GBT 440-031 Study
by Dr Jo Howard, GSTT
The Sickle Cell Teams at Guys’ and St Thomas’ and King’s College London are delighted to be participating in the GBT 440-031 study. This study opened in July 2017 in the UK and is an international multicentre study recruiting over 400 patients with sickle cell disease (SCD) from all around the world.
The GBT 440-031 trial is investigating whether a new medication (GBT 440-031) is safe and effective in reducing anaemia, pain crises, fatigue and other day-to-day symptoms in adolescents and adults with SCD. The medication is taken by mouth every day and is designed to keep the haemoglobin molecules in red blood cells from sticking to each other. This may help the red blood cells keep their normal shape and stop sickling. Early trials have shown that the medication is safe and that it may improve anaemia.
You can participate in this trial if you are between 12 and 65 years of age with SCD. You must have had at least one pain crisis in the past year. The study doctor and staff can explain other requirements for participation. If you enter the trial you will be randomly allocated treatment with the trial medication or a placebo (a ‘sugar-pill’). You will be in the trial for between 2.5 months and 1.5 years, depending when you start.
If you would like to find out more about the trial and if you are eligible to join please talk to your sickle doctor or nurses. You can find more information here.