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

Launched by CSL BEHRING · Feb 3, 2025

Keywords

ClinConnect Summary

This clinical trial is looking at the safety of a new treatment called garadacimab for people aged 12 and older who have hereditary angioedema (HAE). The study aims to see how well patients do after switching from their current treatment, which could be another medication like a kallikrein inhibitor or a plasma-derived C1-esterase inhibitor. Participants will receive garadacimab once a month for about three months while the researchers monitor their health and any side effects.

To join the study, participants must be at least 12 years old and have a confirmed diagnosis of HAE, showing specific symptoms and laboratory results in their medical records. They should also have been using a stable dose of their current HAE medication for at least three months before the trial starts. It’s important to note that individuals with other types of angioedema or certain allergies may not be eligible for this study. While the trial is not yet recruiting, it represents a step toward finding effective treatments for those living with hereditary angioedema.

Gender

ALL

Eligibility criteria

• 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.