Pharmacokinetic (PK)Research on Chinese Children of Hemophilia

Sponsor

Beijing Children's Hospital (Other)

Overall Status

Unknown status

CT.gov ID

NCT03622476

Collaborator

(none)

155

Enrollment

Location

Arm

Anticipated Duration (Months)

4.1

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Pharmacokinetics	Drug: concentrated FVIII	Phase 4

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 Design

Study Type:

Interventional

Anticipated Enrollment :

155 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Jun 30, 2018

Anticipated Primary Completion Date :

Jul 30, 2021

Anticipated Study Completion Date :

Aug 30, 2021

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

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

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 14 Years

Sexes Eligible for Study:

Male

Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Beijing Children's Hospital	Beijing	Beijing	China	100045

Sponsors and Collaborators

Beijing Children's Hospital

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Runhui WU, Capital Medical Univercity, Beijing Children's Hospital

ClinicalTrials.gov Identifier:

NCT03622476

Other Study ID Numbers:

BCH-PK-20180630

First Posted:

Aug 9, 2018

Last Update Posted:

Aug 14, 2018

Last Verified:

Aug 1, 2018

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Runhui WU, Capital Medical Univercity, Beijing Children's Hospital

Hemophilia A，pharmacokinetics

Additional relevant MeSH terms:

Hemophilia A

Study Results

No Results Posted as of Aug 14, 2018