Hemophilia ANCT02750085
Evaluation of a Simple Pharmacokinetic Tool (myPKFiT™) to Guide Personalized Factor VIII Dosing in Patients With Hemophilia
An observational study of Hemophilia A, run by Victor Blanchette.
Trial summary
This is an investigator-initiated, industry-funded, multi-centre, international study that will be carried out prospectively at hemophilia treatment centres across Canada, the Czech Republic and Australia with SickKids as the coordinating site. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- How many people
- 39 participants
- Where
- No sites listed yet
- Study dates
- Apr 2016to Dec 2020
- 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: Patients with severe Hemophilia A
Who may be able to join
- Confirmed diagnosis of Hemophilia A;
- Severe disease (FVIII <2%);
- Receiving ADVATE for prevention of bleeding (prophylaxis) or receiving ADVATE on demand and a candidate for prophylaxis;
- Body weight ≤120 kg; and ≥12kg;
Who cannot join
- FVIII inhibitor positive (level of ≥0.6 Bethesda Units [BU] per mL using the Nijmegen modification of the Bethesda assay). Inhibitor status to be documented as negative prior to study enrollment according to the two most recent, consecutive inhibitor assays on record. If patients have < 50 exposure days, an assay will be completed centrally within a reasonable timeframe (approximately 8 weeks suggested) to make sure that they are negative.
- Body weight >120 kg or <12kg;
- Human immunodeficiency virus (HIV) positivity with cluster of differentiation 4 (CD4) count < 200 / microliter;
- Significant hepatic dysfunction, alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >5 times upper limit of normal
- History of recent events that might affect FVIII half-life (e.g., infection, surgery or an invasive procedure) within 2 weeks of blood sampling.
About this study
This is an investigator-initiated, industry-funded, multi-centre, international study that will be carried out prospectively at hemophilia treatment centres across Canada, the Czech Republic and Australia with SickKids as the coordinating site. The study will use a central laboratory not directly affiliated with any of the participating sites. Enrollment target is 50 participants, both adult and pediatric with severe hemophilia A receiving Advate, who will each complete a 2-point and 6-point pharmacokinetic (PK) sampling. The main aim is to compare the results of a 2 sample PK using clinically practical time points and myPKFiT™ (a web-based, population PK Bayesian tool) to a 6 sample population PK to determine whether the results obtained are in good agreement.
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, pharmacokinetics, myPKFiT
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 2-point and 6-point PK sampling | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Terminal half-life (t1/2)
2-point PK sampling protocol against 6-point PK sampling protocol
Measured: 2 years
area under the plasma concentration versus time curve (AUC)
2-point PK sampling protocol against 6-point PK sampling protocol
Measured: 2 years
Area under the moment curve (AUMC)
2-point PK sampling protocol against 6-point PK sampling protocol
Measured: 2 years
In vivo recovery (IVR)
2-point PK sampling protocol against 6-point PK sampling protocol
Measured: 2 years
Maximum concentration (Cmax)
2-point PK sampling protocol against 6-point PK sampling protocol
Measured: 2 years
Clearance (Cl)
2-point PK sampling protocol against 6-point PK sampling protocol
Measured: 2 years
Volume of distribution at steady state (Vss)
2-point PK sampling protocol against 6-point PK sampling protocol
Measured: 2 years
Mean residence time (MRT)
2-point PK sampling protocol against 6-point PK sampling protocol
Measured: 2 years
Time to factor VIII concentration of 1% over baseline
2-point PK sampling protocol against 6-point PK sampling protocol
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2016 (estimated)
- Study startApr 2016
- Primary completionJan 2019
- Study completionDec 2020 (estimated)
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.
- 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Victor Blanchette
- Sponsor type
- University, hospital or other
- Collaborators
- St. Paul's Hospital, Montreal Children's Hospital of the MUHC, Queen's University, University Hospital, Motol, Brno University Hospital, Royal Children's Hospital, Sydney Children's Hospitals Network, Royal Prince Alfred Hospital, Sydney, Australia
- Responsible party
- Sponsor-investigator, Victor Blanchette, The Hospital for Sick Children
- Investigators
- Victor S Blanchette, MD, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02750085
- Sponsor's protocol ID
- myPKFiT™ Study
- Status last verified
- May 2020
- First submitted
- 7 Apr 2016
- Last updated
- 15 May 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of a Simple Pharmacokinetic Tool (myPKFiT™) to Guide Personalized Factor VIII Dosing in Patients With Hemophilia
- ClinicalTrials.gov ID
- NCT02750085
- Acronym / sponsor ID
- myPKFiT™ Study
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, pharmacokinetics, myPKFiT
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Victor BlanchetteUniversity, hospital or other
- Collaborators
- St. Paul's Hospital, Montreal Children's Hospital of the MUHC, Queen's University, University Hospital, Motol, Brno University Hospital, Royal Children's Hospital, Sydney Children's Hospitals Network, Royal Prince Alfred Hospital, Sydney, Australia
- Study start
- Apr 2016 (actual)
- Primary completion
- Jan 2019 (actual)
- Study completion
- Dec 2020 (estimated)
- First posted
- 25 Apr 2016 (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 is an investigator-initiated, industry-funded, multi-centre, international study that will be carried out prospectively at hemophilia treatment centres across Canada, the Czech Republic and Australia with SickKids as the coordinating site. The study will use a central laboratory not directly affiliated with any of the participating sites. Enrollment target is 50 participants, both adult and pediatric with severe hemophilia A receiving Advate, who will each complete a 2-point and 6-point pharmacokinetic (PK) sampling. The main aim is to compare the results of a 2 sample PK using clinically practical time points and myPKFiT™ (a web-based, population PK Bayesian tool) to a 6 sample population PK to determine whether the results obtained are in good agreement.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 2-point and 6-point PK sampling | – | – |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with severe Hemophilia A
Key inclusion criteria (4 listed)
- Confirmed diagnosis of Hemophilia A;
- Severe disease (FVIII <2%);
- Receiving ADVATE for prevention of bleeding (prophylaxis) or receiving ADVATE on demand and a candidate for prophylaxis;
- Body weight ≤120 kg; and ≥12kg;
Key exclusion criteria (5 listed)
- FVIII inhibitor positive (level of ≥0.6 Bethesda Units [BU] per mL using the Nijmegen modification of the Bethesda assay). Inhibitor status to be documented as negative prior to study enrollment according to the two most recent, consecutive inhibitor assays on record. If patients have < 50 exposure days, an assay will be completed centrally within a reasonable timeframe (approximately 8 weeks suggested) to make sure that they are negative.
- Body weight >120 kg or <12kg;
- Human immunodeficiency virus (HIV) positivity with cluster of differentiation 4 (CD4) count < 200 / microliter;
- Significant hepatic dysfunction, alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >5 times upper limit of normal
- History of recent events that might affect FVIII half-life (e.g., infection, surgery or an invasive procedure) within 2 weeks of blood sampling.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Terminal half-life (t1/2)
Time frame: 2 years
2-point PK sampling protocol against 6-point PK sampling protocol
area under the plasma concentration versus time curve (AUC)
Time frame: 2 years
2-point PK sampling protocol against 6-point PK sampling protocol
Area under the moment curve (AUMC)
Time frame: 2 years
2-point PK sampling protocol against 6-point PK sampling protocol
In vivo recovery (IVR)
Time frame: 2 years
2-point PK sampling protocol against 6-point PK sampling protocol
Maximum concentration (Cmax)
Time frame: 2 years
2-point PK sampling protocol against 6-point PK sampling protocol
Clearance (Cl)
Time frame: 2 years
2-point PK sampling protocol against 6-point PK sampling protocol
Volume of distribution at steady state (Vss)
Time frame: 2 years
2-point PK sampling protocol against 6-point PK sampling protocol
Mean residence time (MRT)
Time frame: 2 years
2-point PK sampling protocol against 6-point PK sampling protocol
Time to factor VIII concentration of 1% over baseline
Time frame: 2 years
2-point PK sampling protocol against 6-point PK sampling protocol
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2020.
- Individual participant dataSharing planned: No
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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.
Victor S Blanchette, MD principal investigator
The Hospital for Sick Children
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Victor Blanchette.
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 4 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 15 May 2020. Status can change between updates, so confirm with the study contact before making plans.
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?
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?
Terminal half-life (t1/2) (time frame: 2 years). The study lists 9 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 15 May 2020.