Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A

Chulalongkorn University

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Joint Bleed; Severe Hemophilia A Without Inhibitor

Treatments: Drug: "Emicizumab", "HEMLIBRA®"

Study type: Interventional

Funder types: Other

Identifiers

NCT06155955

KingChulalongkorn

Details and patient eligibility

About

• To outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration
• To study pharmacokinetic, side effect of low dose Emicizumab

Full description

• To compare outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration using ABR, AJBR and HJHS, HaemoQoL
• To study pharmacokinetic, side effect and effectiveness of low dose Emicizumab

Enrollment: 15 estimated patients

Sex: Male

Ages: 3 to 30 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Severe or moderate Hemophilia A with baseline FVIII:C of <3 IU/dl or severe bleeding phenotype
• Receiving low dose FVIII prophylaxis for at least 6 months

Exclusion criteria

• Detectable FVIII inhibitor at screening
• Detectable FVIII inhibitor at screening Having other underlying diseases: Juvenile rheumatoid arthritis, metabolic bone diseases, or other conditions mimicking or causing joint diseases

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 2 patient groups

low dose emicizumab

Experimental group

Description:

2 mg per kilograms per dose in first month, loading every 2 weeks then every 4 weeks

Treatment:

Drug: "Emicizumab", "HEMLIBRA®"

low dose factor VIII prophylaxis

No Intervention group

Description:

factor VIII prophylaxis