UK - EHL Outcomes Registry

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

500 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-12-17

Study Completion Date

2019-10-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Hemophilia A Hemophilia B

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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.

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Responsible Party

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Pratima Chowdary

Consultant Haematologist

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Pratima Chowdary

Role: PRINCIPAL_INVESTIGATOR

Royal Free Hospitals NHS Foundation Trust

Sheffield, , United Kingdom

Site Status RECRUITING

Royal Cornwall Hospital

Truro, , United Kingdom

Site Status RECRUITING

Countries

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United Kingdom

Central Contacts

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Emal Waqif

Role: CONTACT

[email protected]

Mark Phillips

Role: CONTACT

Mark Phillips

Role: backup

Other Identifiers

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RFH/9782

Identifier Type: -

Identifier Source: org_study_id

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UK - EHL Outcomes Registry

Study Results

Basic Information

Brief Summary

Related Clinical Trials

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Detailed Description

Conditions

Study Design

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Royal Free Hospital NHS Foundation Trust

Responsible Party

Pratima Chowdary

Principal Investigators

Pratima Chowdary

Locations

Basingstoke and North Hampshire Hospital

Birmingham Women and Childrens

Queen Elizabeth Hospital

University Hospital Bristol

Kent & Canterbury Hospital

University Hospital of Wales

University of Coventry & Warwickshire

Glasgow Royal Hospital for Children

Royal Infirmary

Lincoln County Hospital

Liverpool University Hospital

Royal Free Hospital NHS Foundation Trust

Great Ormond Street Hospital

Hammersmith Hospital

St George's Hospital

Royal Victoria Hospital

Nottingham University Hospital

Churchill Hospital Oxford

Royal Hallamshire Hospital

Sheffield Children's Hospital

Royal Cornwall Hospital

Countries

Central Contacts

Emal Waqif

Mark Phillips

Facility Contacts

Chelsie Williams

Davina Patel

Elizabeth Dwenger

Emma Phillips

Sylvia Westrup

Stuart Cunningham

Lauren Homer

Alison Spence

Nancy Brodie

Sandra Lee

Joanne Bell

Emal Waqif

Mark Phillips

Anja Griffoen

Zainab Alashe

Pearl Quartey

Jane Ashby

Charlotte Grimley

Simon Fletcher

Branwen Ellison-Handley

Shaun Emmitt

Sarah Johns

References

Other Identifiers

RFH/9782

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