Pharmacokinetic (PK)Research on Chinese Children of Hemophilia (PK)

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

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.

Study Overview

• Status: Unknown
• Conditions: Pharmacokinetics
• Intervention / Treatment: Drug: concentrated FVIII

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.

• Study Type: Interventional
• Enrollment (Anticipated): 155
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100045
      • Recruiting
      • Beijing Children's Hospital
      • Contact: chen zhenping, Ph.D

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 14 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

EXPERIMENTAL: PK research; 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; Intervention if the assessment considers that the treatment is inadequate

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Beijing Children's Hospital

Study record dates

Study Major Dates

• Study Start (ACTUAL): June 30, 2018
• Primary Completion (ANTICIPATED): July 30, 2021
• Study Completion (ANTICIPATED): August 30, 2021

Study Registration Dates

• First Submitted: July 30, 2018
• First Submitted That Met QC Criteria: August 5, 2018
• First Posted (ACTUAL): August 9, 2018

Study Record Updates

• Last Update Posted (ACTUAL): August 14, 2018
• Last Update Submitted That Met QC Criteria: August 11, 2018
• Last Verified: August 1, 2018

More Information

Terms related to this study

• Keywords: Hemophilia A，pharmacokinetics
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: BCH-PK-20180630

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No