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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/NCT05819775
Registration number
NCT05819775
Ethics application status
Date submitted
4/04/2023
Date registered
19/04/2023
Date last updated
4/06/2024
Titles & IDs
Public title
CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema
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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
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Secondary ID [1]
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2022-502386-13-00
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Secondary ID [2]
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CSL312_3003
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema (HAE)
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Condition category
Condition code
Cardiovascular
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Diseases of the vasculature and circulation including the lymphatic system
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Blood
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Other blood disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - CSL312
Experimental: CSL312 - Ages 2-5 years and 6-11 years will have specific subcutaneous dosing schedules
Other interventions: CSL312
Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody administered subcutaneously (SC)
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of subjects with treatment emergent adverse events (TEAEs)
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Assessment method [1]
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Timepoint [1]
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At least 14 months
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Primary outcome [2]
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Percent of subjects with TEAEs
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Assessment method [2]
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Timepoint [2]
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At lease 14 months
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Primary outcome [3]
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Number of TEAEs
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Assessment method [3]
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Timepoint [3]
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At least 14 months
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Primary outcome [4]
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TEAE rates per injection
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Assessment method [4]
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Timepoint [4]
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At least 14 months
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Primary outcome [5]
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TEAE rates per subject year
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Assessment method [5]
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Timepoint [5]
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At least 14 months
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Primary outcome [6]
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Maximum concentration (Cmax) of CSL312 at steady-state
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Assessment method [6]
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Timepoint [6]
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At least 12 months
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Primary outcome [7]
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Trough concentration (Ctrough) of CSL312 at steady-state
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Assessment method [7]
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Timepoint [7]
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At least 12 months
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Primary outcome [8]
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Time to maximum concentration (Tmax) of CSL312 at steady-state
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Assessment method [8]
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Timepoint [8]
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At least 12 months
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Secondary outcome [1]
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Time-normalized number of HAE attacks per month and per year
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Assessment method [1]
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Timepoint [1]
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At least 12 months
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Secondary outcome [2]
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Time-normalized number of HAE attacks treated with on-demand treatment per month and per year
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Assessment method [2]
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Timepoint [2]
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At least 12 months
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Secondary outcome [3]
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Time-normalized number of moderate and / or severe HAE attacks per month and per year
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Assessment method [3]
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Timepoint [3]
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At least 12 months
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Secondary outcome [4]
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Percentage reduction in the time-normalized number of HAE attacks
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Assessment method [4]
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Timepoint [4]
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At least 12 months
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Secondary outcome [5]
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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
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Assessment method [5]
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Timepoint [5]
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At least 12 months
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Secondary outcome [6]
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Number of subjects with serious adverse events (SAEs)
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Assessment method [6]
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Timepoint [6]
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At least 14 months
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Secondary outcome [7]
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Percent of subjects with SAEs
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Assessment method [7]
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Timepoint [7]
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At least 14 months
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Secondary outcome [8]
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Number of subjects experiencing death
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Assessment method [8]
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Timepoint [8]
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At least 14 months
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Secondary outcome [9]
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Percent of subjects experiencing death
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Assessment method [9]
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Timepoint [9]
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At least 14 months
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Secondary outcome [10]
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Number of subjects with related TEAEs
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Assessment method [10]
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Timepoint [10]
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At least 14 months
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Secondary outcome [11]
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Percent of subjects with related TEAEs
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Assessment method [11]
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Timepoint [11]
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At least 14 months
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Secondary outcome [12]
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Number of subjects with TEAEs leading to study discontinuation
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Assessment method [12]
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Timepoint [12]
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At least 14 months
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Secondary outcome [13]
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Percent of subjects with TEAEs leading to study discontinuation
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Assessment method [13]
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Timepoint [13]
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At least 14 months
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Secondary outcome [14]
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Number of subjects with TEAEs by severity
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Assessment method [14]
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Timepoint [14]
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At least 14 months
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Secondary outcome [15]
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Percent of subjects with TEAEs by severity
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Assessment method [15]
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Timepoint [15]
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At least 14 months
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Secondary outcome [16]
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Number of subjects with Anti-CSL312 antibodies
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Assessment method [16]
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Timepoint [16]
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At least 14 months
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Secondary outcome [17]
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Percent of subjects with Anti-CSL312 antibodies
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Assessment method [17]
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Timepoint [17]
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At least 14 months
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Secondary outcome [18]
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Number of subjects with adverse events of special interest (AESIs)
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Assessment method [18]
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Timepoint [18]
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At least 14 months
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Secondary outcome [19]
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Percent of subjects with AESIs
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Assessment method [19]
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Timepoint [19]
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At least 14 months
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Secondary outcome [20]
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FXIIa-mediated kallikrein activity
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Assessment method [20]
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Blood samples will be collected on the same day as CSL312 administration for assessment of FXIIa-mediated kallikrein activity
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Timepoint [20]
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At least 12 months
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Secondary outcome [21]
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Number of subjects with laboratory findings reported as AEs
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Assessment method [21]
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Timepoint [21]
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At least 14 months
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Secondary outcome [22]
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Percent of subjects with laboratory findings reported as AEs
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Assessment method [22]
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Timepoint [22]
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At least 14 months
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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
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Minimum age
2
Years
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Maximum age
11
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
30/05/2023
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
11/11/2026
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Actual
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Sample size
Target
20
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Campbelltown Hospital, Western Sydney University - Campbelltown
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Recruitment postcode(s) [1]
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NSW 2560 - Campbelltown
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Arizona
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Country [2]
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United States of America
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State/province [2]
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California
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Country [3]
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United States of America
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State/province [3]
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Ohio
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Country [4]
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United States of America
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State/province [4]
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Texas
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Country [5]
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Canada
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State/province [5]
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Ottawa
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Country [6]
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Germany
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State/province [6]
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Hesse
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Country [7]
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Germany
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State/province [7]
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Berlin
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Country [8]
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Israel
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State/province [8]
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Ashkelon
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
CSL Behring
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to investigate the safety, PK / PD, and efficacy of SC CSL312
for prophylactic treatment of pediatric subjects with HAE.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05819775
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Study Director
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Address
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CSL Behring
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Trial Registration Coordinator
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Address
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Country
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Phone
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1-610-878-4000
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05819775
Download to PDF