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

Phase 3

Active, not recruiting

• 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

Study Timeline

• Study First Submitted: 2023-04-04
• Study First Submitted QC: 2023-04-17
• Study First Posted: 2023-04-19
• Study Start: 2023-05-30
• Status Verified: 2024-11
• Last Update Submitted: 2024-12-06
• Last Update Posted: 2024-12-09
• Primary Completion: 2026-11-11
• Study Completion: 2026-11-11

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 20

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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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

• 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