HemophiliaNCT01817868
Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients
An observational study of Hemophilia, run by Bayer at 1 site in Turkey (Türkiye).
Trial 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%). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemophilia
- What is being tested?
- Recombinant Factor VIII (Kogenate, BAY14-2222)
- How many people
- 73 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 4 Jan 2013to 23 May 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult haemophilia patients already on treatment with commercially available recombinant FVIII products.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
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
- Being tested
- Recombinant Factor VIII (Kogenate, BAY14-2222)
- Keywords
- Hemophilia, Turkey, Observational, Adult
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Factor VIII (Kogenate, BAY14-2222)Other
All dosage, frequency and duration for drugs will be under the decision of the treating physician.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Not described Receives: Other: Recombinant Factor VIII (Kogenate, BAY14-2222) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group
Measured: After 4 years
Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group
Measured: After 4 years
Secondary outcomes (9)
Number of overall bleeding episodes
Measured: After 4 years
Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score)
Measured: After 4 years
Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score)
Measured: After 4 years
Cost-effectiveness (cost of additional joint bleed)
Measured: After 4 years
Cost-utility
The costs of care of subjects with haemophilia will be evaluated adopting the perspective of the payer, i.e. the Social Security Institution [SSI].
Measured: After 4 years
Comparison of patient compliance between prophylaxis and on-demand therapy groups
Measured: After 4 years
Number of spontaneous bleeds
Measured: After 4 years
Quality of life as measured with the SF-36
Measured: Baseline and after 4 years
Quality of life as measured with Hemo-QoL
Measured: Baseline and after 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Mar 2013 (estimated)
- Study start4 Jan 2013
- Primary completion16 Jul 2018
- Study completion23 May 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- More trials run by BayerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 73 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bayer Study Director, Study director, Bayer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01817868
- Sponsor's protocol ID
- 16368
- Other IDs
- KG1210TR
- Status last verified
- May 2020
- First submitted
- 12 Mar 2013
- Last updated
- 27 May 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01817868
- Acronym / sponsor ID
- 16368
- Conditions
- Hemophilia
- Keywords
- Hemophilia, Turkey, Observational, Adult
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 73 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 4 Jan 2013 (actual)
- Primary completion
- 16 Jul 2018 (actual)
- Study completion
- 23 May 2019 (actual)
- First posted
- 26 Mar 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Group 1 | – | Other: Recombinant Factor VIII (Kogenate, BAY14-2222) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult haemophilia patients already on treatment with commercially available recombinant FVIII products.
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Recombinant Factor VIII (Kogenate, BAY14-2222)All dosage, frequency and duration for drugs will be under the decision of the treating physician. | Other | Group 1 |
Primary outcome
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
9 secondary outcomes
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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2020.
- Individual participant dataSharing planned: Undecided. Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.
Related trials for Hemophilia
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bayer Study Director study director
Bayer
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia, run by Bayer at 1 site in Turkey (Türkiye).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group (time frame: After 4 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 May 2020.