NCT03287999

Brief 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. 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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
250

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Sep 2017

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 13, 2017

Completed
6 days until next milestone

First Posted

Study publicly available on registry

September 19, 2017

Completed
Same day until next milestone

Study Start

First participant enrolled

September 19, 2017

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 2, 2019

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

October 2, 2020

Completed
Last Updated

March 27, 2019

Status Verified

March 1, 2019

Enrollment Period

2 years

First QC Date

September 13, 2017

Last Update Submit

March 25, 2019

Conditions

Haemophilia

Outcome Measures

Primary Outcomes (1)

  • Initiation pathway correlation with clinical phenotype

    Correlate lab assays that characterise initiation pathway with clinical phenotype.

    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.

    Within 18 months of consent

  • Evaluation the sensitivity and specificity of global assays for disease severity and clinical phenotype.

    Within 18 months of consent

  • Correlation analysis between activation threshold of initiation pathway to thrombin generation and clinical phenotype

    Within 18 months of consent

Study Arms (2)

Haemophilia patients

Persons with haemophilia A or B - 240 to be recruited

Diagnostic Test: Thrombophilia screenDiagnostic Test: Initiation pathway analysis

Healthy volunteers

Healthy volunteers - 10 to be recruited

Diagnostic Test: Thrombophilia screenDiagnostic Test: Initiation pathway analysis

Interventions

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.

Haemophilia patientsHealthy volunteers
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)

Haemophilia patientsHealthy volunteers

Eligibility Criteria

Age18 Years+
Sexmale
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
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

You may qualify if:

  • Patients with haemophilia A or B (baseline FVIII/FIX level \<30%)
  • Age ≥ 18 years
  • Written informed consent in accordance with local and institutional guidelines.

You may not qualify if:

  • \. Patients currently enrolled into a clinical trial of investigational medicinal product for haemophilia.
  • Healthy Volunteers
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Royal Free Hospital

London, United Kingdom

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Blood plasma

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Pratima Chowdary

    Royal Free Hospitals NHS Foundation Trust

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Thomas Roberts

CONTACT

02078302068thomas.roberts1@nhs.net

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Dr Pratima Chowdary

Study Record Dates

First Submitted

September 13, 2017

First Posted

September 19, 2017

Study Start

September 19, 2017

Primary Completion

October 2, 2019

Study Completion

October 2, 2020

Last Updated

March 27, 2019

Record last verified: 2019-03

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)