Understanding Treatment Outcomes and Immunologic Mechanisms in Altuviiio Immune Tolerance Induction

NCT ID: NCT07158606

Last Updated: 2025-09-08

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-10-01
• Study Completion Date: 2032-08-31

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

Conditions

Hemophilia A With Inhibitor

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

ITI

ITI therapy will involve subjects receiving efanesoctacog alpha at a determined dose.

Group Type: EXPERIMENTAL

efanesoctacog alpha

Intervention Type: 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.

Emicizumab

Intervention Type: DRUG

Emicizumab will be prescribed as standard of care bleed prevention.

Interventions

efanesoctacog 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.

Intervention Type: DRUG

Emicizumab

Emicizumab will be prescribed as standard of care bleed prevention.

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 0 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Versiti

OTHER

Sponsor Role: lead

Responsible Party

Lynn Malec

Medical Director, Versiti Comprehensive Center for Bleeding Disorders

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Versiti Wisconsin

Milwaukee, Wisconsin, United States

Countries

United States

Central Contacts

Clinical Trial Manager

Role: CONTACT

CTRO@versiti.org

414-257-2424

Clinical Trials and Research Office Supervisor

Role: CONTACT

CTRO@versiti.org

Facility Contacts

Courtney Conway

Role: primary

cmconway@versiti.org

414-257-2424

Megan Lemanczyk

Role: backup

414-257-2424

Other Identifiers

PRO00052811

Identifier Type: -

Identifier Source: org_study_id