Clinical trial NCT00292981: C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)
Official title: Open-label Extension Study of CE1145 (Human Pasteurized C1 Esterase Inhibitor Concentrate) in Subjects With Congenital C1-INH Deficiency and Acute HAE Attacks
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hereditary Angioedema
Keywords: C1 Inhibitor
Hereditary angioedema
Acute HAE attack
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2006-02-16
Enrollment: 57
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Hereditary angioedema (HAE) is a rare disorder characterized by congenital lack of functional C1 esterase inhibitor. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of involvement of the larynx.The planned extension study is designed to enrol subjects that participated in the pivotal study in order to provide them with C1-INH for treatment of acute HAE attacks for 24 months or until the licensing procedure for C1-INH is finalized, whatever comes first.


Eligibility criteria: Key Inclusion Criteria:

* Documented congenital C1-INH deficiency
* Acute HAE attack
* Participation in base study CE1145_3001 (NCT00168103)

Key Exclusion Criteria:

* Acquired angioedema
* Treatment with any other investigational drug besides CE1145 in the last 30 days before study entry

Primary outcomes:
measure: Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population)
timeFrame: Up to 24 h after start of study treatment
measure: Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population)
timeFrame: Up to 24 h after start of study treatment
Secondary outcomes:
measure: Time to Complete Resolution of All HAE Symptoms (ITT Subject Population)
timeFrame: Up to Day 9 following an attack
measure: Time to Complete Resolution of All HAE Symptoms (ITT Attack Population)
timeFrame: Up to Day 9 following an attack
Other outcomes:
Locations:
city: Weston
country: United States
facility: Contact CSL Behring for facility details
state: Florida
city: Atlanta
country: United States
facility: Contact CSL Behring for facility details
state: Georgia
city: Idaho Falls
country: United States
facility: Contact CSL Behring for facility details
state: Idaho
city: Chicago
country: United States
facility: Contact CSL Behring for facility details
state: Illinois
city: Shreveport
country: United States
facility: Contact CSL Behring for facility details
state: Louisiana
city: Boston
country: United States
facility: Contact CSL Behring for facility details
state: Massachusetts
city: Plymouth
country: United States
facility: Contact CSL Behring for facility details
state: Minnesota
city: Omaha
country: United States
facility: Contact CSL Behring for facility details
state: Nebraska
city: Tulsa
country: United States
facility: Contact CSL Behring for facility details
state: Oklahoma
city: Eugene
country: United States
facility: Contact CSL Behring for facility details
state: Oregon
city: Hershey
country: United States
facility: Contact CSL Behring for facility details
state: Pennsylvania
city: Rapid City
country: United States
facility: Contact CSL Behring for facility details
state: South Dakota
city: Dallas
country: United States
facility: Contact CSL Behring for facility details
state: Texas
city: Ottawa
country: Canada
facility: Contact CSL Behring for facility details
state: Ontario
Study officials:
affiliation: CSL Behring
name: Program Director
role: STUDY_DIRECTOR
References:
citation: Craig TJ, Wasserman RL, Levy RJ, Bewtra AK, Schneider L, Packer F, Yang WH, Keinecke HO, Kiessling PC. Prospective study of rapid relief provided by C1 esterase inhibitor in emergency treatment of acute laryngeal attacks in hereditary angioedema. J Clin Immunol. 2010 Nov;30(6):823-9. doi: 10.1007/s10875-010-9442-1. Epub 2010 Jul 16.
citation: Bernstein JA, Machnig T, Keinecke HO, Whelan GJ, Craig TJ. The effect of weight on the efficacy and safety of C1 esterase inhibitor concentrate for the treatment of acute hereditary angioedema. Clin Ther. 2014 Apr 1;36(4):518-25. doi: 10.1016/j.clinthera.2014.02.005. Epub 2014 Mar 21.
citation: Bork K, Craig TJ, Bernstein JA, Feuersenger H, Machnig T, Staubach P. Efficacy of C1 esterase inhibitor concentrate in treatment of cutaneous attacks of hereditary angioedema. Allergy Asthma Proc. 2015 May-Jun;36(3):218-24. doi: 10.2500/aap.2015.36.3844. Epub 2015 Mar 23.
citation: Wasserman RL, Levy RJ, Bewtra AK, Hurewitz D, Craig TJ, Kiessling PC, Keinecke HO, Bernstein JA. Prospective study of C1 esterase inhibitor in the treatment of successive acute abdominal and facial hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2011 Jan;106(1):62-8. doi: 10.1016/j.anai.2010.10.012. Epub 2010 Nov 20.
citation: Craig TJ, Bewtra AK, Bahna SL, Hurewitz D, Schneider LC, Levy RJ, Moy JN, Offenberger J, Jacobson KW, Yang WH, Eidelman F, Janss G, Packer FR, Rojavin MA, Machnig T, Keinecke HO, Wasserman RL. C1 esterase inhibitor concentrate in 1085 Hereditary Angioedema attacks--final results of the I.M.P.A.C.T.2 study. Allergy. 2011 Dec;66(12):1604-11. doi: 10.1111/j.1398-9995.2011.02702.x. Epub 2011 Sep 2.
citation: Craig TJ, Rojavin MA, Machnig T, Keinecke HO, Bernstein JA. Effect of time to treatment on response to C1 esterase inhibitor concentrate for hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2013 Sep;111(3):211-5. doi: 10.1016/j.anai.2013.06.021. Epub 2013 Jul 16.
citation: Craig TJ, Bewtra AK, Hurewitz D, Levy R, Janss G, Jacobson KW, Packer F, Bernstein JA, Rojavin MA, Machnig T, Keinecke HO, Wasserman RL. Treatment response after repeated administration of C1 esterase inhibitor for successive acute hereditary angioedema attacks. Allergy Asthma Proc. 2012 Jul-Aug;33(4):354-61. doi: 10.2500/aap.2012.33.3589.