Back to resultsCSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema
Primary Purpose
Hereditary Angioedema (HAE)
Status
Recruiting
Phase
Phase 3
Locations
Israel
Study Type
Interventional
Intervention
CSL312
Sponsored by
About this trial
This is an interventional treatment trial for Hereditary Angioedema (HAE)
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type III Any preplanned major surgeries or procedures during the clinical study Use of C1-INH products, androgens, antifibrinolytics, approved or future approved medications, or other small molecule medications for routine prophylaxis against HAE attacks Participation in another interventional clinical study
Sites / Locations
- Barzilai University Medical CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
CSL312
Arm Description
Ages 2-5 years and 6-11 years will have specific subcutaneous dosing schedules
Outcomes
Primary Outcome Measures
Number of subjects with treatment emergent adverse events (TEAEs)
Percent of subjects with TEAEs
Number of TEAEs
TEAE rates per injection
TEAE rates per subject year
Maximum concentration (Cmax) of CSL312 at steady-state
Trough concentration (Ctrough) of CSL312 at steady-state
Time to maximum concentration (Tmax) of CSL312 at steady-state
Secondary Outcome Measures
Time-normalized number of HAE attacks per month and per year
Time-normalized number of HAE attacks treated with on-demand treatment per month and per year
Time-normalized number of moderate and / or severe HAE attacks per month and per year
Percentage reduction in the time-normalized number of HAE attacks
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
Number of subjects with serious adverse events (SAEs)
Percent of subjects with SAEs
Number of subjects experiencing death
Percent of subjects experiencing death
Number of subjects with related TEAEs
Percent of subjects with related TEAEs
Number of subjects with TEAEs leading to study discontinuation
Percent of subjects with TEAEs leading to study discontinuation
Number of subjects with TEAEs by severity
Percent of subjects with TEAEs by severity
Number of subjects with Anti-CSL312 antibodies
Percent of subjects with Anti-CSL312 antibodies
Number of subjects with adverse events of special interest (AESIs)
Percent of subjects with AESIs
FXIIa-mediated kallikrein activity
Blood samples will be collected on the same day as CSL312 administration for assessment of FXIIa-mediated kallikrein activity
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05819775
Brief Title
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
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
May 24, 2023 (Actual)
Primary Completion Date
June 11, 2026 (Anticipated)
Study Completion Date
June 11, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CSL Behring
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema (HAE)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
CSL312
Arm Type
Experimental
Arm Description
Ages 2-5 years and 6-11 years will have specific subcutaneous dosing schedules
Intervention Type
Biological
Intervention Name(s)
CSL312
Other Intervention Name(s)
Garadacimab
Intervention Description
Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody administered subcutaneously (SC)
Primary Outcome Measure Information:
Title
Number of subjects with treatment emergent adverse events (TEAEs)
Time Frame
At least 14 months
Title
Percent of subjects with TEAEs
Time Frame
At lease 14 months
Title
Number of TEAEs
Time Frame
At least 14 months
Title
TEAE rates per injection
Time Frame
At least 14 months
Title
TEAE rates per subject year
Time Frame
At least 14 months
Title
Maximum concentration (Cmax) of CSL312 at steady-state
Time Frame
At least 12 months
Title
Trough concentration (Ctrough) of CSL312 at steady-state
Time Frame
At least 12 months
Title
Time to maximum concentration (Tmax) of CSL312 at steady-state
Time Frame
At least 12 months
Secondary Outcome Measure Information:
Title
Time-normalized number of HAE attacks per month and per year
Time Frame
At least 12 months
Title
Time-normalized number of HAE attacks treated with on-demand treatment per month and per year
Time Frame
At least 12 months
Title
Time-normalized number of moderate and / or severe HAE attacks per month and per year
Time Frame
At least 12 months
Title
Percentage reduction in the time-normalized number of HAE attacks
Time Frame
At least 12 months
Title
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
Time Frame
At least 12 months
Title
Number of subjects with serious adverse events (SAEs)
Time Frame
At least 14 months
Title
Percent of subjects with SAEs
Time Frame
At least 14 months
Title
Number of subjects experiencing death
Time Frame
At least 14 months
Title
Percent of subjects experiencing death
Time Frame
At least 14 months
Title
Number of subjects with related TEAEs
Time Frame
At least 14 months
Title
Percent of subjects with related TEAEs
Time Frame
At least 14 months
Title
Number of subjects with TEAEs leading to study discontinuation
Time Frame
At least 14 months
Title
Percent of subjects with TEAEs leading to study discontinuation
Time Frame
At least 14 months
Title
Number of subjects with TEAEs by severity
Time Frame
At least 14 months
Title
Percent of subjects with TEAEs by severity
Time Frame
At least 14 months
Title
Number of subjects with Anti-CSL312 antibodies
Time Frame
At least 14 months
Title
Percent of subjects with Anti-CSL312 antibodies
Time Frame
At least 14 months
Title
Number of subjects with adverse events of special interest (AESIs)
Time Frame
At least 14 months
Title
Percent of subjects with AESIs
Time Frame
At least 14 months
Title
FXIIa-mediated kallikrein activity
Description
Blood samples will be collected on the same day as CSL312 administration for assessment of FXIIa-mediated kallikrein activity
Time Frame
At least 12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type III
Any preplanned major surgeries or procedures during the clinical study
Use of C1-INH products, androgens, antifibrinolytics, approved or future approved medications, or other small molecule medications for routine prophylaxis against HAE attacks
Participation in another interventional clinical study
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Trial Registration Coordinator
Phone
1-610-878-4000
Email
clinicaltrials@cslbehring.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
CSL Behring
Official's Role
Study Director
Facility Information:
Facility Name
Barzilai University Medical Center
City
Ashkelon
ZIP/Postal Code
7830604
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Use Central Contact
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
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 clinicaltrials@cslbehring.com.
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.
IPD Sharing Time Frame
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.
IPD Sharing Access Criteria
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.
Learn more about this trial
CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema
We'll reach out to this number within 24 hrs