Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks
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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. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04739059 |
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Recruitment Status :
Active, not recruiting
First Posted : February 4, 2021
Last Update Posted : April 5, 2024
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hereditary Angioedema | Biological: CSL312 | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 171 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 : | November 2025 |
| Estimated Study Completion Date : | November 2025 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: CSL312
Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody administered subcutaneously
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Biological: CSL312
Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody
Other Names:
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- Number of subjects with treatment emergent adverse events (TEAEs) [ Time Frame: Up to 45 months ]
- Percentage of subjects with TEAEs [ Time Frame: Up to 45 months ]
- TEAEs rates per injection [ Time Frame: Up to 45 months ]
- TEAEs rates per subject year [ Time Frame: Up to 45 months ]
- The time-normalized number (per month and year) of Hereditary Angioedema (HAE attacks) for the entire study [ Time Frame: Up to 43 months ]
- 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 43 months ]
- The time-normalized number (per month and year) of HAE attacks requiring on-demand treatment in subjects on treatment [ Time Frame: Up to 43 months ]
- The time-normalized number (per month and year) of moderate and/or severe HAE attacks in subjects on treatment [ Time Frame: Up to 43 months ]
- Number and percentage of subjects rating their response to therapy as good or excellent [ Time Frame: Up to 43 months ]
- The number and percentage of subjects experiencing TEAEs [ Time Frame: Up to 45 months ]
- The number and percentage of subjects experiencing adverse events of special interest (AESIs) [ Time Frame: Up to 45 months ]
- The number and percentage of subjects experiencing serious adverse events (SAEs), including deaths [ Time Frame: Up to 45 months ]
- The number and percentage of subjects experiencing CSL312 induced anti-CSL312 antibodies [ Time Frame: Up to 45 months ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
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
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| Study Director: | Study Director | CSL Behring |
| 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: | April 5, 2024 |
| Last Verified: | April 2024 |
| 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. |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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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 |