Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Pharming Healthcare

Status and phase: Completed; Phase 3

Conditions: Angioneurotic Edema; Genetic Disorders; Hereditary Angioedema

Treatments: Drug: recombinant human C1 inhibitor; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT00262301

C1 1304-01

Details and patient eligibility

About

Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that leads to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy and pharmacodynamics/ pharmacokinetics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.

Full description

A prospectively planned interim analysis will be performed on the double-blind data.

Enrollment: 75 patients

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Clear clinical and laboratory diagnosis of HAE
• Baseline plasma level of functional C1INH of less than 50% of normal
• Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack

Exclusion criteria

• Acquired angioedema
• Pregnancy or breastfeeding
• Participation in another clinical study within prior 3 months

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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75 participants in 2 patient groups, including a placebo group

100 IU/kg "rhC1INH"

Experimental group

Description:

100 IU/kg recombinant human C1 inhibitor

Treatment:

Drug: recombinant human C1 inhibitor

Saline

Placebo Comparator group

Description:

Saline solution

Treatment:

Drug: Placebo