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)

Phase 3

Active, not recruiting

• Conditions: Hemophilia A

• Registration Number: 2023-505964-13-00
• Lead Sponsor: F. Hoffmann-La Roche AG

Brief Summary

To evaluate the efficacy, safety, pharmacokinetic (PK) profile, pharmacodynamics (PD) parameters and immune response to treatment of emicizumab

Detailed Description

Not available

Study Timeline

• Study First Posted: 2024-03-06
• Last Update Posted: 2025-05-12

Recruitment & Eligibility

• Status: Ongoing, recruitment ended
• Sex: Male
• Target Recruitment: 31

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

• Study Type: Not specified
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
1. Number of treated bleeds over time		1. Number of treated bleeds over time
2. Number of all bleeds over time		2. Number of all bleeds over time
3. Number of treated spontaneous bleeds over time		3. Number of treated spontaneous bleeds over time
4. Number of treated joint bleeds over time		4. Number of treated joint bleeds over time
5. Joint health, as assessed through use of the Hemophilia Joint Health Score (HJHS) and magnetic resonance imaging (MRI) score of specific joints at specified timepoints only during the 7-year long-term follow-up (LTFU) period		5. Joint health, as assessed through use of the Hemophilia Joint Health Score (HJHS) and magnetic resonance imaging (MRI) score of specific joints at specified timepoints only during the 7-year long-term follow-up (LTFU) period
6. Incidence and severity of adverse events, with severity determined according to WHO Toxicity Grading Scale		6. Incidence and severity of adverse events, with severity determined according to WHO Toxicity Grading Scale
7. Incidence of thromboembolic events and thrombotic microangiopathy (TMA)		7. Incidence of thromboembolic events and thrombotic microangiopathy (TMA)
8. Change from baseline in physical examination findings		8. Change from baseline in physical examination findings
9. Change from baseline in vital signs		9. Change from baseline in vital signs
10. Incidence of laboratory abnormalities		10. Incidence of laboratory abnormalities
11. Incidence and severity of injection-site reactions		11. Incidence and severity of injection-site reactions
12. Incidence of adverse events leading to study drug discontinuation		12. Incidence of adverse events leading to study drug discontinuation
13. Incidence of severe hypersensitivity, anaphylaxis, and anaphylactoid events		13. Incidence of severe hypersensitivity, anaphylaxis, and anaphylactoid events
14. Plasma trough concentrations (Ctrough) of emicizumab prior to study drug administration		14. Plasma trough concentrations (Ctrough) of emicizumab prior to study drug administration
15. Effect of emicizumab on PD parameters, including aPTT, thrombin generation (TG), and reported FVIII activity, as well as FIX antigen and FX antigen (emicizumab substrates) levels prior to study drug		15. Effect of emicizumab on PD parameters, including aPTT, thrombin generation (TG), and reported FVIII activity, as well as FIX antigen and FX antigen (emicizumab substrates) levels prior to study drug

Secondary Outcome Measures

Name	Time	Method
Not applicable		Not applicable

Trial Locations

Locations (12)

Medical University Of Vienna; 🇦🇹 Vienna, Austria

Careggi University Hospital; 🇮🇹 Florence, Italy

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico; 🇮🇹 Milan, Italy

L’Azienda Ospedaliera Di Rilievo Nazionale Santobono-Pausilipon; 🇮🇹 Naples, Italy

Azienda Ospedaliero Universitaria Parma; 🇮🇹 Parma, Italy

University Hospital Virgen Del Rocio S.L.; 🇪🇸 Sevilla, Spain

Sant Joan De Deu Barcelona Hospital; 🇪🇸 Esplugues De Llobregat, Spain

Hospital Universitario La Paz; 🇪🇸 Madrid, Spain

Universitaetsklinikum Bonn AöR; 🇩🇪 Bonn, Germany

Hopital Necker Enfants Malades; 🇫🇷 Paris, France

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Medical University Of Vienna

🇦🇹Vienna, Austria

Christoph Male

Site contact

+4314040032310

kikli@meduniwien.ac.at