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

Phase 3RecruitingNCT06155955

Chulalongkorn University15 enrolled

Overview

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

* 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

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Severe Hemophilia A Without Inhibitor Joint Bleed

Interventions

"Emicizumab", "HEMLIBRA®"DRUG

low dose

Eligibility

Sex: MALEMin age: 3 YearsMax age: 30 YearsHealthy volunteers:

Medical Language ↔ Plain English

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

Locations (1)

King Chulalongkorn Memorial hospital

Bangkok, Thailand

RECRUITING

Outcomes

Primary Outcomes

spontaneous and traumatic bleeding rate

Using "Annualized bleeding rate" (ABR)

Time frame: 6 months after start low dose emicizumab as secondary prophylaxis treatment

treated spontaneous bleeds, treated joint bleeds

Using "Annualized joint bleeding rate" (AJBR)

Time frame: 6 months after start low dose emicizumab as secondary prophylaxis treatment

The function of the knee, elbow, and ankle joints

Using "Hemophilia Joint Health Score" (HJHS)

Time frame: 6 months after start low dose emicizumab as secondary prophylaxis treatment

Central Contacts

NUCHANUN KESSAKORN, MD

CONTACT

662-5054639 nuchanun.ks@gmail.com

Data from ClinicalTrials.gov

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