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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05819775
Recruitment Status : Recruiting
First Posted : April 19, 2023
Last Update Posted : April 10, 2024
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Study Description
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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
Hereditary Angioedema (HAE) Biological: CSL312 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 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
Actual Study Start Date : May 30, 2023
Estimated Primary Completion Date : November 11, 2026
Estimated Study Completion Date : November 11, 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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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 Name: Garadacimab


Outcome Measures
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Primary Outcome Measures :
  1. Number of subjects with treatment emergent adverse events (TEAEs) [ Time Frame: At least 14 months ]
  2. Percent of subjects with TEAEs [ Time Frame: At lease 14 months ]
  3. Number of TEAEs [ Time Frame: At least 14 months ]
  4. TEAE rates per injection [ Time Frame: At least 14 months ]
  5. TEAE rates per subject year [ Time Frame: At least 14 months ]
  6. Maximum concentration (Cmax) of CSL312 at steady-state [ Time Frame: At least 12 months ]
  7. Trough concentration (Ctrough) of CSL312 at steady-state [ Time Frame: At least 12 months ]
  8. Time to maximum concentration (Tmax) of CSL312 at steady-state [ Time Frame: At least 12 months ]

Secondary Outcome Measures :
  1. Time-normalized number of HAE attacks per month and per year [ Time Frame: At least 12 months ]
  2. Time-normalized number of HAE attacks treated with on-demand treatment per month and per year [ Time Frame: At least 12 months ]
  3. Time-normalized number of moderate and / or severe HAE attacks per month and per year [ Time Frame: At least 12 months ]
  4. Percentage reduction in the time-normalized number of HAE attacks [ Time Frame: 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 [ Time Frame: At least 12 months ]
  6. Number of subjects with serious adverse events (SAEs) [ Time Frame: At least 14 months ]
  7. Percent of subjects with SAEs [ Time Frame: At least 14 months ]
  8. Number of subjects experiencing death [ Time Frame: At least 14 months ]
  9. Percent of subjects experiencing death [ Time Frame: At least 14 months ]
  10. Number of subjects with related TEAEs [ Time Frame: At least 14 months ]
  11. Percent of subjects with related TEAEs [ Time Frame: At least 14 months ]
  12. Number of subjects with TEAEs leading to study discontinuation [ Time Frame: At least 14 months ]
  13. Percent of subjects with TEAEs leading to study discontinuation [ Time Frame: At least 14 months ]
  14. Number of subjects with TEAEs by severity [ Time Frame: At least 14 months ]
  15. Percent of subjects with TEAEs by severity [ Time Frame: At least 14 months ]
  16. Number of subjects with Anti-CSL312 antibodies [ Time Frame: At least 14 months ]
  17. Percent of subjects with Anti-CSL312 antibodies [ Time Frame: At least 14 months ]
  18. Number of subjects with adverse events of special interest (AESIs) [ Time Frame: At least 14 months ]
  19. Percent of subjects with AESIs [ Time Frame: At least 14 months ]
  20. FXIIa-mediated kallikrein activity [ Time Frame: At least 12 months ]
    Blood samples will be collected on the same day as CSL312 administration for assessment of FXIIa-mediated kallikrein activity

  21. Number of subjects with laboratory findings reported as AEs [ Time Frame: At least 14 months ]
  22. Percent of subjects with laboratory findings reported as AEs [ Time Frame: At least 14 months ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 3
  2. 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
  3. 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
  4. Having laboratory clinical abnormalities assessed as clinically significant by the investigator in results of hematology or chemistry assessments performed during Screening
  5. Currently receiving a therapy not permitted during the study
  6. Being pregnant or breastfeeding.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05819775


Contacts
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Contact: Trial Registration Coordinator 1-610-878-4000 clinicaltrials@cslbehring.com

Locations
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United States, Arizona
Research Solutions of Arizona Recruiting
Litchfield Park, Arizona, United States, 85340
Contact: Use Central Contact         
Medical Research of Arizona Recruiting
Scottsdale, Arizona, United States, 85251
Contact: Use Central Contact         
United States, California
Donald S. Levy M.D. Recruiting
Orange, California, United States, 92868
Contact: Use Central Contact         
Raffi Tachdjian MD, Inc. Recruiting
Santa Monica, California, United States, 90404
Contact: Use Central Contact         
United States, Ohio
Bernstein Clinical Research Recruiting
Cincinnati, Ohio, United States, 45236
Contact: Use Central Contact         
Israel
Barzilai University Medical Center Recruiting
Ashkelon, Israel, 7830604
Contact: Use Central Contact         
Sponsors and Collaborators
CSL Behring
Investigators
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Study Director: Study Director CSL Behring
More Information
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Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT05819775    
Other Study ID Numbers: CSL312_3003
2022-502386-13-00 ( EU Trial (CTIS) Number )
First Posted: April 19, 2023    Key Record Dates
Last Update Posted: April 10, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Time Frame: Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
Access Criteria:

Proposed research should seek to answer a previously unanswered important medical or scientific question.

Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.

If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
 Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Hereditary Complement Deficiency Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes