Hemophilia ANCT02938156
UK - EHL Outcomes Registry
An observational study of Hemophilia A and Hemophilia B, run by Royal Free Hospital NHS Foundation Trust at 21 sites in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- How many people
- 500 participants
- Where
- 21 sitesUnited Kingdom
- Study dates
- 17 Dec 2016to Oct 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with Haemophilia A or B requiring replacement therapy or being considered for use of EHL - CFC.
Who may be able to join
- Patients with Haemophilia A or B requiring replacement therapy
- Patients or parents able to provide informed consent
- Patients being considered for use of EHL - CFC.
Who cannot join
- 1. Patients currently enrolled into a clinical trial of investigational medicinal product.
Trial locations and contacts
21 sites in 1 country.
Emal Waqif contact
Mark Phillips contact
21 sites in 1 country
United Kingdom21 sites, 21 recruiting
- Recruiting
Basingstoke and North Hampshire Hospital
Basingstoke
Chelsie Williams
Sarah Mangles
- Recruiting
Birmingham Women and Childrens
Birmingham
Davina Patel
Jayashree Motwani
- Recruiting
Queen Elizabeth Hospital
Birmingham
Elizabeth Dwenger
Charles Percy
- Recruiting
University Hospital Bristol
Bristol
Emma Phillips
Emma Phillips
- Recruiting
Kent & Canterbury Hospital
Canterbury
Sylvia Westrup
Gillian Evans
- Recruiting
University Hospital of Wales
Cardiff
Stuart Cunningham
Peter Collins
- Recruiting
University of Coventry & Warwickshire
Coventry
Lauren Homer
Benjamin Bailiff
- Recruiting
Glasgow Royal Hospital for Children
Glasgow
Alison Spence
Elizabeth Chalmers
- Recruiting
Royal Infirmary
Glasgow
Nancy Brodie
Campbell Tate
- Recruiting
Lincoln County Hospital
Lincoln
Sandra Lee
Bethan Myers
- Recruiting
Liverpool University Hospital
Liverpool
Joanne Bell
Cheng Toh
- Recruiting
Royal Free Hospital NHS Foundation Trust
London, NW3 2QG
Emal Waqif, emal.waqif@nhs.net
Mark Phillips, mark.phillips@ucl.ac.uk
- Recruiting
Great Ormond Street Hospital
London
Anja Griffoen
Mary Mathias, MBBS
- Recruiting
Hammersmith Hospital
London
Zainab Alashe
Michael Laffan
- Recruiting
St George's Hospital
London
Pearl Quartey
Steve Austin
- Recruiting
Royal Victoria Hospital
Newcastle upon Tyne
Jane Ashby
John Hanley
- Recruiting
Nottingham University Hospital
Nottingham
Charlotte Grimley
Charlotte Grimley
- Recruiting
Churchill Hospital Oxford
Oxford
Simon Fletcher
Nicola Curry
- Recruiting
Royal Hallamshire Hospital
Sheffield
Branwen Ellison-Handley
Mike Makris
- Recruiting
Sheffield Children's Hospital
Sheffield
Shaun Emmitt
Jeanette Payne
- Recruiting
Royal Cornwall Hospital
Truro
Sarah Johns
Darren Beech
About this study
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
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.
- Conditions
- Hemophilia A, Hemophilia B
- Keywords
- Hemophilia
- Medical subject headings
- Hemophilia A, Hemophilia B
What is being measured
The primary outcome is the main question the study is designed to answer.
Bleed Control
Investigate changes to bleed control using questionnaire \& Haemtrack (software package to record therapy received
Measured: 2 years post enrolment
Joint Health
Target joint assessment and questionnaire
Measured: 2 years post enrolment
Secondary outcomes (6)
EQ-5D-5L
Patient Questionnaire
Measured: 2 years post enrolment
HAEM-A-QoL
Patient Questionnaire
Measured: 2 years post enrolment
Haemo-QoL
Patient Questionnaire
Measured: 2 years post enrolment
Physical Activity QoL
Patient Questionnaire
Measured: 2 years post enrolment
Haemoprefer
Patient Questionnaire
Measured: 2 years post enrolment
Identify the value of individualised prophylaxis
Patient questionnaire
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2016 (estimated)
- Study start17 Dec 2016
- Primary completionOct 2019 (estimated)
- Study completionOct 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Royal Free Hospital NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Pratima Chowdary, Royal Free Hospital NHS Foundation Trust
- Investigators
- Pratima Chowdary, Principal investigator, Royal Free Hospitals NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02938156
- Sponsor's protocol ID
- RFH/9782
- Status last verified
- Aug 2018
- First submitted
- 12 Oct 2016
- Last updated
- 4 Sep 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
Koopman SF, Goedhart TMHJ, Bukkems LH, Mulders TM, Leebeek FWG, Fijnvandraat K, Coppens M, Mathias M, Collins PW, Tait RC, Bagot CN, Curry N, Payne J, Chowdary P, Cnossen MH, Mathot RAA; OPTI-CLOT study group and SYMPHONY consortium. A new population pharmacokinetic model for recombinant factor IX-Fc fusion concentrate including young children with haemophilia B. Br J Clin Pharmacol. 2024 Jan;90(1):220-231. doi: 10.1111/bcp.15881. Epub 2023 Sep 12.
PubMed 37567779
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02938156
- Acronym / sponsor ID
- RFH/9782
- Conditions
- Hemophilia A, Hemophilia B
- Keywords
- Hemophilia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- Royal Free Hospital NHS Foundation TrustUniversity, hospital or other
- Study start
- 17 Dec 2016 (actual)
- Primary completion
- Oct 2019 (estimated)
- Study completion
- Oct 2019 (estimated)
- First posted
- 19 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Haemophilia A or B requiring replacement therapy or being considered for use of EHL - CFC.
Key inclusion criteria (3 listed)
- Patients with Haemophilia A or B requiring replacement therapy
- Patients or parents able to provide informed consent
- Patients being considered for use of EHL - CFC.
Key exclusion criteria (1 listed)
- 1. Patients currently enrolled into a clinical trial of investigational medicinal product.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Bleed Control
Time frame: 2 years post enrolment
Investigate changes to bleed control using questionnaire \& Haemtrack (software package to record therapy received
Joint Health
Time frame: 2 years post enrolment
Target joint assessment and questionnaire
6 secondary outcomes
EQ-5D-5L
Time frame: 2 years post enrolment
HAEM-A-QoL
Time frame: 2 years post enrolment
Haemo-QoL
Time frame: 2 years post enrolment
Physical Activity QoL
Time frame: 2 years post enrolment
Haemoprefer
Time frame: 2 years post enrolment
Identify the value of individualised prophylaxis
Time frame: 5 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Sep 2018.
- Derived from this study: PubMed 37567779Koopman SF, Goedhart TMHJ, Bukkems LH, Mulders TM, Leebeek FWG, Fijnvandraat K, Coppens M, Mathias M, Collins PW, Tait RC, Bagot CN, Curry N, Payne J, Chowdary P, Cnossen MH, Mathot RAA; OPTI-CLOT study group and SYMPHONY consortium. A new population pharmacokinetic model for recombinant factor IX-Fc fusion concentrate including young children with haemophilia B. Br J Clin Pharmacol. 2024 Jan;90(1):220-231. doi: 10.1111/bcp.15881. Epub 2023 Sep 12.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Emal Waqif contact
Mark Phillips contact
Pratima Chowdary principal investigator
Royal Free Hospitals NHS Foundation Trust
21 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A and Hemophilia B, run by Royal Free Hospital NHS Foundation Trust at 21 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 4 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Emal Waqif (emal.waqif@nhs.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Bleed Control (time frame: 2 years post enrolment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02938156, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Sep 2018.