Comparing low dose Emicizumab and Factor VIII for severe Hemophilia A
Comparison of Outcomes Between Low Dose Emicizumab and Extended Half-life Factor VIII With Pharmacokinetic-guided Prophylaxis in Clinically Severe Hemophilia A
This study is testing if a low dose of Emicizumab can work better than a low dose of Factor VIII to help people with severe Hemophilia A have fewer bleeding episodes and improve their quality of life.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 3 Years to 30 Years |
| Sex | Male |
| Sponsor | Chulalongkorn University Academic / other |
| Drugs / interventions | Emicizumab |
| Locations | 1 site (Bangkok) |
| Trial ID | NCT06155955 on ClinicalTrials.gov |
What this trial studies
This study aims to compare the outcomes of low dose Emicizumab with low dose prophylaxis using Factor VIII in patients with severe Hemophilia A. It will evaluate various metrics such as annualized bleeding rate (ABR), joint bleeding rates (AJBR), and health-related quality of life (HaemoQoL). Additionally, the study will investigate the pharmacokinetics and side effects associated with low dose Emicizumab treatment. The research is designed to provide insights into the effectiveness and safety of this alternative treatment approach.
Who should consider this trial
Good fit: Ideal candidates include individuals with severe or moderate Hemophilia A who have been on low dose Factor VIII prophylaxis for at least six months.
Not a fit: Patients with detectable FVIII inhibitors or other underlying diseases that affect joint health may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could offer a more effective and safer treatment option for patients with severe Hemophilia A.
How similar studies have performed: Other studies have shown promising results with Emicizumab, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Bangkok
- King Chulalongkorn Memorial hospital — Bangkok, Thailand (Recruiting)
Study contacts
- Principal investigator: Nuchanun Kessakorn — King Chulalongkorn Memorial Hospital
- Study coordinator: Nuchanun Kessakorn, Md
- Email: nuchanun.ks@gmail.com
- Phone: 662-5054639
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.