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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00168103




Registration number
NCT00168103
Ethics application status
Date submitted
12/09/2005
Date registered
14/09/2005
Date last updated
31/03/2015

Titles & IDs
Public title
Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks
Scientific title
Human Pasteurized C1 Esterase Inhibitor Concentrate (CE1145) in Subjects With Congenital C1-INH Deficiency and Acute Abdominal or Facial HAE Attacks
Secondary ID [1] 0 0
2004-001186-17
Secondary ID [2] 0 0
CE1145_3001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - C1 Esterase Inhibitor
Treatment: Other - Placebo

Experimental: C1-INH 10 U/kg bw - 10 Units (U)/kg body weight (bw) dose

Experimental: C1-INH 20 U/kg bw - 20 U/kg bw dose

Placebo comparator: Placebo -


Treatment: Other: C1 Esterase Inhibitor
Single application of C1-INH administered intravenously by slow injection or infusion at a recommended rate of 4mL/min.

Treatment: Other: Placebo
Single application of physiological saline solution equivalent to the volume calculated for subjects in the C1-INH 20 U/kg bw arm.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time to Start of Relief of Symptoms From HAE Attack
Timepoint [1] 0 0
Up to 24 h after start of study treatment
Secondary outcome [1] 0 0
Number of Subjects With Worsened Intensity of Clinical HAE Symptoms
Timepoint [1] 0 0
Baseline and between 2 and 4 h after start of study treatment
Secondary outcome [2] 0 0
Number of Vomiting Episodes
Timepoint [2] 0 0
Within 4 h after start of study treatment

Eligibility
Key inclusion criteria
Key

* Documented congenital C1-INH deficiency
* Acute facial or abdominal HAE attack

Key
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Acquired angioedema
* Treatment with any other investigational drug within the last 30 days before study entry
* Treatment with any C1-INH concentrate within the previous 7 days

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2005
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/12/2007
Sample size
Target
Accrual to date
Final
126
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Study Site - Westmead
Recruitment postcode(s) [1] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Idaho
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Louisiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
Nebraska
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Oklahoma
Country [13] 0 0
United States of America
State/province [13] 0 0
Oregon
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
South Dakota
Country [16] 0 0
United States of America
State/province [16] 0 0
Texas
Country [17] 0 0
United States of America
State/province [17] 0 0
Washington
Country [18] 0 0
Argentina
State/province [18] 0 0
Buenos Aires
Country [19] 0 0
Bulgaria
State/province [19] 0 0
Plovdiv
Country [20] 0 0
Bulgaria
State/province [20] 0 0
Sofia
Country [21] 0 0
Canada
State/province [21] 0 0
Edmonton
Country [22] 0 0
Canada
State/province [22] 0 0
Ottawa
Country [23] 0 0
Czech Republic
State/province [23] 0 0
Brno
Country [24] 0 0
Hungary
State/province [24] 0 0
Budapest
Country [25] 0 0
Israel
State/province [25] 0 0
Tel Hashomer
Country [26] 0 0
Macedonia, The Former Yugoslav Republic of
State/province [26] 0 0
Skopje
Country [27] 0 0
Poland
State/province [27] 0 0
Grodzisk Mazowiecki
Country [28] 0 0
Poland
State/province [28] 0 0
Krakow
Country [29] 0 0
Romania
State/province [29] 0 0
Tirgu-Mures
Country [30] 0 0
Russian Federation
State/province [30] 0 0
Moscow
Country [31] 0 0
Spain
State/province [31] 0 0
Madrid
Country [32] 0 0
Sweden
State/province [32] 0 0
Goeteborg
Country [33] 0 0
United Kingdom
State/province [33] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
CSL Behring
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
HAE is a rare disorder characterized by functional C1 esterase inhibitor deficiency. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of swelling of the larynx. This clinical Phase 2/Phase 3 study was designed to provide clinically relevant data on dosing, efficacy and safety in subjects with HAE.
Trial website
https://clinicaltrials.gov/study/NCT00168103
Trial related presentations / publications
Bernstein JA, Ritchie B, Levy RJ, Wasserman RL, Bewtra AK, Hurewitz DS, Obtulowicz K, Reshef A, Moldovan D, Shirov T, Grivcheva-Panovska V, Kiessling PC, Keinecke HO, Craig TJ. Hereditary angioedema: Validation of the end point time to onset of relief by correlation with symptom intensity. Allergy Asthma Proc. 2011 Jan-Feb;32(1):36-42. doi: 10.2500/aap.2011.32.3404.
Craig TJ, Levy RJ, Wasserman RL, Bewtra AK, Hurewitz D, Obtulowicz K, Reshef A, Ritchie B, Moldovan D, Shirov T, Grivcheva-Panovska V, Kiessling PC, Keinecke HO, Bernstein JA. Efficacy of human C1 esterase inhibitor concentrate compared with placebo in acute hereditary angioedema attacks. J Allergy Clin Immunol. 2009 Oct;124(4):801-8. doi: 10.1016/j.jaci.2009.07.017. Epub 2009 Sep 19.
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.
Bernstein JA, Ritchie B, Levy RJ, Wasserman RL, Bewtra AK, Hurewitz DS, Obtulowicz K, Reshef A, Moldovan D, Shirov T, Grivcheva-Panovska V, Kiessling PC, Schindel F, Craig TJ. Population pharmacokinetics of plasma-derived C1 esterase inhibitor concentrate used to treat acute hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2010 Aug;105(2):149-54. doi: 10.1016/j.anai.2010.06.005. Erratum In: Ann Allergy Asthma Immunol. 2011 Jan;106(1):78.
Public notes

Contacts
Principal investigator
Name 0 0
Program Director, Clinical R&D
Address 0 0
CSL Behring
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00168103