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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05819775




Registration number
NCT05819775
Ethics application status
Date submitted
4/04/2023
Date registered
19/04/2023

Titles & IDs
Public title
CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema
Scientific title
A Phase 3 Open-label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema in Pediatric Subjects 2 to 11 Years of Age
Secondary ID [1] 0 0
2022-502386-13-00
Secondary ID [2] 0 0
CSL312_3003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema (HAE) 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 - Ages 2-5 years and 6-11 years will have specific subcutaneous dosing schedules


Treatment: Other: CSL312
Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody administered subcutaneously (SC)
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
At least 14 months
Primary outcome [2] 0 0
Percent of subjects with TEAEs
Timepoint [2] 0 0
At lease 14 months
Primary outcome [3] 0 0
Number of TEAEs
Timepoint [3] 0 0
At least 14 months
Primary outcome [4] 0 0
TEAE rates per injection
Timepoint [4] 0 0
At least 14 months
Primary outcome [5] 0 0
TEAE rates per subject year
Timepoint [5] 0 0
At least 14 months
Primary outcome [6] 0 0
Maximum concentration (Cmax) of CSL312 at steady-state
Timepoint [6] 0 0
At least 12 months
Primary outcome [7] 0 0
Trough concentration (Ctrough) of CSL312 at steady-state
Timepoint [7] 0 0
At least 12 months
Primary outcome [8] 0 0
Time to maximum concentration (Tmax) of CSL312 at steady-state
Timepoint [8] 0 0
At least 12 months
Secondary outcome [1] 0 0
Time-normalized number of HAE attacks per month and per year
Timepoint [1] 0 0
At least 12 months
Secondary outcome [2] 0 0
Time-normalized number of HAE attacks treated with on-demand treatment per month and per year
Timepoint [2] 0 0
At least 12 months
Secondary outcome [3] 0 0
Time-normalized number of moderate and / or severe HAE attacks per month and per year
Timepoint [3] 0 0
At least 12 months
Secondary outcome [4] 0 0
Percentage reduction in the time-normalized number of HAE attacks
Timepoint [4] 0 0
At least 12 months
Secondary outcome [5] 0 0
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
Timepoint [5] 0 0
At least 12 months
Secondary outcome [6] 0 0
Number of subjects with serious adverse events (SAEs)
Timepoint [6] 0 0
At least 14 months
Secondary outcome [7] 0 0
Percent of subjects with SAEs
Timepoint [7] 0 0
At least 14 months
Secondary outcome [8] 0 0
Number of subjects experiencing death
Timepoint [8] 0 0
At least 14 months
Secondary outcome [9] 0 0
Percent of subjects experiencing death
Timepoint [9] 0 0
At least 14 months
Secondary outcome [10] 0 0
Number of subjects with related TEAEs
Timepoint [10] 0 0
At least 14 months
Secondary outcome [11] 0 0
Percent of subjects with related TEAEs
Timepoint [11] 0 0
At least 14 months
Secondary outcome [12] 0 0
Number of subjects with TEAEs leading to study discontinuation
Timepoint [12] 0 0
At least 14 months
Secondary outcome [13] 0 0
Percent of subjects with TEAEs leading to study discontinuation
Timepoint [13] 0 0
At least 14 months
Secondary outcome [14] 0 0
Number of subjects with TEAEs by severity
Timepoint [14] 0 0
At least 14 months
Secondary outcome [15] 0 0
Percent of subjects with TEAEs by severity
Timepoint [15] 0 0
At least 14 months
Secondary outcome [16] 0 0
Number of subjects with Anti-CSL312 antibodies
Timepoint [16] 0 0
At least 14 months
Secondary outcome [17] 0 0
Percent of subjects with Anti-CSL312 antibodies
Timepoint [17] 0 0
At least 14 months
Secondary outcome [18] 0 0
Number of subjects with adverse events of special interest (AESIs)
Timepoint [18] 0 0
At least 14 months
Secondary outcome [19] 0 0
Percent of subjects with AESIs
Timepoint [19] 0 0
At least 14 months
Secondary outcome [20] 0 0
FXIIa-mediated kallikrein activity
Timepoint [20] 0 0
At least 12 months
Secondary outcome [21] 0 0
Number of subjects with laboratory findings reported as AEs
Timepoint [21] 0 0
At least 14 months
Secondary outcome [22] 0 0
Percent of subjects with laboratory findings reported as AEs
Timepoint [22] 0 0
At least 14 months

Eligibility
Key inclusion criteria
1. Male or female
2. Aged 2 to 11 years, inclusive, with body weight = 10th percentile based on age
3. Diagnosed with clinically confirmed C1-INH HAE
4. Experienced = 2 HAE attacks during the 6 months before Screening
Minimum age
2 Years
Maximum age
11 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type 3
2. Use of C1-INH products, androgens, antifibrinolytics, approved or future approved medications, or other small molecule medications for routine prophylaxis against HAE attacks within a minimum of 2 weeks before the Treatment Period
3. Participation in another interventional clinical study during the 30 days before the Treatment Period or within 5 half-lives of the final dose of the investigational product administered during the previous interventional study, whichever is longer
4. Having laboratory clinical abnormalities assessed as clinically significant by the investigator in results of hematology or chemistry assessments performed during Screening
5. Currently receiving a therapy not permitted during the study
6. Being pregnant or breastfeeding.

Study design
Purpose of the study
Treatment
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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/05/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
11/11/2026
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Campbelltown Hospital, Western Sydney University - Campbelltown
Recruitment postcode(s) [1] 0 0
NSW 2560 - Campbelltown
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Ohio
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
Canada
State/province [6] 0 0
Ottawa
Country [7] 0 0
Germany
State/province [7] 0 0
Hesse
Country [8] 0 0
Germany
State/province [8] 0 0
Berlin
Country [9] 0 0
Germany
State/province [9] 0 0
Frankfurt am Main
Country [10] 0 0
Israel
State/province [10] 0 0
Ashkelon

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
Trial Registration Coordinator
Address 0 0
Country 0 0
Phone 0 0
1-610-878-4000
Fax 0 0
Email 0 0
[email protected]
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 on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at [email protected].

Supporting document/s available: Study protocol, Statistical analysis plan (SAP)
When will data be available (start and end dates)?
Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
Available to whom?
Proposed research should seek to answer a previously unanswered important medical or scientific question.

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.
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/NCT05819775