HemophiliaNCT02908243
Outcomes of Prophylaxis and On-demand Treatment for Severe Hemophiliacs and Collection of Baseline Data in Hemophiliacs in Taiwan
An observational study of Hemophilia, run by Taiwan Society of Thrombosis and Hemostasis.
Trial summary
Primary prophylaxis in severe hemophiliacs is defined that prophylaxis therapy starts before 2 years of age and prior to any clinically evident joint bleeding or after first joint bleeding and prior to the onset of joint damage irrespective of age, joints can be kept normal or very mildly damaged till teenage or… Read more about this study
- Who is it for?
- Ages 2 to 80 years, men only
- Conditions the study is for
- Hemophilia
- What is being tested?
- collection of baseline data
- Type of treatment
- Injection or infusion
- How many people
- 350 participants
- Where
- No sites listed yet
- Study dates
- Feb 2015to Dec 2018
- How long will I take part?
- 3 Years
- 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 2 to 80 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: 200 Severe hemophilia A patients
Who may be able to join
- all severe hemophilia A and B patients more than 2 years of age and without inhibitor
Who cannot join
- female hemophilia
- hemophiliac patients with inhibitor > = 0.6 Bethesda unit (BU)
About this study
Primary prophylaxis in severe hemophiliacs is defined that prophylaxis therapy starts before 2 years of age and prior to any clinically evident joint bleeding or after first joint bleeding and prior to the onset of joint damage irrespective of age, joints can be kept normal or very mildly damaged till teenage or adulthood. Primary prophylaxis has been proved to be more beneficial and cause less damage to joint than "on-demand" therapy. Primary prophylaxis is also known to be able to decrease the occurrence of factor VIII inhibitor and is the most advanced and useful, cost-effective therapy for hemophilia care. However, it requires 2 to 3 injections of factor VIII or IX of 20-50 IU/Kg doses every week, it costs a lot of expenses. In the year 2013, we tried hard to discuss with Bureau of National Health Insurance (BNHI) and have meeting a couple of time, eventually a guideline of an intermediate-dose prophylaxis for severe hemophilia was established and a consensus was reached that this prophylactic treatment will be cost effective without increased burden of total budget. This guideline was finally approved by BNHI and will be implemented from July 1st, 2014. In oder to evaluate the efficacy of prophylaxis treatment, patients will be arranged to come back to each hemophilia center at least once a year to have investigation of doses and annual consumption of clotting factors, frequencies and causes of bleedings, especially joint bleedings, joint outcome by studies of hemophilia joint health score (HJHS), hemophilia actives list and health-related quality of life. These results will be collected and compared between intermediate-dose prophylaxis group of patients and on demand treatment group of patients.
The life span of hemophiliacs has been improved remarkably in recent years due to sufficient and adequate treatments, especially prophylaxis treatment, therefore comorbidities in the hemophilic population, e.g. hypertension, diabetes, hyperlipidemia and cancer, etc, have been found with prevalences close to those in non-hemophilic population. It is worth that the prevalence of these comorbidity will also be investigated. In addition, basic data of the patients including age, sex, severity, the development of inhibitor and viral infection etc will also be collected for analysis.
- Conditions
- Hemophilia
- Being tested
- collection of baseline data
- Keywords
- Hemophilia, Primary prophylaxis, Pettersson Score, Joint function study, Comorbidity
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
collection of baseline dataOther
collection of baseline data including ages, types, severity, inhibitors and comorbidities
| Study group | Role | What they receive |
|---|---|---|
| Prophylaxis | Severe hemophilia A patients who receive factor VIII with dose of 15-25 IU/Kg, 2-3 times/week Severe hemophilia B patients who receive factor IX with dose of 30-50 IU/Kg, 1-2 times/week | |
| On-demand | Severe hemophilia A patients who receive factor VIII injection when bleeding occurs with the dose according to the criteria of World Federation of Hemophilia (WFH); Severe hemophilia B patients who receive factor IX injection when bleeding occurs with the dose according to the criteria of World Federation of Hemophilia (WFH) | |
| Collection of baseline data in all hemophilia A and B patients | All severe, moderate and mild types of hemophilia A and B patients Receives: Other: collection of baseline data |
What is being measured
The primary outcome is the main question the study is designed to answer.
annual bleeding rate
spontaneous and traumatic joint and other site bleedings
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2016 (estimated)
- Study startFeb 2015
- Primary completionDec 2018 (estimated)
- Study completionDec 2018 (estimated)
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.
- 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
- Enrolment
- 350 participants (estimated)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- Taiwan Society of Thrombosis and Hemostasis
- Sponsor type
- University, hospital or other
- Collaborators
- Changhua Christian Hospital, Taipei Medical University Hospital, Taipei Veterans General Hospital, Taiwan, Taipei Medical University Shuang Ho Hospital, Taichung Veterans General Hospital, China Medical University Hospital, Kaohsiung Medical University Chung-Ho Memorial Hospital
- Responsible party
- Sponsor
- Investigators
- Ming-Ching Shen, BS, Study chair, Changhua Christian Hospital, Changhua, Taiwan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02908243
- Sponsor's protocol ID
- 141115
- Status last verified
- Sep 2016
- First submitted
- 8 Sep 2016
- Last updated
- 20 Sep 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Plan to share whole data
Study at a glance
From the registry record, last updated by the sponsor on 20 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Outcomes of Prophylaxis and On-demand Treatment for Severe Hemophiliacs and Collection of Baseline Data in Hemophiliacs in Taiwan
- ClinicalTrials.gov ID
- NCT02908243
- Acronym / sponsor ID
- 141115
- Conditions
- Hemophilia
- Keywords
- Hemophilia, Primary prophylaxis, Pettersson Score, Joint function study, Comorbidity
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 350 participants (estimated)
- Lead sponsor
- Taiwan Society of Thrombosis and HemostasisUniversity, hospital or other
- Collaborators
- Changhua Christian Hospital, Taipei Medical University Hospital, Taipei Veterans General Hospital, Taiwan, Taipei Medical University Shuang Ho Hospital, Taichung Veterans General Hospital, China Medical University Hospital, Kaohsiung Medical University Chung-Ho Memorial Hospital
- Study start
- Feb 2015
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 20 Sep 2016 (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.
Primary prophylaxis in severe hemophiliacs is defined that prophylaxis therapy starts before 2 years of age and prior to any clinically evident joint bleeding or after first joint bleeding and prior to the onset of joint damage irrespective of age, joints can be kept normal or very mildly damaged till teenage or adulthood. Primary prophylaxis has been proved to be more beneficial and cause less damage to joint than "on-demand" therapy. Primary prophylaxis is also known to be able to decrease the occurrence of factor VIII inhibitor and is the most advanced and useful, cost-effective therapy for hemophilia care. However, it requires 2 to 3 injections of factor VIII or IX of 20-50 IU/Kg doses every week, it costs a lot of expenses. In the year 2013, we tried hard to discuss with Bureau of National Health Insurance (BNHI) and have meeting a couple of time, eventually a guideline of an intermediate-dose prophylaxis for severe hemophilia was established and a consensus was reached that this prophylactic treatment will be cost effective without increased burden of total budget. This guideline was finally approved by BNHI and will be implemented from July 1st, 2014. In oder to evaluate the efficacy of prophylaxis treatment, patients will be arranged to come back to each hemophilia center at least once a year to have investigation of doses and annual consumption of clotting factors, frequencies and causes of bleedings, especially joint bleedings, joint outcome by studies of hemophilia joint health score (HJHS), hemophilia actives list and health-related quality of life. These results will be collected and compared between intermediate-dose prophylaxis group of patients and on demand treatment group of patients.
The life span of hemophiliacs has been improved remarkably in recent years due to sufficient and adequate treatments, especially prophylaxis treatment, therefore comorbidities in the hemophilic population, e.g. hypertension, diabetes, hyperlipidemia and cancer, etc, have been found with prevalences close to those in non-hemophilic population. It is worth that the prevalence of these comorbidity will also be investigated. In addition, basic data of the patients including age, sex, severity, the development of inhibitor and viral infection etc will also be collected for analysis.
Study design
- Observational model
- Cohort
- Target follow-up
- 3 Years
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Prophylaxis | – | –Severe hemophilia A patients who receive factor VIII with dose of 15-25 IU/Kg, 2-3 times/week Severe hemophilia B patients who receive factor IX with dose of 30-50 IU/Kg, 1-2 times/week |
| On-demand | – | –Severe hemophilia A patients who receive factor VIII injection when bleeding occurs with the dose according to the criteria of World Federation of Hemophilia (WFH); Severe hemophilia B patients who receive factor IX injection when bleeding occurs with the dose according to the criteria of World Federation of Hemophilia (WFH) |
| Collection of baseline data in all hemophilia A and B patients | – | Other: collection of baseline dataAll severe, moderate and mild types of hemophilia A and B patients |
Who may qualify
- Ages
- 2 to 80 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 200 Severe hemophilia A patients
Key inclusion criteria (1 listed)
- all severe hemophilia A and B patients more than 2 years of age and without inhibitor
Key exclusion criteria (2 listed)
- female hemophilia
- hemophiliac patients with inhibitor > = 0.6 Bethesda unit (BU)
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 |
|---|---|---|
| collection of baseline datacollection of baseline data including ages, types, severity, inhibitors and comorbidities | Other | Collection of baseline data in all hemophilia A and B patients |
Primary outcome
annual bleeding rate
Time frame: through study completion, an average of 1 year
spontaneous and traumatic joint and other site bleedings
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Sep 2016.
- Individual participant dataSharing planned: Yes. Plan to share whole data
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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.
Ming-Ching Shen, BS study chair
Changhua Christian Hospital, Changhua, Taiwan
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia, run by Taiwan Society of Thrombosis and Hemostasis.
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 1 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 20 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
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?
annual bleeding rate (time frame: through study completion, an average of 1 year). 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 20 Sep 2016.