Emicizumab for Von Willebrand Disease

(WILL-EMI Trial)

Recruiting at 22 trial locations

Overseen By Reference Study ID Number: WP45338 https://forpatients.roche.com/

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Hoffmann-La Roche

Must not be taking: Systemic immunomodulators

Disqualifiers: Bleeding disorders, Thromboembolic disease, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

75 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called emicizumab for individuals with Type 3 von Willebrand disease, a condition that hinders blood clotting. Emicizumab aids in more effective blood clotting. The study compares emicizumab to the usual treatment to determine its effectiveness in reducing bleeding episodes. Participants will either switch to emicizumab or continue their current treatment to assess which is more effective. Those experiencing frequent bleeding despite treatment might be well-suited for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Do I need to stop my current medications for the trial?

The trial does not specify if you need to stop your current medications, but it does mention that you cannot use systemic immunomodulators (medications that affect the immune system) during the study, except for anti-retroviral therapy.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that emicizumab has been studied for safety in individuals with severe blood disorders like Von Willebrand Disease (VWD). In one study, a child with severe VWD used emicizumab for 51 weeks, effectively preventing bleeding without major safety issues.

Emicizumab is already approved for treating other bleeding disorders, such as hemophilia A, and patients have generally tolerated it well. This suggests it might also be safe for people with similar conditions. However, like any treatment, side effects can occur. Discuss these with the trial team or your doctor. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for von Willebrand disease?

Researchers are excited about emicizumab for Von Willebrand Disease because it offers a new approach compared to existing treatments. Unlike standard therapies, which often involve replacing missing clotting factors through infusions, emicizumab is a monoclonal antibody delivered via subcutaneous injection. This convenient delivery method means fewer hospital visits and potentially greater adherence. Additionally, emicizumab works by bridging activated factors IX and X, bypassing the need for factor VIII, which can provide a more consistent bleeding control experience for patients.

What evidence suggests that this trial's treatments could be effective for Von Willebrand Disease?

Studies have shown that emicizumab can improve blood clotting in people with von Willebrand disease (VWD). Research indicates that it enhances the body's ability to form clots, helping to prevent bleeding. In patients with VWD, emicizumab has effectively reduced bleeding episodes and improved quality of life. One study found that three out of four patients experienced significant improvement. In this trial, participants in Arm A and Arm C will receive emicizumab prophylaxis, while those in Arm B will continue with their current on-demand standard of care treatment. Emicizumab's known effectiveness in similar bleeding disorders supports its potential for VWD. ^{1 2 6 7 8}

Who Is on the Research Team?

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Are You a Good Fit for This Trial?

This trial is for people aged 2 and older with Type 3 von Willebrand Disease who've had at least two bleeds treated with factor concentrate in the last 24 weeks. They must have a confirmed diagnosis, stable organ function, and if of childbearing potential, agree to use contraception. Those already on standard prophylactic or on-demand therapies are eligible.

Inclusion Criteria

I agree to follow the study's rules on not having a child or using birth control.

I have followed a standard treatment plan for VWD as prescribed.

I have followed a specific study protocol for at least 24 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive emicizumab prophylaxis or continue SOC on-demand therapy for 24 weeks

24 weeks

Treatment Extension

Participants who benefit from emicizumab can continue prophylaxis in the extension period

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Emicizumab

Trial Overview The study tests emicizumab's effectiveness and safety as a preventive treatment compared to the current standard care for Type 3 VWD. Participants will either continue their usual therapy or switch to emicizumab, with results from both groups being analyzed.

How Is the Trial Designed?

Group I: Treatment Extension Period for All Arms: Emicizumab Prophylaxis Experimental Treatment 1 Intervention

Participants in Arms A and C who have completed 24 weeks of emicizumab prophylaxis and who derive benefit from emicizumab will have the opportunity to continue to receive emicizumab prophylaxis in the extension period. Participants in Arm B who have completed 24 weeks of SOC on-demand treatment will have the opportunity to receive emicizumab prophylaxis in the extension period.

Group II: Arm C (Prior Prophylaxis SOC): Emicizumab Prophylaxis for 24 Weeks Experimental Treatment 1 Intervention

Participants who enroll in Arm C, taking SOC prophylactic treatment at the time of study entry and for at least 24 weeks of observation during the preceding NIS WP45335, will receive emicizumab SC prophylaxis.

Group III: Arm A (Prior On-Demand SOC): Emicizumab Prophylaxis for 24 Weeks Experimental Treatment 1 Intervention

Participants who are randomized to Arm A, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will receive emicizumab SC prophylaxis.

Group IV: Arm B (Prior On-Demand SOC): On-Demand SOC for 24 Weeks Active Control 4 Interventions

Participants who are randomized to Arm B, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will continue to receive their current SOC on-demand treatment until Week 24.

Who Is Running the Clinical Trial?

Hoffmann-La Roche

Lead Sponsor

Trials

2,482

Recruited

1,107,000+

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Avastin, Herceptin, Rituxan, Accu-Chek

Dr. Levi Garraway

Hoffmann-La Roche

Chief Medical Officer since 2019

MD from the University of Basel

Dr. Thomas Schinecker

Hoffmann-La Roche

Chief Executive Officer since 2023

PhD in Molecular Biology from New York University

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06998524

NCT06998524 | A Study to Assess the Efficacy and Safety ... A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease (WILL-EMI). ClinicalTrials.gov ID NCT06998524.

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35014121/

Efficacy of emicizumab in von Willebrand disease (VWD) ... In vitro studies and in VWD patients on emicizumab, showed improvement in thrombin generation and fibrin formation. Among four patients, three ...

3. genentech-clinicaltrials.com genentech-clinicaltrials.com/content/patient-platform/na/us/en/trials/blood-disorder/blood-platelet-disorder/a-study-to-assess-the-efficacy-and-safety-of-emicizumab-28219.pdf

A study to look at how safe and well emicizumab treatment ... This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2475037924002942

Prophylaxis in von Willebrand disease with ... Despite a lack of comparison with the preprophylaxis bleeding event rate, the authors concluded that prophylaxis was effective in preventing recurrent bleeding ...

5. ashpublications.org ashpublications.org/bloodadvances/article/3/18/2748/374944/Efficacy-of-emicizumab-in-a-pediatric-patient-with

Efficacy of emicizumab in a pediatric patient with type 3 von ... Emicizumab prophylaxis in such patients may result in improved hemarthrosis control, lower cost, and enhanced quality of life. Topics:.

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/33341070/

The potential role of emicizumab prophylaxis in severe von ... We report 51 weeks of successful off label emicizumab prophylaxis in a child with severe VWD and recurrent hemarthroses and progressive ...

7. forpatients.roche.com forpatients.roche.com/en/trials/blood-disorder/blood-platelet-disorder/a-study-to-assess-the-efficacy-and-safety-of-emicizumab-28219.html

Clinical Study | WP45338 | Severe Bleeding Disorders | Fo... A study to look at how safe and well emicizumab treatment works to prevent bleeds for people living with Von Willebrand disease and understand what happens ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT05500807

Emicizumab for Severe Von Willebrand Disease (VWD) ... The hypothesis of this study is that Emicizumab is safe and efficacious for prophylaxis in severe VWD and concomitant VWD/hemophilia patients. Detailed ...