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

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ClinicalTrials.gov Identifier: NCT03622476
Recruitment Status : Recruiting
First Posted : August 9, 2018
Last Update Posted : August 14, 2018
Sponsor:
Information provided by (Responsible Party):
Runhui WU, 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.

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 155 participants
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 : June 30, 2018
Estimated Primary Completion Date : July 30, 2021
Estimated Study Completion Date : August 30, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

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




Primary Outcome Measures :
  1. Ultrasound evaluation [ Time Frame: 3 years ]
    the sonographer will use ultrasound to assess whether the patient's joint lesions become more severe than when they were enrolled.

  2. Imaging evaluation [ Time Frame: 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 :
  1. Annualized Bleeding Rate [ Time Frame: 3 years ]
    How many times for all types of bleeding

  2. Annualized Joint Bleeding Rate [ Time Frame: 3 years ]
    How many times for joint bleeding



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Ages Eligible for Study:   1 Year to 14 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03622476


Contacts
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Contact: chen zhen ping, Ph.D 15011221677 chenzhenping@outlook.com

Locations
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China, Beijing
Beijing Children's Hospital Recruiting
Beijing, Beijing, China, 100045
Contact: chen zhenping, Ph.D         
Sponsors and Collaborators
Beijing Children's Hospital

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Responsible Party: Runhui WU, Capital Medical Univercity, Beijing Children's Hospital
ClinicalTrials.gov Identifier: NCT03622476     History of Changes
Other Study ID Numbers: BCH-PK-20180630
First Posted: August 9, 2018    Key Record Dates
Last Update Posted: August 14, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Runhui WU, Beijing Children's Hospital:
Hemophilia A,pharmacokinetics
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn