ClinicalTrials.gov
ClinicalTrials.gov Menu

UK - EHL Outcomes Registry

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02938156
Recruitment Status : Recruiting
First Posted : October 19, 2016
Last Update Posted : September 3, 2018
Sponsor:
Information provided by (Responsible Party):
Pratima Chowdary, Royal Free Hospital NHS Foundation Trust

Brief Summary:

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.


Condition or disease
Hemophilia A Hemophilia B

Study Type : Observational
Estimated Enrollment : 500 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Evaluation of Real World Outcomes With Extended Half-Life Concentrates for Routine Clinical Use in Haemophilia A and B: UK - EHL Outcomes Registry
Actual Study Start Date : December 17, 2016
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : October 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia




Primary Outcome Measures :
  1. Bleed Control [ Time Frame: 2 years post enrolment ]
    Investigate changes to bleed control using questionnaire & Haemtrack (software package to record therapy received

  2. Joint Health [ Time Frame: 2 years post enrolment ]
    Target joint assessment and questionnaire


Secondary Outcome Measures :
  1. EQ-5D-5L [ Time Frame: 2 years post enrolment ]
    Patient Questionnaire

  2. HAEM-A-QoL [ Time Frame: 2 years post enrolment ]
    Patient Questionnaire

  3. Haemo-QoL [ Time Frame: 2 years post enrolment ]
    Patient Questionnaire

  4. Physical Activity QoL [ Time Frame: 2 years post enrolment ]
    Patient Questionnaire

  5. Haemoprefer [ Time Frame: 2 years post enrolment ]
    Patient Questionnaire

  6. Identify the value of individualised prophylaxis [ Time Frame: 5 years ]
    Patient questionnaire



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with Haemophilia A or B requiring replacement therapy or being considered for use of EHL - CFC.
Criteria

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02938156


Contacts
Contact: Emal Waqif emal.waqif@nhs.net
Contact: Mark Phillips mark.phillips@ucl.ac.uk

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

Responsible Party: Pratima Chowdary, Consultant Haematologist, Royal Free Hospital NHS Foundation Trust
ClinicalTrials.gov Identifier: NCT02938156     History of Changes
Other Study ID Numbers: RFH/9782
First Posted: October 19, 2016    Key Record Dates
Last Update Posted: September 3, 2018
Last Verified: August 2018

Keywords provided by Pratima Chowdary, Royal Free Hospital NHS Foundation Trust:
Hemophilia

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked