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:
1. A screening period - 1 visit up to 8 weeks before the Treatment Period starts 2. ITI Treatment Period - 1 Baseline Visit plus Interim visits that occur every 4 weeks for up to 52 weeks. (Some of the interim visits may be done via phone) 3. Tapering Period - 5 visits at weeks 2, 4, 8, 12 and 16 4. Ongoing Monitoring Period - 4 visits at weeks 20, 30, 40 and 50
None
von Willebrand disease or a known second bleeding diathesis besides SHA
prior course of ITI
inhibitor present for > 2 years without prior attempts at eradication
Single group
efanesoctacog alpha
DrugEfanesoctacog 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.
Emicizumab
DrugEmicizumab will be prescribed as standard of care bleed prevention.
ITI Success
Time from initial dose of efanesoctacog alpha for ITI to ITI success or failure.
Events
All bleeding events and factor and bypassing agent consumption from the time of ITI initiation until a patient has achieved tolerance.
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This trial is not recruiting at the moment. You can still explore other options:
