Clinical trial NCT05819775: CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema
Official 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Hereditary Angioedema (HAE)
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2023-04-19
Enrollment: 20
Sex: ALL
Minimum age (years): 2
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

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.

Primary outcomes:
measure: Number of subjects with treatment emergent adverse events (TEAEs)
timeFrame: At least 14 months
measure: Percent of subjects with TEAEs
timeFrame: At lease 14 months
measure: Number of TEAEs
timeFrame: At least 14 months
measure: TEAE rates per injection
timeFrame: At least 14 months
measure: TEAE rates per subject year
timeFrame: At least 14 months
measure: Maximum concentration (Cmax) of CSL312 at steady-state
timeFrame: At least 12 months
measure: Trough concentration (Ctrough) of CSL312 at steady-state
timeFrame: At least 12 months
measure: Time to maximum concentration (Tmax) of CSL312 at steady-state
timeFrame: At least 12 months
Secondary outcomes:
measure: Time-normalized number of HAE attacks per month and per year
timeFrame: At least 12 months
measure: Time-normalized number of HAE attacks treated with on-demand treatment per month and per year
timeFrame: At least 12 months
measure: Time-normalized number of moderate and / or severe HAE attacks per month and per year
timeFrame: At least 12 months
measure: Percentage reduction in the time-normalized number of HAE attacks
timeFrame: At least 12 months
measure: 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
timeFrame: At least 12 months
measure: Number of subjects with serious adverse events (SAEs)
timeFrame: At least 14 months
measure: Percent of subjects with SAEs
timeFrame: At least 14 months
measure: Number of subjects experiencing death
timeFrame: At least 14 months
measure: Percent of subjects experiencing death
timeFrame: At least 14 months
measure: Number of subjects with related TEAEs
timeFrame: At least 14 months
measure: Percent of subjects with related TEAEs
timeFrame: At least 14 months
measure: Number of subjects with TEAEs leading to study discontinuation
timeFrame: At least 14 months
measure: Percent of subjects with TEAEs leading to study discontinuation
timeFrame: At least 14 months
measure: Number of subjects with TEAEs by severity
timeFrame: At least 14 months
measure: Percent of subjects with TEAEs by severity
timeFrame: At least 14 months
measure: Number of subjects with Anti-CSL312 antibodies
timeFrame: At least 14 months
measure: Percent of subjects with Anti-CSL312 antibodies
timeFrame: At least 14 months
measure: Number of subjects with adverse events of special interest (AESIs)
timeFrame: At least 14 months
measure: Percent of subjects with AESIs
timeFrame: At least 14 months
measure: FXIIa-mediated kallikrein activity
timeFrame: At least 12 months
measure: Number of subjects with laboratory findings reported as AEs
timeFrame: At least 14 months
measure: Percent of subjects with laboratory findings reported as AEs
timeFrame: At least 14 months
Other outcomes:
Locations:
city: Litchfield Park
country: United States
facility: Research Solutions of Arizona
state: Arizona
city: Scottsdale
country: United States
facility: Medical Research of Arizona
state: Arizona
city: Orange
country: United States
facility: Donald S. Levy M.D.
state: California
city: Santa Monica
country: United States
facility: Raffi Tachdjian MD, Inc.
state: California
city: Cincinnati
country: United States
facility: Bernstein Clinical Research
state: Ohio
city: Hershey
country: United States
facility: PennState Health Milton S. Hershey Medical Center
state: Pennsylvania
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
city: Campbelltown
country: Australia
facility: Campbelltown Hospital, Western Sydney University
city: Ottawa
country: Canada
facility: Ottawa Allergy Research Corp
city: Frankfurt am Main
country: Germany
facility: HZRM Hämophilie Zentrum Rhein Main GmbH
state: Hesse
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Frankfurt am Main
country: Germany
facility: Universitätsklinikum Frankfurt
city: Ashkelon
country: Israel
facility: Barzilai University Medical Center
Study officials:
affiliation: CSL Behring
name: Study Director
role: STUDY_DIRECTOR
References: