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

Sponsor
CSL Behring (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05819775
Collaborator
(none)
12
Enrollment
1
Arm
36.8
Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Biological: CSL312
 Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
12 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official 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
Anticipated Study Start Date :
May 18, 2023
Anticipated Primary Completion Date :
Jun 11, 2026
Anticipated Study Completion Date :
Jun 11, 2026

Arms and Interventions

Arm Intervention/Treatment
Experimental: CSL312

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

Biological: CSL312
Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody administered subcutaneously (SC)
Other Names:
  • Garadacimab

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Number of subjects with treatment emergent adverse events (TEAEs) [At least 14 months]

    2. Percent of subjects with TEAEs [At lease 14 months]

    3. Number of TEAEs [At least 14 months]

    4. TEAE rates per injection [At least 14 months]

    5. TEAE rates per subject year [At least 14 months]

    6. Maximum concentration (Cmax) of CSL312 at steady-state [At least 12 months]

    7. Trough concentration (Ctrough) of CSL312 at steady-state [At least 12 months]

    8. Time to maximum concentration (Tmax) of CSL312 at steady-state [At least 12 months]

    Secondary Outcome Measures

    1. Time-normalized number of HAE attacks per month and per year [At least 12 months]

    2. Time-normalized number of HAE attacks treated with on-demand treatment per month and per year [At least 12 months]

    3. Time-normalized number of moderate and / or severe HAE attacks per month and per year [At least 12 months]

    4. Percentage reduction in the time-normalized number of HAE attacks [At least 12 months]

    5. The number of subjects experiencing at least ≥ 50%, ≥ 70%, ≥ 90%, or equal to 100% (attack-free) reduction in the time-normalized number of HAE attacks [At least 12 months]

    6. Number of subjects with serious adverse events (SAEs) [At least 14 months]

    7. Percent of subjects with SAEs [At least 14 months]

    8. Number of subjects experiencing death [At least 14 months]

    9. Percent of subjects experiencing death [At least 14 months]

    10. Number of subjects with related TEAEs [At least 14 months]

    11. Percent of subjects with related TEAEs [At least 14 months]

    12. Number of subjects with TEAEs leading to study discontinuation [At least 14 months]

    13. Percent of subjects with TEAEs leading to study discontinuation [At least 14 months]

    14. Number of subjects with TEAEs by severity [At least 14 months]

    15. Percent of subjects with TEAEs by severity [At least 14 months]

    16. Number of subjects with Anti-CSL312 antibodies [At least 14 months]

    17. Percent of subjects with Anti-CSL312 antibodies [At least 14 months]

    18. Number of subjects with adverse events of special interest (AESIs) [At least 14 months]

    19. Percent of subjects with AESIs [At least 14 months]

    20. FXIIa-mediated kallikrein activity [At least 12 months]

      Blood samples will be collected on the same day as CSL312 administration for assessment of FXIIa-mediated kallikrein activity

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 11 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. Male or female

    2. Aged 2 to 11 years, inclusive, with body weight ≥ 10th percentile based on age

    3. Diagnosed with clinically confirmed C1-INH HAE

    4. Experienced ≥ 2 HAE attacks during the 6 months before Screening

    Exclusion Criteria:

    1. Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type III

    2. Any preplanned major surgeries or procedures during the clinical study

    3. Use of C1-INH products, androgens, antifibrinolytics, approved or future approved medications, or other small molecule medications for routine prophylaxis against HAE attacks

    4. Participation in another interventional clinical study

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • CSL Behring

    Investigators

    • Study Director: Study Director, CSL Behring

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    CSL Behring
    ClinicalTrials.gov Identifier:
    NCT05819775
    Other Study ID Numbers:
    • CSL312_3003
    First Posted:
    Apr 19, 2023
    Last Update Posted:
    Apr 19, 2023
    Last Verified:
    Apr 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Angioedema
    Angioedemas, Hereditary

    Study Results

    No Results Posted as of Apr 19, 2023