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 1

• Conditions: Hemophilia A; MedDRA version: 20.0Level: LLTClassification code: 10053753Term: Hemophilia A without inhibitors Class: 10010331; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

• Registration Number: CTIS2023-505964-13-00
• Lead Sponsor: F. Hoffmann-La Roche AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-02-01
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 65

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: Interventional clinical trial of medicinal product
• Study Design: Not specified