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

• Status: Completed
• Trial end date: 2010-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Pharming Technologies B.V.
• Treatments: Complement C1 Inactivator Proteins; Complement C1 Inhibitor Protein

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