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 III Study of the Efficacy and Safety 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: NCT00262301
• Other study ID #: C1 1304-01
• Status: Completed
• Phase: Phase 3
• First received: December 1, 2005
• Last updated: September 27, 2012
• Start date: June 2004
• Est. completion date: October 2009

Study information

• Verified date: September 2012
• Source: Pharming Technologies B.V.
• Contact: n/a
• Is FDA regulated: No
• Health authority: Netherlands: Independent Ethics Committee
• 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 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.

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 75
• Est. completion date: October 2009
• Est. primary completion date: July 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years and older

Eligibility

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

Exclusion Criteria:

• Acquired angioedema

• Pregnancy or breastfeeding

• Participation in another clinical study within prior 3 months

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
• Genetic Disorders
• Hereditary Angioedema

Intervention

Drug:
• recombinant human C1 inhibitor
IV
• Placebo
IV

Locations

Country	Name	City	State
Netherlands	For information on sites, please contact Pharming Medical Affairs Deparment	Leiden
Romania	Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department	Tirgu Mures

Sponsors (1)

Lead Sponsor	Collaborator
Pharming Technologies B.V.

Countries where clinical trial is conducted

Netherlands,  Romania, 

References & Publications (2)

Moldovan D, Reshef A, Fabiani J, Kivity S, Toubi E, Shlesinger M, Triggiani M, Montinaro V, Cillari E, Realdi G, Cancian M, Visscher S, Zanichelli A, Relan A, Cicardi M. Efficacy and safety of recombinant human C1-inhibitor for the treatment of attacks of — View Citation

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 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.	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 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.	up to 48 hours after study drug administration	No