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

Phase 3

Completed

• Conditions: Hereditary Angioedema; Angioneurotic Edema; Genetic Disorders
• Interventions: Drug: Placebo; Drug: recombinant human C1 inhibitor

• Registration Number: NCT00262301
• 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 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.

Detailed Description

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

Study Timeline

• Study Start: 2004-06
• Study First Submitted: 2005-12-01
• Study First Submitted QC: 2005-12-05
• Study First Posted: 2005-12-06
• Primary Completion: 2009-07
• Study Completion: 2009-10
• Results First Submitted: 2012-07-27
• Results First Submitted QC: 2012-07-27
• Results First Posted: 2012-08-30
• Status Verified: 2012-09
• Last Update Submitted: 2012-09-27
• Last Update Posted: 2012-10-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 75

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

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Exclusion Criteria

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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Saline	Placebo	Saline solution
100 IU/kg "rhC1INH"	recombinant human C1 inhibitor	100 IU/kg recombinant human C1 inhibitor

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 has been assessed by using a patient-reported visual analogue scale ("VAS") ranging from 0 mm (no symptoms at all) to 100 mm (extremely disabling). Time to beginning of relief of symptoms at the location that showed first "VAS" score decrease of at least 20 mm from baseline score (t= 0 min) to the next assessment time-point). Assessment time-points were taken on pre-scheduled time-points after drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to beginning of relief has been calculated as median time, by using the exact time-points 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 time-points were: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to minimal symptoms has been calculated by using the exact time-points on which each assessment was performed.

Trial Locations

Locations (2)

Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department; 🇷🇴 Tirgu Mures, Romania

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