Validation Study of a cOmputer Pharmacokinetic Tool to assIst in the Follow up Care of haeMophilia A Patients

A Validation Study of the OPTIMS Pharmacokinetic Calculator for Clinician Use in Prophylactic Treatment of Patients With Haemophilia A.

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01436825

Acronym

OPTIMS

Enrollment

Registered

2011-09-20

Start date

2011-10-31

Completion date

2015-07-31

Last updated

2015-08-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Haemophilia A

Keywords

Haemophilia A, Factor VIII, Pharmacokinetic, Computer Tool validation

Brief summary

OPTIMS is a non interventional validation study of the calculator developed by Bayer for clinician's use in the prophylactic treatment by factor VIII of patients with severe or moderate Haemophilia A with a severe clinical profile. The study takes place during a single visit, at the time of patient enrollment in the study

Interventions

DRUGRecombinant Factor VIII (Kogenate FS, BAY14-2222)

Prophylactic treatment : 20-40 UI /Kg administered all 2 in 3 days .

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

Inclusion criteria

* Patient with severe haemophilia A defined as residual factor VIII activity \< 1% * or Patient with moderate haemophilia A (residual factor VIII activity \>1% and \<5%) with severe clinical profile * Patient treated in prevention with the same plasma or recombinant factor VIII for at least 6 months * Patient whose inclusion visit is performed during a routine visit including a measurement of plasma residual factor VIII :c level * In the medical file (retrospective data) the pharmacokinetic parameters are available and have been obtained from a PK analysis of factor VIII level performed with the same anti haemophilic factor than the one used on the inclusion day

Exclusion criteria

* Patients with haemophilia B

Design outcomes

Primary

Measure	Time frame
The difference of the recovery rate calculated by the physician and the recovery rate calculated by an independent calculator With OPTIMS Tool	after 1 month
The difference of the elimination half-life calculated by the physician and the ones calculated by an independent calculator With OPTIMS Tool	after 1 month
The difference of the clearance calculated by the physician and the ones calculated by an independent calculator With OPTIMS Tool	after 1 month

Secondary

Measure	Time frame
The dosage of factor VIII calculated by OPTIMS calculator	within 48 hours after enrollment
The practicality of the OPTIMS calculator by a physician's satisfaction questionnaire	within 48 hours after enrollment

Countries

France, Italy

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026