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

CSL Behring

Status and phase

Enrolling

Phase 4

Conditions

Hereditary Angioedema

Treatments

Biological: Garadacimab

Study type

Interventional

Funder types

Industry

Identifiers

NCT06806657

CSL312_4002

2024-517757-27-00 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment

30 estimated patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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.