CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

Hereditary Angioedema (HAE) Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05819775
• Other study ID #: CSL312_3003
• Secondary ID: 2022-502386-13-0
• Status: Recruiting
• Phase: Phase 3
• Start date: May 30, 2023
• Est. completion date: November 11, 2026

Study information

• Verified date: April 2024
• Source: CSL Behring
• Contact: Trial Registration Coordinator
• Phone: 1-610-878-4000
• Email: clinicaltrials[@]cslbehring.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: November 11, 2026
• Est. primary completion date: November 11, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 11 Years

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema (HAE)

Intervention

Biological:
• CSL312
Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody administered subcutaneously (SC)

Locations

Country	Name	City	State
Israel	Barzilai University Medical Center	Ashkelon
United States	Bernstein Clinical Research	Cincinnati	Ohio
United States	Research Solutions of Arizona	Litchfield Park	Arizona
United States	Donald S. Levy M.D.	Orange	California
United States	Raffi Tachdjian MD, Inc.	Santa Monica	California
United States	Medical Research of Arizona	Scottsdale	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

United States,  Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of subjects with treatment emergent adverse events (TEAEs)		At least 14 months
Primary	Percent of subjects with TEAEs		At lease 14 months
Primary	Number of TEAEs		At least 14 months
Primary	TEAE rates per injection		At least 14 months
Primary	TEAE rates per subject year		At least 14 months
Primary	Maximum concentration (Cmax) of CSL312 at steady-state		At least 12 months
Primary	Trough concentration (Ctrough) of CSL312 at steady-state		At least 12 months
Primary	Time to maximum concentration (Tmax) of CSL312 at steady-state		At least 12 months
Secondary	Time-normalized number of HAE attacks per month and per year		At least 12 months
Secondary	Time-normalized number of HAE attacks treated with on-demand treatment per month and per year		At least 12 months
Secondary	Time-normalized number of moderate and / or severe HAE attacks per month and per year		At least 12 months
Secondary	Percentage reduction in the time-normalized number of HAE attacks		At least 12 months
Secondary	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
Secondary	Number of subjects with serious adverse events (SAEs)		At least 14 months
Secondary	Percent of subjects with SAEs		At least 14 months
Secondary	Number of subjects experiencing death		At least 14 months
Secondary	Percent of subjects experiencing death		At least 14 months
Secondary	Number of subjects with related TEAEs		At least 14 months
Secondary	Percent of subjects with related TEAEs		At least 14 months
Secondary	Number of subjects with TEAEs leading to study discontinuation		At least 14 months
Secondary	Percent of subjects with TEAEs leading to study discontinuation		At least 14 months
Secondary	Number of subjects with TEAEs by severity		At least 14 months
Secondary	Percent of subjects with TEAEs by severity		At least 14 months
Secondary	Number of subjects with Anti-CSL312 antibodies		At least 14 months
Secondary	Percent of subjects with Anti-CSL312 antibodies		At least 14 months
Secondary	Number of subjects with adverse events of special interest (AESIs)		At least 14 months
Secondary	Percent of subjects with AESIs		At least 14 months
Secondary	FXIIa-mediated kallikrein activity	Blood samples will be collected on the same day as CSL312 administration for assessment of FXIIa-mediated kallikrein activity	At least 12 months
Secondary	Number of subjects with laboratory findings reported as AEs		At least 14 months
Secondary	Percent of subjects with laboratory findings reported as AEs		At least 14 months