Pharmacokinetic (PK)Research on Chinese Children of Hemophilia
- Conditions
- Pharmacokinetics
- Interventions
- Drug: concentrated FVIII
- Registration Number
- NCT03622476
- Lead Sponsor
- Beijing Children's Hospital
- Brief Summary
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
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.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- Male
- Target Recruitment
- 155
- 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.
- Combining other disease researchers believes that it is not suitable for enrollment.
- FVIII inhibitor was found.
- Refuse to participate in research
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description PK research concentrated FVIII 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.
- Primary Outcome Measures
Name Time Method Ultrasound evaluation 3 years the sonographer will use ultrasound to assess whether the patient's joint lesions become more severe than when they were enrolled.
Imaging evaluation 3 years the imaging specialist will use MRI to assess whether the patient's joint lesions are more severe than when they were enrolled.
- Secondary Outcome Measures
Name Time Method Annualized Bleeding Rate 3 years How many times for all types of bleeding
Annualized Joint Bleeding Rate 3 years How many times for joint bleeding
Trial Locations
- Locations (1)
Beijing Children's Hospital
🇨🇳Beijing, Beijing, China