---
title: Real-World Joint Outcomes After Selective Transition to Low-Dose Emicizumab in Pediatric Hemophilia A
nct_id: NCT07439055
overall_status: ACTIVE_NOT_RECRUITING
phase: PHASE2, PHASE3
sponsor: Chulalongkorn University
study_type: INTERVENTIONAL
primary_condition: Hemophilia A Patient
countries: Thailand
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT07439055.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT07439055"
ct_last_update_post_date: 2026-02-27
last_seen_at: "2026-05-12T06:44:40.885Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Real-World Joint Outcomes After Selective Transition to Low-Dose Emicizumab in Pediatric Hemophilia A

**Official 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

**NCT ID:** [NCT07439055](https://clinicaltrials.gov/study/NCT07439055)

## Key Facts

- **Status:** ACTIVE_NOT_RECRUITING
- **Phase:** PHASE2, PHASE3
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 18
- **Lead Sponsor:** Chulalongkorn University
- **Conditions:** Hemophilia A Patient
- **Start Date:** 2025-06-01
- **Completion Date:** 2026-04-01
- **CT.gov Last Update:** 2026-02-27

## Brief 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.

## Eligibility

- **Minimum age:** 3 Years
- **Maximum age:** 30 Years
- **Sex:** MALE
- **Healthy Volunteers:** No

```
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
```

## Arms

- **Hemophilia A patients with HJHS more than 12** (EXPERIMENTAL) — 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
- **Hemophilia A patients with HJHS less than 12** (NO_INTERVENTION) — Hemophilia A patients whose HJHS was less than 12 continue the same PK-guided EHL Factor VIII

## Interventions

- **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

- **Annual bleeding rate** _(time frame: 8 months)_ — Annual bleeding rate report as times per year
- **Annual joint bleeding rate** _(time frame: 8 months)_ — Annual joint bleeding rate report as times per year
- **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.
- **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.

## Locations (1)

- Chulalongkorn University, Bangkok, Thailand

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.chulalongkorn university|bangkok||thailand` — added _(2026-05-12)_

---

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