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Completed

NCT Number: NCT01817868

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

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.

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Key information

Conditions

Hemophilia Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhagic Disorders

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Multiple Locations, Turkey (Türkiye)

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Recombinant Factor VIII (Kogenate, BAY14-2222)

Other

All dosage, frequency and duration for drugs will be under the decision of the treating physician.

Primary outcomes

  1. Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group

    Time frame: After 4 years

  2. Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group

    Time frame: After 4 years

Secondary outcomes

  1. Number of overall bleeding episodes

    Time frame: After 4 years

  2. Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score)

    Time frame: After 4 years

  3. Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score)

    Time frame: After 4 years

  4. Cost-effectiveness (cost of additional joint bleed)

    Time frame: After 4 years

  5. 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].

  6. Comparison of patient compliance between prophylaxis and on-demand therapy groups

    Time frame: After 4 years

  7. Number of spontaneous bleeds

    Time frame: After 4 years

  8. Quality of life as measured with the SF-36

    Time frame: Baseline and after 4 years

  9. Quality of life as measured with Hemo-QoL

    Time frame: Baseline and after 4 years

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study 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

Important dates

Study start
2013
Primary completion
2018
Study completion
2019
First posted
Mar 26, 2013
Registry last updated
May 27, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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