Hemophilia ANCT03006965
Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®
An observational study of Hemophilia A and Hemophilia, run by Spanish Society of Thrombosis and Haemostasis at 14 sites in Spain.
Trial summary
The purpose of this study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis in Spain using myPKFit® Read more about this study
- Who is it for?
- Ages 1 year to 65 years, men only
- Conditions the study is for
- Hemophilia A, Hemophilia, Factor VIII Deficiency
- What is being tested?
- octocog alfa and rurioctocog alfa pegol
- Type of treatment
- Injection or infusion
- How many people
- 50 participants
- Where
- 14 sitesSpain
- Study dates
- 11 Nov 2016to 22 Dec 2022
- 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 1 year to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients diagnosed with hemophilia A on prophylactic treatment with Advate® or Adynovi®
Who may be able to join
- Male patients.
- Age between 1 and 65 years old.
- Patients diagnosed with Hemophilia A who are on prophylactic treatment with Advate® or Adynovi® or adjusted with myPKFIT.
- Patients older than 18 who have signed the informed consent form.
- In the case of mature minors, in addition to the consent signed by the legal guardian, an assent of the minor must be obtain.
- In the case of patients legally incapable for giving their consent, their primary caregiver will consent as the patient's legal guardian.
Who cannot join
- Withdrawal of informed consent.
- Patients with any medical or psychological condition that according to the researcher's criteria prevents them from following the usual clinical practice procedures.
- Patients with concomitant diagnosis of other haemostasis disorders.
- Patients being treated for induction of immunologic tolerance at the time of inclusion.
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
Spain14 sites
Complexo Hospitalario Universitario A Coruña
A Coruña, A Coruña, 15006
Hospital Universitario Cruces
Barakaldo, Vizcaya, 48903
Hospital Vall D'Hebron
Barcelona, 08035
Hospital Universitario Virgen de la Arrixaca
El Palmar, Murcia, 30120
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, 08950
Hospital Universitario La Paz
Madrid, 28046
Hospital Regional Universitario de Málaga
Málaga, 29010
Hospital Materno-infantil de Málaga
Málaga, 29011
Hospital Universitario Central de Asturias
Oviedo, Principality of Asturias, 33011
Hospital Universitario Son Espases
Palma, Balearic Islands, 07120
Hospital Virgen del Camino
Pamplona, Navarre, 31008
Hospital Universitario Virgen del Rocío
Seville, 41013
Hospital Universitari i Politècnic La Fe
Valencia, 46026
Hospital Xeral de Vigo
Vigo, Pontevedra, 36212
About this study
Official title: Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using an Online Medical Application Based on a Published Population Model and a Bayesian Algorithm
The purpose of this study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis in Spain using myPKFit®
Read the detailed description
This pharmacokinetic profile will be obtained in only in patient using Advate® (INN-octocog alfa) or Adynovi® (INN- rurioctocog alfa pegol), both recombinant intravenous FVIII products, with myPKFiT®. This patients will be followed during the period of 12 months, and the pharmacokinetic will be measured collecting at least 2 blood samples, in which the presence of factor VIII will be determined by the usual clinical methods: chromogenic method or coagulative method.
- Conditions
- Hemophilia A, Hemophilia, Factor VIII Deficiency
- Being tested
- octocog alfa, rurioctocog alfa pegol
- Keywords
- Hemophilia, Hemophilia A, Factor VIII, Advate, Antithrombotic factor, Pharmacokinetic dosing, Hemarthrosis, myPKFiT
- Medical subject headings
- Hemophilia A, Hemarthrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
octocog alfaDrug
Also called Advate®, recombinant intravenous factor VIII
Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software
rurioctocog alfa pegolDrug
Also called recombinant intravenous factor VIII
Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A patients | Group of patients in prophylactic treatment with Advate® (octocog alfa) or Adynovi® (rurioctocog alfa pegol), or patients using already myPKFit®.
Patients will be given a dose of octocog alfa or rurioctocog alfa pegol according to usual clinical practice, and two blood samples will be taken in case of octocog alfa: one sample will be extracted 3-4h postdose (+/- 30 minutes), and the second sample will be extracted 24-32h postdose (+/- 60 minutes). In case of rurioctocog alfa pegol, the first sample is taken in the same conditions than octocog alfa, and the second sample will be extracted 48h postdose (+/- 120 minutes), and other sample post 72h(+/- 120 minutes) optional. Receives: Drug: octocog alfa; Drug: rurioctocog alfa pegol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
Change from Baseline to 12 months
Measured: 3-4 hours postdose +/- 30 minutes
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
Change from Baseline to 12 months
Measured: 24-32 hours postdose +/- 60 minutes
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
Change from Baseline to 12 months
Measured: 48 hours postdose +/- 60 minutes
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
Change from Baseline to 12 months
Measured: 72 hours postdose
Pharmacokinetic profile: Clearance measured with MyPKFiT
Change from Baseline to 12 months
Measured: 3-4 hours postdose +/- 30 minutes
Pharmacokinetic profile: Clearance measured with MyPKFiT
Change from Baseline to 12 months
Measured: 24-32 hours postdose +/- 60 minutes
Pharmacokinetic profile: Clearance measured with MyPKFiT
Change from Baseline to 12 months
Measured: 48 hours postdose +/- 60 minutes
Pharmacokinetic profile: Clearance measured with MyPKFiT
Change from Baseline to 12 months
Measured: 72 hours postdose
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
Change from Baseline to 12 months
Measured: 3-4 hours postdose +/- 30 minutes
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
Change from Baseline to 12 months
Measured: 24-32 hours postdose +/- 60 minutes
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
Change from Baseline to 12 months
Measured: 48 hours postdose +/- 60 minutes
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
Change from Baseline to 12 months
Measured: 72 hours postdose
Secondary outcomes (11)
Hemorrhagic risk of physical activity
Measured with Broderick scale
Measured: baseline
Hemorrhagic risk of physical activity
Measured with Broderick scale
Measured: month 12
Joint condition
Hemophilia Joint Health Score
Measured: baseline
Joint condition
Hemophilia Joint Health Score
Measured: month 12
Number of total bleeding episodes in a year
Measured by annual bleeding record
Measured: baseline
Number of total bleeding episodes in a year
Measured by annual bleeding record
Measured: month 12
Annual Joint Bleeding Record
Hemophilia Joint Health Score
Measured: baseline
Annual Joint Bleeding Record
Hemophilia Joint Health Score
Measured: month 12
Self-adherence to treatment evaluation
Measured by VERITAS- PRO scale
Measured: month 12
Adverse Events
Collect and identify adverse events
Measured: baseline
Adverse Events
Collect and identify adverse events
Measured: month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2016 (estimated)
- Study start11 Nov 2016
- Primary completion22 Dec 2022
- Study completion22 Dec 2022
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
- Cohort
- Time perspective
- Other
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Spanish Society of Thrombosis and Haemostasis
- Sponsor type
- University, hospital or other
- Collaborators
- Takeda, Instituto de Investigación Hospital Universitario La Paz
- Responsible party
- Sponsor
- Investigators
- M Teresa Álvarez Román, MD,PhD, Principal investigator, Hospital Universitario La Paz, IdiPAZ, Universidad Autónoma de Madrid
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03006965
- Sponsor's protocol ID
- PK HemoA-SP
- Other IDs
- STH-OCT-2016-01
- Status last verified
- Jun 2023
- First submitted
- 22 Dec 2016
- Last updated
- 8 Jun 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using an Online Medical Application Based on a Published Population Model and a Bayesian Algorithm
- ClinicalTrials.gov ID
- NCT03006965
- Acronym / sponsor ID
- PK HemoA-SP
- Conditions
- Hemophilia A, Hemophilia, Factor VIII Deficiency
- Keywords
- Hemophilia, Hemophilia A, Factor VIII, Advate, Antithrombotic factor, Pharmacokinetic dosing, Hemarthrosis, myPKFiT
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Spanish Society of Thrombosis and HaemostasisUniversity, hospital or other
- Collaborators
- Takeda, Instituto de Investigación Hospital Universitario La Paz
- Study start
- 11 Nov 2016 (actual)
- Primary completion
- 22 Dec 2022 (actual)
- Study completion
- 22 Dec 2022 (actual)
- First posted
- 30 Dec 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.
The purpose of this study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis in Spain using myPKFit®
Detailed description
This pharmacokinetic profile will be obtained in only in patient using Advate® (INN-octocog alfa) or Adynovi® (INN- rurioctocog alfa pegol), both recombinant intravenous FVIII products, with myPKFiT®. This patients will be followed during the period of 12 months, and the pharmacokinetic will be measured collecting at least 2 blood samples, in which the presence of factor VIII will be determined by the usual clinical methods: chromogenic method or coagulative method.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A patients | – | Drug: octocog alfa, Drug: rurioctocog alfa pegolGroup of patients in prophylactic treatment with Advate® (octocog alfa) or Adynovi® (rurioctocog alfa pegol), or patients using already myPKFit®. Patients will be given a dose of octocog alfa or rurioctocog alfa pegol according to usual clinical practice, and two blood samples will be taken in case of octocog alfa: one sample will be extracted 3-4h postdose (+/- 30 minutes), and the second sample will be extracted 24-32h postdose (+/- 60 minutes). In case of rurioctocog alfa pegol, the first sample is taken in the same conditions than octocog alfa, and the second sample will be extracted 48h postdose (+/- 120 minutes), and other sample post 72h(+/- 120 minutes) optional. |
Who may qualify
- Ages
- 1 year to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients diagnosed with hemophilia A on prophylactic treatment with Advate® or Adynovi®
Key inclusion criteria (6 listed)
- Male patients.
- Age between 1 and 65 years old.
- Patients diagnosed with Hemophilia A who are on prophylactic treatment with Advate® or Adynovi® or adjusted with myPKFIT.
- Patients older than 18 who have signed the informed consent form.
- In the case of mature minors, in addition to the consent signed by the legal guardian, an assent of the minor must be obtain.
Key exclusion criteria (4 listed)
- Withdrawal of informed consent.
- Patients with any medical or psychological condition that according to the researcher's criteria prevents them from following the usual clinical practice procedures.
- Patients with concomitant diagnosis of other haemostasis disorders.
- Patients being treated for induction of immunologic tolerance at the time of inclusion.
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 |
|---|---|---|
| octocog alfaAlso: Advate®, recombinant intravenous factor VIIIPharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software | Drug | Hemophilia A patients |
| rurioctocog alfa pegolAlso: recombinant intravenous factor VIIIPharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software | Drug | Hemophilia A patients |
Primary outcome
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
Time frame: 3-4 hours postdose +/- 30 minutes
Change from Baseline to 12 months
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
Time frame: 24-32 hours postdose +/- 60 minutes
Change from Baseline to 12 months
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
Time frame: 48 hours postdose +/- 60 minutes
Change from Baseline to 12 months
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
Time frame: 72 hours postdose
Change from Baseline to 12 months
Pharmacokinetic profile: Clearance measured with MyPKFiT
Time frame: 3-4 hours postdose +/- 30 minutes
Change from Baseline to 12 months
Pharmacokinetic profile: Clearance measured with MyPKFiT
Time frame: 24-32 hours postdose +/- 60 minutes
Change from Baseline to 12 months
Pharmacokinetic profile: Clearance measured with MyPKFiT
Time frame: 48 hours postdose +/- 60 minutes
Change from Baseline to 12 months
Pharmacokinetic profile: Clearance measured with MyPKFiT
Time frame: 72 hours postdose
Change from Baseline to 12 months
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
Time frame: 3-4 hours postdose +/- 30 minutes
Change from Baseline to 12 months
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
Time frame: 24-32 hours postdose +/- 60 minutes
Change from Baseline to 12 months
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
Time frame: 48 hours postdose +/- 60 minutes
Change from Baseline to 12 months
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
Time frame: 72 hours postdose
Change from Baseline to 12 months
11 secondary outcomes
Hemorrhagic risk of physical activity
Time frame: baseline
Hemorrhagic risk of physical activity
Time frame: month 12
Joint condition
Time frame: baseline
Joint condition
Time frame: month 12
Number of total bleeding episodes in a year
Time frame: baseline
Number of total bleeding episodes in a year
Time frame: month 12
Annual Joint Bleeding Record
Time frame: baseline
Annual Joint Bleeding Record
Time frame: month 12
Self-adherence to treatment evaluation
Time frame: month 12
Adverse Events
Time frame: baseline
Adverse Events
Time frame: month 12
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 8 Jun 2023.
- Individual participant dataSharing planned: Undecided
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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.
M Teresa Álvarez Román, MD,PhD principal investigator
Hospital Universitario La Paz, IdiPAZ, Universidad Autónoma de Madrid
14 sites 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 and Hemophilia, run by Spanish Society of Thrombosis and Haemostasis at 14 sites in Spain.
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 6 inclusion and 4 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 8 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in Spain. 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?
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT (time frame: 3-4 hours postdose +/- 30 minutes). The study lists 12 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 8 Jun 2023.