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

Sponsor
Chulalongkorn University (Other)
Overall Status
Recruiting
CT.gov ID
NCT06155955
Collaborator
(none)
15
Enrollment
1
Location
2
Arms
12
Anticipated Duration (Months)
1.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

  • To study pharmacokinetic, side effect of low dose Emicizumab

Condition or Disease Intervention/Treatment Phase
  • Drug: "Emicizumab", "HEMLIBRA®"
 Phase 2/Phase 3

Detailed 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

Study Design

Study Type:
Interventional
Anticipated Enrollment :
15 participants
Allocation:
Non-Randomized
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Comparison of Outcomes Between Low Dose Emicizumab and Extended Half-life Factor VIII With Pharmacokinetic-guided Prophylaxis in Clinically Severe Hemophilia A
Actual Study Start Date :
Jun 22, 2023
Anticipated Primary Completion Date :
Feb 21, 2024
Anticipated Study Completion Date :
Jun 21, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: low dose emicizumab

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

Drug: "Emicizumab", "HEMLIBRA®"
low dose
Other Names:
  • emicizumab-kxwh

    		•
    No Intervention: low dose factor VIII prophylaxis

    factor VIII prophylaxis

    Outcome Measures

    Primary Outcome Measures

    1. spontaneous and traumatic bleeding rate [6 months after start low dose emicizumab as secondary prophylaxis treatment]

      Using "Annualized bleeding rate" (ABR)

    2. treated spontaneous bleeds, treated joint bleeds [6 months after start low dose emicizumab as secondary prophylaxis treatment]

      Using "Annualized joint bleeding rate" (AJBR)

    3. The function of the knee, elbow, and ankle joints [6 months after start low dose emicizumab as secondary prophylaxis treatment]

      Using "Hemophilia Joint Health Score" (HJHS)

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    3 Years to 30 Years
    Sexes Eligible for Study:
    Male
    Accepts Healthy Volunteers:
    Yes
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 King Chulalongkorn Memorial hospital Bangkok Thailand 10330

    Sponsors and Collaborators

    • Chulalongkorn University

    Investigators

    • Principal Investigator: NUCHANUN KESSAKORN, King Chulalongkorn Memorial Hospital

    Study Documents (Full-Text)

    More Information

    Publications

    None provided.
    Responsible Party:
    Nuchanun Kessakorn, MD, MD, Chulalongkorn University
    ClinicalTrials.gov Identifier:
    NCT06155955
    Other Study ID Numbers:
    • KingChulalongkorn
    First Posted:
    Dec 5, 2023
    Last Update Posted:
    Dec 5, 2023
    Last Verified:
    Nov 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Nuchanun Kessakorn, MD, MD, Chulalongkorn University
    emicizumab
    hemophilia A
    Additional relevant MeSH terms:
    Hemophilia A
    Antibodies, Bispecific

    Study Results

    No Results Posted as of Dec 5, 2023