PK Driven Prophylaxis for Hemophilia A

NCT02634424 | Terminated

• 1 other identifier: P140701
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Hemophilia A

Keywords

Hemophilia A; Prophylaxy; Pharmacokinetic; Bayesian

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (7)

• Total annual bleeding rate

  during 6 months before (retrospectively) and up to 12 months after using of myPKFiT

• Quality of life

  up to 12 months

• Joint function

  up to 12 months

• Adherence to treatment estimated as the quantity of Facteur VIII consumed reported to the prescribed quantity.

  up to 6 and 12 months

• 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

Study Arms (1)

MyPKFiT

EXPERIMENTAL

Personalized prophylaxis : Treatment is adjustment according to PK modeling

Device: MyPKFiT

Interventions

MyPKFiT DEVICE

Adjustment of the treatment dose using a decision support software according to the PK values

Also known as: Personalized prophylaxis by PK modeling

MyPKFiT

Eligibility Criteria

• Age: 6 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• Baxter Healthcare Corporation: collaborator

Study Sites (1)

AP-HP, Bicêtre Hospital

Le Kremlin-Bicêtre, 94275, France

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Thierry LAMBERT, MD

  AP-HP, Bicêtre Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 8, 2015
• First Posted: December 18, 2015
• Study Start: November 25, 2015
• Primary Completion: May 12, 2016
• Study Completion: May 12, 2016
• Last Updated: March 16, 2020

Record last verified: 2016-08

Locations

FR (1)