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

Pharming Healthcare

Status and phase: Completed; Phase 3; Phase 2

Conditions: Angioneurotic Edema; Hereditary Angioedema

Treatments: Drug: placebo; Drug: Recombinant Human C1 Inhibitor

Study type: Interventional

Funder types: Industry

Identifiers

NCT00225147

C1 1205-01

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 lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.

Funding Source - FDA OOPD

Full description

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

Enrollment: 77 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Main Inclusion Criteria:

• Clear clinical and laboratory diagnosis of HAE
• Plasma level of functional C1INH of less than 50% of normal
• Acute abdominal, urogenital, peripheral, and/or oro-facial/pharyngeal/laryngeal HAE attack

Main Exclusion Criteria:

• Acquired angioedema
• Pregnancy or breastfeeding
• Treatment with any investigational drug within prior 30 days
• Body weight >120 kg

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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77 participants in 3 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

50 IU/kg rhC1INH

Experimental group

Description:

50 IU/kg Recombinant human C1 inhibitor

Treatment:

Drug: Recombinant Human C1 Inhibitor

Saline

Placebo Comparator group

Treatment:

Drug: placebo