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Completed

NCT Number: NCT03712228

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

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.

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Key information

Conditions

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

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Campbelltown Hospital, Campbelltown, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Factor XIIa antagonist monoclonal antibody

Biological

Factor XIIa antagonist monoclonal antibody for intravenous and subcutaneous use

Other names: CSL312

Placebo

Drug

Buffer without active ingredient

Primary outcomes

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

    Time frame: 13 weeks

    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

Secondary outcomes

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

    Time frame: 13 weeks

    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

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

    Time frame: 13 weeks

    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.

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

    Time frame: 13 weeks

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

    Time frame: 13 weeks

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

    Time frame: 13 weeks

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

    Time frame: 13 weeks

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

    Time frame: 13 weeks

    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

  8. 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

  9. 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

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

    Time frame: 13 weeks

  11. 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

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

    Time frame: 13 weeks

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

    Time frame: 13 weeks

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

    Time frame: 13 weeks

  15. 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

  16. 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

    Time frame: 13 weeks

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

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

A Multicenter, Randomized, Placebo-controlled, Parallel-arm Study to Investigate the Efficacy, Pharmacokinetics, and Safety of CSL312 in Subjects With Hereditary Angioedema

Important dates

Study start
2018
Primary completion
2021
Study completion
2021
First posted
Oct 19, 2018
Registry last updated
Nov 8, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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