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

NCT05819775 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: CSL312_30032022-502386-13-00
• Study Type: interventional
• Target: 22
• Locations: 5 countries
• Sites: 13

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.

Conditions

Hereditary Angioedema (HAE)

Outcome Measures

Primary Outcomes (8)

• Number of Participants With Treatment Emergent Adverse Events (TEAE)

  Up to Month 12

• Percentage of Participants With TEAE

  The percentage of participants was rounded to one place of decimal.

  Up to Month 12

• Number of TEAE

  Up to Month 12

• TEAE Rates Per Injection

  The TEAE rate per injection was calculated as the number of TEAE/ number of injections. The number of injections was defined as the total injections a participant received during the Safety Evaluation Period under the dosing regimen to which the TEAE was assigned.

  Up to Month 12

• TEAE Rates Per Participant-Year

  The TEAE rate per participant year was calculated as number of TEAEs/ participant years. Participant-years of exposure were calculated as the sum of each participant's exposure duration (in years) under the specified dosing regimen or overall. For the time assigned to a dosing regimen, each study day was counted under the corresponding regimen.

  Up to Month 12

• Maximum Concentration (Cmax) of CSL312 at Steady-state

  Up to Month 12

• Trough Concentration (Ctrough) of CSL312 at Steady-state

  At Months 3, 4, 6, 9, 10, and 12

• Time to Maximum Concentration (Tmax) of CSL312 at Steady-State

  Up to Month 12

Secondary Outcomes (20)

• Time-normalized Number of HAE Attacks Per Month

  Up to Month 12

• Time-normalized Number of HAE Attacks Per Year

  Up to Month 12

• Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Month

  Up to Month 12

• Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Year

  Up to Month 12

• Time-normalized Number of Moderate and/or Severe HAE Attacks Per Month

  Up to Month 12

Study Arms (1)

CSL312

EXPERIMENTAL

Ages 2-5 years and 6-11 years will have specific subcutaneous dosing schedules

Biological: CSL312

Interventions

CSL312 BIOLOGICAL

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

Also known as: Garadacimab

CSL312

Eligibility Criteria

• Age: 2 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type 3
• 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
• 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
• Having laboratory clinical abnormalities assessed as clinically significant by the investigator in results of hematology or chemistry assessments performed during Screening
• Currently receiving a therapy not permitted during the study
• Being pregnant or breastfeeding.

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (13)

Research Solutions of Arizona

Litchfield Park, Arizona, 85340, United States

Location

Medical Research of Arizona

Scottsdale, Arizona, 85251, United States

Location

Donald S. Levy M.D.

Orange, California, 92868, United States

Location

Raffi Tachdjian MD, Inc.

Santa Monica, California, 90404, United States

Location

Bernstein Clinical Research

Cincinnati, Ohio, 45236, United States

Location

PennState Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

AARA Research Center

Dallas, Texas, 75231, United States

Location

Campbelltown Hospital, Western Sydney University

Campbelltown, NSW 2560, Australia

Location

Ottawa Allergy Research Corp

Ottawa, K1H1E4, Canada

Location

HZRM Hämophilie Zentrum Rhein Main GmbH

Frankfurt am Main, Hesse, 60596, Germany

Location

Charité - Universitätsmedizin Berlin

Berlin, 12203, Germany

Location

Universitätsklinikum Frankfurt

Frankfurt am Main, 60590, Germany

Location

Barzilai University Medical Center

Ashkelon, 7830604, Israel

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Results Point of Contact

• Title: Clinical Study Disclosure Manager
• Organization: CSL Behring

clinicaltrials@cslbehring.com

1-610-878-4697

Study Officials

• Study Director

  CSL Behring

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2023
• First Posted: April 19, 2023
• Study Start: May 30, 2023
• Primary Completion: November 19, 2025
• Study Completion: November 19, 2025
• Last Updated: June 2, 2026
• Results First Posted: June 2, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
• Access Criteria: Proposed research should seek to answer a previously unanswered important medical or scientific question. 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.

Locations

US (7); CA (1); AU (1); IL (1); DE (3)