Pharmacokinetic (PK)Research on Chinese Children of Hemophilia

Study Overview
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Brief Summary
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The study start on June 30, 2018. The Severe(F Ⅷ<1%) hemophilia A children without F Ⅷ inhibitor combining were recruited to Test the concentration of the drug in the blood to provide better treatment.

Detailed Description
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Through the integration of two technologies, pharmacokinetics and comprehensive evaluation system, we can improve the individualized prevention and treatment of hemophilia in Chinese children and achieve precise customization of treatment plans. Rational use of medical resources, but also fully achieve the prevention and treatment goals.

Official Title
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Capital Characteristic Application: Pharmacokinetic（PK） Research on Chinese Children of Hemophilia

Conditions
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Pharmacokinetics

Intervention / Treatment
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* Drug: concentrated FVIII


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: FⅧ<1% , 1-14years old, There have been at least one history of bleeding in any joint of the knee, elbow, or ankle. A blood product containing FVIII is applied. There were no inhibitors at the time of enrollment, and there was no inhibitor-positive history and family history. Clinical visits are available on a regular basis and data is available, and preventive treatment is available prior to enrollment. The child was enrolled in the group and the guardian agreed. Exclusion Criteria: Combining other disease researchers believes that it is not suitable for enrollment. FVIII inhibitor was found. Refuse to participate in research

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 14 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PK research<br>Interventional studies were performed in the 1-7 year old subgroup, and the children received a comprehensive assessment and PK test every 3 months. After the 72-hour washout period, 50 IU/kg of concentrated FVIII was administered in a single dose, and blood was taken within half an hour before the infusion and 1 h, 9 h, 24 h, and 48 h after the infusion, and the samples were centrifuged. If the assessment considers that the treatment is inadequate, then the valley concentration target is upgraded. | Drug: concentrated FVIII<br>* Intervention if the assessment considers that the treatment is inadequate<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ultrasound evaluation | the sonographer will use ultrasound to assess whether the patient's joint lesions become more severe than when they were enrolled. | 3 years | 
| Imaging evaluation | the imaging specialist will use MRI to assess whether the patient's joint lesions are more severe than when they were enrolled. | 3 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annualized Bleeding Rate | How many times for all types of bleeding | 3 years | 
| Annualized Joint Bleeding Rate | How many times for joint bleeding | 3 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemophilia A，pharmacokinetics