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NCT Number: NCT07439055

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

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.

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Key information

Conditions

Hemophilia A Patient Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hemarthrosis Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhage Hemorrhagic Disorders Joint Diseases Musculoskeletal Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

3 year–30 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Chulalongkorn University

Bangkok, 10330, Thailand

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • hemophilia A patients aged 3-30 years who received treatment with PK-guided EHL Factor VIII for more than 8 months

Exclusion criteria

  • hemophilia A patients with presence of factor VIII inhibitors

Treatment and study plan

Switching to low dose emicizumab

Drug

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

Primary outcomes

  1. Annual bleeding rate

    Time frame: 8 months

    Annual bleeding rate report as times per year

  2. Annual joint bleeding rate

    Time frame: 8 months

    Annual joint bleeding rate report as times per year

  3. Hemophilia Joint Health Score (HJHS)

    Time frame: 8 months

    HJHS is the physical examination assessment tool for hemophilia patients evaluated most commonly 6 joints affected by bleeding (both elbows, both knees and both ankles). The scores range from 0 (minimum) to 125 (maximum). The lowest score means the best joint health.

  4. The quality of life assessment instrument for children and adolescents with haemophilia (Hae-QoL)

    Time frame: 8 months

    The assessment tools evaluate physical, psychological, family, social aspect of Hemophilia patients. There are 35 questions. Each questions has multiple choices (never, seldom, somtimes, often, always) represent score 1 (never) to 5 (always). The total raw score range from 35 to 175 and then must be transform to "transformed score" ranged from 0 to 100. 0 is the best and 100 is the worst score.

Sponsors and collaborators

Lead sponsor

Chulalongkorn University

Other

Registry information

Official study title

The Study of Clinical Outcomes, Cartilage Biomarkers and Hemophilic Arthropathy (HEAD-US) in Hemophilia A Patients : Low Dose Emicizumab vs. Pharmacokinetic-Guided Extended Half-Life FVIII Concentrates

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Feb 27, 2026
Registry last updated
Feb 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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