GARADACIMAB for Hereditary angioedema (HAE) | HAE-C1INH (type 1 or type 2) | HAE-nC1INH

Inclusion criteria

• {"criterion_text":"- 1. Aged ≥ 12 years at the time of providing written informed consent / assent."}
• {"criterion_text":"- 2. Have a history of response to on-demand HAE treatment for the treatment of acute HAE attacks."}
• {"criterion_text":"- 3. Documented laboratory diagnosis in medical records of HAE-C1INH type 1 or type 2: o Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling episodes without accompanying urticaria), o C1INH antigen concentration or functional activity < 50% of normal as documented in the subject’s medical record, or o C4-antigen concentration below the lower limit of the reference range as documented in the subject’s medical record, or o 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 subject’s medical record, OR an HAE-associated plasminogen gene mutation (PLG) gene mutation (eg, PLG point mutation Lys330Glu), as documented in the subject’s medical record; C1INH antigen concentration or functional activity 70 to 120% of the normal level, as documented in the subject’s medical record."}
• {"criterion_text":"- 4. 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

• {"criterion_text":"- 1. Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema or recurrent angioedema associated with urticaria."}
• {"criterion_text":"- 2. Use of androgens, antifibrinolytics, or investigational products (other than garadacimab) for routine prophylaxis against HAE attacks."}
• {"criterion_text":"- 3. Known or suspected hypersensitivity to monoclonal antibody therapy or hypersensitivity to the active substance (garadacimab) or to any of the excipients."}

Primary endpoints

• {"endpoint_text":"- 1. Number of Participants with Treatment Emergent Adverse Events (TEAEs)","definition_or_measurement_approach":"Count of participants experiencing treatment-emergent adverse events as recorded during study follow-up (safety reporting)."}
• {"endpoint_text":"- 2. Percentage of Participants with TEAEs","definition_or_measurement_approach":"Proportion (percentage) of enrolled participants who experienced TEAEs during the study period."}
• {"endpoint_text":"- 3. Number of TEAEs","definition_or_measurement_approach":"Total count of treatment-emergent adverse events reported in the study."}
• {"endpoint_text":"- 4. Rate of TEAEs per injection","definition_or_measurement_approach":"Number of TEAEs divided by number of injections administered (TEAEs per injection)."}
• {"endpoint_text":"- 5. Rate of TEAEs per participant year","definition_or_measurement_approach":"Number of TEAEs normalized to participant-years of exposure (TEAEs per participant-year)."}

Secondary endpoints

• {"endpoint_text":"- 1. 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)","definition_or_measurement_approach":"Counts of participants with each listed safety outcome as recorded during study follow-up."}
• {"endpoint_text":"- 2. 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","definition_or_measurement_approach":"Proportions (percentages) of participants with each listed safety outcome."}
• {"endpoint_text":"- 3. 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","definition_or_measurement_approach":"Counts of events for each listed category (event-level counts)."}
• {"endpoint_text":"- 4. 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","definition_or_measurement_approach":"Event counts normalized per injection administered (rates per injection) for each listed category."}
• {"endpoint_text":"- 5. 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","definition_or_measurement_approach":"Event counts normalized to participant-years for each listed category."}
• {"endpoint_text":"- 6. Number of Participants with Anti-garadacimab Antibodies","definition_or_measurement_approach":"Count of participants with detected anti-garadacimab antibodies (immunogenicity testing results)."}
• {"endpoint_text":"- 7. Percentage of Participants with Anti-garadacimab Antibodies","definition_or_measurement_approach":"Proportion (percentage) of participants with detected anti-garadacimab antibodies."}
• {"endpoint_text":"- 8. Plasma Concentrations of Garadacimab","definition_or_measurement_approach":"Measured plasma concentration levels of garadacimab (pharmacokinetic sampling)."}
• {"endpoint_text":"- 9. Percentage of Participants who Indicated Their Preference for Garadacimab","definition_or_measurement_approach":"Proportion (percentage) of participants reporting a stated preference for garadacimab (patient-reported preference measure)."}

Germany

Earliest CTIS Part Ii Submission Date

24-07-2025

Latest Decision Or Authorization Date

06-10-2025

Processing Time Days

74

Number Of Sites

2

Number Of Participants

5

Primary sponsor

Full Name

CSL Behring LLC

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

PAREXEL International GmbH

Responsibilities

sponsorDuties codes: 10 (id 748042)

Name

Syneos Health Inc.

Responsibilities

sponsorDuties codes: 1, 12, 5, 8 (ids 748036-748039)

Name

Fortrea Inc.

Responsibilities

sponsorDuties codes: 6, 7 (ids 748044, 748045)

Name

MEDPACE LABORATORIES

Responsibilities

sponsorDuties code: 4 (id 748040) - laboratory services

Name

Suvoda LLC

Responsibilities

sponsorDuties code: 3 (id 748043) - eClinical services

Third parties

• {"country":"United States","full_name":"Fisher Clinical Services Inc.","duties_or_roles":"Clinical Trial Supplies (sponsorDuties id 748041, value: Clinical Trial Supplies)","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"PAREXEL International GmbH","duties_or_roles":"sponsorDuties codes: 10 (id 748042)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"sponsorDuties codes: 1, 12, 5, 8 (ids 748036-748039)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Kcas LLC","duties_or_roles":"sponsorDuties code: 4 (id 748047)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"sponsorDuties code: 3 (id 748043)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"sponsorDuties code: 7 (id 748035)","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"MEDPACE LABORATORIES","duties_or_roles":"sponsorDuties code: 4 (id 748040)","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"sponsorDuties codes: 6, 7 (ids 748044, 748045)","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"Clinical Trial Supplies (sponsorDuties id 748046, value: Clinical Trial Supplies)","organisation_type":"Pharmaceutical company"}