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NCT Number: NCT07158606

Understanding Treatment Outcomes and Immunologic Mechanisms in Altuviiio Immune Tolerance Induction

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

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Key information

Conditions

Hemophilia A With Inhibitor

Age range

0 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Versiti Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location contact

Courtney Conway

CONTACT

[email protected]

414-257-2424

Lisa Baumann Kreuziger, MD, MS

PRINCIPAL_INVESTIGATOR

Megan Lemanczyk

CONTACT

414-257-2424

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

  • 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

Treatment and study plan

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.

Emicizumab

Drug

Emicizumab will be prescribed as standard of care bleed prevention.

Primary outcomes

  1. ITI Success

    Time frame: 12 months

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

Secondary outcomes

  1. Events

    Time frame: 12 months

    All bleeding events and factor and bypassing agent consumption from the time of ITI initiation until a patient has achieved tolerance.

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Trial Manager

CONTACT

[email protected]

414-257-2424

Clinical Trials and Research Office Supervisor

CONTACT

[email protected]

Sponsors and collaborators

Lead sponsor

Versiti Blood Health

Other

Registry information

Acronym: UTILITI

Important dates

Study start
2026
Primary completion
2031
Study completion
2033
First posted
Sep 8, 2025
Registry last updated
Jul 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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