PK Driven Prophylaxis for Hemophilia A

Not Applicable

Terminated

• Conditions: Hemophilia A
• Interventions: Device: MyPKFiT

• Registration Number: NCT02634424
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Brief Summary

The purpose of this study is to evaluate the implementation of pharmacokinetic (PK) driven prophylaxis in current clinical practice

Detailed Description

Starting from 3 pharmacokinetic (PK) points, data are entered in the PK calculator device, and treatment dose is adjusted following the results of PK values obtained and clinical bleeding data

Study Timeline

• Study Start: 2015-11-25
• Study First Submitted: 2015-12-08
• Study First Submitted QC: 2015-12-15
• Study First Posted: 2015-12-18
• Primary Completion: 2016-05-12
• Study Completion: 2016-05-12
• Status Verified: 2016-08
• Last Update Submitted: 2020-03-12
• Last Update Posted: 2020-03-16

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 12

Inclusion Criteria

• Male Patient
• Severe or moderately severe haemophilia A (FVIII = 2 %), preventively treated (processing) by ADVATE for at least 6 months,
• At last 6 years old,
• An informed consent must be signed by the patient or his legal representative for the patients minor.
• Affiliated to a national insurance scheme

Exclusion Criteria

• Previous or actual treatment with FVIII inhibitors > 0,6 UB at the selection
• Induction of immune tolerance
• Planned orthopedic surgery for the 18 next months
• Any other haemostatic pathology
• Any treatment interacting on the haemostasis
• Patient under guardianship
• Patient participating in another biomedical research

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
MyPKFiT	MyPKFiT	Personalized prophylaxis : Treatment is adjustment according to PK modeling

Primary Outcome Measures

Name	Time	Method
Joint Annual Bleeding Rate	during 6 months before (retrospectively) and up to 12 months after using of myPKFiT	Joint Annual Bleeding Rate assessed by usual patient book

Secondary Outcome Measures

Name	Time	Method
Adherence to treatment estimated as the quantity of Facteur VIII consumed reported to the prescribed quantity.	up to 6 and 12 months	Quantity of Facteur VIII consumed will be assessed by usual patient book
Consumption of Factor VIII estimated by the Quantity of Facteur VIII consumed by thr patient.	during 6 months before (retrospectively) and up to 12 months after using of myPKFiT	Consumption of Factor VIII assessed by usual patient book
Hemorrhagic Risk Sport	up to 6 and 12 months	Hemorrhagic Risk Sport assessed by the scale "Hemophilia and sport"
Evolution between the annualized incidence of intercurrent events appeared or worsened under each treatment period (with and without myPKFit), globally, by System Organ ( SO) and Preferred Term ( PT) of the code MedDRA	during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Total annual bleeding rate	during 6 months before (retrospectively) and up to 12 months after using of myPKFiT	Total Annual Bleeding Rate assessed by usual patient book
Quality of life	up to 12 months	Quality of life assessed by QoL-A-Haemo questionnaire for Adult and Haemo-QoL for children aged 6-17 years
Joint function	up to 12 months	Joint function assessed thanks to score of joint state "Haemophilia Joint Health Score" (HJHS)

Trial Locations

Locations (1)

AP-HP, Bicêtre Hospital; 🇫🇷 Le Kremlin Bicêtre, France