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Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Bayer Identifier:
First received: March 12, 2013
Last updated: March 16, 2017
Last verified: March 2017

The project is an observational, multi-central, prospective, non-interventional and open-label data collection study on secondary prophylaxis with recombinant FVIII products in adolescents and adults with severe hemophilia A (FVIII < 1%).

It will be a controlled observation of patients on secondary prophylaxis versus on-demand treatment regimen. Patients will be enrolled preferably on a 1:1 basis with regards to prophylaxis and on-demand treatment.

The patient enrollment period will be 2 years with a follow-up (observation period) of 2 years for each patient. Based on the primary effectiveness parameters (joint bleeds and overall bleeds per year) an observation period of 2 years is considered sufficient although it has to be admitted that it is rather short to assess the progression of orthopedic status. Previously treated prophylaxis patients with at least 50 exposure days and patients with continuing prophylaxis treatment will be included.

Condition Intervention
Other: Recombinant Factor VIII (Kogenate, BAY14-2222)

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Evaluation of Efficacy, Safety and Costs of Recombinant FVIII Products Applied to Severe Hemophilia A Patients: Observational Data Collection Study Evaluating On-demand Treatment and Secondary Prophylaxis

Resource links provided by NLM:

Further study details as provided by Bayer:

Primary Outcome Measures:
  • Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group [ Time Frame: After 4 years ]
  • Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group [ Time Frame: After 4 years ]

Secondary Outcome Measures:
  • Number of overall bleeding episodes [ Time Frame: After 4 years ]
  • Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score) [ Time Frame: After 4 years ]
  • Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score) [ Time Frame: After 4 years ]
  • Cost-effectiveness (cost of additional joint bleed) [ Time Frame: After 4 years ]
  • Cost-utility [ Time Frame: After 4 years ]
    The costs of care of subjects with haemophilia will be evaluated adopting the perspective of the payer, i.e. the Social Security Institution [SSI].

  • Comparison of patient compliance between prophylaxis and on-demand therapy groups [ Time Frame: After 4 years ]
  • Number of spontaneous bleeds [ Time Frame: After 4 years ]
  • Quality of life as measured with the SF-36 [ Time Frame: Baseline and after 4 years ]
  • Quality of life as measured with Hemo-QoL [ Time Frame: Baseline and after 4 years ]

Enrollment: 73
Actual Study Start Date: January 4, 2013
Estimated Study Completion Date: August 30, 2018
Estimated Primary Completion Date: June 28, 2018 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Group 1 Other: Recombinant Factor VIII (Kogenate, BAY14-2222)
All dosage, frequency and duration for drugs will be under the decision of the treating physician.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Adult haemophilia patients already on treatment with commercially available recombinant FVIII products.

Inclusion Criteria:

  • Age ≥ 18 years
  • Severe hemophilia A (FVIII<1%) diagnosis
  • Prior treatment or ongoing treatment with on-demand or prophylaxis regimens according to Turkish guidelines and requirements
  • Previously treated patients with at least >50 exposure days
  • Written informed consent signed by patient/legal representative

Exclusion Criteria:

  • Currently on immune tolerance treatment
  • Platelet count < 75,000/mm3
  • Participation in another study
  • Existence of inhibitors in the past and in the last currently available blood sample prior to study start (Bethesda titer < 0.6 BU/ml)
  • Existence of inhibitor history in family members who also are diagnosed with hemophilia A
  • Having been on primary prophylaxis as defined in the introduction
  • Participation in another study
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01817868

Many Locations, Turkey
Sponsors and Collaborators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
Responsible Party: Bayer Identifier: NCT01817868     History of Changes
Other Study ID Numbers: 16368
KG1210TR ( Other Identifier: Company Internal )
Study First Received: March 12, 2013
Last Updated: March 16, 2017

Keywords provided by Bayer:

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants processed this record on May 25, 2017