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PK Driven Prophylaxis for Hemophilia A (OBTC)

Primary Purpose

Hemophilia A

Status
Terminated
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
MyPKFiT
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Hemophilia A focused on measuring Hemophilia A, Prophylaxy, Pharmacokinetic, Bayesian

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • AP-HP, Bicêtre Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

MyPKFiT

Arm Description

Personalized prophylaxis : Treatment is adjustment according to PK modeling

Outcomes

Primary Outcome Measures

Joint Annual Bleeding Rate
Joint Annual Bleeding Rate assessed by usual patient book

Secondary Outcome Measures

Total annual bleeding rate
Total Annual Bleeding Rate assessed by usual patient book
Quality of life
Quality of life assessed by QoL-A-Haemo questionnaire for Adult and Haemo-QoL for children aged 6-17 years
Joint function
Joint function assessed thanks to score of joint state "Haemophilia Joint Health Score" (HJHS)
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
Consumption of Factor VIII estimated by the Quantity of Facteur VIII consumed by thr patient.
Consumption of Factor VIII assessed by usual patient book
Hemorrhagic Risk Sport
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

Full Information

First Posted
December 8, 2015
Last Updated
March 12, 2020
Sponsor
Assistance Publique - Hôpitaux de Paris
Collaborators
Baxter Healthcare Corporation
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1. Study Identification

Unique Protocol Identification Number
NCT02634424
Brief Title
PK Driven Prophylaxis for Hemophilia A
Acronym
OBTC
Official Title
Interest of Preventive Personalized Treatment in Hemophilia A
Study Type
Interventional

2. Study Status

Record Verification Date
August 2016
Overall Recruitment Status
Terminated
Why Stopped
lack of inclusion and prophylaxy plateform being free to use for everyone in septembre 2016
Study Start Date
November 25, 2015 (Actual)
Primary Completion Date
May 12, 2016 (Actual)
Study Completion Date
May 12, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris
Collaborators
Baxter Healthcare Corporation

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
Keywords
Hemophilia A, Prophylaxy, Pharmacokinetic, Bayesian

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
MyPKFiT
Arm Type
Experimental
Arm Description
Personalized prophylaxis : Treatment is adjustment according to PK modeling
Intervention Type
Device
Intervention Name(s)
MyPKFiT
Other Intervention Name(s)
Personalized prophylaxis by PK modeling
Intervention Description
Adjustment of the treatment dose using a decision support software according to the PK values
Primary Outcome Measure Information:
Title
Joint Annual Bleeding Rate
Description
Joint Annual Bleeding Rate assessed by usual patient book
Time Frame
during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Secondary Outcome Measure Information:
Title
Total annual bleeding rate
Description
Total Annual Bleeding Rate assessed by usual patient book
Time Frame
during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Title
Quality of life
Description
Quality of life assessed by QoL-A-Haemo questionnaire for Adult and Haemo-QoL for children aged 6-17 years
Time Frame
up to 12 months
Title
Joint function
Description
Joint function assessed thanks to score of joint state "Haemophilia Joint Health Score" (HJHS)
Time Frame
up to 12 months
Title
Adherence to treatment estimated as the quantity of Facteur VIII consumed reported to the prescribed quantity.
Description
Quantity of Facteur VIII consumed will be assessed by usual patient book
Time Frame
up to 6 and 12 months
Title
Consumption of Factor VIII estimated by the Quantity of Facteur VIII consumed by thr patient.
Description
Consumption of Factor VIII assessed by usual patient book
Time Frame
during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Title
Hemorrhagic Risk Sport
Description
Hemorrhagic Risk Sport assessed by the scale "Hemophilia and sport"
Time Frame
up to 6 and 12 months
Title
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
Time Frame
during 6 months before (retrospectively) and up to 12 months after using of myPKFiT

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Thierry LAMBERT, MD
Organizational Affiliation
AP-HP, Bicêtre Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
AP-HP, Bicêtre Hospital
City
Le Kremlin Bicêtre
ZIP/Postal Code
94275
Country
France

12. IPD Sharing Statement

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PK Driven Prophylaxis for Hemophilia A

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