Subcutaneous administration of human plasma-derived C1-esterase inhibitor for prevention of hereditary angioedema attacks in Japanese subjects

An open-label, single-arm, non-randomized phase 3 study to evaluate clinical efficacy, safety, and pharmacokinetics of subcutaneous administration of human plasma-derived C1-esterase inhibitor in the prophylactic treatment of hereditary angioedema in Japanese subjects

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080225177

Enrollment

Registered

2020-04-24

Start date

2020-06-17

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hereditary angioedema (HAE) Types I and II

Interventions

investigational material(s) Generic name etc : C1-esterase inhibitor (C1-INH) INN of investigational material : - Therapeutic category code : 634 Human blood preparations Dosage and Administration for

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Males or females aged 12 years or older. - Japanese with a confirmed diagnosis of HAE type I or II. - At least 4 HAE attacks over a consecutive 2-month period as documented in the subject's medical records in the 3 months before the Screening Visit - C1-INH functional activity of less than 50% norm, AND C4 antigen concentration below the laboratory reference range - During their participation in the Run-In Period experienced >= 2 HAE attacks within any consecutive 4-week period, OR experienced >= 1 HAE attack during the first 2 weeks

Exclusion criteria

Exclusion criteria: - History of arterial or venous thrombosis requiring anticoagulant therapy, or current, clinically significant prothrombotic risk - Known malignancies at the time of the Screening Visit - Have a history of allergic reaction to C1-INH products, including Cinryze and Berinert P or other blood products - Unable to discontinue use of intravenous (IV) C1-INH for routine prophylaxis against HAE attacks or plans to use IV C1-INH for routine prophylaxis against HAE attacks by Day 1 of the Run-in Period - Assessed by the investigator as unable to have their HAE adequately managed with on-demand treatment, administered either independently or with assistance

Design outcomes

Primary

Measure	Time frame
efficacy Time-normalized number of HAE attacks during treatment with CSL830: Up to 14 weeks. pharmacokinetics C1-INH functional activity (PK: AUC0-tau, AUC0-last, Cmax, Tmax, T1/2) after the last dose of CSL830 Treatment: Up to 11 days after the last dose	—

Secondary

Measure	Time frame
efficacy - The percentage of subjects who achieved >= 90%, >= 70%, and >= 50% relative reduction of monthly HAE attack rate: Up to 14 weeks - The relative reduction in the time-normalized number of rescue medication uses: Up to 14 weeks - Mean trough C1-INH functional activity during treatment with CSL830: Up to 16 weeks - Mean trough C1-INH and C4 antigens during treatment with CSL830: Up to 16 weeks - Subject-reported Angioedema Quality of Life (AEQoL) outcome scores: Up to 16 weeks - Subject-reported Global Assessments of Response to Therapy outcomes (SGART): Up to 16 weeks - Investigator-reported Global Assessments of Response to Therapy outcomes (IGART): Up to 16 weeks safety - The number of reported adverse Events: Up to 18 weeks - Percentage of subjects reporting adverse events (AEs) and injection site reactions that begin within 24 hours of CSL830 administration: Up to 24 hours after dose	—

Measure

Time frame

efficacy - The percentage of subjects who achieved >= 90%, >= 70%, and >= 50% relative reduction of monthly HAE attack rate: Up to 14 weeks - The relative reduction in the time-normalized number of rescue medication uses: Up to 14 weeks - Mean trough C1-INH functional activity during treatment with CSL830: Up to 16 weeks - Mean trough C1-INH and C4 antigens during treatment with CSL830: Up to 16 weeks - Subject-reported Angioedema Quality of Life (AEQoL) outcome scores: Up to 16 weeks - Subject-reported Global Assessments of Response to Therapy outcomes (SGART): Up to 16 weeks - Investigator-reported Global Assessments of Response to Therapy outcomes (IGART): Up to 16 weeks safety - The number of reported adverse Events: Up to 18 weeks - Percentage of subjects reporting adverse events (AEs) and injection site reactions that begin within 24 hours of CSL830 administration: Up to 24 hours after dose

—

Countries

Japan

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026