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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/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
Other interventions - C1 Esterase Inhibitor
Other interventions - 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 -


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

Other interventions: 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
Other interventions
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/Phase 3
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/ct2/show/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.
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/ct2/show/NCT00168103