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

Phase 3CompletedNCT05819775

CSL Behring22 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hereditary Angioedema (HAE)

Interventions

CSL312BIOLOGICAL

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

Eligibility

Sex: ALLMin age: 2 YearsMax age: 11 Years

Medical Language ↔ Plain English

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.

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

Outcomes

Primary Outcomes

Number of subjects with treatment emergent adverse events (TEAEs)

Time frame: At least 14 months

Percent of subjects with TEAEs

Time frame: At lease 14 months

Number of TEAEs

Time frame: At least 14 months

TEAE rates per injection

Time frame: At least 14 months

TEAE rates per subject year

Time frame: At least 14 months

Maximum concentration (Cmax) of CSL312 at steady-state

Time frame: At least 12 months

Trough concentration (Ctrough) of CSL312 at steady-state

Time frame: At least 12 months

Time to maximum concentration (Tmax) of CSL312 at steady-state

Time frame: At least 12 months

Secondary Outcomes

Time-normalized number of HAE attacks per month and per year

Time frame: At least 12 months

Time-normalized number of HAE attacks treated with on-demand treatment per month and per year

Time frame: At least 12 months

Time-normalized number of moderate and / or severe HAE attacks per month and per year

Time frame: At least 12 months

Data from ClinicalTrials.gov

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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

...and 3 more locations

Percentage reduction in the time-normalized number of HAE attacks

Time frame: 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

Time frame: At least 12 months

Number of subjects with serious adverse events (SAEs)

Time frame: At least 14 months

Percent of subjects with SAEs

Time frame: At least 14 months

Number of subjects experiencing death

Time frame: At least 14 months

Percent of subjects experiencing death

Time frame: At least 14 months

Number of subjects with related TEAEs

Time frame: At least 14 months

Percent of subjects with related TEAEs

Time frame: At least 14 months

Number of subjects with TEAEs leading to study discontinuation

Time frame: At least 14 months

Percent of subjects with TEAEs leading to study discontinuation

Time frame: At least 14 months

Number of subjects with TEAEs by severity

Time frame: At least 14 months

Percent of subjects with TEAEs by severity

Time frame: At least 14 months

Number of subjects with Anti-CSL312 antibodies

Time frame: At least 14 months

Percent of subjects with Anti-CSL312 antibodies

Time frame: At least 14 months

Number of subjects with adverse events of special interest (AESIs)

Time frame: At least 14 months

Percent of subjects with AESIs

Time frame: At least 14 months

FXIIa-mediated kallikrein activity

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

Number of subjects with laboratory findings reported as AEs

Time frame: At least 14 months

Percent of subjects with laboratory findings reported as AEs

Time frame: At least 14 months