73 Participants Needed

Emicizumab for Hemophilia A

(HAVEN 6 Trial)

Recruiting at 36 trial locations
RS
Overseen ByReference Study ID Number: BO41423 www.roche.com/about_roche/roche_worldwide.htm
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: Hoffmann-La Roche
No Placebo GroupAll 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 DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you are on any investigational drugs or treatments for hemophilia, you may need to stop them before joining the trial. It's best to discuss your specific medications with the trial team.

What data supports the effectiveness of the drug Emicizumab for Hemophilia A?

Research shows that Emicizumab is effective in improving blood clotting in people with Hemophilia A by mimicking the function of a missing protein, leading to better outcomes in both those with and without inhibitors.12345

Is emicizumab safe for humans?

Emicizumab has been shown to be safe in humans, with its safety proven in multiple clinical trials for patients with hemophilia A, including those with and without inhibitors. Long-term studies have also supported its safety over several years of use.12467

How is the drug Emicizumab different from other treatments for Hemophilia A?

Emicizumab is unique because it is a bispecific antibody that mimics the function of missing factor VIII by bridging activated factor IX and factor X, helping to prevent bleeding in Hemophilia A patients. Unlike traditional treatments that require intravenous infusions, Emicizumab is administered subcutaneously (under the skin), making it more convenient for patients.128910

What is the purpose of this trial?

This trial tests emicizumab, a medication that helps blood clot, in people with mild or moderate hemophilia A. It works by mimicking a missing protein to improve blood clotting. Emicizumab has been approved for treatment of hemophilia A patients.

Research Team

CT

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Eligibility Criteria

This trial is for individuals with mild or moderate Hemophilia A without inhibitors to factor VIII. Participants must weigh at least 3 kg, need prophylaxis, have documented treatment and bleeding episodes history, and proper organ function. Women of childbearing age should agree to use contraception.

Inclusion Criteria

I have mild or moderate Hemophilia A without inhibitors.
Need for prophylaxis based on investigator assessment
I weigh at least 3 kilograms.
See 5 more

Exclusion Criteria

Inability to comply with the study protocol in the opinion of the investigator
Pregnant or breastfeeding, or intending to become pregnant during the study (women of childbearing potential must have a negative serum pregnancy test result within 7 days prior to initiation of study drug)
I am not planning any surgery during the initial phase of my emicizumab treatment.
See 8 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive emicizumab with a loading dose regimen followed by one of three maintenance dose regimens

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term follow-up

Participants continue to be monitored for long-term safety and efficacy outcomes

up to approximately 48 months

Treatment Details

Interventions

  • Emicizumab
Trial Overview The study tests Emicizumab's safety, effectiveness, how the body processes it (pharmacokinetics), and its impact on the body (pharmacodynamics) in those with mild/moderate Hemophilia A who don't have FVIII inhibitors.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: EmicizumabExperimental Treatment1 Intervention
Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors will be enrolled to receive the emicizumab loading dose regimen followed by the participant's preference of one of 3 maintenance dose regimens.

Emicizumab is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Hemlibra for:
  • Hemophilia A
  • Hemophilia A with inhibitors
🇪🇺
Approved in European Union as Hemlibra for:
  • Hemophilia A
  • Hemophilia A with inhibitors

Find a Clinic Near You

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 profile image

Dr. Levi Garraway

Hoffmann-La Roche

Chief Medical Officer since 2019

MD from the University of Basel

Dr. Thomas Schinecker profile image

Dr. Thomas Schinecker

Hoffmann-La Roche

Chief Executive Officer since 2023

PhD in Molecular Biology from New York University

Findings from Research

Emicizumab significantly shortened the activated partial thromboplastin time (APTT) in patients with severe hemophilia A after the first dose, indicating its effectiveness in improving blood clotting.
The standard one-stage APTT-based FVIII activity assay (sOSA) produced inaccurately high FVIII activity levels when using emicizumab, suggesting it should not be used for monitoring, while modified OSA (mOSA) effectively quantified emicizumab concentration.
Effects of emicizumab on APTT, one-stage and chromogenic assays of factor VIII in artificially spiked plasma and in samples from haemophilia A patients with inhibitors.Bowyer, A., Kitchen, S., Maclean, R.[2020]
In a phase 3 trial with 152 participants, emicizumab significantly reduced the annualized bleeding rate in hemophilia A patients without factor VIII inhibitors, showing a 96% reduction compared to no prophylaxis.
Emicizumab was well-tolerated, with the most common side effect being low-grade injection-site reactions, and it did not lead to serious complications like thrombotic events or the development of factor VIII inhibitors.
Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors.Mahlangu, J., Oldenburg, J., Paz-Priel, I., et al.[2022]
Emicizumab is an effective treatment for hemophilia A, significantly reducing bleeding events compared to on-demand treatment and traditional factor VIII prophylaxis, making it a valuable alternative for patients.
The safety profile of emicizumab is generally excellent, with only a few reported cases of thrombotic events, indicating it is a well-tolerated option for managing hemophilia A.
Safety evaluation of emicizumab prophylaxis in individuals with haemophilia A.Wang, CP., Young, G., Thornburg, CD.[2022]

References

Effects of emicizumab on APTT, one-stage and chromogenic assays of factor VIII in artificially spiked plasma and in samples from haemophilia A patients with inhibitors. [2020]
Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors. [2022]
Safety evaluation of emicizumab prophylaxis in individuals with haemophilia A. [2022]
Emicizumab prophylaxis among infants and toddlers with severe hemophilia A and inhibitors-a single-center cohort. [2020]
Safety and efficacy of long-term emicizumab prophylaxis in hemophilia A with factor VIII inhibitors: A phase 3b, multicenter, single-arm study (STASEY). [2023]
Emicizumab prophylaxis in haemophilia patients older than 50 years with cardiovascular risk factors: Real-world data. [2021]
Long-term safety and efficacy of emicizumab for up to 5.8 years and patients' perceptions of symptoms and daily life: A phase 1/2 study in patients with severe haemophilia A. [2021]
Emicizumab-kxwh: First Global Approval. [2019]
Detailed analysis of anti-emicizumab antibody decreasing drug efficacy, using plasma samples from a patient with hemophilia A. [2023]
Real-world use of emicizumab in patients with haemophilia A: Bleeding outcomes and surgical procedures. [2021]
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