Clinical trial NCT02938156: UK - EHL Outcomes Registry
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
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Hemophilia A
Hemophilia B
Keywords: Hemophilia
Lead sponsor: Royal Free Hospital NHS Foundation Trust
Sponsor class: OTHER
First posted: 2016-10-19
Enrollment: 500
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Haemophilia A or B requiring replacement therapy or being considered for use of EHL - CFC.

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.


Eligibility 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.

Primary outcomes:
measure: Bleed Control
timeFrame: 2 years post enrolment
measure: Joint Health
timeFrame: 2 years post enrolment
Secondary outcomes:
measure: EQ-5D-5L
timeFrame: 2 years post enrolment
measure: HAEM-A-QoL
timeFrame: 2 years post enrolment
measure: Haemo-QoL
timeFrame: 2 years post enrolment
measure: Physical Activity QoL
timeFrame: 2 years post enrolment
measure: Haemoprefer
timeFrame: 2 years post enrolment
measure: Identify the value of individualised prophylaxis
timeFrame: 5 years
Other outcomes:
Locations:
city: Basingstoke
country: United Kingdom
facility: Basingstoke and North Hampshire Hospital
city: Birmingham
country: United Kingdom
facility: Birmingham Women and Childrens
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital
city: Bristol
country: United Kingdom
facility: University Hospital Bristol
city: Canterbury
country: United Kingdom
facility: Kent & Canterbury Hospital
city: Cardiff
country: United Kingdom
facility: University Hospital of Wales
city: Coventry
country: United Kingdom
facility: University of Coventry & Warwickshire
city: Glasgow
country: United Kingdom
facility: Glasgow Royal Hospital for Children
city: Glasgow
country: United Kingdom
facility: Royal Infirmary
city: Lincoln
country: United Kingdom
facility: Lincoln County Hospital
city: Liverpool
country: United Kingdom
facility: Liverpool University Hospital
city: London
country: United Kingdom
facility: Royal Free Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital
city: London
country: United Kingdom
facility: Hammersmith Hospital
city: London
country: United Kingdom
facility: St George's Hospital
city: Newcastle upon Tyne
country: United Kingdom
facility: Royal Victoria Hospital
city: Nottingham
country: United Kingdom
facility: Nottingham University Hospital
city: Oxford
country: United Kingdom
facility: Churchill Hospital Oxford
city: Sheffield
country: United Kingdom
facility: Royal Hallamshire Hospital
city: Sheffield
country: United Kingdom
facility: Sheffield Children's Hospital
city: Truro
country: United Kingdom
facility: Royal Cornwall Hospital
Study officials:
affiliation: Royal Free Hospitals NHS Foundation Trust
name: Pratima Chowdary
role: PRINCIPAL_INVESTIGATOR
References: