Understanding Treatment Outcomes and Immunologic Mechanisms in Altuviiio Immune Tolerance Induction

NCT07158606 | Not Yet Recruiting Phase 4 DMC FDA Drug

• 1 other identifier: PRO00052811
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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. 1.A screening period - 1 visit up to 8 weeks before the Treatment Period starts
2. 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. 3.Tapering Period - 5 visits at weeks 2, 4, 8, 12 and 16
4. 4.Ongoing Monitoring Period - 4 visits at weeks 20, 30, 40 and 50

Conditions

Hemophilia A With Inhibitor

Outcome Measures

Primary Outcomes (1)

• ITI Success

  Time from initial dose of efanesoctacog alpha for ITI to ITI success or failure.

  12 months

Secondary Outcomes (1)

• Events

  12 months

Study Arms (1)

ITI

EXPERIMENTAL

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

Drug: efanesoctacog alpha; Drug: Emicizumab

Interventions

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.

ITI

Emicizumab DRUG

Emicizumab will be prescribed as standard of care bleed prevention.

ITI

Eligibility Criteria

• Age: 0 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• \) 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

You may not qualify if:

• 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
• \) 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

Sponsors & Collaborators

• Versiti Blood Health: lead

Study Sites (1)

Versiti Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Interventions

emicizumab

Central Study Contacts

Clinical Trial Manager

CONTACT

414-257-2424; CTRO@versiti.org

Clinical Trials and Research Office Supervisor

CONTACT

CTRO@versiti.org

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical Director, Versiti Comprehensive Center for Bleeding Disorders

Study Record Dates

• First Submitted: August 28, 2025
• First Posted: September 8, 2025
• Study Start: October 1, 2025
• Primary Completion (Estimated): December 31, 2030
• Study Completion (Estimated): August 31, 2032
• Last Updated: September 8, 2025

Record last verified: 2025-08

Locations

US (1)