CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema
- Conditions
- Hereditary Angioedema (HAE)
- Interventions
- Biological: CSL312
- Registration Number
- NCT05819775
- Lead Sponsor
- CSL Behring
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 20
- Male or female
- Aged 2 to 11 years, inclusive, with body weight ≥ 10th percentile based on age
- Diagnosed with clinically confirmed C1-INH HAE
- Experienced ≥ 2 HAE attacks during the 6 months before Screening
- Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type 3
- 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
- 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
- Having laboratory clinical abnormalities assessed as clinically significant by the investigator in results of hematology or chemistry assessments performed during Screening
- Currently receiving a therapy not permitted during the study
- Being pregnant or breastfeeding.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description CSL312 CSL312 Ages 2-5 years and 6-11 years will have specific subcutaneous dosing schedules
- Primary Outcome Measures
Name Time Method TEAE rates per injection At least 14 months Number of subjects with treatment emergent adverse events (TEAEs) At least 14 months Percent of subjects with TEAEs At lease 14 months Number of TEAEs At least 14 months TEAE rates per subject year At least 14 months Maximum concentration (Cmax) of CSL312 at steady-state At least 12 months Trough concentration (Ctrough) of CSL312 at steady-state At least 12 months Time to maximum concentration (Tmax) of CSL312 at steady-state At least 12 months
- Secondary Outcome Measures
Name Time Method Percent of subjects with AESIs At least 14 months Number of subjects with adverse events of special interest (AESIs) At least 14 months Time-normalized number of HAE attacks treated with on-demand treatment per month and per year At least 12 months Percentage reduction in the time-normalized number of HAE attacks At least 12 months 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 At least 12 months Percent of subjects with SAEs At least 14 months Number of subjects experiencing death At least 14 months Percent of subjects experiencing death At least 14 months FXIIa-mediated kallikrein activity At least 12 months Blood samples will be collected on the same day as CSL312 administration for assessment of FXIIa-mediated kallikrein activity
Number of subjects with laboratory findings reported as AEs At least 14 months Time-normalized number of HAE attacks per month and per year At least 12 months Number of subjects with related TEAEs At least 14 months Percent of subjects with related TEAEs At least 14 months Number of subjects with TEAEs leading to study discontinuation At least 14 months Number of subjects with Anti-CSL312 antibodies At least 14 months Time-normalized number of moderate and / or severe HAE attacks per month and per year At least 12 months Number of subjects with serious adverse events (SAEs) At least 14 months Percent of subjects with TEAEs leading to study discontinuation At least 14 months Number of subjects with TEAEs by severity At least 14 months Percent of subjects with TEAEs by severity At least 14 months Percent of subjects with Anti-CSL312 antibodies At least 14 months Percent of subjects with laboratory findings reported as AEs At least 14 months
Trial Locations
- Locations (13)
Research Solutions of Arizona
🇺🇸Litchfield Park, Arizona, United States
Medical Research of Arizona
🇺🇸Scottsdale, Arizona, United States
Donald S. Levy M.D.
🇺🇸Orange, California, United States
Raffi Tachdjian MD, Inc.
🇺🇸Santa Monica, California, United States
Bernstein Clinical Research
🇺🇸Cincinnati, Ohio, United States
PennState Health Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
AARA Research Center
🇺🇸Dallas, Texas, United States
Campbelltown Hospital, Western Sydney University
🇦🇺Campbelltown, Australia
Ottawa Allergy Research Corp
🇨🇦Ottawa, Canada
HZRM Hämophilie Zentrum Rhein Main GmbH
🇩🇪Frankfurt am Main, Hesse, Germany
Charité - Universitätsmedizin Berlin
🇩🇪Berlin, Germany
Universitätsklinikum Frankfurt
🇩🇪Frankfurt am Main, Germany
Barzilai University Medical Center
🇮🇱Ashkelon, Israel