Pharmacokinetic (PK)Research on Chinese Children of Hemophilia

NCT03622476 | Unknown Phase 4

• 1 other identifier: BCH-PK-20180630
• Study Type: interventional
• Target: 155
• Locations: 1 country
• Sites: 1

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.

Conditions

Pharmacokinetics

Keywords

Hemophilia A，pharmacokinetics

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (2)

• Annualized Bleeding Rate

  3 years

• Annualized Joint Bleeding Rate

  3 years

Study Arms (1)

PK research

EXPERIMENTAL

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

Interventions

concentrated FVIII DRUG

Intervention if the assessment considers that the treatment is inadequate

PK research

Eligibility Criteria

• Age: 1 Year - 14 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• FⅧ\<1% ,
• years 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.

You may not qualify if:

• Combining other disease researchers believes that it is not suitable for enrollment.
• FVIII inhibitor was found.
• Refuse to participate in research

Sponsors & Collaborators

• Beijing Children's Hospital: lead

Study Sites (1)

Beijing Children's Hospital

Beijing, Beijing Municipality, 100045, China

RECRUITING

Central Study Contacts

chen zhen ping, Ph.D

CONTACT

15011221677; chenzhenping@outlook.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Capital Medical Univercity

Study Record Dates

• First Submitted: July 30, 2018
• First Posted: August 9, 2018
• Study Start: June 30, 2018
• Primary Completion: July 30, 2021
• Study Completion: August 30, 2021
• Last Updated: August 14, 2018

Record last verified: 2018-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)