Hemophilia AHAMLETNCT02319070
2-cohort Study of Adult Patients With Severe Hemophilia A in Greece
An observational study of Hemophilia A, run by Bayer at 1 site in Greece.
Trial summary
This study is a prospective, single center, observational, 2-cohort study of adult patients with severe Hemophilia A. There is no randomization procedure and all patients will be treated as per usual clinical practice. Patients will be followed up for 18 months after enrolment. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Survey
- How many people
- 72 participants
- Where
- 1 siteGreece
- Study dates
- 13 Jan 2015to 28 Feb 2018
- 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
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: This will be a single center prospective two-cohort observational study conducted in a hospital setting.Patients will be enrolled from the biggest specialty management center in Greece
Who may be able to join
- Patients diagnosed with severe Hemophilia A.
- Age ≥18 years old
- Patients able to read and write
- Patients receiving Factor VIII substitute therapy, either on-demand or as secondary prophylaxis.
- Patients under the same therapeutic strategy (On Demand or Secondary Prophylaxis) for at least 6 months prior to enrolment.
Who cannot join
- Patients that have developed inhibitors against factor VIII.
- Patients participating in an investigational program with interventions outside of routine clinical practice.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Prospective, Observational, 2-cohort Study of Adult Patients With Severe Hemophilia A in Greece. Cost,Clinical Outcomes and Quality of Life Comparison Between on Demand and Secondary Prophylaxis Treatment Strategies
This study is a prospective, single center, observational, 2-cohort study of adult patients with severe Hemophilia A. There is no randomization procedure and all patients will be treated as per usual clinical practice. Patients will be followed up for 18 months after enrolment.
Read the detailed description
The primary therapy for hemophilia is coagulation factor replacement, given either episodically on demand (OD) for the treatment of acute bleeds or prophylactically to prevent them.
The last years there has been an interest to study the comparative effectiveness of OD vs. SP (Secondary Prophylaxis) treatment strategies so as to create an evidence based platform to guide both clinical decision making and quantify the economic aspects of each treatment alternative. In Greece relevant information is lacking, thus it is imperative to study how patients on different treatment strategies are managed in a real-life setting in Greece, including the main factors associated with increased health care utilization, clinical success and patient well being.
- Conditions
- Hemophilia A
- Being tested
- Survey
- Keywords
- Cost, Clinical Outcomes, Quality of Life, On Demand Treatment, Secondary Prophylaxis
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SurveyOther
Recombinant antihemophilic factor VIII (FVIII) on Secondary Prophylaxis treatment scheduled according to daily routine
SurveyOther
Recombinant antihemophilic factor VIII (FVIII) On Demand treatment scheduled according to daily routine
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Adult patients with severe Hemophilia A.(25 patients on Secondary Prophylaxis treatment) Receives: Other: Survey | |
| Cohort 2 | Adult patients with severe Hemophilia A.(50 patients on On Demand treatment) Receives: Other: Survey |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy
Measured: At 18 months
Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis
Measured: At 18 months
Secondary outcomes (9)
Mean cost per joint bleed episodes for overall patients
Measured: At 18 months
Mean cost per joint bleed episodes by cohort for patients
Measured: At 18 months
Number of joint bleed episodes for overall patients
Measured: At 18 months
Number of joint bleed episodes by cohort for patients.
Measured: At 18 months
Severity of joint bleed episodes for overall patients.
Measured: At 18 months
Severity of joint bleed episodes by cohort for patients.
Measured: At 18 months
Mean EQ-VAS score as reported from patients at the time of a major bleed.
EQ-VAS score is Standardized questionnaire for hemophilia
Measured: At 18 months
Mean EQ-5D score as reported from patients at the time of a major bleed.
EQ-5D The EuroQol standardized instrument of health outcome
Measured: At 18 months
Mean change from baseline in Quality of Life scores derived from the Haem-A-Qol and EQ-5D questionnaires
Haem-A-Qol Specific questionnaire for hemophilia and EQ-5D The EuroQol standardized instrument of health outcome
Measured: Baseline and at 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Dec 2014 (estimated)
- Study start13 Jan 2015
- Primary completion28 May 2017
- Study completion28 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies 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
- 72 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
- NCT02319070
- Sponsor's protocol ID
- 17493
- Other IDs
- KG1411GR
- Acronym
- HAMLET
- Status last verified
- Jun 2018
- First submitted
- 12 Dec 2014
- Last updated
- 20 Jun 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Observational, 2-cohort Study of Adult Patients With Severe Hemophilia A in Greece. Cost,Clinical Outcomes and Quality of Life Comparison Between on Demand and Secondary Prophylaxis Treatment Strategies
- ClinicalTrials.gov ID
- NCT02319070
- Acronym / sponsor ID
- HAMLET / 17493
- Conditions
- Hemophilia A
- Keywords
- Cost, Clinical Outcomes, Quality of Life, On Demand Treatment, Secondary Prophylaxis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 72 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 13 Jan 2015 (actual)
- Primary completion
- 28 May 2017 (actual)
- Study completion
- 28 Feb 2018 (actual)
- First posted
- 18 Dec 2014 (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.
This study is a prospective, single center, observational, 2-cohort study of adult patients with severe Hemophilia A. There is no randomization procedure and all patients will be treated as per usual clinical practice. Patients will be followed up for 18 months after enrolment.
Detailed description
The primary therapy for hemophilia is coagulation factor replacement, given either episodically on demand (OD) for the treatment of acute bleeds or prophylactically to prevent them.
The last years there has been an interest to study the comparative effectiveness of OD vs. SP (Secondary Prophylaxis) treatment strategies so as to create an evidence based platform to guide both clinical decision making and quantify the economic aspects of each treatment alternative. In Greece relevant information is lacking, thus it is imperative to study how patients on different treatment strategies are managed in a real-life setting in Greece, including the main factors associated with increased health care utilization, clinical success and patient well being.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | Other: SurveyAdult patients with severe Hemophilia A.(25 patients on Secondary Prophylaxis treatment) |
| Cohort 2 | – | Other: SurveyAdult patients with severe Hemophilia A.(50 patients on On Demand treatment) |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- This will be a single center prospective two-cohort observational study conducted in a hospital setting.Patients will be enrolled from the biggest specialty management center in Greece
Key inclusion criteria (5 listed)
- Patients diagnosed with severe Hemophilia A.
- Age ≥18 years old
- Patients able to read and write
- Patients receiving Factor VIII substitute therapy, either on-demand or as secondary prophylaxis.
- Patients under the same therapeutic strategy (On Demand or Secondary Prophylaxis) for at least 6 months prior to enrolment.
Key exclusion criteria (2 listed)
- Patients that have developed inhibitors against factor VIII.
- Patients participating in an investigational program with interventions outside of routine clinical practice.
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 |
|---|---|---|
| SurveyRecombinant antihemophilic factor VIII (FVIII) on Secondary Prophylaxis treatment scheduled according to daily routine | Other | Cohort 1 |
| SurveyRecombinant antihemophilic factor VIII (FVIII) On Demand treatment scheduled according to daily routine | Other | Cohort 2 |
Primary outcome
Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy
Time frame: At 18 months
Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis
Time frame: At 18 months
9 secondary outcomes
Mean cost per joint bleed episodes for overall patients
Time frame: At 18 months
Mean cost per joint bleed episodes by cohort for patients
Time frame: At 18 months
Number of joint bleed episodes for overall patients
Time frame: At 18 months
Number of joint bleed episodes by cohort for patients.
Time frame: At 18 months
Severity of joint bleed episodes for overall patients.
Time frame: At 18 months
Severity of joint bleed episodes by cohort for patients.
Time frame: At 18 months
Mean EQ-VAS score as reported from patients at the time of a major bleed.
Time frame: At 18 months
Mean EQ-5D score as reported from patients at the time of a major bleed.
Time frame: At 18 months
Mean change from baseline in Quality of Life scores derived from the Haem-A-Qol and EQ-5D questionnaires
Time frame: Baseline and at 18 months
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 20 Jun 2018.
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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.
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 A, run by Bayer at 1 site in Greece.
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 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: finished, as of the sponsor's last update on 20 Jun 2018. 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 Greece. 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?
Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy (time frame: At 18 months). 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 20 Jun 2018.