RecruitingPHASE3INTERVENTIONAL

RElated Haplo-DonoR Haematopoietic stEm Cell Transplantation for Adults With Severe Sickle Cell Disease

This important study is investigating a new type of stem cell transplant, called a haploidentical stem cell transplant, for adults living with severe sickle cell disease. Researchers want to find out if this transplant is a safe and effective treatment option, particularly for people whose existing treatments haven't worked well, cause difficult side effects, or who need regular blood transfusions to manage their condition. The study also aims to understand the cost-effectiveness of this new treatment approach. By comparing it with standard care, the study hopes to offer new hope and improved quality of life for adults severely affected by sickle cell disease.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

King's College Hospital NHS Trust

Enrolment target

120

Start

23 Feb 2023

Estimated completion

01 Mar 2027

Sickle Cell Disease

What is this study about?

Sickle cell disease is a serious blood condition that can cause a lot of pain and health problems. While there are treatments available, sometimes they don't work well enough, or people experience difficult side effects. This study is looking at a treatment called a 'haploidentical stem cell transplant'. This is a special type of transplant where stem cells are taken from a donor who is only a partial match for the patient, often a family member. These new, healthy stem cells are then given to the patient to replace their own sickled cells.

The main goal of this study is to see how well this haploidentical stem cell transplant works for adults with severe sickle cell disease. Researchers want to understand if it can lead to better health outcomes and a better quality of life compared to the usual treatments. They also want to check if it's a good use of healthcare resources. The study is particularly focused on people whose sickle cell disease is causing severe problems, despite other treatments, or who need regular blood transfusions.

By carefully studying this transplant, doctors hope to gather strong evidence to show if it can be a valuable new treatment option. If successful, this could mean significant improvements for adults dealing with the most challenging forms of sickle cell disease, potentially offering a more lasting solution than current therapies.

Key takeaways

This study evaluates a stem cell transplant for severe sickle cell disease.
It's for adults with severe symptoms who haven't responded to other treatments.
It compares a 'haploidentical' transplant (from a partial match) against usual care.
The study aims to find out if the transplant is effective and good value for money.
Close medical monitoring is provided for all participants.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To join this study, you need to be an adult aged 18 or over and have a confirmed family member who is a 'haploidentical' donor (meaning they are a partial match for you). You must also have severe sickle cell disease, which means your condition is causing significant health issues despite trying other treatments. This could include having had a stroke, frequent severe pain crises or chest problems, or needing regular blood transfusions.

There are also some health requirements to make sure you are well enough for the transplant. Your heart, lung, kidney, and liver function will be checked to confirm they are strong enough. You will also need to have a good general level of fitness, as measured by a specific health score.

You cannot join the study if you already have a fully matched sibling donor, have had a bone marrow transplant before, or if you are pregnant or breastfeeding. If you can get pregnant, you must also be willing to use effective contraception if you join the study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a family member who might be a partial match (haploidentical) for a stem cell donation?
Has your sickle cell disease caused severe problems despite current treatments (e.g., strokes, many pain crises, or needing regular blood transfusions)?
Are you generally well enough for a major medical procedure, with healthy heart, lung, and kidney function?
Are you not pregnant or breastfeeding, and willing to use contraception if you can get pregnant?

Answer every question to see your result.

What does participation involve?

This study is a clinical trial comparing a new transplant procedure with the standard care you would usually receive for your sickle cell disease. If you join, you would either receive the haploidentical stem cell transplant from your partially matched family donor, or you would continue with your current standard medical care. The study will involve a series of medical assessments, tests, and regular check-ups to monitor your health and how you respond to your assigned treatment. The exact number of visits and tests would be explained in detail if you are found to be eligible. The total duration of your participation in the study would depend on which treatment path you are on and how you are responding, but it would involve long-term follow-up to understand the lasting effects.

Potential risks and benefits

Participating in this study might offer you the potential benefit of a new treatment that could improve your severe sickle cell disease. However, like all medical treatments, the haploidentical stem cell transplant carries potential risks, including side effects from the procedure and potential complications, which your medical team will discuss with you in detail. There are also risks involved with standard medical care, which you would continue to receive if you were in that arm of the study. You will receive close medical monitoring throughout your participation. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

King's College Hospital
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is 'haploidentical'?

It means the donor is a partial match for you, often a family member, not a complete match like a full sibling.

What does 'severe sickle cell disease' mean in this study?

It means your condition has caused major problems like strokes, frequent pain crises, or needing regular blood transfusions.

Will I definitely receive the transplant if I join?

Not necessarily. The study compares the transplant with standard care, so you might receive either.

Is the transplant safe?

All medical procedures have risks. The study will carefully monitor your health, and your doctors will explain all potential risks to you.

How long will I be in the study?

Your participation will involve long-term follow-up, with frequent assessments and check-ups over a period of time that will be fully explained to you.

How to find out more

Victoria Potter, BSc, MBBS, FRACP, FRCPA

victoriapotter@nhs.net +44 20 3299 3730 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.