Hemophilia APOTTER 10NCT02581969
Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A."
An observational study of Hemophilia A, run by Bayer at 1 site in Italy.
Trial summary
Following the performance of the POTTER observational study, whose primary objective was to collect data on the benefits of secondary prophylaxis versus on demand treatment in terms of prevention of bleeding episodes, the present study aims to extend the observation time up to 10 years, of the same population involved… Read more about this study
- Who is it for?
- Ages 17 to 60 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Type of treatment
- Injection or infusion
- How many people
- 43 participants
- Where
- 1 siteItaly
- Study dates
- 14 Apr 2016to 21 Jun 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 17 to 60 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: A total of approximately 50 patients with a diagnosis of severe haemophilia A (FVIII < 1%) , who are already on Kogenate Bayer treatment ongoing for long-term secondary prophylaxis or on-demand therapy and have properly completed the follow-up in the original Potter study are expected to be enrolled.
Who may be able to join
- Completion of follow-up in the original Potter study without having being excluded from the Intention To Treat efficacy analysis of the primary endpoint;
- Written informed consent specifically issued for the 5-year extension.
Who cannot join
- Switching of treatment to a Factor VIII concentrate different from Kogenate Bayer/Helixate NexGen after the end of the previous follow-up period.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A." POTTER-10
Following the performance of the POTTER observational study, whose primary objective was to collect data on the benefits of secondary prophylaxis versus on demand treatment in terms of prevention of bleeding episodes, the present study aims to extend the observation time up to 10 years, of the same population involved in the previous study.
The present study has been designed to allow a focus on long term disease-related damage at joint level in subjects with severe haemophilia A, as well as on the pharmacoeconomics impact of the two different treatment regimens.
- Conditions
- Hemophilia A
- Being tested
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Keywords
- Hemophilia A, Congenital
- 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 FS, BAY14-2222)Drug
Administered 20-30 IU/kg 3 times a week
Recombinant Factor VIII (Kogenate FS, BAY14-2222)Drug
Administered only for bleeding episodes
| Study group | Role | What they receive |
|---|---|---|
| Prophylaxis | Prophylaxis group: treatment is based on regularly repeated infusions of clotting factor, 20-30 IU/kg -3 times a week Receives: Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222) | |
| On-demand | On-demand group: treatment administered when bleeding episode occur Receives: Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of joint bleeding episodes per year
Measured: 5 years period
Secondary outcomes (13)
Total number of bleeding episodes per year
Measured: 5 years period
Total amount of Recombinant Factor VIII consumption
Measured: 5 years period
Changes of prophylaxis dose
Measured: 5 years period
Changes of frequency of infusions
Measured: 5 years period
Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score)
Measured: 5 years period
Proportions of patients who will change the therapeutic regimen during the study
Measured: 5 years period
Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score)
Measured: 5 years period
Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol)
Measured: 5 years period
Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36)
Measured: 5 years period
Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D)
Measured: 5 years period
Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related
Measured: 5 years period
Number of patients with poor compliance
Measured: 5 years period
Number of patients with adverse events
Measured: 5 years period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2015 (estimated)
- Study start14 Apr 2016
- Primary completion16 Mar 2017
- Study completion21 Jun 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
- Retrospective
- Enrolment
- 43 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
- NCT02581969
- Sponsor's protocol ID
- 18251
- Acronym
- POTTER 10
- Status last verified
- Nov 2020
- First submitted
- 20 Oct 2015
- Last updated
- 18 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A." POTTER-10
- ClinicalTrials.gov ID
- NCT02581969
- Acronym / sponsor ID
- POTTER 10 / 18251
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, Congenital
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 14 Apr 2016 (actual)
- Primary completion
- 16 Mar 2017 (actual)
- Study completion
- 21 Jun 2018 (actual)
- First posted
- 21 Oct 2015 (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.
Following the performance of the POTTER observational study, whose primary objective was to collect data on the benefits of secondary prophylaxis versus on demand treatment in terms of prevention of bleeding episodes, the present study aims to extend the observation time up to 10 years, of the same population involved in the previous study.
The present study has been designed to allow a focus on long term disease-related damage at joint level in subjects with severe haemophilia A, as well as on the pharmacoeconomics impact of the two different treatment regimens.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prophylaxis | – | Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)Prophylaxis group: treatment is based on regularly repeated infusions of clotting factor, 20-30 IU/kg -3 times a week |
| On-demand | – | Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)On-demand group: treatment administered when bleeding episode occur |
Who may qualify
- Ages
- 17 to 60 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- A total of approximately 50 patients with a diagnosis of severe haemophilia A (FVIII < 1%) , who are already on Kogenate Bayer treatment ongoing for long-term secondary prophylaxis or on-demand therapy and have properly completed the follow-up in the original Potter study are expected to be enrolled.
Key inclusion criteria (2 listed)
- Completion of follow-up in the original Potter study without having being excluded from the Intention To Treat efficacy analysis of the primary endpoint;
- Written informed consent specifically issued for the 5-year extension.
Key exclusion criteria (1 listed)
- Switching of treatment to a Factor VIII concentrate different from Kogenate Bayer/Helixate NexGen after the end of the previous follow-up period.
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 FS, BAY14-2222)Administered 20-30 IU/kg 3 times a week | Drug | Prophylaxis |
| Recombinant Factor VIII (Kogenate FS, BAY14-2222)Administered only for bleeding episodes | Drug | On-demand |
Primary outcome
Number of joint bleeding episodes per year
Time frame: 5 years period
13 secondary outcomes
Total number of bleeding episodes per year
Time frame: 5 years period
Total amount of Recombinant Factor VIII consumption
Time frame: 5 years period
Changes of prophylaxis dose
Time frame: 5 years period
Changes of frequency of infusions
Time frame: 5 years period
Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score)
Time frame: 5 years period
Proportions of patients who will change the therapeutic regimen during the study
Time frame: 5 years period
Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score)
Time frame: 5 years period
Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol)
Time frame: 5 years period
Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36)
Time frame: 5 years period
Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D)
Time frame: 5 years period
Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related
Time frame: 5 years period
Number of patients with poor compliance
Time frame: 5 years period
Number of patients with adverse events
Time frame: 5 years period
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 18 Nov 2020.
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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 Italy.
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 2 inclusion and 1 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 18 Nov 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 Italy. 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?
Number of joint bleeding episodes per year (time frame: 5 years period). 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 18 Nov 2020.