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

NCT01817868 | Completed

• 2 other identifiers: 16368KG1210TR
• Study Type: observational
• Target: 73
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Hemophilia

Keywords

Hemophilia; Turkey; Observational; Adult

Outcome Measures

Primary Outcomes (2)

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

  After 4 years

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

  After 4 years

Secondary Outcomes (9)

• Number of overall bleeding episodes

  After 4 years

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

  After 4 years

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

  After 4 years

• Cost-effectiveness (cost of additional joint bleed)

  After 4 years

• Cost-utility

  After 4 years

Study Arms (1)

Group 1

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

Interventions

Recombinant Factor VIII (Kogenate, BAY14-2222) OTHER

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

Group 1

Eligibility Criteria

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

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Bayer: lead

Study Sites (1)

Unknown Facility

Multiple Locations, Turkey (Türkiye)

Location

Related Links

• Click here to find results for studies related to Bayer products.

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: March 12, 2013
• First Posted: March 26, 2013
• Study Start: January 4, 2013
• Primary Completion: July 16, 2018
• Study Completion: May 23, 2019
• Last Updated: May 27, 2020

Record last verified: 2020-05

Locations

TU (1)