Hemophilia A With Inhibitor
Conditions
Brief summary
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
Interventions
DRUGefanesoctacog alpha
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.
DRUGEmicizumab
Emicizumab will be prescribed as standard of care bleed prevention.
Sponsors
Versiti Blood Health
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
0 Years to 17 Years
Healthy volunteers
No
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
1. von Willebrand disease or a known second bleeding diathesis besides SHA 2. prior course of ITI 3. inhibitor present for \> 2 years without prior attempts at eradication 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ITI Success | 12 months | Time from initial dose of efanesoctacog alpha for ITI to ITI success or failure. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Events | 12 months | All bleeding events and factor and bypassing agent consumption from the time of ITI initiation until a patient has achieved tolerance. |
Countries
United States
Contacts
CONTACTClinical Trial Manager
CONTACTClinical Trials and Research Office Supervisor
Outcome results
None listed