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Completed

NCT Number: NCT05819775

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

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.

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Key information

Conditions

Hereditary Angioedema (HAE) 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

2 year–11 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Campbelltown Hospital, Western Sydney University, Campbelltown, 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 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

Exclusion criteria

  • 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.

Treatment and study plan

CSL312

Biological

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

Other names: Garadacimab

Primary outcomes

  1. Number of Participants With Treatment Emergent Adverse Events (TEAE)

    Time frame: Up to Month 12

  2. Percentage of Participants With TEAE

    Time frame: Up to Month 12

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

  3. Number of TEAE

    Time frame: Up to Month 12

  4. TEAE Rates Per Injection

    Time frame: Up to Month 12

    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.

  5. TEAE Rates Per Participant-Year

    Time frame: Up to Month 12

    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.

  6. Maximum Concentration (Cmax) of CSL312 at Steady-state

    Time frame: Up to Month 12

  7. Trough Concentration (Ctrough) of CSL312 at Steady-state

    Time frame: At Months 3, 4, 6, 9, 10, and 12

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

    Time frame: Up to Month 12

Secondary outcomes

  1. Time-normalized Number of HAE Attacks Per Month

    Time frame: Up to Month 12

    Time-normalized number of HAE attacks per month during treatment was calculated per participant as: [Number of HAE attacks / Length of participant treatment in days] * 30.4375.

  2. Time-normalized Number of HAE Attacks Per Year

    Time frame: Up to Month 12

    Time-normalized number of HAE attacks per year during treatment was calculated per participant as: [Number of HAE attacks / Length of participant treatment in days] * 365.25.

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

    Time frame: Up to Month 12

    The time-normalized number of HAE attacks per month treated with on-demand treatment were calculated as follows:

    [(Number of HAE attacks treated with on - demand treatment during treatment period)/ Length of participant treatment in days] ∗ 30.4375.

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

    Time frame: Up to Month 12

    The time-normalized number of HAE attacks per year treated with on-demand treatment were calculated as follows:

    [(Number of HAE attacks treated with on - demand treatment during treatment period)/ Length of participant treatment in days] ∗ 365.25.

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

    Time frame: Up to Month 12

    Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: [number of moderate or severe HAE attacks / length of participant treatment in days] * 30.4375.

  6. Time-normalized Number of Moderate and/or Severe HAE Attacks Per Year

    Time frame: Up to Month 12

    Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: [number of moderate or severe HAE attacks / length of participant treatment in days] * 365.25.

  7. Percentage Reduction in the Time-normalized Number of HAE Attacks

    Time frame: Up to Month 12

    The percentage reduction in the time-normalized number of HAE attacks was calculated within a participant as follows: 100*[ 1 - (Time-normalized number of HAE attacks per month during treatment period/Time-normalized number of HAE attacks per month from historical data)].

  8. Number of Participants Experiencing at Least Greater Than or Equal to (>=) 50 Percent (%), >= 70%, >= 90%, or Equal to 100% (Attack-free) Reduction in the Time-normalized Number of HAE Attacks

    Time frame: Up to Month 12

    A participant was classified as a responder if the percentage reduction in the time-normalized number of HAE attacks under treatment compared to the time-normalized number of HAE attacks documented in the medical records was >= 50%. Percent Reduction = 100 * [1 - (time-normalized number of HAE attacks during corresponding time window / time-normalized number of HAE attacks based on historical data)]. Here number of participants experiencing at least >= 50%, >= 70%, >= 90%, or equal to 100% (Attack-free) reduction in the time-normalized number of HAE attacks are reported. The number of responders at each reduction category have been reported.

  9. Number of Participants Experiencing Serious Adverse Events (SAE), Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation

    Time frame: Up to Month 12

  10. Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation

    Time frame: Up to Month 12

    The percentage of participants was rounded to one decimal place.

  11. Number of Participants With TEAE by Severity

    Time frame: Up to Month 12

    Severity of AE was assessed by the investigator and categorized as mild, moderate and severe where:

    Mild: AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. Moderate: AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention.

  12. Percentage of Participants With TEAE by Severity

    Time frame: Up to Month 12

    Severity of AE was assessed by the investigator and categorized as mild, moderate and severe where:

    Mild: AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. Moderate: AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention. The percentage of participants was rounded to one place of decimal.

  13. Number of Participants With Anti-CSL312 Antibodies

    Time frame: At Day 1, Months 6 and 12

  14. Percentage of Participants With Anti-CSL312 Antibodies

    Time frame: At Day 1, Months 6 and 12

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

  15. Number of Participants With Adverse Events of Special Interest (AESI)

    Time frame: Up to Month 12

    AESI included severe hypersensitivity including anaphylaxis. The AESI reported have been identified by investigators and suggestive events were independently identified for further review with a Standardized MedDRA Query (SMQ).

  16. Percentage of Participants With AESI

    Time frame: Up to Month 12

    AESI included severe hypersensitivity including anaphylaxis. The AESI reported have been identified by investigators and suggestive events were independently identified for further review with an SMQ. The percentage of participants was rounded to one place of decimal.

  17. FXIIa-mediated Kallikrein Activity

    Time frame: At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10

  18. Percent of Baseline FXIIa-mediated Kallikrein Activity

    Time frame: At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10

    Percent of Baseline at Visit [i] = 100 * (actual value at Visit [i] / Baseline value), where Baseline is defined as the most recent, non-missing value before the first IP administration (including unscheduled visits). Here unit of measure is Percent (%) of FXIIa-mediated Kallikrein Activity.

  19. Number of Participants With Laboratory Findings Reported as AE

    Time frame: Up to Month 12

  20. Percentage of Participants With Laboratory Findings Reported as AE

    Time frame: Up to Month 12

    The participant data were rounded to one decimal place.

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

A Phase 3 Open-label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema in Pediatric Subjects 2 to 11 Years of Age

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Apr 19, 2023
Registry last updated
Jun 2, 2026

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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