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Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks

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: NCT04739059
Recruitment Status : Recruiting
First Posted : February 4, 2021
Last Update Posted : May 13, 2022
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Brief Summary:
This phase 3b study will evaluate long-term safety and efficacy of CSL312 (also known as garadacimab) when administered subcutaneously (SC)

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Biological: CSL312 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 150 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema
Actual Study Start Date : March 29, 2021
Estimated Primary Completion Date : September 2023
Estimated Study Completion Date : September 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: CSL312
Fully human immunoglobulin G subclass 4/lambda recombinant monoclonal antibody administered subcutaneously
Biological: CSL312
Fully human immunoglobulin G subclass 4/lambda recombinant monoclonal antibody
Other Names:
  • Factor XIIa antagonist monoclonal antibody
  • garadacimab




Primary Outcome Measures :
  1. Number of subjects with treatment emergent adverse events (TEAEs) [ Time Frame: Up to 32 months ]
  2. Percentage of subjects with TEAEs [ Time Frame: Up to 32 months ]
  3. TEAEs rates per injection [ Time Frame: Up to 32 months ]
  4. TEAEs rates per subject year [ Time Frame: Up to 32 months ]

Secondary Outcome Measures :
  1. The time-normalized number (per month and year) of Hereditary Angioedema (HAE attacks) for the entire study [ Time Frame: Up to 34 months ]
  2. The percentage reduction and 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 on Treatment compared to Run-in Period [ Time Frame: Up to 34 months ]
  3. The time-normalized number (per month and year) of HAE attacks requiring on-demand treatment in subjects on treatment [ Time Frame: Up to 32 months ]
  4. The time-normalized number (per month and year) of moderate and/or severe HAE attacks in subjects on treatment [ Time Frame: Up to 32 months ]
  5. Number and percentage of subjects rating their response to therapy as good or excellent [ Time Frame: Up to 32 months ]
  6. The number and percentage of subjects experiencing TEAEs [ Time Frame: Up to 32 months ]
  7. The number and percentage of subjects experiencing adverse events of special interest (AESIs) [ Time Frame: Up to 32 months ]
  8. The number and percentage of subjects experiencing TEAEs for naïve C1-INH subjects only [ Time Frame: Up to 32 months ]
  9. The number and percentage of subjects experiencing serious adverse events (SAEs), including deaths [ Time Frame: Up to 32 months ]
  10. The number and percentage of subjects experiencing CSL312 induced anti-CSL312 antibodies [ Time Frame: Up to 32 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males and females aged ≥ 12 years
  • Diagnosed with clinically confirmed C1-INH HAE
  • Experienced ≥ 3 HAE attacks during the 3 months before Screening
  • Participated in the Run-in Period for at least 1 month (CSL312-naïve subjects only)
  • Experienced at least an average of 1 HAE attack per month during the Run-in Period

Exclusion Criteria:

  • Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema or recurrent angioedema associated with urticaria
  • Use of C1-INH products, androgens, antifibrinolytics or other small molecule medications for routine prophylaxis against HAE attacks at least 2 weeks before the first day of the Run-in Period
  • Use of monoclonal antibodies such as lanadelumab (Takhzyro®) 3 months before the first day of the Run-in Period.
  • Female subjects use estrogen-containing oral contraceptives or hormone replacement therapy within 4 weeks prior to screening
  • Female or male subjects who are fertile and sexually active not using or not willing to use an acceptable method of contraception to avoid pregnancy during the study and for 30 days after receipt of the last dose of CSL312
  • Pregnant, breastfeeding, or not willing to cease breastfeeding

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): NCT04739059


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

Locations
Show Show 48 study locations
Sponsors and Collaborators
CSL Behring
Investigators
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Study Director: Study Director CSL Behring
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Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT04739059    
Other Study ID Numbers: CSL312_3002
2020-003918-12 ( EudraCT Number )
First Posted: February 4, 2021    Key Record Dates
Last Update Posted: May 13, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

CSL will consider requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.

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.

Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Time Frame: IPD requests may be submitted to CSL no earlier than 12 months after publication of the results of this study via an article made available on a public website.
Access Criteria:

Requests may only be made by systematic review groups or bona-fide researchers whose proposed use of the IPD is non-commercial in nature and has been approved by an internal review committee.

An IPD request will not be considered by CSL unless the proposed research question seeks to answer a significant and unknown medical science or patient care question as determined by CSL's internal review committee.

The requesting party must execute an appropriate data sharing agreement before IPD will be made 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