A study to evaluate the long-term clinical safety and efficacy of subcutaneously administered C1-esterase inhibitor in the prevention of hereditary angioedema

An open-label, randomized study to evaluate the long-term clinical safety and efficacy of subcutaneous administration of human plasma-derived C1-esterase inhibitor in the prophylactic treatment of hereditary angioedema

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-001054-42-DE

Enrollment

110

Registered

2014-10-27

Start date

2015-03-04

Completion date

Unknown

Last updated

2018-01-08

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

Conditions

Hereditary Angioedema Types I and II MedDRA version: 18.0 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: C1-esterase inhibitor Product Code: CSL830 Pharmaceutical Form: Powder and solvent for solution for injection Current Sponsor code: CSL830 Other descriptive name: COMPLEMENT C1 ESTERASE

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: "• Males or females aged 6 years or older. • A confirmed diagnosis of HAE type I or II. • HAE attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment. • For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of first study visit: use of a stable regimen within 3 months of the first study visit." Are the trial subjects under 18? yes Number of subjects for this age range: 11 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 88 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 11

Exclusion criteria

Exclusion criteria: • Incurable malignancies. • Any clinical condition that will interfere with the evaluation of C1-INH therapy. • Clinically significant history of poor response to C1-esterase therapy for the management of HAE. • Suspected or confirmed diagnosis of acquired HAE or HAE with normal C1-INH. • Inability to have HAE managed pharmacologically with on-demand treatment."

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the clinical safety of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE. ;Secondary Objective: • To further characterize the clinical safety of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE. • To characterize the clinical efficacy of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE." ;Primary end point(s): The person-time incidence rates of specified safety events. ;Timepoint(s) of evaluation of this end point: During the treatment phase, up to 52 weeks.	—

Measure

Time frame

Main Objective: To assess the clinical safety of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE. ;Secondary Objective: • To further characterize the clinical safety of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE. • To characterize the clinical efficacy of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE." ;Primary end point(s): The person-time incidence rates of specified safety events. ;Timepoint(s) of evaluation of this end point: During the treatment phase, up to 52 weeks.

—

Secondary

Measure	Time frame
Secondary end point(s): - Percentage of subjects with SAEs or other specified safety events. - Percentage of C1-INH injections resulting in solicited AEs (injection site reactions). - Percentage of subjects with at least 1 solicited AE (injection site reaction). - Percentage of subjects who become seropositive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus. - Percentage of subjects who experience < 1 HAE attack per 4-week period. - Percentage of subjects with a = 50% reduction in the time-normalized number of HAE attacks. ;Timepoint(s) of evaluation of this end point: - During the treatment phase, up to 52 weeks. - During the treatment phase, up to 52 weeks. - During the treatment phase, up to 52 weeks. - From baseline through the treatment phase, up to 52 weeks. - During the treatment phase, up to 52 weeks. - From baseline through the treatment phase, up to 52 weeks.	—

Measure

Time frame

Secondary end point(s): - Percentage of subjects with SAEs or other specified safety events. - Percentage of C1-INH injections resulting in solicited AEs (injection site reactions). - Percentage of subjects with at least 1 solicited AE (injection site reaction). - Percentage of subjects who become seropositive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus. - Percentage of subjects who experience < 1 HAE attack per 4-week period. - Percentage of subjects with a = 50% reduction in the time-normalized number of HAE attacks. ;Timepoint(s) of evaluation of this end point: - During the treatment phase, up to 52 weeks. - During the treatment phase, up to 52 weeks. - During the treatment phase, up to 52 weeks. - From baseline through the treatment phase, up to 52 weeks. - During the treatment phase, up to 52 weeks. - From baseline through the treatment phase, up to 52 weeks.

—

Countries

Australia, Canada, Czech Republic, European Union, Germany, Hungary, Israel, Italy, Spain, United Kingdom, United States

Contacts

Public ContactTrial Registration Coordinator

CSL Behring GmbH

clinicaltrials@cslbehring.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026