Hemophilia AOBTCNCT02634424
PK Driven Prophylaxis for Hemophilia A
A study testing MyPKFiT in people with Hemophilia A, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The purpose of this study is to evaluate the implementation of pharmacokinetic (PK) driven prophylaxis in current clinical practice Read more about this study
- Who is it for?
- Ages 6 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- MyPKFiT
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteFrance
- Study dates
- 25 Nov 2015to 12 May 2016
- Phase
- Interventional
- 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 6 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male Patient
- Severe or moderately severe haemophilia A (FVIII = 2 %), preventively treated (processing) by ADVATE for at least 6 months,
- At last 6 years old,
- An informed consent must be signed by the patient or his legal representative for the patients minor.
- Affiliated to a national insurance scheme
Who cannot join
- Previous or actual treatment with FVIII inhibitors > 0,6 UB at the selection
- Induction of immune tolerance
- Planned orthopedic surgery for the 18 next months
- Any other haemostatic pathology
- Any treatment interacting on the haemostasis
- Patient under guardianship
- Patient participating in another biomedical research
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Interest of Preventive Personalized Treatment in Hemophilia A
The purpose of this study is to evaluate the implementation of pharmacokinetic (PK) driven prophylaxis in current clinical practice
Read the detailed description
Starting from 3 pharmacokinetic (PK) points, data are entered in the PK calculator device, and treatment dose is adjusted following the results of PK values obtained and clinical bleeding data
- Conditions
- Hemophilia A
- Being tested
- MyPKFiT
- Keywords
- Hemophilia A, Prophylaxy, Pharmacokinetic, Bayesian
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MyPKFiTDevice
Also called Personalized prophylaxis by PK modeling
Adjustment of the treatment dose using a decision support software according to the PK values
| Study group | Role | What they receive |
|---|---|---|
| MyPKFiT | Experimental | Personalized prophylaxis : Treatment is adjustment according to PK modeling Receives: Device: MyPKFiT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Joint Annual Bleeding Rate
Joint Annual Bleeding Rate assessed by usual patient book
Measured: during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Secondary outcomes (7)
Total annual bleeding rate
Total Annual Bleeding Rate assessed by usual patient book
Measured: during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Quality of life
Quality of life assessed by QoL-A-Haemo questionnaire for Adult and Haemo-QoL for children aged 6-17 years
Measured: up to 12 months
Joint function
Joint function assessed thanks to score of joint state "Haemophilia Joint Health Score" (HJHS)
Measured: up to 12 months
Adherence to treatment estimated as the quantity of Facteur VIII consumed reported to the prescribed quantity.
Quantity of Facteur VIII consumed will be assessed by usual patient book
Measured: up to 6 and 12 months
Consumption of Factor VIII estimated by the Quantity of Facteur VIII consumed by thr patient.
Consumption of Factor VIII assessed by usual patient book
Measured: during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Hemorrhagic Risk Sport
Hemorrhagic Risk Sport assessed by the scale "Hemophilia and sport"
Measured: up to 6 and 12 months
Evolution between the annualized incidence of intercurrent events appeared or worsened under each treatment period (with and without myPKFit), globally, by System Organ ( SO) and Preferred Term ( PT) of the code MedDRA
Measured: during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Dec 2015 (estimated)
- Study start25 Nov 2015
- Primary completion12 May 2016
- Study completion12 May 2016
Why it stopped: lack of inclusion and prophylaxy plateform being free to use for everyone in septembre 2016
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 Assistance Publique - Hôpitaux de ParisThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Baxter Healthcare Corporation
- Responsible party
- Sponsor
- Investigators
- Thierry LAMBERT, MD, Principal investigator, AP-HP, Bicêtre Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02634424
- Sponsor's protocol ID
- P140701
- Acronym
- OBTC
- Status last verified
- Aug 2016
- First submitted
- 8 Dec 2015
- Last updated
- 16 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Interest of Preventive Personalized Treatment in Hemophilia A
- ClinicalTrials.gov ID
- NCT02634424
- Acronym / sponsor ID
- OBTC / P140701
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, Prophylaxy, Pharmacokinetic, Bayesian
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: lack of inclusion and prophylaxy plateform being free to use for everyone in septembre 2016
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Baxter Healthcare Corporation
- Study start
- 25 Nov 2015 (actual)
- Primary completion
- 12 May 2016 (actual)
- Study completion
- 12 May 2016 (actual)
- First posted
- 18 Dec 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.
The purpose of this study is to evaluate the implementation of pharmacokinetic (PK) driven prophylaxis in current clinical practice
Detailed description
Starting from 3 pharmacokinetic (PK) points, data are entered in the PK calculator device, and treatment dose is adjusted following the results of PK values obtained and clinical bleeding data
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MyPKFiT | Experimental | Device: MyPKFiTPersonalized prophylaxis : Treatment is adjustment according to PK modeling |
Who may qualify
- Ages
- 6 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male Patient
- Severe or moderately severe haemophilia A (FVIII = 2 %), preventively treated (processing) by ADVATE for at least 6 months,
- At last 6 years old,
- An informed consent must be signed by the patient or his legal representative for the patients minor.
- Affiliated to a national insurance scheme
Key exclusion criteria (7 listed)
- Previous or actual treatment with FVIII inhibitors > 0,6 UB at the selection
- Induction of immune tolerance
- Planned orthopedic surgery for the 18 next months
- Any other haemostatic pathology
- Any treatment interacting on the haemostasis
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 |
|---|---|---|
| MyPKFiTAlso: Personalized prophylaxis by PK modelingAdjustment of the treatment dose using a decision support software according to the PK values | Device | MyPKFiT |
Primary outcome
Joint Annual Bleeding Rate
Time frame: during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Joint Annual Bleeding Rate assessed by usual patient book
7 secondary outcomes
Total annual bleeding rate
Time frame: during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Quality of life
Time frame: up to 12 months
Joint function
Time frame: up to 12 months
Adherence to treatment estimated as the quantity of Facteur VIII consumed reported to the prescribed quantity.
Time frame: up to 6 and 12 months
Consumption of Factor VIII estimated by the Quantity of Facteur VIII consumed by thr patient.
Time frame: during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Hemorrhagic Risk Sport
Time frame: up to 6 and 12 months
Evolution between the annualized incidence of intercurrent events appeared or worsened under each treatment period (with and without myPKFit), globally, by System Organ ( SO) and Preferred Term ( PT) of the code MedDRA
Time frame: during 6 months before (retrospectively) and up to 12 months after using of myPKFiT
Results and safety
The study was stopped early (reason given: lack of inclusion and prophylaxy plateform being free to use for everyone in septembre 2016). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 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.
Thierry LAMBERT, MD principal investigator
AP-HP, Bicêtre Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing MyPKFiT in people with Hemophilia A, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 7 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: stopped early, as of the sponsor's last update on 16 Mar 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 France. 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 was stopped early (reason given: lack of inclusion and prophylaxy plateform being free to use for everyone in septembre 2016). 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?
Joint Annual Bleeding Rate (time frame: during 6 months before (retrospectively) and up to 12 months after using of myPKFiT). 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 16 Mar 2020.