PK Driven Prophylaxis for Hemophilia A

Hemophilia A Clinical Trial

— OBTC  

Official title:

Interest of Preventive Personalized Treatment in Hemophilia A

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02634424
• Other study ID #: P140701
• Status: Terminated
• Phase: N/A
• Start date: November 25, 2015
• Est. completion date: May 12, 2016

Study information

• Verified date: August 2016
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 12
• Est. completion date: May 12, 2016
• Est. primary completion date: May 12, 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Device:
• MyPKFiT
Adjustment of the treatment dose using a decision support software according to the PK values

Locations

Country	Name	City	State
France	AP-HP, Bicêtre Hospital	Le Kremlin Bicêtre

Sponsors (2)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	Baxter Healthcare Corporation

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	Joint function	Joint function assessed thanks to score of joint state "Haemophilia Joint Health Score" (HJHS)	up to 12 months
Secondary	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
Secondary	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
Secondary	Hemorrhagic Risk Sport	Hemorrhagic Risk Sport assessed by the scale "Hemophilia and sport"	up to 6 and 12 months
Secondary	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