Hemophilia A With InhibitorPhase 4UTILITINCT07158606
Understanding Treatment Outcomes and Immunologic Mechanisms in Altuviiio Immune Tolerance Induction
A Phase 4 study testing efanesoctacog alpha and Emicizumab in people with Hemophilia A With Inhibitor, run by Versiti Blood Health at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 17 years
- Conditions the study is for
- Hemophilia A With Inhibitor
- What is being tested?
- efanesoctacog alpha and Emicizumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Dec 2026to 31 Aug 2033
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Clinical Trials and Research Office Supervisor contact
United States1 site
Versiti Wisconsin
Milwaukee, Wisconsin, 53226
Courtney Conway, 414-257-2424, cmconway@versiti.org
Megan Lemanczyk, 414-257-2424
About this study
Official title: 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.
Read more
Participants will need to attend visits for checkups and tests. These visits are divided into 4 periods:
- A screening period - 1 visit up to 8 weeks before the Treatment Period starts
- 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)
- Tapering Period - 5 visits at weeks 2, 4, 8, 12 and 16
- Ongoing Monitoring Period - 4 visits at weeks 20, 30, 40 and 50
- Conditions
- Hemophilia A With Inhibitor
- Being tested
- efanesoctacog alpha, Emicizumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
efanesoctacog alphaDrug
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.
EmicizumabDrug
Emicizumab will be prescribed as standard of care bleed prevention.
| Study group | Role | What they receive |
|---|---|---|
| ITI | Experimental | ITI therapy will involve subjects receiving efanesoctacog alpha at a determined dose. Receives: Drug: efanesoctacog alpha; Drug: Emicizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
ITI Success
Time from initial dose of efanesoctacog alpha for ITI to ITI success or failure.
Measured: 12 months
Secondary outcomes (1)
Events
All bleeding events and factor and bypassing agent consumption from the time of ITI initiation until a patient has achieved tolerance.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2025
- Study start1 Dec 2026 (estimated)
- Primary completion31 Dec 2031 (estimated)
- Study completion31 Aug 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A With InhibitorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Versiti Blood Health
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Lisa Baumann Kreuziger, Versiti Blood Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07158606
- Sponsor's protocol ID
- PRO00052811
- Acronym
- UTILITI
- Status last verified
- Jul 2026
- First submitted
- 28 Aug 2025
- Last updated
- 27 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Understanding Treatment Outcomes and Immunologic Mechanisms in ALtuviiio Immune Tolerance Induction
- ClinicalTrials.gov ID
- NCT07158606
- Acronym / sponsor ID
- UTILITI / PRO00052811
- Conditions
- Hemophilia A With Inhibitor
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Versiti Blood HealthUniversity, hospital or other
- Study start
- 1 Dec 2026 (estimated)
- Primary completion
- 31 Dec 2031 (estimated)
- Study completion
- 31 Aug 2033 (estimated)
- First posted
- 8 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- A screening period - 1 visit up to 8 weeks before the Treatment Period starts
- 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)
- Tapering Period - 5 visits at weeks 2, 4, 8, 12 and 16
- Ongoing Monitoring Period - 4 visits at weeks 20, 30, 40 and 50
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ITI | Experimental | Drug: efanesoctacog alpha, Drug: EmicizumabITI therapy will involve subjects receiving efanesoctacog alpha at a determined dose. |
Who may qualify
- Ages
- 0 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- 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
Key exclusion criteria (4 listed)
- 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
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| efanesoctacog alphaEfanesoctacog 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. | Drug | ITI |
| EmicizumabEmicizumab will be prescribed as standard of care bleed prevention. | Drug | ITI |
Primary outcome
ITI Success
Time frame: 12 months
Time from initial dose of efanesoctacog alpha for ITI to ITI success or failure.
1 secondary outcome
Events
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Hemophilia A With Inhibitor
RecruitingPhase 3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Hemophilia A, Hemophilia B
RecruitingPhase 3
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Hemophilia A, Hemophilia B
RecruitingObservational study
Treatment of Hemophilia A Patients With FVIII Inhibitors
Hemophilia A
Not yet recruitingObservational study
Prevalence of Inhibitor Development in Hemophilia A Patients Receiving Coageight: A Multicenter Cross-sectional Observational Study
Hemophilia A, Hemophilia
Not yet recruiting
A Study of Elranatamab for Inhibitor Eradication in Patients With Hemophilia A
Hemophilia A With Inhibitors
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Clinical Trials and Research Office Supervisor contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing efanesoctacog alpha and Emicizumab in people with Hemophilia A With Inhibitor, run by Versiti Blood Health at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 27 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trial Manager (414-257-2424, CTRO@versiti.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ITI Success (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2026.