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

March 4, 2024 updated by: Nuchanun Kessakorn, MD, Chulalongkorn University

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

15

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Thailand
      • Bangkok, Thailand, 10330
        • Recruiting
        • King Chulalongkorn Memorial Hospital
        • Contact:
        • Contact:
        • Principal Investigator:
          • NUCHANUN KESSAKORN, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

Yes

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chulalongkorn University

Investigators

  • Principal Investigator: NUCHANUN KESSAKORN, King Chulalongkorn Memorial Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 22, 2023

Primary Completion (Estimated)

March 21, 2024

Study Completion (Estimated)

June 21, 2024

Study Registration Dates

First Submitted

November 11, 2023

First Submitted That Met QC Criteria

November 25, 2023

First Posted (Actual)

December 5, 2023

Study Record Updates

Last Update Posted (Estimated)

March 5, 2024

Last Update Submitted That Met QC Criteria

March 4, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KingChulalongkorn

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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