A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of emicizumab in patients with mild or moderate hemophilia A without FVIII inhibitors

Phase 1

• Conditions: Mild or moderate hemophilia A without FVIII inhibitors; MedDRA version: 20.0Level: LLTClassification code: 10060612Term: Hemophilia A Class: 10010331; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

• Registration Number: CTIS2023-506610-52-00
• Lead Sponsor: F. Hoffmann-La Roche AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-06-13
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 22

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 kg, Need for prophylaxis based on investigator assessment, A negative test for inhibitor (i.e., < 0.6 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

Exclusion Criteria

Inherited or acquired bleeding disorder other than mild (FVIII level between > 5% and < 40%) or moderate (FVIII level between >= 1% and <= 5%) 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, 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 patients on emicizumab from Week 5 onwards are allowed, Known HIV infection with CD4 counts < 200 cells/micro

Study & Design

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