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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02316353




Registration number
NCT02316353
Ethics application status
Date submitted
10/12/2014
Date registered
12/12/2014
Date last updated
14/11/2018

Titles & IDs
Public title
A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema
Scientific title
An Open-label, Randomized Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Hereditary Angioedema
Secondary ID [1] 0 0
2014-001054-42
Secondary ID [2] 0 0
CSL830_3002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema Types I and II 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
Cardiovascular 0 0 0 0
Other cardiovascular diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Experimental: C1-INH - low-volume dose - A low-volume dose of C1-INH will be administered subcutaneously twice a week for up to 52 weeks (up to 146 weeks extension period).

Experimental: C1-INH - medium-volume dose - A medium-volume dose of C1-INH will be administered subcutaneously twice a week for up to 52 weeks (up to 146 weeks extension period).
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Person-time Incidence Rates (Subject Based)
Timepoint [1] 0 0
Up to 146 weeks.
Primary outcome [2] 0 0
The Person-time Incidence Rates (Event Based)
Timepoint [2] 0 0
Up to 146 weeks.
Secondary outcome [1] 0 0
Percentage of Subjects Who Have Solicited Adverse Events (AEs)
Timepoint [1] 0 0
Up to 146 weeks
Secondary outcome [2] 0 0
Percentage of Injections Followed by At Least One Solicited Adverse Event
Timepoint [2] 0 0
Up to 146 weeks
Secondary outcome [3] 0 0
Percentage of Subjects Who Become Seropositive for Human Immunodeficiency Virus (HIV-1/-2), Hepatitis B Virus, or Hepatitis C Virus.
Timepoint [3] 0 0
Up to 146 weeks
Secondary outcome [4] 0 0
Percentage of Subjects Who Experience a Time-normalized HAE Attack Frequency of <1 HAE Attack Per 4-Week Period
Timepoint [4] 0 0
Up to 146 weeks
Secondary outcome [5] 0 0
Percentage of Subjects Who Are Responders
Timepoint [5] 0 0
Up to 146 weeks

Eligibility
Key inclusion criteria
* Males or females aged 6 years or older.
* A confirmed diagnosis of HAE type I or II.
* HAE attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment.
* For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of first study visit: use of a stable regimen within 3 months of the first study visit.
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Incurable malignancies.
* Any clinical condition that will interfere with the evaluation of C1-INH therapy.
* Clinically significant history of poor response to C1-esterase therapy for the management of HAE.
* Suspected or confirmed diagnosis of acquired HAE or HAE with normal C1-INH.
* Inability to have HAE managed pharmacologically with on-demand treatment.

Study design
Purpose of the study
Prevention
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
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
31/12/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
21/09/2017
Sample size
Target
Accrual to date
Final
126
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Study Site - Campbelltown
Recruitment postcode(s) [1] 0 0
2560 - Campbelltown
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
Oklahoma
Country [7] 0 0
United States of America
State/province [7] 0 0
Oregon
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
United States of America
State/province [10] 0 0
Virginia
Country [11] 0 0
Canada
State/province [11] 0 0
Ontario
Country [12] 0 0
Canada
State/province [12] 0 0
Quebec
Country [13] 0 0
Czechia
State/province [13] 0 0
Plzen
Country [14] 0 0
Germany
State/province [14] 0 0
Hesse
Country [15] 0 0
Germany
State/province [15] 0 0
Berlin
Country [16] 0 0
Germany
State/province [16] 0 0
Frankfurt
Country [17] 0 0
Germany
State/province [17] 0 0
Mainz
Country [18] 0 0
Hungary
State/province [18] 0 0
Budapest
Country [19] 0 0
Israel
State/province [19] 0 0
Tel Aviv
Country [20] 0 0
Israel
State/province [20] 0 0
Tel Hashomer
Country [21] 0 0
Italy
State/province [21] 0 0
Catania
Country [22] 0 0
Italy
State/province [22] 0 0
Milano
Country [23] 0 0
Romania
State/province [23] 0 0
Cluj Napoca
Country [24] 0 0
Spain
State/province [24] 0 0
Madrid
Country [25] 0 0
Spain
State/province [25] 0 0
Valencia
Country [26] 0 0
United Kingdom
State/province [26] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Global Clinical Program Director
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02316353