Understanding Treatment Outcomes and Immunologic Mechanisms in Altuviiio Immune Tolerance Induction

Not Applicable

Not yet recruiting

• Conditions: Hemophilia A With Inhibitor
• Interventions: Drug: efanesoctacog alpha; Drug: Emicizumab

• Registration Number: NCT07158606
• Lead Sponsor: Versiti

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

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-08
• Study First Submitted: 2025-08-28
• Study First Submitted QC: 2025-08-28
• Last Update Submitted: 2025-08-28
• Study First Posted: 2025-09-08
• Last Update Posted: 2025-09-08
• Study Start: 2025-10-01
• Primary Completion: 2030-12-31
• Study Completion: 2032-08-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 15

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ITI	efanesoctacog alpha	ITI therapy will involve subjects receiving efanesoctacog alpha at a determined dose.
ITI	Emicizumab	ITI therapy will involve subjects receiving efanesoctacog alpha at a determined dose.

Primary Outcome Measures

Name	Time	Method
ITI Success	12 months	Time from initial dose of efanesoctacog alpha for ITI to ITI success or failure.

Secondary Outcome Measures

Name	Time	Method
Events	12 months	All bleeding events and factor and bypassing agent consumption from the time of ITI initiation until a patient has achieved tolerance.

Trial Locations

Locations (1)

Versiti Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States