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

Phase 2

Completed

• Conditions: Hereditary Angioedema; Angioneurotic Edema
• Interventions: Drug: Recombinant Human C1 Inhibitor; Drug: placebo

• Registration Number: NCT00225147
• Lead Sponsor: Pharming Technologies B.V.

Brief 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

Detailed Description

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

Study Timeline

• Study Start: 2005-07
• Study First Submitted: 2005-09-20
• Study First Submitted QC: 2005-09-21
• Study First Posted: 2005-09-23
• Primary Completion: 2009-10
• Study Completion: 2010-01
• Results First Submitted: 2012-02-22
• Results First Submitted QC: 2012-07-27
• Results First Posted: 2012-08-31
• Status Verified: 2013-02
• Last Update Submitted: 2013-02-20
• Last Update Posted: 2013-02-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 77

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
100 IU/kg rhC1INH	Recombinant Human C1 Inhibitor	100 IU/kg Recombinant human C1 inhibitor
50 IU/kg rhC1INH	Recombinant Human C1 Inhibitor	50 IU/kg Recombinant human C1 inhibitor
Saline	placebo	-

Primary Outcome Measures

Name	Time	Method
Time to Beginning of Relief of Symptoms	up to 48 hours after study drug administration	The time to beginning of relief of symptoms at the location that showed the first visual analogue scale ("VAS") score decrease of at least 20 mm from baseline score with persistence to the next timepoint, assessment timepoints were taken on pre-scheduled time-points after study drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to beginning of relief has been calculated as median time, by using the exact timepoints on which each assessment was performed.

Secondary Outcome Measures

Name	Time	Method
Time to Minimal Symptoms	up to 48 hours after study drug administration	The time to minimal symptoms was the time to minimal symptoms for an attack, assessed using the Visual Analogue Scale ("VAS") score. Symptoms were said to be minimal when the "VAS" score at all locations was below 20 mm. Assessment timepoints were: baseline, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to minimal symtoms has been calculated by using the exact timepoints on which each assessment was performed.

Trial Locations

Locations (1)

For information on sites please contact Pharming Medical Affairs Department; 🇳🇱 Leiden, Netherlands