UK - EHL Outcomes Registry

August 30, 2018 updated by: Pratima Chowdary, Royal Free Hospital NHS Foundation Trust

Evaluation of Real World Outcomes With Extended Half-Life Concentrates for Routine Clinical Use in Haemophilia A and B: UK - EHL Outcomes Registry

Severe haemophilia A and B (SHA, SHB) are inherited bleeding disorders affecting male patients and are characterised by low levels of circulating clotting factors VIII and IX respectively. Clinically low levels present with multiple recurrent bleeds into joints and muscle from the first couple of years of life. In addition patients may present with spontaneous and potentially fatal bleeding into any organ. The mainstay of treatment is replacement with the missing factor in the form of intravenous injections of factor VIII and IX. Clotting factors can be given to treat a bleed or can be given to prevent a bleed, and the latter is termed prophylaxis. Regular prophylaxis is the current standard of care and aims to decrease spontaneous bleeding events and resulting joint damage, and this requires patients to self-infuse factor into their veins two to four times week. Patient's compliance with prescribed regimen and recommendations has a significant influence on outcomes.

Advances in biomolecular and protein engineering have extended the duration of the effect of clotting factor VIII and IX through multiple mechanisms. This extension of the duration of the effect presents the clinician and patients with opportunities to tailor the treatment to their particular needs, circumstances and body other characteristics. It has been suggested that decreasing the frequency of infusions will improve adherence and thus contribute to improved outcomes.

In rare disorders, it is an accepted fact that post-marketing studies are crucial to understand the generalisability of the efficacy and safety outcomes and identify any new safety and efficacy concerns in relation to specific population group. The investigators propose the development of a registry for systematic collection of information with the dual aim of analysing the relationship between patient and treatment characteristics, and outcomes, and simultaneously identify areas for practice development that can improve the overall quality of life experienced by the haemophilia patient community.

Study Overview

Status

Unknown

Conditions

Study Type

Observational

Enrollment (Anticipated)

500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United Kingdom
      • Basingstoke, United Kingdom
        • Recruiting
        • Basingstoke and North Hampshire Hospital
        • Contact:
          • Chelsie Williams
        • Principal Investigator:
          • Sarah Mangles
      • Birmingham, United Kingdom
        • Recruiting
        • Queen Elizabeth Hospital
        • Contact:
          • Elizabeth Dwenger
        • Principal Investigator:
          • Charles Percy
      • Birmingham, United Kingdom
        • Recruiting
        • Birmingham Women and Childrens
        • Principal Investigator:
          • Jayashree Motwani
        • Contact:
          • Davina Patel
      • Bristol, United Kingdom
        • Recruiting
        • University Hospital Bristol
        • Contact:
          • Emma Phillips
        • Principal Investigator:
          • Emma Phillips
      • Canterbury, United Kingdom
        • Recruiting
        • Kent & Canterbury Hospital
        • Contact:
          • Sylvia Westrup
        • Principal Investigator:
          • Gillian Evans
      • Cardiff, United Kingdom
        • Recruiting
        • University Hospital of Wales
        • Contact:
          • Stuart Cunningham
        • Principal Investigator:
          • Peter Collins
      • Coventry, United Kingdom
        • Recruiting
        • University of Coventry & Warwickshire
        • Contact:
          • Lauren Homer
        • Principal Investigator:
          • Benjamin Bailiff
      • Glasgow, United Kingdom
        • Recruiting
        • Glasgow Royal Hospital for Children
        • Contact:
          • Alison Spence
        • Principal Investigator:
          • Elizabeth Chalmers
      • Glasgow, United Kingdom
        • Recruiting
        • Royal Infirmary
        • Contact:
          • Nancy Brodie
        • Principal Investigator:
          • Campbell Tate
      • Lincoln, United Kingdom
        • Recruiting
        • Lincoln County Hospital
        • Contact:
          • Sandra Lee
        • Principal Investigator:
          • Bethan Myers
      • Liverpool, United Kingdom
        • Recruiting
        • Liverpool University Hospital
        • Contact:
          • Joanne Bell
        • Principal Investigator:
          • Cheng Toh
      • London, United Kingdom
        • Recruiting
        • Great Ormond Street Hospital
        • Contact:
          • Anja Griffoen
        • Principal Investigator:
          • Mary Mathias, MBBS
      • London, United Kingdom, NW3 2QG
      • London, United Kingdom
        • Recruiting
        • St George's Hospital
        • Contact:
          • Pearl Quartey
        • Principal Investigator:
          • Steve Austin
      • London, United Kingdom
        • Recruiting
        • Hammersmith Hospital
        • Contact:
          • Zainab Alashe
        • Principal Investigator:
          • Michael Laffan
      • Newcastle upon Tyne, United Kingdom
        • Recruiting
        • Royal Victoria Hospital
        • Contact:
          • Jane Ashby
        • Principal Investigator:
          • John Hanley
      • Nottingham, United Kingdom
        • Recruiting
        • Nottingham University Hospital
        • Contact:
          • Charlotte Grimley
        • Principal Investigator:
          • Charlotte Grimley
      • Oxford, United Kingdom
        • Recruiting
        • Churchill Hospital Oxford
        • Contact:
          • Simon Fletcher
        • Principal Investigator:
          • Nicola Curry
      • Sheffield, United Kingdom
        • Recruiting
        • Royal Hallamshire Hospital
        • Contact:
          • Branwen Ellison-Handley
        • Principal Investigator:
          • Mike Makris
      • Sheffield, United Kingdom
        • Recruiting
        • Sheffield Children's Hospital
        • Contact:
          • Shaun Emmitt
        • Principal Investigator:
          • Jeanette Payne
      • Truro, United Kingdom
        • Recruiting
        • Royal Cornwall Hospital
        • Contact:
          • Sarah Johns
        • Principal Investigator:
          • Darren Beech

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Non-Probability Sample

Study Population

Patients with Haemophilia A or B requiring replacement therapy or being considered for use of EHL - CFC.

Description

Inclusion Criteria:

  1. Patients with Haemophilia A or B requiring replacement therapy
  2. Patients or parents able to provide informed consent
  3. Patients being considered for use of EHL - CFC.

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Bleed Control
Time Frame: 2 years post enrolment
Investigate changes to bleed control using questionnaire & Haemtrack (software package to record therapy received
2 years post enrolment
Joint Health
Time Frame: 2 years post enrolment
Target joint assessment and questionnaire
2 years post enrolment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
EQ-5D-5L
Time Frame: 2 years post enrolment
Patient Questionnaire
2 years post enrolment
HAEM-A-QoL
Time Frame: 2 years post enrolment
Patient Questionnaire
2 years post enrolment
Haemo-QoL
Time Frame: 2 years post enrolment
Patient Questionnaire
2 years post enrolment
Physical Activity QoL
Time Frame: 2 years post enrolment
Patient Questionnaire
2 years post enrolment
Haemoprefer
Time Frame: 2 years post enrolment
Patient Questionnaire
2 years post enrolment
Identify the value of individualised prophylaxis
Time Frame: 5 years
Patient questionnaire
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Royal Free Hospital NHS Foundation Trust

Investigators

  • Principal Investigator: Pratima Chowdary, Royal Free Hospitals NHS Foundation Trust

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 17, 2016

Primary Completion (Anticipated)

October 1, 2019

Study Completion (Anticipated)

October 1, 2019

Study Registration Dates

First Submitted

October 12, 2016

First Submitted That Met QC Criteria

October 17, 2016

First Posted (Estimate)

October 19, 2016

Study Record Updates

Last Update Posted (Actual)

September 4, 2018

Last Update Submitted That Met QC Criteria

August 30, 2018

Last Verified

August 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RFH/9782

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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