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

Primary Purpose

Pharmacokinetics

Status
Unknown status
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
concentrated FVIII
Sponsored by
Beijing Children's Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pharmacokinetics focused on measuring Hemophilia A，pharmacokinetics

Eligibility Criteria

1 Year - 14 Years (Child)MaleDoes not accept 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

Sites / Locations

  • Beijing Children's HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PK research

Arm 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.

Outcomes

Primary Outcome Measures

Ultrasound evaluation
the sonographer will use ultrasound to assess whether the patient's joint lesions become more severe than when they were enrolled.
Imaging evaluation
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

Annualized Bleeding Rate
How many times for all types of bleeding
Annualized Joint Bleeding Rate
How many times for joint bleeding

Full Information

First Posted
July 30, 2018
Last Updated
August 11, 2018
Sponsor
Beijing Children's Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT03622476
Brief Title
Pharmacokinetic (PK)Research on Chinese Children of Hemophilia
Acronym
PK
Official Title
Capital Characteristic Application: Pharmacokinetic（PK） Research on Chinese Children of Hemophilia
Study Type
Interventional

2. Study Status

Record Verification Date
August 2018
Overall Recruitment Status
Unknown status
Study Start Date
June 30, 2018 (Actual)
Primary Completion Date
July 30, 2021 (Anticipated)
Study Completion Date
August 30, 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Beijing Children's Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pharmacokinetics
Keywords
Hemophilia A，pharmacokinetics

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
155 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
PK research
Arm Type
Experimental
Arm 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.
Intervention Type
Drug
Intervention Name(s)
concentrated FVIII
Intervention Description
Intervention if the assessment considers that the treatment is inadequate
Primary Outcome Measure Information:
Title
Ultrasound evaluation
Description
the sonographer will use ultrasound to assess whether the patient's joint lesions become more severe than when they were enrolled.
Time Frame
3 years
Title
Imaging evaluation
Description
the imaging specialist will use MRI to assess whether the patient's joint lesions are more severe than when they were enrolled.
Time Frame
3 years
Secondary Outcome Measure Information:
Title
Annualized Bleeding Rate
Description
How many times for all types of bleeding
Time Frame
3 years
Title
Annualized Joint Bleeding Rate
Description
How many times for joint bleeding
Time Frame
3 years

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
chen zhen ping, Ph.D
Phone
15011221677
Email
chenzhenping@outlook.com
Facility Information:
Facility Name
Beijing Children's Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100045
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
chen zhenping, Ph.D

12. IPD Sharing Statement

Plan to Share IPD
No

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Pharmacokinetic (PK)Research on Chinese Children of Hemophilia

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