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

Hereditary Angioedema Clinical Trial

Official title:

A Randomized, Placebo-controlled, Double Blind Phase II/III Study of the Safety and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00225147
• Other study ID #: C1 1205-01
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: September 20, 2005
• Last updated: February 20, 2013
• Start date: July 2005
• Est. completion date: January 2010

Study information

• Verified date: February 2013
• Source: Pharming Technologies B.V.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationCanada: Health Canada
• Study type: Interventional

Clinical Trial 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

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 77
• Est. completion date: January 2010
• Est. primary completion date: October 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Angioedema
• Angioneurotic Edema
• Hereditary Angioedema

Intervention

Drug:
• Recombinant Human C1 Inhibitor
IV
• placebo
saline solution

Locations

Country	Name	City	State
Netherlands	For information on sites please contact Pharming Medical Affairs Department	Leiden

Sponsors (1)

Lead Sponsor	Collaborator
Pharming Technologies B.V.

Country where clinical trial is conducted

Netherlands, 

References & Publications (1)

Zuraw B, Cicardi M, Levy RJ, Nuijens JH, Relan A, Visscher S, Haase G, Kaufman L, Hack CE. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010 Oct;126(4):821-827 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time to Beginning of Relief of Symptoms	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.	up to 48 hours after study drug administration	No
Secondary	Time to Minimal Symptoms	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.	up to 48 hours after study drug administration	No