Haemophilia AOPTIMSNCT01436825
Validation Study of a cOmputer Pharmacokinetic Tool to assIst in the Follow up Care of haeMophilia A Patients
An observational study of Haemophilia A, run by Bayer at 2 sites in 2 countries.
Trial summary
OPTIMS is a non interventional validation study of the calculator developed by Bayer for clinician's use in the prophylactic treatment by factor VIII of patients with severe or moderate Haemophilia A with a severe clinical profile. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haemophilia A
- What is being tested?
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- How many people
- 69 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Oct 2011to Jul 2015
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: * Patient with severe haemophilia A defined as residual factor VIII activity < 1% * or Patient with moderate haemophilia A (residual factor VIII activity >1% and <5%) with clinical signs of frequent, severe bleeding episodes * Patient treated prophylatically with the same plasma or recombinant factor VIII for at least 6 months
Who may be able to join
- Patient with severe haemophilia A defined as residual factor VIII activity < 1%
- or Patient with moderate haemophilia A (residual factor VIII activity >1% and <5%) with severe clinical profile
- Patient treated in prevention with the same plasma or recombinant factor VIII for at least 6 months
- Patient whose inclusion visit is performed during a routine visit including a measurement of plasma residual factor VIII :c level
- In the medical file (retrospective data) the pharmacokinetic parameters are available and have been obtained from a PK analysis of factor VIII level performed with the same anti haemophilic factor than the one used on the inclusion day
Who cannot join
- Patients with haemophilia B
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Validation Study of the OPTIMS Pharmacokinetic Calculator for Clinician Use in Prophylactic Treatment of Patients With Haemophilia A.
OPTIMS is a non interventional validation study of the calculator developed by Bayer for clinician's use in the prophylactic treatment by factor VIII of patients with severe or moderate Haemophilia A with a severe clinical profile.
The study takes place during a single visit, at the time of patient enrollment in the study
- Conditions
- Haemophilia A
- Being tested
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Keywords
- Haemophilia A, Factor VIII, Pharmacokinetic, Computer Tool validation
- 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
Prophylactic treatment : 20-40 UI /Kg administered all 2 in 3 days .
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | 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.
The difference of the recovery rate calculated by the physician and the recovery rate calculated by an independent calculator With OPTIMS Tool
Measured: after 1 month
The difference of the elimination half-life calculated by the physician and the ones calculated by an independent calculator With OPTIMS Tool
Measured: after 1 month
The difference of the clearance calculated by the physician and the ones calculated by an independent calculator With OPTIMS Tool
Measured: after 1 month
Secondary outcomes (2)
The dosage of factor VIII calculated by OPTIMS calculator
Measured: within 48 hours after enrollment
The practicality of the OPTIMS calculator by a physician's satisfaction questionnaire
Measured: within 48 hours after enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2011 (estimated)
- Study startOct 2011
- Primary completionJun 2014
- Study completionJul 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia 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
- 69 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
- NCT01436825
- Sponsor's protocol ID
- 15453
- Other IDs
- KG110FR
- Acronym
- OPTIMS
- Status last verified
- Aug 2015
- First submitted
- 14 Sep 2011
- Last updated
- 10 Aug 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Validation Study of the OPTIMS Pharmacokinetic Calculator for Clinician Use in Prophylactic Treatment of Patients With Haemophilia A.
- ClinicalTrials.gov ID
- NCT01436825
- Acronym / sponsor ID
- OPTIMS / 15453
- Conditions
- Haemophilia A
- Keywords
- Haemophilia A, Factor VIII, Pharmacokinetic, Computer Tool validation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 69 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- Oct 2011
- Primary completion
- Jun 2014 (actual)
- Study completion
- Jul 2015 (actual)
- First posted
- 20 Sep 2011 (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.
OPTIMS is a non interventional validation study of the calculator developed by Bayer for clinician's use in the prophylactic treatment by factor VIII of patients with severe or moderate Haemophilia A with a severe clinical profile.
The study takes place during a single visit, at the time of patient enrollment in the study
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Group 1 | – | Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- * Patient with severe haemophilia A defined as residual factor VIII activity < 1% * or Patient with moderate haemophilia A (residual factor VIII activity >1% and <5%) with clinical signs of frequent, severe bleeding episodes * Patient treated prophylatically with the same plasma or recombinant factor VIII for at least 6 months
Key inclusion criteria (5 listed)
- Patient with severe haemophilia A defined as residual factor VIII activity < 1%
- or Patient with moderate haemophilia A (residual factor VIII activity >1% and <5%) with severe clinical profile
- Patient treated in prevention with the same plasma or recombinant factor VIII for at least 6 months
- Patient whose inclusion visit is performed during a routine visit including a measurement of plasma residual factor VIII :c level
- In the medical file (retrospective data) the pharmacokinetic parameters are available and have been obtained from a PK analysis of factor VIII level performed with the same anti haemophilic factor than the one used on the inclusion day
Key exclusion criteria (1 listed)
- Patients with haemophilia B
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)Prophylactic treatment : 20-40 UI /Kg administered all 2 in 3 days . | Drug | Group 1 |
Primary outcome
The difference of the recovery rate calculated by the physician and the recovery rate calculated by an independent calculator With OPTIMS Tool
Time frame: after 1 month
The difference of the elimination half-life calculated by the physician and the ones calculated by an independent calculator With OPTIMS Tool
Time frame: after 1 month
The difference of the clearance calculated by the physician and the ones calculated by an independent calculator With OPTIMS Tool
Time frame: after 1 month
2 secondary outcomes
The dosage of factor VIII calculated by OPTIMS calculator
Time frame: within 48 hours after enrollment
The practicality of the OPTIMS calculator by a physician's satisfaction questionnaire
Time frame: within 48 hours after enrollment
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 10 Aug 2015.
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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
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Haemophilia A, run by Bayer at 2 sites in 2 countries.
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 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 10 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France, 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?
The difference of the recovery rate calculated by the physician and the recovery rate calculated by an independent calculator With OPTIMS Tool (time frame: after 1 month). The study lists 3 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 10 Aug 2015.