Phase 2 N=44 Randomized Quadruple-blind Prevention

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

Hereditary Angioedema

Bottom Line

View on ClinicalTrials.gov: NCT03712228 ↗

Enrolled (actual)

Serious AEs

2.9%

Results posted

Nov 2022

Primary outcome: Primary: The Mean Time Normalized Number of HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1 — 4.24; 0.48; 0.05; 0.35 Number of HAE attacks per month

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Factor XIIa antagonist monoclonal antibody (Biological); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: CSL Behring
Primary completion: Oct 2021

Outcome Measures

Outcome	Result	p-value
PRIMARY The Mean Time Normalized Number of HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1	4.24; 0.48; 0.05; 0.35; 0.14	—
SECONDARY The Number of Responder Subjects With C1-INH HAE During Treatment Period 1	0; 9; 8; 6; 6	—
SECONDARY The Percentage of Responder Subjects With C1-INH HAE During Treatment Period 1	0; 100.0; 100.0; 85.7; 100.0	—
SECONDARY The Number of HAE Attack-free Subjects With C1-INH HAE During Treatment Period 1	0; 5; 7; 3; 4	—
SECONDARY The Percentage of HAE Attack-free Subjects With C1-INH HAE During Treatment Period 1	0; 55.6; 87.5; 42.9; 66.7	—
SECONDARY The Number of Mild, Moderate or Severe HAE Attacks in Subjects With C1-INH HAE During Treatment Period 1	32; 3; 0; 2; 2; 43	—
SECONDARY The Percentage of Mild, Moderate or Severe HAE Attacks in Subjects With C1-INH HAE During Treatment Period 1	33.7; 25.0; 0; 28.6; 100.0; 45.3	—
SECONDARY The Mean Time-normalized Number of Mild, Moderate or Severe HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1	1.42; 0.12; 0.0; 0.10; 0.14; 1.93	—
SECONDARY 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	8; 3; 1; 2; 0	—
SECONDARY 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	100.0; 33.3; 12.5; 28.6; 0	—
SECONDARY Maximum Concentration (Cmax) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1	10.6; 15.9; 56.4	—
SECONDARY Area Under the Concentration-time Curve in 1 Dosing Interval (AUC0-tau) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1	4507; 7166; 26514	—
SECONDARY Time of Maximum Concentration (Tmax) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1	143.38; 165.51; 165.63	—
SECONDARY Terminal Elimination Half-life (T1/2) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1	411.7; 394.0; 443.5	—
SECONDARY Clearance (CL/F) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1	0.0198; 0.0303; 0.0246	—
SECONDARY Volume of Distribution During the Elimination Phase (Vz/F) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1	10.6; 17.0; 17.1	—
SECONDARY 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	7; 7; 7; 7; 4; 0	—

Summary

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.

Eligibility Criteria

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

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Data sourced from ClinicalTrials.gov (NCT03712228). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.