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

Severe Hemophilia A Without Inhibitor Clinical Trial

Official title:

Comparison of Outcomes Between Low Dose Emicizumab and Extended Half-life Factor VIII With Pharmacokinetic-guided Prophylaxis in Clinically Severe Hemophilia A

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06155955
• Other study ID #: KingChulalongkorn
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: June 22, 2023
• Est. completion date: June 21, 2024

Study information

• Verified date: March 2024
• Source: Chulalongkorn University
• Contact: NUCHANUN KESSAKORN, MD
• Phone: 662-5054639
• Email: nuchanun.ks[@]gmail.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

• To outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration

• To study pharmacokinetic, side effect of low dose Emicizumab

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 15
• Est. completion date: June 21, 2024
• Est. primary completion date: March 21, 2024
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Male
• Age group: 3 Years to 30 Years

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A
• Joint Bleed

Intervention

Drug:
• "Emicizumab", "HEMLIBRA®"
low dose

Locations

Country	Name	City	State
Thailand	King Chulalongkorn Memorial hospital	Bangkok

Sponsors (1)

Lead Sponsor	Collaborator
Chulalongkorn University

Country where clinical trial is conducted

Thailand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	spontaneous and traumatic bleeding rate	Using "Annualized bleeding rate" (ABR)	6 months after start low dose emicizumab as secondary prophylaxis treatment
Primary	treated spontaneous bleeds, treated joint bleeds	Using "Annualized joint bleeding rate" (AJBR)	6 months after start low dose emicizumab as secondary prophylaxis treatment
Primary	The function of the knee, elbow, and ankle joints	Using "Hemophilia Joint Health Score" (HJHS)	6 months after start low dose emicizumab as secondary prophylaxis treatment