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Real-World Joint Outcomes After Selective Transition to Low-Dose Emicizumab in Pediatric Hemophilia A

NCT07439055 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2026-02-27

No results posted yet for this study

Summary

Recent studies have shown that pharmacokinetic (PK)-guided extended half-life (EHL) factor VIII can improve joint health in patients with hemophilia A. However, some patients experience suboptimal joint outcomes despite optimized PK-guided therapy. Low-dose emicizumab has emerged as a potential option for improving bleeding control and joint health in patients who do not respond adequately to PK-guided EHL factor VIII.

The objectives of the study is to compare clinical bleeding outcomes, joint health, cartilage biomarkers, and musculoskeletal ultrasound findings (HEAD-US) in hemophilia A patients in Thailand during PK-guided EHL factor VIII therapy and after switching to low-dose emicizumab.

Eighteen patients with hemophilia A from King Chulalongkorn Memorial Hospital, Thailand, aged 8-28 years, who were receiving PK-guided EHL factor VIII were enrolled. Patients with a Hemophilia Joint Health Score (HJHS) ≥ 12 were switched to low-dose emicizumab. A loading dose of 2 mcg/kg was administered, followed by dosing every 2 weeks during the first month and every 4 weeks thereafter. Clinical bleeding, annual bleeding rate (ABR), annual joint bleeding rate (AJBR), HJHS, cartilage biomarkers, and musculoskeletal ultrasound findings (HEAD-US) were evaluated every 4 months for up to 8 months after switching.

Conditions

  • Hemophilia A Patient

Interventions

DRUG

Switching to low dose emicizumab

All Hemophilia A patients in the study had received treatment with PK-guided EHL factor VIII. The patients who had HJHS more than 12 were switched to treatment with low dose emicizumab

Sponsors & Collaborators

  • Chulalongkorn University

    lead OTHER

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
3 Years
Max Age
30 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-01
Primary Completion
2026-03-01
Completion
2026-04-01

Countries

  • Thailand

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07439055 on ClinicalTrials.gov