In this section
This section of the HCC website focuses on research in to haemoglobinopathies. Research produces valuable knowledge for understanding human diseases/conditions, preventing, and treating illness and comorbidities, and promoting health.
Please note many of the clinical trials listed below will be located at Manchester University NHS Foundation Trust (MFT), as it is one of the key centres outside of London. Other trials, within the HCC, will have links to external websites (where the trial is being conducted).
Click on the links below or scroll down to articles:
Clinical trials and other research projects for sickle cell and thalassaemia patients
Taking part in research at MFT
Health and care research can only improve and save lives with the help of people like you. Clinical trials are carefully designed and conducted to minimise the risks and maximise the benefits to all who take part.
Taking part in research is a positive experience and entirely voluntary. Different studies and trials look for different people to take part, such as because of their age or aspects of the condition they are experiencing. Some explore new treatments, others seek to better understand how the conditions work. All health and social care research has to go through very strict ethical and regulatory checks before it can go ahead and participants to our research are very closely monitored throughout.
At MFT we have a number of research projects taking place to better understand and improve treatments for sickle cell and thalassaemia. We want to give people in Greater Manchester and beyond the opportunity to be part of our research.
What is Sickle Cell and Thalassaemia?
Approximately 15,000 people in the UK have sickle cell disease, an inherited condition, mainly affecting people of African and Caribbean background. People with this condition produce unusually shaped red blood cells that can cause problems because they do not live as long as healthy blood cells and can block blood vessels.
Thalassaemia is the name for a group of inherited conditions that affect a substance in the blood called haemoglobin. People with thalassaemia produce either no or too little haemoglobin, which is used by red blood cells to carry oxygen around the body. This can make them very anaemic (tired, short of breath and pale). This condition mainly affects people of Mediterranean, south Asian, southeast Asian and Middle Eastern origin.
Find out more about sickle cell disease and thalassaemia on the NHS England website.
Get in touch to find out more
If you would like more information or you are thinking of taking part in research in these areas, please contact the Clinical Trials team at MFT. Whether or not you take part in a study is always entirely up to you, but a member of the team will be happy to discuss our current studies with you.
Email: NMH.Trials@mft.nhs.uk
Telephone: 0161 276 6603 / 0161 906 7510
(Updated September 2024)
Current Studies
Now Recruiting For The Following:
IBHO & Genomics England Sickle Cell Initiative
Study Investigators
Dr Joseph Sharif, Dr Nandini Sadasivam & Dr Louza Al-Mashaykhi
Outline
Improving Black Health Outcomes (IBHO) Project & Research (NIHR) Bioresource Research Tissue Bank and the Diverse Data Initiative hope to get a better understanding of common physical and mental health disorders that affect people in Black communities including Sickle Cell Disease and Thalassaemia. This could help people get earlier and better treatment before developing more severe complications. Patients who have been diagnosed with Sickle Cell Disease and/or Thalassaemia from any ethnicity are invited to take part in this initiative so that findings from the research can benefit everyone and advance the understanding of both conditions.
Evaluation of the Urgent Care Sickle Cell Unit
Study Investigators
Dr Joseph Sharif, Dr Nandini Sadasivam & Dr Louza Al-Mashaykhi
Outline
Researchers from the University of Manchester are exploring the implementation of and experiences of patients who have attended the Urgent Emergency Care Unit (Ward 10).
They aim to understand your experiences of your care which you have received during your visit to the unit from the moment you engaged with the unit to discharge from hospital.
The REDRESS Study
Study Investigators
Dr Eleni Tholouli, Dr Joseph Sharif, Dr Nandini Sadasivam & Dr Louza Al-Mashaykhi
Outline
The REDRESS Study is a clinical trial researching the use of stem cell transplant (aka bone marrow transplant) to treat patients with Severe Sickle Cell Disease.
This study is being led by a group of Researchers based at Kings College London NHS Foundation Trust, University College London Hospitals NHS Foundation Trust and Guys & St Thomas’ NHS Foundation Trust.
The GLADIOLUS Study
Study Investigators
Dr Nandini Sadasivam, Dr Joseph Sharif & Dr Louza Al-Mashaykhi
Outline
The Gladiolus Study is a Clinical trial whose main purpose is to evaluate how safe and effective the study medication Etavopivat is on improving Haemoglobin levels, anaemia, and reducing the need of Red Blood Cell transfusion. The study is enrolling both Sickle Cell and Thalassaemia patients
This study is made up of 2 parts:
48 weeks of primary treatment with Etavopivat
60 weeks of optional extension treatment period
Thalassaemia (NOW CLOSED) & Sickle Cell Disease (OPEN)
Young Migrants Living with Sickle Cell Disease
Study Investigators
Dr Joseph Sharif & Dr John Grainger
Outline
Young Migrants, Chronic illness and disability: The case of Children and young people with Sickle Cell Disease who Migrate to England
This study is sponsored by the University of Nottingham and led by the Chief Investigator – Dr Brenda Poku, Research Fellow based at the University of Nottingham.
This study hopes to better understand the health and care experiences of children/young people (CYP) living with Sickle Cell Disease who migrate to the UK and identify ways of enhancing, improving and/or integrating health services to support their transition and integration post-migration.
This is a qualitative study and will involve individual interviews and focus group discus
Studies Opening Soon
Natural History of Sickle Cell Disease Study
Study Investigators
Dr Joseph Sharif, Dr Nandini Sadasivam & Dr Louza Al-Mashaykhi
Outline
This study aims to set up a database of patients with Sickle Cell Disease to follow patients over a period of 5 years during routine care.
Adults with Sickle Cell Disease (SCD) may develop long term complications of their SCD. Current treatment options for individuals with SCD are supportive care, blood transfusions or hydroxycarbamide (hydroxyurea). We currently only have limited information on how many adults will develop sickle-related complications and on the long-term outcomes of the available treatments. This limits the advice we are able to give you on your prognosis and on the best treatment options for you.
Past Studies and Studies in Follow-up
The RISE-UP Phase 3 Study
Study Investigators
Dr Joseph Sharif, Dr Nandini Sadasivam & Dr Louza Al-Mashaykhi
Outline
The RISE UP study is a Phase 2 & Phase 3 double-blinded, randomised study which is to determining the effect of a new medication known as Mitapivat in participants with Sickle Cell Disease Vs Placebo.
The study is divided into 3 parts, Phase 2 (which is now completed), Phase 3 (now recruiting) and Open Label Extension.
For participants enrolled onto the Phase 3 study, they will be followed up for a total of 52 weeks including an up to 4 week screening period and double blind – treatment period over 10 visits and will be offered to continue on the Open Label phase of the study where they will receive the study medication Mitapivat.
The ENERGIZE Study
Study Investigators
Dr Nandini Sadasivam, Dr Louza Al-Mashaykhi & Dr Joseph Sharif
Outline
ENERGIZE is a Phase 3, double blind, randomised study with the purpose of assessing whether Mitapivat helps to improve haemoglobin levels in non-transfusion-dependent Alpha- or Beta-thalassemia patients and whether it is safe.
Participants enrolled onto this study received either a dose of Mitapivat or placebo for a period of 24 weeks in the Double-blind period and after this had the option to continue to receive Mitapivat for an additional 5 years as part of the Open Label Extension.
TAPS 2 – Transfusion Antenatally in Pregnant Women with Sickle Cell Disease
Study Investigators
Dr Joseph Sharif
Outline
TAPS 2 is a randomised, two arm feasibility trial assessing whether pregnant women with Sickle Cell Disease would be willing to take part in a randomised controlled trial comparing Serial Prophylactic Exchange blood transfusion (SPEBT) to Standard of care.
Participants which were randomised to receive SPEBT completed this treatment from between 6- and 20-weeks’ gestation every 6-10 weeks until the end of pregnancy either in the haematology day unit or antenatal day units/wards in accordance with local practices.
The FiSCD Study – Fatigue in Children & Young People with Sickle Cell Disease
Study Investigators
Dr Joseph Sharif
Outline
Understanding fatigue experiences of children and young people with Sickle Cell Disease to guide the co-development of a fatigue self-management intervention.
This study was conducted by the Research Team at the University of Manchester, University of York, MFT and the Sickle Cell Society with the aim to better understand how children and young people with Sickle Cell Disease experience fatigue and how they can be supported. Participants were asked to complete an interview and Questionnaire with the Researcher of the Study – Dr Brenda Poku.
A short report of the study findings can be found on the Haemoglobinopathy webpage.
The RISE UP Phase 2 Study
Study Investigators
Dr Joseph Sharif, Dr Nandini Sadasivam & Dr Louza Al-Mashaykhi
Outline
The RISE UP study is a Phase 2 & Phase 3 double-blinded, randomised study which is to determine the effect of a new medication known as Mitapivat in participants with Sickle Cell Disease Vs Placebo.
The study is divided into 3 parts, Phase 2 (which is now completed), Phase 3 (now recruiting) and Open Label Extension.
The IMR-BTL-201 Study & The IMR-SCD-301 Study
Study Investigators
Dr Joseph Sharif & Dr Nandini Sadasivam
Outline
Phase 2 &2b studies evaluating a medication known as IMR-687 to treat participants with β-Thalassemia as well as Sickle Cell Disease.
The purpose of these studies was to evaluate the how well IMR-687 is tolerated by both groups of patients, how safe it is and to establish what the medication does to red blood cells and other biomarkers associated with these diseases.
The ViSion Serenity Study
Study Investigators
Dr Joseph Sharif & Dr Nandini Sadasivam
Outline
A Phase 2a (assessing dose requirements), Double blind, randomised study investigating a new medication known as VIT-2763 or Vamifeport and its safety, tolerability and beneficial effects on two forms of Sickle Cell Disease – HbS/S and HbSβT0.
The ENERGIZE-T Study
Study Investigators
Dr Nandini Sadasivam
Outline
ENERGIZE-T is a Clinical trial testing the study medication Mitapivat to see how safe and effective is for patients with Thalassaemia.
The trials’ main goal is to see whether Mitapivat decreases the need for transfusion and only included patients who require regular transfusions.
The CSL 889 Study
Study Investigators
Dr Joseph Sharif & Dr Nandini Sadasivam
Outline
CSL889 is being developed for the treatment of the pain episodes (vaso-occlusive crisis or VOCs) experienced by patients with SCD. In patients with SCD, heme, a molecule that is released by red blood cells, is thought to be important in the development of a pain episode. Hemopexin is a protein that is produced naturally in the body, which normally mops up excess heme. In patients with SCD, levels of hemopexin are decreased.
CSL889 is a plasma-derived hemopexin that aims to correct this deficiency of hemopexin, thereby improving the symptoms of a pain episode.
Future Research
Our team continues to work closely with our links at the University, other Hospital Trusts as well as pharmaceutical companies and have a number of studies in the pipeline within Haemoglobinopathies.
The team also plans to host its first Patient & Public Involvement and Engagement Event in November at the Manchester Sickle Cell and Thalassaemia Centre with the possibility of holding further events in the Future.
You can find more research trials available in other locations through this link: Search Results | Beta ClinicalTrials.gov
News stories
A Mancunian woman revives her dream to visit the North Pole after receiving new NHS treatment for Sickle Cell.
In May 2022, Gloria started a new treatment at the Manchester Royal Infirmary that has significantly improved her quality of life and allowed her to revive her long-cherished dream. She said: “I’m one of those people who’s always open to trying new things to help myself and others it may benefit, like my brother, so I was excited to get started. I was just like – let’s do it! I didn’t even think twice.”
First in Europe to recruit patient to Sickle Cell Disease study
The adult non-malignant haematology research team at Manchester Royal Infirmary recruited the first patient in Europe to a study researching the effect of GBT440, in increasing red blood cells and reducing the severity of symptoms of sickle cell disease.
A Manchester woman, whose sickle cell condition was so painful she once thought it might end her life – has told of a new revolutionary NHS treatment which has left her ‘feeling as if she has wings.
Sanah Shaikh is one of the first patients in the UK to receive a Crizanlizumab. Sanah who is from Indian heritage, also hopes to help dispel a widespread myth about sickle cell – that it only affects people from Black or Afro-Caribbean backgrounds.
Useful links
Research and Innovation at MFT website
Manchester University NHS Foundation Trust website
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- Twitter: @MFT_Research
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Fatigue in Sickle Cell Disease Study funded by the National Institute for Health Research
This study (report in link below) looked at understanding fatigue in children and young people with sickle cell (SCD). The research team plan to use the findings to develop and evaluate a fatigue self-management programme. This will be done in collaboration with children/young people with SCD, adults with SCD, parents and care provider.
The FiSCD Study _Final Short Report
Study carried out by Dr Brenda Poku (University of Nottingham), Professor Susan Kirk (University of Manchester), Professor Karl Atkin (University of York), Dr John Grainger (Manchester NHS Foundation Trust) and Iyamide Thomas (Sickle Cell Society).