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

NCT01436825 | Completed

• 2 other identifiers: 15453KG110FR
• Study Type: observational
• Target: 69
• Locations: 2 countries
• Sites: 2

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

Conditions

Haemophilia A

Keywords

Haemophilia A; Factor VIII; Pharmacokinetic; Computer Tool validation

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (2)

• 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

Study Arms (1)

Group 1

Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Interventions

Recombinant Factor VIII (Kogenate FS, BAY14-2222) DRUG

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

Group 1

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

* 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 clinical signs of frequent, severe bleeding episodes * Patient treated prophylatically with the same plasma or recombinant factor VIII for at least 6 months

You may qualify if:

• 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

You may not qualify if:

• Patients with haemophilia B

Sponsors & Collaborators

• Bayer: lead

Study Sites (2)

Unknown Facility

Many Locations, France

Location

Unknown Facility

Many Locations, Italy

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII; F8 protein, human; BAY 14-2222

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

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Bayer Study Director

  Bayer

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 14, 2011
• First Posted: September 20, 2011
• Study Start: October 1, 2011
• Primary Completion: June 1, 2014
• Study Completion: July 1, 2015
• Last Updated: August 10, 2015

Record last verified: 2015-08

Locations

FR (1); IT (1)