Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02938156
Recruitment Status : Unknown
Verified August 2018 by Pratima Chowdary, Royal Free Hospital NHS Foundation Trust.
Recruitment status was:  Recruiting
First Posted : October 19, 2016
Last Update Posted : September 4, 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

Layout table for study information
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.


Layout table for eligibility information
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
Layout table for location contacts
Contact: Emal Waqif emal.waqif@nhs.net
Contact: Mark Phillips mark.phillips@ucl.ac.uk

Locations
Show Show 21 study locations
Sponsors and Collaborators
Royal Free Hospital NHS Foundation Trust
Investigators
Layout table for investigator information
Principal Investigator: Pratima Chowdary Royal Free Hospitals NHS Foundation Trust
Layout table for additonal information
Responsible Party: Pratima Chowdary, Consultant Haematologist, Royal Free Hospital NHS Foundation Trust
ClinicalTrials.gov Identifier: NCT02938156    
Other Study ID Numbers: RFH/9782
First Posted: October 19, 2016    Key Record Dates
Last Update Posted: September 4, 2018
Last Verified: August 2018
Keywords provided by Pratima Chowdary, Royal Free Hospital NHS Foundation Trust:
Hemophilia
Additional relevant MeSH terms:
Layout table for 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