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Inter Individual Variability in Initiation Pathway Activation and Regulation and Phenotypic Heterogeneity in Patients With Haemophilia A and B (InPath)

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ClinicalTrials.gov Identifier: NCT03287999
Recruitment Status : Recruiting
First Posted : September 19, 2017
Last Update Posted : March 27, 2019
Sponsor:
Information provided by (Responsible Party):
Pratima Chowdary, Royal Free Hospital NHS Foundation Trust

Tracking Information
First Submitted Date September 13, 2017
First Posted Date September 19, 2017
Last Update Posted Date March 27, 2019
Actual Study Start Date September 19, 2017
Estimated Primary Completion Date October 2, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: September 15, 2017)
Initiation pathway correlation with clinical phenotype [ Time Frame: Within 18 months of consent ]
Correlate lab assays that characterise initiation pathway with clinical phenotype.
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT03287999 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: September 15, 2017)
  • 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 ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Inter Individual Variability in Initiation Pathway Activation and Regulation and Phenotypic Heterogeneity in Patients With Haemophilia A and B
Official Title Inter Individual Variability in Initiation Pathway Activation and Regulation and Phenotypic Heterogeneity in Patients With Haemophilia A and B
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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Blood plasma
Sampling Method Non-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
Condition Haemophilia
Intervention
  • Diagnostic Test: Thrombophilia screen
    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.
  • Diagnostic Test: Initiation pathway analysis
    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 Groups/Cohorts
  • Haemophilia patients
    Persons with haemophilia A or B - 240 to be recruited
    Interventions:
    • Diagnostic Test: Thrombophilia screen
    • Diagnostic Test: Initiation pathway analysis
  • Healthy volunteers
    Healthy volunteers - 10 to be recruited
    Interventions:
    • Diagnostic Test: Thrombophilia screen
    • Diagnostic Test: Initiation pathway analysis
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: September 15, 2017)
250
Original Estimated Enrollment Same as current
Estimated Study Completion Date October 2, 2020
Estimated Primary Completion Date October 2, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

Patients

Inclusion Criteria:

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

Exclusion Criteria:

1. Patients currently enrolled into a clinical trial of investigational medicinal product for haemophilia.

Healthy Volunteers

Inclusion Criteria:

  1. Currently not receiving any antiplatelet or anticoagulant therapy or other drugs that can affect the coagulation system.
  2. Age ≥ 18 years
  3. Written informed consent in accordance with local and institutional guidelines.
Sex/Gender
Sexes Eligible for Study: Male
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers Yes
Contacts
Contact: Thomas Roberts 02078302068 thomas.roberts1@nhs.net
Listed Location Countries United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number NCT03287999
Other Study ID Numbers 11296
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Pratima Chowdary, Royal Free Hospital NHS Foundation Trust
Study Sponsor Royal Free Hospital NHS Foundation Trust
Collaborators Not Provided
Investigators
Principal Investigator: Pratima Chowdary Royal Free Hospitals NHS Foundation Trust
PRS Account Royal Free Hospital NHS Foundation Trust
Verification Date March 2019