Hemophilia APOTTERNCT01159587
Prophylaxis Versus On-demand Therapy Through Economic Report
An observational study of Hemophilia A, run by Bayer at 1 site in Italy.
Trial summary
The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII < 1%). The observational period will cover at least 5 years per patient. Read more about this study
- Who is it for?
- Ages 12 to 55 years
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- How many people
- 58 participants
- Where
- 1 siteItaly
- Study dates
- Jul 2004to Dec 2010
- 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 12 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Haemophilics
Who may be able to join
- age ≥ 12 years and ≤ 55 years
- severe haemophilia A (FVIII < 1%)
- absence of inhibitors (Bethesda titre < 0.6 BU/ml)
- Previous Treated Patients (prior exposure days > 200)
- Kogenate Bayer administered 20-30 IU/kg -3 times a week, for the prophylaxis group
- ≥ 6 joint bleeds requiring treatment with FVIII concentrates in the previous 6 months before enrollment, for on-demand group
- written informed consent
Who cannot join
- concomitant severe and chronic diseases or congenital skeletal malformation
- unreliability of patient or likelihood of follow-up failure
- presence of inhibitors or history of inhibitors (in the previous 2 years)
- currently on immune tolerance treatment
- hepatic cirrhosis or liver disease in rapid progression
- AIDS
Show all 9Show fewer
- platelet count < 75,000/mm3
- presence of conditions that influence negatively patient´s compliance
- participation in another study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A. POTTER
The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII < 1%).
The observational period will cover at least 5 years per patient.
The long-term secondary prophylaxis group will be compared versus on-demand treatment group by the assessment of orthopedic status progression and pharmacoeconomics evaluation.
- Conditions
- Hemophilia A
- Being tested
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- 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
Long-term secondary prophylaxis with product administered 20-30 UI /kg three times weekly
Recombinant Factor VIII (Kogenate FS, BAY14-2222)Drug
On-demand treatment with product given only for bleeding episodes
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Not described Receives: Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222) | |
| Group 2 | Not described 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.
Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per year
Measured: Every 6 months
Secondary outcomes (5)
Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes
Measured: Every 6 months
Muscolo skeletal evaluation by Orthopedic Joint Score
Measured: Every 12 months
Radiological Evaluation by Pettersson Score
Measured: Baseline and after 3-5 years
Health related quality of life
Measured: Every 12 months
cost-effectiveness and utility, patient compliance, adverse events
Measured: Every 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2010 (estimated)
- Study startJul 2004
- Primary completionDec 2010
- Study completionDec 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Bayer Study Director, Study director, Bayer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01159587
- Sponsor's protocol ID
- 11856
- Acronym
- POTTER
- Status last verified
- Apr 2011
- First submitted
- 8 Jul 2010
- Last updated
- 13 Apr 2011
- Results posted
- No
- Data monitoring committee
- No
Publications
Tagliaferri A, Feola G, Molinari AC, Santoro C, Rivolta GF, Cultrera DB, Gagliano F, Zanon E, Mancuso ME, Valdre L, Mameli L, Amoresano S, Mathew P, Coppola A; POTTER Study Group. Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study. Thromb Haemost. 2015 Jul;114(1):35-45. doi: 10.1160/TH14-05-0407. Epub 2015 Apr 9.
PubMed 25855376
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A. POTTER
- ClinicalTrials.gov ID
- NCT01159587
- Acronym / sponsor ID
- POTTER / 11856
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 58 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- Jul 2004
- Primary completion
- Dec 2010 (actual)
- Study completion
- Dec 2010 (actual)
- First posted
- 9 Jul 2010 (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 a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII < 1%).
The observational period will cover at least 5 years per patient.
The long-term secondary prophylaxis group will be compared versus on-demand treatment group by the assessment of orthopedic status progression and pharmacoeconomics evaluation.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | – | Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222) |
| Group 2 | – | Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222) |
Who may qualify
- Ages
- 12 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Haemophilics
Key inclusion criteria (7 listed)
- age ≥ 12 years and ≤ 55 years
- severe haemophilia A (FVIII < 1%)
- absence of inhibitors (Bethesda titre < 0.6 BU/ml)
- Previous Treated Patients (prior exposure days > 200)
- Kogenate Bayer administered 20-30 IU/kg -3 times a week, for the prophylaxis group
Key exclusion criteria (9 listed)
- concomitant severe and chronic diseases or congenital skeletal malformation
- unreliability of patient or likelihood of follow-up failure
- presence of inhibitors or history of inhibitors (in the previous 2 years)
- currently on immune tolerance treatment
- hepatic cirrhosis or liver disease in rapid progression
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)Long-term secondary prophylaxis with product administered 20-30 UI /kg three times weekly | Drug | Group 1 |
| Recombinant Factor VIII (Kogenate FS, BAY14-2222)On-demand treatment with product given only for bleeding episodes | Drug | Group 2 |
Primary outcome
Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per year
Time frame: Every 6 months
5 secondary outcomes
Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes
Time frame: Every 6 months
Muscolo skeletal evaluation by Orthopedic Joint Score
Time frame: Every 12 months
Radiological Evaluation by Pettersson Score
Time frame: Baseline and after 3-5 years
Health related quality of life
Time frame: Every 12 months
cost-effectiveness and utility, patient compliance, adverse events
Time frame: Every 6 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 13 Apr 2011.
- Derived from this study: PubMed 25855376Tagliaferri A, Feola G, Molinari AC, Santoro C, Rivolta GF, Cultrera DB, Gagliano F, Zanon E, Mancuso ME, Valdre L, Mameli L, Amoresano S, Mathew P, Coppola A; POTTER Study Group. Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study. Thromb Haemost. 2015 Jul;114(1):35-45. doi: 10.1160/TH14-05-0407. Epub 2015 Apr 9.
Related trials for Hemophilia A
RecruitingPhase 3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Hemophilia A, Hemophilia B
RecruitingObservational study
An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
Hemophilia A
RecruitingPhase 3
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
Hemophilia A
Not yet recruitingObservational study
A Study to Describe Patient REal-world FitusiRan ExpeRience
Hemophilia A or B
Active, not recruitingPhase 4
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Severe Hemophilia A, Moderate Hemophilia A
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 7 inclusion and 9 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 13 Apr 2011. 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?
Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per year (time frame: Every 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01159587, which also lists 1 linked publication.
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 13 Apr 2011; AzTrials last refreshed its copy on 27 Oct 2026.