A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE)

CSL Behring

Status and phase: Completed; Phase 2

Conditions: Hereditary Angioedema

Treatments: Drug: Placebo; Biological: Factor XIIa antagonist monoclonal antibody

Study type: Interventional

Funder types: Industry

Identifiers

NCT03712228

2018-000605-24 (EudraCT Number)

CSL312_2001

Details and patient eligibility

About

This is a multicenter, randomized, placebo-controlled, parallel-arm, phase 2 study to investigate the clinical efficacy, pharmacokinetics, and safety of CSL312 as prophylaxis to prevent attacks in subjects with HAE.

Enrollment: 44 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female
• Aged ≥ 18 to ≤ 65 years
• A diagnosis of C1-INH HAE or FXII/PLG HAE;
• For subjects with C1-INH HAE: ≥ 4 HAE attacks over a consecutive 2-month period during the 3 months before Screening, as documented in the subject's medical record.

Exclusion criteria

• History of clinically significant arterial or venous thrombosis, or current clinically significant prothrombotic risk
• History of an uncontrolled, abnormal bleeding event due to a coagulopathy, or a current clinically significant coagulopathy or clinically significant risks for bleeding events
• Known incurable malignancies

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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44 participants in 5 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Subjects with C1-INH HAE receiving buffer only

Treatment:

Drug: Placebo

CSL312 (low)

Active Comparator group

Description:

Subjects with C1-INH HAE receiving low dose CSL312

Treatment:

Biological: Factor XIIa antagonist monoclonal antibody

CSL312 (med)

Active Comparator group

Description:

Subjects with C1-INH HAE receiving medium dose CSL312

Treatment:

Biological: Factor XIIa antagonist monoclonal antibody

CSL312 (high)

Active Comparator group

Description:

Subjects with C1-INH HAE receiving high dose CSL312

Treatment:

Biological: Factor XIIa antagonist monoclonal antibody

CSL312 (med/high)

Active Comparator group

Description:

Subjects with C1-INH HAE receiving medium/high dose CSL312

Treatment:

Biological: Factor XIIa antagonist monoclonal antibody