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A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors (HAVEN 7)
EU CTIS ID: 2023-505964-13-00
What this study is testing
To evaluate the efficacy, safety, pharmacokinetic (PK) profile, pharmacodynamics (PD) parameters and immune response to treatment of emicizumab
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. No history of documented FVIII inhibitor (i.e., < 0.6 BU/mL), FVIII drug-elimination half-life < 6 hours, or FVIII recovery < 66%
- 2. Mandatory receipt of vitamin K prophylaxis according to local standard practice
- 3. Diagnosis of severe congenital hemophilia A (intrinsic FVIII level < 1%)
- 4. A negative test for FVIII inhibitor (i.e., < 0.6 Bethesda units [BU]/mL) locally assessed during the 2-week screening period for all patients
- 5. Previously untreated patients (PUPs) or minimally treated patient (MTPs) (i.e., up to 5 days of exposure with hemophilia-related treatments, such as plasma-derived FVIII, recombinant FVIII, fresh frozen plasma, cryoprecipitate, or whole blood products)
- 6. Documentation of the details of the hemophilia-related treatments received since birth and documentation of the details of the bleeding episodes since birth
You likely can't join if
- 1. Inherited or acquired bleeding disorder other than severe hemophilia A
- 2. Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study
- 3. Current active severe bleed, such as intracranial hemorrhage (ICH)
- 4. History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
- 5. Patients who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA, such as thrombotic thrombocytopenic purpura, atypical hemolytic uremic syndrome) in the investigator's judgment
- 6. Previous or current treatment for thromboembolic disease or signs of thromboembolic disease
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Brazil
- Canada
- Israel
- Australia
- United Kingdom
- Turkey
- South Africa
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Brazil; Canada; Israel; Australia; United Kingdom and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.