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

This study is currently recruiting participants.
Verified March 2014 by Bayer
Sponsor:
Information provided by (Responsible Party):
Bayer
ClinicalTrials.gov Identifier:
NCT01817868
First received: March 12, 2013
Last updated: March 24, 2014
Last verified: March 2014
  Purpose

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
Hemophilia
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 ] [ Designated as safety issue: No ]
  • Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group [ Time Frame: After 4 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number of overall bleeding episodes [ Time Frame: After 4 years ] [ Designated as safety issue: No ]
  • Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score) [ Time Frame: After 4 years ] [ Designated as safety issue: No ]
  • Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score) [ Time Frame: After 4 years ] [ Designated as safety issue: No ]
  • Cost-effectiveness (cost of additional joint bleed) [ Time Frame: After 4 years ] [ Designated as safety issue: No ]
  • Cost-utility [ Time Frame: After 4 years ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
  • Number of spontaneous bleeds [ Time Frame: After 4 years ] [ Designated as safety issue: No ]
  • Quality of life as measured with the SF-36 [ Time Frame: Baseline and after 4 years ] [ Designated as safety issue: No ]
  • Quality of life as measured with Hemo-QoL [ Time Frame: Baseline and after 4 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 102
Study Start Date: January 2013
Estimated Study Completion Date: June 2017
Estimated Primary Completion Date: December 2016 (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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Adult haemophilia patients already on treatment with commercially available recombinant FVIII products.

Criteria

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01817868

Contacts
Contact: Bayer Clinical Trials Contact clinical-trials-contact@bayerhealthcare.com

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

Additional Information:
No publications provided

Responsible Party: Bayer
ClinicalTrials.gov Identifier: NCT01817868     History of Changes
Other Study ID Numbers: 16368, KG1210TR
Study First Received: March 12, 2013
Last Updated: March 24, 2014
Health Authority: Turkey: Ministry of Health General Directorate of Pharmaceuticals and Pharmacy

Keywords provided by Bayer:
Hemophilia
Turkey
Observational
Adult

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
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 17, 2014