Learning to Live (L2L)
Research type
Research Study
Full title
Learning to live with non-severe haemophilia.
IRAS ID
315092
Contact name
Simon Fletcher
Contact email
Sponsor organisation
Haemnet
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
CT.Gov, NCT05314751
Duration of Study in the UK
0 years, 11 months, 27 days
Research summary
Haemophilia is a rare congenital disorder caused by an inherited genetic defect of the X chromosome. It affects approximately one in every 5,000 males. Different subtypes (haemophilia A or FVIII deficiency) and haemophilia B (factor IX deficiency) and severities of haemophilia: “severe” (factor level < 1%), “moderate” (factor level 1-5% and “mild” (factor level 6-25%) are reported. For all affected individuals painful joint bleeds occur which are treated by replacing the missing factor by injection into the veins. For the most severely affected this can be as often as three times per week.
Over the past 10 years strides have been made in novel therapies given as injections under the skin as infrequently as monthly and gene therapy, to replace the missing gene which then produces factor VIII or IX. Both of these treatments are given to those with severe haemophilia only and 'convert' their blood levels to a milder form of haemophilia. However for those with established joint disease from bleeds, the damage is not repair, merely halted; their pain and mobility issues remain.
In this study we want to compare four groups of men with haemophilia - those who have severe haemophilia and who remain on factor therapy, those with moderate or mild haemophilia since birth and those who have received new therapy to convert them from severe to mild.
We will do this is two ways, firstly a survey using a haemophilia specific validated quality of life tool, and then through in-depth interviews with 10 men in each group. We hope that this will shed light on living with non-severe haemophilia form birth, and that this can help those with severe haemophilia to adapt to a new way of living following treatment with new therapies.
REC name
West Midlands - South Birmingham Research Ethics Committee
REC reference
22/WM/0205
Date of REC Opinion
6 Sep 2022
REC opinion
Further Information Favourable Opinion