Pharmacokinetic (PK)Research on Chinese Children of Hemophilia

Capital Medical University

Status and phase: Unknown; Phase 4

Conditions: Pharmacokinetics

Treatments: Drug: concentrated FVIII

Study type: Interventional

Funder types: Other

Identifiers

NCT03622476

BCH-PK-20180630

Details and patient eligibility

About

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.

Full 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.

Enrollment: 155 estimated patients

Sex: Male

Ages: 1 to 14 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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155 participants in 1 patient group

PK research

Experimental group

Description:

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.

Treatment:

Drug: concentrated FVIII