PK Driven Prophylaxis for Hemophilia A | NCT02634424

Studies
PK Driven Prophylaxis for Hemophilia A

NCT02634424

Sponsor

Assistance Publique - Hôpitaux de Paris

Status

Terminated

Last Update Posted

Mar 16, 2020Actual

Enrollment

12Actual

Phase

Not Applicable

Conditions

Hemophilia A

Interventions

MyPKFiT

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2016

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

lack of inclusion and prophylaxy plateform being free to use for everyone in septembre 2016

Expanded Access Info

Start Date

Nov 25, 2015Actual

Primary Completion Date

May 12, 2016Actual

Completion Date

May 12, 2016Actual

First Submitted Date

Dec 8, 2015

First Submission Date that Met QC Criteria

Dec 15, 2015

First Posted Date

Dec 18, 2015Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 12, 2020

Last Update Posted Date

Mar 16, 2020Actual

Name	Class
Baxter Healthcare Corporation	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

12Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
MyPKFiT	Experimental	Personalized prophylaxis : Treatment is adjustment according to PK modeling	Device: MyPKFiT

Interventions

Name	Type	Description	Arm Group Labels	Other Names
MyPKFiT	Device	Adjustment of the treatment dose using a decision support software according to the PK values	MyPKFiT

Outcomes Module

Primary Outcomes

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 Outcomes

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

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Thierry LAMBERT, MD	AP-HP, Bicêtre Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
AP-HP, Bicêtre Hospital		Le Kremlin-Bicêtre		94275	France

Condition Browse Module

MeSH Terms

ID	Term
D006467	Hemophilia A

Ancestor Terms

ID	Term
D025861	Blood Coagulation Disorders, Inherited
D001778	Blood Coagulation Disorders
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases

Browse Leaves

Not provided

Browse Branches

Not provided