Emicizumab + Altuviiio for Hemophilia A

(UTILITI Trial)

Overseen By Clinical Trials and Research Office Supervisor

Age: < 18

Sex: Any

Trial Phase: Phase 4

Sponsor: Versiti

Must be taking: Emicizumab

Disqualifiers: Von Willebrand, Thrombosis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 11

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the effectiveness of a new treatment, Altuviiio (efanesoctacog alpha), for individuals with severe hemophilia A—a condition where blood doesn't clot properly—who also use Hemlibra to prevent bleeding. Participants will receive Altuviiio alongside their regular Hemlibra treatment. Those with severe hemophilia A from birth, a history of high inhibitor levels, and current or planned use of Hemlibra may be suitable candidates for this trial. As a Phase 4 trial, this research seeks to understand how the already FDA-approved and proven effective treatment benefits a broader patient population.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications. However, since the trial involves using Hemlibra as a standard treatment, you may need to continue with it. It's best to discuss your current medications with the research doctor.

What is the safety track record for these treatments?

Research shows that Altuviiio (efanesoctacog alpha) is generally well-tolerated by people with severe hemophilia A. Studies have found that weekly doses can effectively reduce bleeding over a year, with no major safety concerns reported.

Long-term research on Hemlibra (emicizumab) indicates it is safe and effective for treating hemophilia A. People have used it for nearly five years without major issues. It helps control bleeding and maintains a strong safety record.

Both treatments have undergone thorough study and are considered safe based on current data. Participants who used these treatments reported few side effects, suggesting they are well-tolerated. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Researchers are excited about Emicizumab and Efanesoctacog Alpha for Hemophilia A because they offer innovative approaches to manage the condition. Unlike most treatments that require frequent intravenous infusions, Emicizumab is administered subcutaneously, making it more convenient for patients. Efanesoctacog Alpha, also known as Altuviiio, is engineered to provide longer-lasting protection with less frequent dosing. These features could significantly improve the quality of life for individuals with Hemophilia A by simplifying their treatment regimen and reducing the frequency of interventions.

What evidence suggests that this trial's treatments could be effective for Hemophilia A?

Research shows that efanesoctacog alpha, also known as Altuviiio, effectively protects against bleeding in people with severe hemophilia A. Studies indicate that it maintains high levels of factor VIII, a protein essential for blood clotting. Early results suggest that regular use of this treatment greatly reduces bleeding episodes. Emicizumab (Hemlibra) has already proven effective in preventing bleeds in hemophilia A patients, supporting its use as a preventive measure. In this trial, participants will receive efanesoctacog alpha as part of ITI therapy. Together, these treatments offer a promising way to manage hemophilia A by preventing bleeds and improving the body's tolerance to treatment. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals with Hemophilia A. Participants will receive Altuviiio for Immune Tolerance Induction and Hemlibra to prevent bleeding. They must attend several checkups and tests over a year, including in-person visits and possibly phone calls.

Inclusion Criteria

Severe hemophilia A (congenital)

History of high titer inhibitor (≥ 5 BU)

Peak inhibitor titer (pre-ITI) < 1,000

Exclusion Criteria

Known contraindication, intolerance, or allergy to either of the investigational agents of study

Inability or unwillingness to provide informed consent and/or assent

Inability to speak or read English

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 8 weeks

1 visit (in-person)

ITI Treatment

Participants receive Altuviiio for ITI therapy with Hemlibra as prophylaxis

up to 52 weeks

1 Baseline Visit, Interim visits every 4 weeks (some via phone)

Tapering

Participants undergo tapering of treatment

16 weeks

5 visits at weeks 2, 4, 8, 12, and 16

Ongoing Monitoring

Participants are monitored for safety and effectiveness after tapering

30 weeks

4 visits at weeks 20, 30, 40, and 50

What Are the Treatments Tested in This Trial?

Interventions

• Efanesoctacog Alpha
• Emicizumab

Trial Overview The study is testing the effectiveness of Altuviiio (efanesoctacog alpha) in inducing immune tolerance while using Hemlibra (emicizumab) as a preventative treatment against bleeds in Hemophilia A patients.

How Is the Trial Designed?

Group I: ITI Experimental Treatment 2 Interventions

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

Who Is Running the Clinical Trial?

Versiti

Lead Sponsor

Trials

16

Recruited

2,500+

Citations

1. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/5495/527825/Clinical-Outcomes-over-2-Years-of-Once-Weekly

Clinical Outcomes over 2 Years of Once-Weekly ... Once-weekly efanesoctocog alfa 50 IU/kg was well tolerated and provided highly effective bleed protection in children with severe hemophilia A in the XTEND- ...

2. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK616635/

Efanesoctocog Alfa (ALTUVIIIO) - NCBI Bookshelf - NIH The cost-effectiveness of ALTUVIIIO for routine prophylaxis in patients with mild and moderate hemophilia A, for treatment and control of bleeding episodes, and ...

3. nejm.org nejm.org/doi/full/10.1056/NEJMoa2209226

Efanesoctocog Alfa Prophylaxis for Patients with Severe ... Efanesoctocog alfa provides high sustained factor VIII activity by overcoming the von Willebrand factor–imposed half-life ceiling.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05911763

A Study to Evaluate Impact of Efanesoctocog Alfa on Long- ... ... efanesoctocog alfa. Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period, Over a 5 year period. Number ...

5. hematologyadvisor.com hematologyadvisor.com/news/efanesoctocog-alfa-not-cost-effective-for-prophylaxis-in-hemophilia-a/

Efanesoctocog Alfa Not Cost-Effective for Prophylaxis in ... Prophylaxis with efanesoctocog alfa is neither conventionally nor distributionally cost-effective, according to a study published online April 22 in the Annals ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04644575

Study Details | NCT04644575 | Long-term Safety and ... Primary Objective: - To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A. Secondary Objectives:.

7. sobi.com sobi.com/en/press-releases/late-breaking-efanesoctocog-alfa-data-presented-isth-demonstrates-highly-effective-bleed-protection-children-severe-haemophilia-once

Late-breaking efanesoctocog alfa data presented at ISTH ... Late-breaking efanesoctocog alfa data presented at ISTH demonstrates highly effective bleed protection in children with severe haemophilia A ...