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The goal of this clinical trial is to learn how well Altuviiio (efanesoctacog alpha) works for Immune Tolerance Induction (ITI) while using Hemlibra (emicizumab) to prevent bleeds.
Participants will be given Altuviiio for their ITI therapy and also be treated with Hemlibra as standard of care prophylaxis to prevent bleeding. The research doctor will decide how much and how often the participant will get Hemlibra.
Participants will need to attend visits for checkups and tests. These visits are divided into 4 periods:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ITI | Experimental | ITI therapy will involve subjects receiving efanesoctacog alpha at a determined dose. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| efanesoctacog alpha | Drug | Efanesoctacog alpha will be administered at a dose of 50 IU/kg two times weekly during the ITI Treatment Period. The site investigator will determine the dose of efanesoctacog alpha once the subjects reaches the Follow Up Period. |
| Measure | Description | Time Frame |
|---|---|---|
| ITI Success | Time from initial dose of efanesoctacog alpha for ITI to ITI success or failure. | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Events | All bleeding events and factor and bypassing agent consumption from the time of ITI initiation until a patient has achieved tolerance. | 12 months |
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Inclusion Criteria:
1) severe hemophilia A (congenital) 2) history of high titer inhibitor (≥ 5 BU) 3) peak inhibitor titer (pre-ITI) < 1,000 3) age < 18 y/o at the time of study enrollment 4) undergoing initial ITI course 5) current or planned concomitant use of emicizumab
Exclusion Criteria:
3) personal history of unprovoked thrombosis 4) known contraindication, intolerance, or allergy to either of the investigational agents of study 5) inability or unwillingness to provide informed consent and/or assent 6) inability to speak or read English 7) Any other condition, that in the opinion of the investigator, would negatively impact the safety of the participant
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Manager | Contact | 414-257-2424 | CTRO@versiti.org | |
| Clinical Trials and Research Office Supervisor | Contact | CTRO@versiti.org |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Versiti Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
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| ID | Term |
|---|---|
| C000608208 | emicizumab |
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| Emicizumab | Drug | Emicizumab will be prescribed as standard of care bleed prevention. |
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