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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04739059




Registration number
NCT04739059
Ethics application status
Date submitted
1/02/2021
Date registered
4/02/2021

Titles & IDs
Public title
Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks
Scientific title
An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema
Secondary ID [1] 0 0
2020-003918-12
Secondary ID [2] 0 0
CSL312_3002
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 - CSL312

Experimental: CSL312 - Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody administered subcutaneously


Treatment: Other: CSL312
Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of subjects with treatment emergent adverse events (TEAEs)
Timepoint [1] 0 0
Up to 45 months
Primary outcome [2] 0 0
Percentage of subjects with TEAEs
Timepoint [2] 0 0
Up to 45 months
Primary outcome [3] 0 0
TEAEs rates per injection
Timepoint [3] 0 0
Up to 45 months
Primary outcome [4] 0 0
TEAEs rates per subject year
Timepoint [4] 0 0
Up to 45 months
Secondary outcome [1] 0 0
The time-normalized number (per month and year) of Hereditary Angioedema (HAE attacks) for the entire study
Timepoint [1] 0 0
Up to 43 months
Secondary outcome [2] 0 0
The percentage reduction and the number of subjects experiencing at least = 50% = 70%, = 90 or equal to 100% (attack free) reduction in the time-normalized number of HAE attacks on Treatment compared to Run-in Period
Timepoint [2] 0 0
Up to 43 months
Secondary outcome [3] 0 0
The time-normalized number (per month and year) of HAE attacks requiring on-demand treatment in subjects on treatment
Timepoint [3] 0 0
Up to 43 months
Secondary outcome [4] 0 0
The time-normalized number (per month and year) of moderate and/or severe HAE attacks in subjects on treatment
Timepoint [4] 0 0
Up to 43 months
Secondary outcome [5] 0 0
Number and percentage of subjects rating their response to therapy as good or excellent
Timepoint [5] 0 0
Up to 43 months
Secondary outcome [6] 0 0
The number and percentage of subjects experiencing TEAEs
Timepoint [6] 0 0
Up to 45 months
Secondary outcome [7] 0 0
The number and percentage of subjects experiencing adverse events of special interest (AESIs)
Timepoint [7] 0 0
Up to 45 months
Secondary outcome [8] 0 0
The number and percentage of subjects experiencing serious adverse events (SAEs), including deaths
Timepoint [8] 0 0
Up to 45 months
Secondary outcome [9] 0 0
The number and percentage of subjects experiencing CSL312 induced anti-CSL312 antibodies
Timepoint [9] 0 0
Up to 45 months

Eligibility
Key inclusion criteria
* Males and females aged = 12 years
* Diagnosed with clinically confirmed C1-INH HAE
* Experienced = 3 HAE attacks during the 3 months before Screening
* Participated in the Run-in Period for at least 1 month (CSL312-naïve subjects only)
* Experienced at least an average of 1 HAE attack per month during the Run-in Period
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema or recurrent angioedema associated with urticaria
* Use of C1-INH products, androgens, antifibrinolytics or other small molecule medications for routine prophylaxis against HAE attacks at least 2 weeks before the first day of the Run-in Period
* Use of monoclonal antibodies such as lanadelumab (Takhzyro®) 3 months before the first day of the Run-in Period.
* Female subjects use estrogen-containing oral contraceptives or hormone replacement therapy within 4 weeks prior to screening
* Female or male subjects who are fertile and sexually active not using or not willing to use an acceptable method of contraception to avoid pregnancy during the study and for 30 days after receipt of the last dose of CSL312
* Pregnant, breastfeeding, or not willing to cease breastfeeding

Study design
Purpose of the study
Prevention
Allocation to intervention
NA
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
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/03/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2025
Actual
Sample size
Target
Accrual to date
Final
171
Recruitment in Australia
Recruitment state(s)
NSW,VIC,West Australi
Recruitment hospital [1] 0 0
Campbelltown Hospital / Western Sydney University - Campbelltown
Recruitment hospital [2] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [3] 0 0
Fiona Stanley Hospital, Department of Clinical Immunology - Murdoch
Recruitment postcode(s) [1] 0 0
2560 - Campbelltown
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment postcode(s) [3] 0 0
6150 - Murdoch
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
Arkansas
Country [4] 0 0
United States of America
State/province [4] 0 0
California
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
Canada
State/province [9] 0 0
Alberta
Country [10] 0 0
Canada
State/province [10] 0 0
Ontario
Country [11] 0 0
Canada
State/province [11] 0 0
Quebec
Country [12] 0 0
Czechia
State/province [12] 0 0
Brno
Country [13] 0 0
Czechia
State/province [13] 0 0
Praha 5
Country [14] 0 0
Germany
State/province [14] 0 0
Berlin
Country [15] 0 0
Germany
State/province [15] 0 0
Frankfurt
Country [16] 0 0
Germany
State/province [16] 0 0
Mainz
Country [17] 0 0
Germany
State/province [17] 0 0
Mörfelden-Walldorf
Country [18] 0 0
Hong Kong
State/province [18] 0 0
Hong Kong
Country [19] 0 0
Hungary
State/province [19] 0 0
Budapest
Country [20] 0 0
Israel
State/province [20] 0 0
Ashkelon
Country [21] 0 0
Japan
State/province [21] 0 0
Daikakuji Yaizu-shi
Country [22] 0 0
Japan
State/province [22] 0 0
Edobashi, Tsu-shi
Country [23] 0 0
Japan
State/province [23] 0 0
Hongo Bunkyo-ku
Country [24] 0 0
Japan
State/province [24] 0 0
Kamoda Kawagoe-shi
Country [25] 0 0
Japan
State/province [25] 0 0
Kawasaki-shi
Country [26] 0 0
Japan
State/province [26] 0 0
Kugenumaishigami, Fujisawa-shi
Country [27] 0 0
Japan
State/province [27] 0 0
Matsubara Soka-shi
Country [28] 0 0
Japan
State/province [28] 0 0
Midoricho, Tachikawa-shi
Country [29] 0 0
Japan
State/province [29] 0 0
Nebeshima, Saga-shi
Country [30] 0 0
Japan
State/province [30] 0 0
Osaka-shi
Country [31] 0 0
Netherlands
State/province [31] 0 0
Amsterdam
Country [32] 0 0
New Zealand
State/province [32] 0 0
Auckland
Country [33] 0 0
Russian Federation
State/province [33] 0 0
Moscow
Country [34] 0 0
Spain
State/province [34] 0 0
Barcelona
Country [35] 0 0
Spain
State/province [35] 0 0
Madrid
Country [36] 0 0
Taiwan
State/province [36] 0 0
Taichung City

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
Study 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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
CSL will consider requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at [email protected].

Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.

If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP)
When will data be available (start and end dates)?
IPD requests may be submitted to CSL no earlier than 12 months after publication of the results of this study via an article made available on a public website.
Available to whom?
Requests may only be made by systematic review groups or bona-fide researchers whose proposed use of the IPD is non-commercial in nature and has been approved by an internal review committee.

An IPD request will not be considered by CSL unless the proposed research question seeks to answer a significant and unknown medical science or patient care question as determined by CSL's internal review committee.

The requesting party must execute an appropriate data sharing agreement before IPD will be made available.
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04739059