A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE)

CSL Behring44 enrolled

Overview

This is a multicenter, randomized, placebo-controlled, parallel-arm, phase 2 study to investigate the clinical efficacy, pharmacokinetics, and safety of CSL312 as prophylaxis to prevent attacks in subjects with HAE.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

QUADRUPLE

Enrollment

Conditions

Hereditary Angioedema

Interventions

Factor XIIa antagonist monoclonal antibodyBIOLOGICAL

Factor XIIa antagonist monoclonal antibody for intravenous and subcutaneous use

PlaceboDRUG

Buffer without active ingredient

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male or female * Aged ≥ 18 to ≤ 65 years * A diagnosis of C1-INH HAE or FXII/PLG HAE; * For subjects with C1-INH HAE: ≥ 4 HAE attacks over a consecutive 2-month period during the 3 months before Screening, as documented in the subject's medical record. Exclusion Criteria: * History of clinically significant arterial or venous thrombosis, or current clinically significant prothrombotic risk * History of an uncontrolled, abnormal bleeding event due to a coagulopathy, or a current clinically significant coagulopathy or clinically significant risks for bleeding events * Known incurable malignancies

Locations (16)

Donald S. Levy

Orange, California, United States

Allergy & Asthma Clinical Research

Walnut Creek, California, United States

Outcomes

Primary Outcomes

The Mean Time Normalized Number of HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1

The time-normalized number of HAE attacks per month during Treatment Period 1 for a subject was calculated as the (number of HAE attacks / length of subject's evaluation period in days) \* 30.4375

Time frame: 13 weeks

Secondary Outcomes

The Number of Responder Subjects With C1-INH HAE During Treatment Period 1

Response is defined as a ≥ 50% relative reduction in the time-normalized number of HAE attacks (per month) during Treatment Period 1 compared to each subject's time-normalized number of HAE attacks (per month) during the Run-in Period

Time frame: 13 weeks

The Percentage of Responder Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

The Number of HAE Attack-free Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

The Percentage of HAE Attack-free Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

The Number of Mild, Moderate or Severe HAE Attacks in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

The Percentage of Mild, Moderate or Severe HAE Attacks in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

Data from ClinicalTrials.gov

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Immunoe Health Centers

Centennial, Colorado, United States

Institute for Asthma and Allergy

Chevy Chase, Maryland, United States

The Mount Sinai Hospital

New York, New York, United States

Pennsylvania State University

Hershey, Pennsylvania, United States

AARA Research Center

Dallas, Texas, United States

Campbelltown Hospital

Campbelltown, New South Wales, Australia

University of Alberta

Edmonton, Alberta, Canada

Allergy and Clinical Immunology McMaster University

Hamilton, Ontario, Canada

...and 6 more locations

The Mean Time-normalized Number of Mild, Moderate or Severe HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1

The time-normalized number of HAE attacks per month during Treatment Period 1 for a subject was calculated as the (number of HAE attacks / length of subject's evaluation period in days) \* 30.4375

Time frame: 13 weeks

The Number of Subjects With at Least One (1) HAE Attack Treated With On-demand HAE Medication, in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

The Percentage of Subjects With at Least One (1) HAE Attack Treated With On-demand HAE Medication, in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

Maximum Concentration (Cmax) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

Area Under the Concentration-time Curve in 1 Dosing Interval (AUC0-tau) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

Time of Maximum Concentration (Tmax) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

Terminal Elimination Half-life (T1/2) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

Clearance (CL/F) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

Volume of Distribution During the Elimination Phase (Vz/F) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1

Time frame: 13 weeks

The Number of Subjects With C1-INH HAE With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESI), Injection Site Reactions (ISRs), Binding Antibodies to CSL312 During Treatment Period 1

Adverse events of special interest is defined as anaphylaxis, thromboembolic events, and bleeding events.

Time frame: 13 weeks