RecruitingPHASE2INTERVENTIONAL

Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor

This study is testing a new, gentler type of stem cell transplant for children aged 1 to 19 with sickle cell disease. Stem cell transplants can cure sickle cell disease, but they’re usually only an option for a small number of patients with a perfectly matched sibling donor. This trial is trying to make the treatment safer and more widely available, especially for children whose sibling donor has a different — but still compatible — blood type. Researchers will use specific medicines like alemtuzumab and sirolimus, along with a low dose of radiation, to prepare the child's body for the transplant. The main goal is to see how safe and effective this new approach is, and to carefully watch for any side effects.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

University of Calgary

Enrolment target

Start

05 Jul 2017

Estimated completion

01 Jul 2028

Sickle Cell Disease Stem Cell Transplant Complications Red Blood Cell Disorder Pure Red Cell Aplasia

What is this study about?

Sickle cell disease is a serious blood condition that can cause a lot of pain and health problems over time. For many years, the only treatment that could cure sickle cell disease was a stem cell transplant. However, this treatment is usually only an option for a small number of children who have a sibling with a very close genetic match and the same blood type. This means many children with sickle cell disease miss out on the chance for this life-changing cure.

This study is testing a new way of doing stem cell transplants, called a 'nonmyeloablative' transplant. This method uses a gentler approach to prepare the child's body for the transplant, using special medications and a lower dose of radiation. The main aim is to make the transplant safer and reduce side effects, especially if the donor's blood type is different from the child's. While blood type differences between donor and recipient aren't usually a problem for transplants, they can sometimes cause specific issues when using this gentler method.

The research team wants to find out if this new, gentler transplant method is safe and effective for children aged 1 to 19 with sickle cell disease whose sibling donor has a different, but still compatible, blood type. By testing this new approach, they hope to make stem cell transplants a possibility for more children who currently can't have one, offering them a chance for a healthier future.

Key takeaways

This study explores a new, gentler stem cell transplant for children with sickle cell disease.
It aims to expand transplant options for children with a specific blood-type mismatch with their sibling donor.
The treatment uses special medicines and low-dose radiation.
Researchers will monitor for safety and effectiveness, including a specific blood side effect.
The goal is to offer more children a potential cure for sickle cell disease.

Who may be eligible?

This study is looking for children with sickle cell disease who are between 1 and 19 years old. They must have a type of sickle cell disease that is confirmed by specific blood tests. It's important that their sickle cell disease has already caused health problems, such as frequent painful crises, a history of stroke, breathing problems, or other serious issues, even if they've been taking medication for it. Some of these issues could include repeated crises, feeling short of breath, or needing many blood transfusions.

For a child to be considered for this study, they must also have a sibling who is a match for them in terms of their immune system, but who has a different blood type (specifically, their blood type isn't a perfect match in a certain way). This specific difference in blood type is what the researchers are focusing on with this new transplant method.

Children would not be able to join the study if they are allergic to certain medications used in the treatment, or if they are unable to follow the study plan carefully. The medical team will check all these details to make sure the study is right for each child.

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.

Is my child between 1 and 19 years old?
Does my child have a confirmed diagnosis of sickle cell disease?
Has my child experienced serious health problems from sickle cell disease?
Does my child have a sibling who could be a donor, but their blood types aren't a perfect match?
Is my child able to attend regular hospital appointments?

Answer every question to see your result.

What does participation involve?

If you or your child are eligible and choose to take part, you would first have various tests and appointments to prepare for the stem cell transplant. This would involve taking specific medicines and having a lower dose of radiation to help the body accept the new stem cells. After the transplant, there would be regular hospital visits for check-ups and monitoring to make sure the new cells are working well and to watch for any side effects. You would also need to take specific medicines, such as sirolimus, to prevent the body from rejecting the transplant. The study will carefully monitor blood counts and overall health. The specific duration of follow-up after the transplant would be discussed by the medical team, but it typically involves several years of monitoring to ensure long-term success and to catch any potential complications early.

Potential risks and benefits

Taking part in this study may offer the potential benefit of a cure for sickle cell disease using a gentler transplant method, which could be an option for children who previously couldn't have this treatment. However, as with any medical treatment, especially transplants, there are potential risks. These can include side effects from the medications used, risks associated with the transplant procedure itself, and a specific concern called 'pure red cell aplasia,' which the study aims to monitor and prevent. You will be given full information about all potential benefits and risks, and you always have the right to withdraw from the study at any time without affecting your or your child's future medical care.

Locations (1)

Alberta Children's Hospital
Verified postcode
Calgary, Canada· Recruiting

Common questions

What is sickle cell disease?

Sickle cell disease is a serious inherited blood condition where red blood cells are shaped like crescents (sickles). These cells can get stuck in blood vessels, causing pain, anaemia, and other health problems.

What is a stem cell transplant?

A stem cell transplant replaces unhealthy blood-forming cells with healthy ones, usually from a donor. This can cure conditions like sickle cell disease.

What does 'nonmyeloablative' mean?

It means the treatment preparing the body for the transplant is gentler, using lower doses of medication and radiation, aiming for fewer side effects compared to traditional transplants.

What if my child's blood type doesn't perfectly match their sibling donor's?

This study is specifically looking at children where the donor's blood type isn't a perfect match in one specific way. The aim is to make transplants safe even with this difference.

Are the study medications safe?

The medicines used, like alemtuzumab and sirolimus, are approved medications. However, like all medicines, they can have side effects, which the medical team will discuss with you in detail.

How to find out more

Tony Truong, MD, MPH

tony.truong@ahs.ca 403-955-7272 Official trial record

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