Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks

CSL Behring

Status and phase: Completed; Phase 3; Phase 2

Conditions: Hereditary Angioedema

Treatments: Biological: C1 Esterase Inhibitor; Biological: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT00168103

2004-001186-17 (EudraCT Number)

CE1145_3001

Details and patient eligibility

About

HAE is a rare disorder characterized by functional C1 esterase inhibitor deficiency. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of swelling of the larynx. This clinical Phase 2/Phase 3 study was designed to provide clinically relevant data on dosing, efficacy and safety in subjects with HAE.

Full description

For each subject, only a single abdominal or facial attack was treated and evaluated. After receiving treatment, subjects were observed for a minimum of 4 hours, after which they could be discharged from the study center if they reported onset of symptom relief. Starting from 4 hours after treatment, subjects who reported insufficient or no symptom relief could receive a second dose of double-blind treatment (called "rescue medication") as follows: C1-INH 20 U/kg bw for subjects initially receiving placebo, C1-INH 10 U/kg bw for subjects initially receiving C1-INH 10 U/kg bw, and placebo for subjects initially receiving C1-INH 20 U/kg bw.

The study was defined to be successful if the primary outcome measure and at least one of the secondary outcome measures were met in the comparison between the C1-INH 20 U/kg bw group and the Placebo group.

Enrollment: 126 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Documented congenital C1-INH deficiency
• Acute facial or abdominal HAE attack

Key Exclusion Criteria:

• Acquired angioedema
• Treatment with any other investigational drug within the last 30 days before study entry
• Treatment with any C1-INH concentrate within the previous 7 days

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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126 participants in 3 patient groups, including a placebo group

C1-INH 10 U/kg bw

Experimental group

Description:

10 Units (U)/kg body weight (bw) dose

Treatment:

Biological: C1 Esterase Inhibitor

C1-INH 20 U/kg bw

Experimental group

Description:

20 U/kg bw dose

Treatment:

Biological: C1 Esterase Inhibitor

Placebo

Placebo Comparator group

Treatment:

Biological: Placebo