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A clinical trial to look at how safe emicizumab is in patients with mild or moderate hemophilia A, and how emicizumab affects bleeding in this patient population.
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
- Hemophilia
- Hemophilia A
Please note that the recruitment status of the trial at your site may differ from the overall study status because some study sites may recruit earlier than others.
Trial Status:
Active, not recruiting
This trial runs in
Countries
- Belgium
- Canada
- France
- Germany
- Netherlands
- Poland
- South Africa
- Spain
- United Kingdom
- United States
Trial Identifier:
NCT04158648 2019-002179-32,2023-506610-52-00 BO41423
Trial Summary
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against factor VIII (FVIII).
Hoffmann-La Roche
Sponsor
Phase 3
Phase
NCT04158648,BO41423,2019-002179-32,2023-506610-52-00
Trial Identifier
All
Gender
No
Healthy Volunteers
How does the HAVEN 6 clinical trial work?
This clinical trial is recruiting people who have hemophilia A. In order to take part, patients must have mild or moderate hemophilia A (without factor FVIII antibodies), and be suitable to receive treatment for bleed prevention (prophylaxis).
The purpose of this study is to test the effects, good and/or bad, of emicizumab in patients with mild or moderate hemophilia A when it is given either once a week, once every 2 weeks, or once every 4 weeks.
How do I take part in this clinical trial?
To be able to take part in this clinical trial, you must have been diagnosed with mild or moderate hemophilia A.
You must not have any other bleeding disorder or be taking certain treatments, and you will not be able to take part if you are pregnant or breastfeeding.
If you think this clinical trial may be suitable for you and you would like to take part, please talk to your doctor. If your doctor is of the opinion that you might be able to take part in this clinical trial, he/she may refer you to the closest clinical trial doctor. They will give you all the information you need to make your decision about taking part. You can also find the clinical trial locations on this page.
You will have some further tests to make sure you will be able to take the treatments given in this clinical trial. Some of these tests or procedures may be part of your regular medical care. They may be done even if you do not take part in the clinical trial. If you have had some of the tests recently, they may not need to be done again.
Before starting the clinical trial, you will be told about any risks and benefits of taking part in the trial. You will also be told what other treatments are available so that you may decide if you still want to take part.
If you are a woman taking part in the clinical trial, who is not currently pregnant but can become pregnant, you will either need to take contraceptive medication or not have heterosexual intercourse. This is for safety reasons.
What treatment will I be given if I join this clinical trial?
You and your doctor will decide how often you should have emicizumab treatments. Everyone who joins this clinical trial will then be assigned to one of the following 3 groups based on this decision:
Group 1
- emicizumab given as an injection under your skin every week for 4 weeks then continue with a lower dose of emicizumab every week
Group 2
- emicizumab given as an injection under your skin every week for 4 weeks then continue with the same dose of emicizumab every 2 weeks
Group 3
- emicizumab given as an injection under your skin every week for 4 weeks then continue with a higher dose of emicizumab every 4 weeks
You will not be allowed to change between groups during the first year of the study.
How often will I be seen in follow-up appointments and for how long?
During the study, you will be seen by the clinical trial doctor every 4 weeks for at least one year. These hospital visits will include regular checks and blood draws to see how you are responding to the treatment and any side effects that you may be having. You will also be given questionnaires to complete at some of your visits. Between these visits you or your caregiver will be asked to provide information on your bleeds and bleed treatments on a weekly basis. You are free to stop this treatment at any time. Your healthcare team will discuss any ongoing treatment with you.
What happens if I am unable to take part in this clinical trial?
If this clinical trial is not suitable for you, you will not be able to take part. Your doctor may suggest other clinical trials that you may be able to take part in or other treatments that you can be given. You will not lose access to any of your regular care.
For more information about this clinical trial see the For Expert tab on the specific For Patient page or follow this link to ClinicalTrials.gov: https://clinicaltrials.gov/ct2/show/NCT04158648
Trial-identifier: NCT04158648
Trial Summary
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against factor VIII (FVIII).
Hoffmann-La Roche
Sponsor
Phase 3
Phase
NCT04158648,BO41423,2019-002179-32,2023-506610-52-00
Trial Identifier
Emicizumab
Treatments
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
Condition
Official Title
A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors
Eligibility Criteria
All
Gender
No
Healthy Volunteers
Inclusion Criteria
- Diagnosis of mild (FVIII level between >5% and <40%) or moderate (FVIII level between ≥1% and ≤5%) congenital Hemophilia A without FVIII inhibitors
- Weight ≥3 kilograms (kg)
- Need for prophylaxis based on investigator assessment
- A negative test for inhibitor (i.e., <0.6 Bethesda Units per milliliter [BU/mL]) within 8 weeks prior to enrollment
- No documented inhibitor (i.e., <0.6 BU/mL), FVIII half-life <6 hours, or FVIII recovery <66% in the last 5 years
- Documentation of the details of prophylactic or episodic FVIII treatment and of number of bleeding episodes for at least the last 24 weeks prior to enrollment
- Adequate hematologic, hepatic, and renal function
- For women of childbearing potential: agreement to remain abstinent or use contraception (as defined in the protocol) during the treatment period and for at least 24 weeks after the final dose of study drug
Exclusion Criteria
- Inherited or acquired bleeding disorder other than mild or moderate congenital hemophilia A
- History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the investigator's judgment
- Previous (within the last 12 months) or current treatment for thromboembolic disease or signs of thromboembolic disease
- Other conditions that may currently increase the risk of bleeding or thrombosis
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
- Planned surgery during the emicizumab loading dose phase (surgeries in participants on emicizumab from Week 5 onwards are allowed)
- Known HIV infection with CD4 counts <200 cells per microlitre (/μL)
- Concomitant disease, condition, significant abnormality on screening evaluation or laboratory tests, or treatment that could interfere with the conduct of the study, or that would in the opinion of the investigator, pose an additional unacceptable risk in administering study drug to the participant
- Receipt of any of the following: An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration with the exception of prior emicizumab prophylaxis; A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter; or Any other investigational drug currently being administered or planned to be administered
- 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)
For the latest version of this information please go to www.forpatients.roche.com