HaemophiliaInPathNCT03287999
Inter Individual Variability in Initiation Pathway Activation and Regulation and Phenotypic Heterogeneity in Patients With Haemophilia A and B
An observational study of Haemophilia, run by Royal Free Hospital NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
Severe haemophilia A and B (SHA, SHB) are X - linked inherited bleeding disorders, characterised by factor VIII and IX levels of <1 IU/dL respectively. The mainstay of treatment in SHA and SHB is replacement therapy with intravenous infusions of factor VIII and IX. Read more about this study
- Who is it for?
- Ages 18 years and older, men onlyHealthy volunteers can take part
- Conditions the study is for
- Haemophilia
- What is being tested?
- Thrombophilia screen and Initiation pathway analysis
- How many people
- 250 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 19 Sep 2017to 2 Oct 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 18 years and older
- Men only
- Healthy volunteers accepted
- Adult, Older adult
Study population: The study population will include patients severe or moderate haemophilia A or B who are currently attending study sites for routine follow up visits. In addition, 10 healthy volunteers will also be recruited to act as controls
Who may be able to join
- Patients with haemophilia A or B (baseline FVIII/FIX level <30%)
- Age ≥ 18 years
- Written informed consent in accordance with local and institutional guidelines.
- Currently not receiving any antiplatelet or anticoagulant therapy or other drugs that can affect the coagulation system.
- Age ≥ 18 years
- Written informed consent in accordance with local and institutional guidelines.
Who cannot join
- 1. Patients currently enrolled into a clinical trial of investigational medicinal product for haemophilia.
- Healthy Volunteers
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site, 1 recruiting
- Recruiting
Royal Free Hospital
London
Thomas Roberts
Pratima Chowdary
About this study
Severe haemophilia A and B (SHA, SHB) are X - linked inherited bleeding disorders, characterised by factor VIII and IX levels of <1 IU/dL respectively. The mainstay of treatment in SHA and SHB is replacement therapy with intravenous infusions of factor VIII and IX. However, there is significant variability in the bleeding phenotype within severe haemophiliacs with some presenting with minimal bleeding episodes even on less intensive treatment regimens. A significant contributor to inter-individual variability in the bleeding phenotype is the coagulation phenotype, but there are no established assays in routine clinical practice that can be used to quantify this. This study aims to study novel assays and characterise the observed phenotypic heterogeneity.
- Conditions
- Haemophilia
- Being tested
- Thrombophilia screen, Initiation pathway analysis
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thrombophilia screenDiagnostic test
Thrombophilia screen (including antithrombin activity (AT:Ac), protein S antigen (PS:free), protein C activity (PC:Ac) , genetic analysis for FV Leiden and Prothrombin 3'UTR mutations and screening for lupus anticoagulant.
Initiation pathway analysisDiagnostic test
Evaluation of inter-individual variability in regulation of TF.VIIa.Xa.TFPI complex (tissue factor, activated Factor VII, activated factor X, tissue factor pathway inhibitor)
| Study group | Role | What they receive |
|---|---|---|
| Haemophilia patients | Persons with haemophilia A or B - 240 to be recruited Receives: Diagnostic Test: Thrombophilia screen; Diagnostic Test: Initiation pathway analysis | |
| Healthy volunteers | Healthy volunteers - 10 to be recruited Receives: Diagnostic Test: Thrombophilia screen; Diagnostic Test: Initiation pathway analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Initiation pathway correlation with clinical phenotype
Correlate lab assays that characterise initiation pathway with clinical phenotype.
Measured: Within 18 months of consent
Secondary outcomes (3)
Correlation analysis between FVIII:C/FIX:C levels and whole blood clotting time, thrombin generation in platelet poor plasma.
Measured: Within 18 months of consent
Evaluation the sensitivity and specificity of global assays for disease severity and clinical phenotype.
Measured: Within 18 months of consent
Correlation analysis between activation threshold of initiation pathway to thrombin generation and clinical phenotype
Measured: Within 18 months of consent
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2017
- Study start19 Sep 2017
- Primary completion2 Oct 2019 (estimated)
- Study completion2 Oct 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HaemophiliaStudies 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
- Prospective
- Enrolment
- 250 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Royal Free Hospital NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Pratima Chowdary, Royal Free Hospital NHS Foundation Trust
- Investigators
- Pratima Chowdary, Principal investigator, Royal Free Hospitals NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03287999
- Sponsor's protocol ID
- 11296
- Acronym
- InPath
- Status last verified
- Mar 2019
- First submitted
- 13 Sep 2017
- Last updated
- 27 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Inter Individual Variability in Initiation Pathway Activation and Regulation and Phenotypic Heterogeneity in Patients With Haemophilia A and B
- ClinicalTrials.gov ID
- NCT03287999
- Acronym / sponsor ID
- InPath / 11296
- Conditions
- Haemophilia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- Royal Free Hospital NHS Foundation TrustUniversity, hospital or other
- Study start
- 19 Sep 2017 (actual)
- Primary completion
- 2 Oct 2019 (estimated)
- Study completion
- 2 Oct 2020 (estimated)
- First posted
- 19 Sep 2017 (actual)
- 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.
Severe haemophilia A and B (SHA, SHB) are X - linked inherited bleeding disorders, characterised by factor VIII and IX levels of <1 IU/dL respectively. The mainstay of treatment in SHA and SHB is replacement therapy with intravenous infusions of factor VIII and IX. However, there is significant variability in the bleeding phenotype within severe haemophiliacs with some presenting with minimal bleeding episodes even on less intensive treatment regimens. A significant contributor to inter-individual variability in the bleeding phenotype is the coagulation phenotype, but there are no established assays in routine clinical practice that can be used to quantify this. This study aims to study novel assays and characterise the observed phenotypic heterogeneity.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Haemophilia patients | – | Diagnostic Test: Thrombophilia screen, Diagnostic Test: Initiation pathway analysisPersons with haemophilia A or B - 240 to be recruited |
| Healthy volunteers | – | Diagnostic Test: Thrombophilia screen, Diagnostic Test: Initiation pathway analysisHealthy volunteers - 10 to be recruited |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- The study population will include patients severe or moderate haemophilia A or B who are currently attending study sites for routine follow up visits. In addition, 10 healthy volunteers will also be recruited to act as controls
Key inclusion criteria (6 listed)
- Patients with haemophilia A or B (baseline FVIII/FIX level <30%)
- Age ≥ 18 years
- Written informed consent in accordance with local and institutional guidelines.
- Currently not receiving any antiplatelet or anticoagulant therapy or other drugs that can affect the coagulation system.
- Age ≥ 18 years
Key exclusion criteria (2 listed)
- 1. Patients currently enrolled into a clinical trial of investigational medicinal product for haemophilia.
- Healthy Volunteers
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 |
|---|---|---|
| Thrombophilia screenThrombophilia screen (including antithrombin activity (AT:Ac), protein S antigen (PS:free), protein C activity (PC:Ac) , genetic analysis for FV Leiden and Prothrombin 3'UTR mutations and screening for lupus anticoagulant. | Diagnostic test | Haemophilia patients, Healthy volunteers |
| Initiation pathway analysisEvaluation of inter-individual variability in regulation of TF.VIIa.Xa.TFPI complex (tissue factor, activated Factor VII, activated factor X, tissue factor pathway inhibitor) | Diagnostic test | Haemophilia patients, Healthy volunteers |
Primary outcome
Initiation pathway correlation with clinical phenotype
Time frame: Within 18 months of consent
Correlate lab assays that characterise initiation pathway with clinical phenotype.
3 secondary outcomes
Correlation analysis between FVIII:C/FIX:C levels and whole blood clotting time, thrombin generation in platelet poor plasma.
Time frame: Within 18 months of consent
Evaluation the sensitivity and specificity of global assays for disease severity and clinical phenotype.
Time frame: Within 18 months of consent
Correlation analysis between activation threshold of initiation pathway to thrombin generation and clinical phenotype
Time frame: Within 18 months of consent
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2019.
- 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.
Pratima Chowdary principal investigator
Royal Free Hospitals NHS Foundation Trust
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 Haemophilia, run by Royal Free Hospital NHS Foundation Trust at 1 site in United Kingdom.
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 2 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 27 Mar 2019. 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 United Kingdom. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Thomas Roberts (02078302068, thomas.roberts1@nhs.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Initiation pathway correlation with clinical phenotype (time frame: Within 18 months of consent). 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 27 Mar 2019.