE and be on a stable (consistent) dose / regimen of such medication for at least 3 months prior to Screening.

Exclusion Criteria

• Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema or recurrent angioedema associated with urticaria.
• Use of androgens, antifibrinolytics, or investigational products (other than garadacimab) for routine prophylaxis against HAE attacks.
• Known or suspected hypersensitivity to monoclonal antibody therapy or hypersensitivity to the active substance (garadacimab) or to any of the excipients.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Garadacimab	Garadacimab	-

Primary Outcome Measures

Name	Time	Method
Percentage of Participants With TEAEs	Up to Day 95 (EoS)
Number of TEAEs	Up to Day 95 (EoS)
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	Up to Day 95 (End of study [EoS])
Rate of TEAEs per injection	Up to Day 95 (EoS)
Rate of TEAEs per participant year	Up to Day 95 (EoS)

Secondary Outcome Measures

Name	Time	Method
Number of Participants With: Serious Adverse Events (SAEs), Deaths, Related TEAEs, TEAEs leading to study discontinuation, TEAEs by severity, Laboratory Findings Reported as an AE, and Adverse Events of Special Interest (AESI)	Up to Day 95 (EoS)	The AESIs for garadacimab are severe hypersensitivity including anaphylaxis.
Percentage of Participants With: SAEs, Deaths, Treatment Related TEAEs, TEAEs leading to study discontinuation, TEAEs by severity, Laboratory Findings Reported as an AE, and AESI	Up to Day 95 (EoS)
Number of SAEs, Deaths, Treatment Related TEAEs, TEAEs leading to study discontinuation, TEAEs by severity, AESI and Laboratory Findings Reported as an AE, and AESI	Up to Day 95 (EoS)
Rate per injection of: SAEs, Deaths, Treatment Related TEAEs, TEAEs leading to study discontinuation, TEAEs by severity, Laboratory Findings Reported as an AE, and AESI	Up to Day 95 (EoS)
Rate per participant year of: SAEs, Deaths, Treatment Related TEAEs, TEAEs leading to study discontinuation, TEAEs by severity, Laboratory Findings Reported as an AE, and AESI	Up to Day 95 (EoS)
Number of Participants with Anti-garadacimab Antibodies	Up to Day 95 (EoS)
Percentage of Participants with Anti-garadacimab Antibodies	Up to Day 95 (EoS)
Plasma Concentrations of Garadacimab	Up to Day 95 (EoS)
Percentage of Participants who Indicated Their Preference for Garadacimab	Up to Day 95 (EoS)

Trial Locations

Locations (4)

Bernstein Clinical Research Center, LLC; 🇺🇸 Cincinnati, Ohio, United States

Research Solutions of Arizona; 🇺🇸 Litchfield Park, Arizona, United States

Donald Levy M.D.; 🇺🇸 Orange, California, United States

Raffi Tachdjian MD, Inc.; 🇺🇸 Santa Monica, California, United States