PK Driven Prophylaxis for Hemophilia A (OBTC)

Interest of Preventive Personalized Treatment in Hemophilia A

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

Study Overview

• Status: Terminated
• Conditions: Hemophilia A
• Intervention / Treatment: Device: MyPKFiT

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 Type: Interventional
• Enrollment (Actual): 12
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • Le Kremlin Bicêtre, France, 94275
    • AP-HP, Bicêtre Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: SUPPORTIVE_CARE
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: MyPKFiT; Personalized prophylaxis : Treatment is adjustment according to PK modeling	Device: MyPKFiT; Adjustment of the treatment dose using a decision support software according to the PK values; Other Names: Personalized prophylaxis by PK modeling

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total annual bleeding rate	Total Annual Bleeding Rate assessed by usual patient book	during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Quality of life	Quality of life assessed by QoL-A-Haemo questionnaire for Adult and Haemo-QoL for children aged 6-17 years	up to 12 months
Joint function	Joint function assessed thanks to score of joint state "Haemophilia Joint Health Score" (HJHS)	up to 12 months
Adherence to treatment estimated as the quantity of Facteur VIII consumed reported to the prescribed quantity.	Quantity of Facteur VIII consumed will be assessed by usual patient book	up to 6 and 12 months
Consumption of Factor VIII estimated by the Quantity of Facteur VIII consumed by thr patient.	Consumption of Factor VIII assessed by usual patient book	during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Hemorrhagic Risk Sport	Hemorrhagic Risk Sport assessed by the scale "Hemophilia and sport"	up to 6 and 12 months
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

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborators: Baxter Healthcare Corporation
• Investigators: Principal Investigator: Thierry LAMBERT, MD, AP-HP, Bicêtre Hospital

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 25, 2015
• Primary Completion (ACTUAL): May 12, 2016
• Study Completion (ACTUAL): May 12, 2016

Study Registration Dates

• First Submitted: December 8, 2015
• First Submitted That Met QC Criteria: December 15, 2015
• First Posted (ESTIMATE): December 18, 2015

Study Record Updates

• Last Update Posted (ACTUAL): March 16, 2020
• Last Update Submitted That Met QC Criteria: March 12, 2020
• Last Verified: August 1, 2016

More Information

Terms related to this study

• Keywords: Pharmacokinetic; Bayesian; Hemophilia A; Prophylaxy
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: P140701