Hemlibra in Mild Hemophilia A

Conditions

• Factor VIII Deficiency, Congenital

Eligibility Criteria

Sex

MALE

Age

5 Years - 45 Years

Healthy Volunteers

Not accepted

Interventions

• Emicizumab — DRUG
  bispecific monoclonal antibody binding to activated Factor IX and Factor X

Study Details

This is a single arm, phase 4, prospective, open-label, United States single-center study to determine the hemostatic characteristics of Hemlibra (emicizumab) as measured by coagulation laboratory parameters in the mild hemophilia A male patient population with endogenous altered FVIII (baseline FVIII activity of \>5% to 30%). The safety and hemostatic efficacy of Hemlibra (emicizumab) in this patient population will be investigated. Secondary outcomes will assess changes in joint health and quality of life in treated patients.

Key Dates

Start date

Jul 25, 2022

Status verified

Mar 2024

Primary completion

Jul 31, 2025

Completion

Jul 31, 2027

Study Design

Enrollment

20 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Single Arm
  Patients with mild hemophilia A (without inhibitors) will be treated with prophylactic emicizumab. The clinical hemostatic efficacy and safety will be assessed. Secondary outcomes will assess changes in quality of life and joint health in treated patients.

Primary Outcome Measure

Interaction of Hemlibra (emicizumab) binding with endogenous altered FVIII protein in an individual with mild hemophilia A and the combined effect on thrombin generation and hemostatic characteristics [ Time Frame: Before treatment, month 4, month 7, and month 13 ]

Central Contacts

• Amy D Shapiro, MD
  3178710000
  [email protected]
• Usha Sirimalle, MS
  3178710000
  [email protected]

Locations (1)

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