A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD
This study is looking at a new way to treat sickle cell disease using a special type of stem cell transplant. Currently, the most effective cure is a transplant from a perfectly matched sibling, but most people don't have one. This trial focuses on using stem cells from a family member who is only a partial match. The main goal is to find out if this new transplant method is safe, prevents the disease, and avoids serious side effects like graft-versus-host disease (GvHD) and rejection. If successful, it could offer a cure to many more people living with sickle cell disease who currently don't have suitable donor options.
At a glance
What is this study about?
Sickle cell disease is a serious blood condition. At the moment, the only way to cure it is through a treatment called a stem cell transplant. However, this is usually only possible if you have a brother or sister who is a perfect match – and unfortunately, less than 20% of people with sickle cell disease have such a match. This leaves many without a good treatment option.
This study is exploring a new type of stem cell transplant that uses cells from a family member who is only a partial match, rather than a perfect one. The main question researchers want to answer is whether this new approach is just as safe and effective as a perfectly matched transplant. They want to make sure it can cure the disease without causing serious problems like the body rejecting the new cells or a condition called graft-versus-host disease, where the new cells attack the patient's body.
If this new type of transplant proves to be successful and safe, it could be a game-changer. It would mean that many more people with sickle cell disease who don't have a perfectly matched sibling could have a chance at a cure. The study is also looking into how well the immune system recovers after the transplant and what effects it might have on future fertility.
Key takeaways
- This study tests a new stem cell transplant method for sickle cell disease.
- It uses cells from a partially matched family member, widening donor options.
- Main goals: safety, cure rates, and preventing severe side effects like GvHD.
- Could offer a cure to more patients currently without a donor.
- Open to patients aged 2-35 with specific sickle cell complications.
- Requires significant commitment and includes potential risks alongside benefits.
Who may be eligible?
This study is open to people aged between 2 and 35 years old who have a specific type of sickle cell disease (either homozygous hemoglobin S disease, hemoglobin SC, or S 0/+). You would need to have experienced some serious complications related to your sickle cell disease in the past. This includes things like having a stroke, frequent pain crises, several hospital stays for your condition, or needing many blood transfusions.
There are also some reasons why you might not be able to join the study. For example, if you have very poor general health, if you have certain antibodies in your blood that would make a transplant difficult, or if you have major differences in blood type with the potential donor. Also, if you have significant heart or kidney problems, you might not be able to take part.
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 between 2 and 35 years old?
- Do you have a specific type of sickle cell disease (Homozygous HbS, HbSC, or S 0/+)?
- Have you had serious sickle cell complications like stroke, frequent pain crises, or many transfusions?
- Do you have a family member who might be a partial match?
- Do you have good heart and kidney function?
- Are you generally well enough to go through a transplant procedure?
What does participation involve?
If you decide to take part in this study, you will undergo a special stem cell transplant. Before the transplant, you'll receive medication to prepare your body for the new cells. After the transplant, you'll be closely monitored by the medical team. This will involve regular hospital visits, blood tests, and other check-ups to make sure the transplant is working well and to look for any side effects. The researchers will also be monitoring your overall health, how your immune system recovers, and your long-term health, including any impact on fertility. The total duration of follow-up will be carefully explained to you by the study team, likely involving checks extending for some time after the transplant itself.
Potential risks and benefits
Locations (9)
- St. Anna KinderspitalVerified postcodeVienna, Austria· Not yet recruiting
- University Hospital Aachen, Children's HospitalVerified postcodeAachen, Germany· Not yet recruiting
- Charité University medicine, Clinic for Hematology, OncologyVerified postcodeBerlin, Germany· Not yet recruiting
- University Hospital Duesseldorf, Clinic for Pediatric Oncology, - Hemtaology and Clinical ImmunologyVerified postcodeDüsseldorf, Germany· Not yet recruiting
- University Hospital of Frankfurt, Clinic for Paediatrics and Adolescent MedicineVerified postcodeFrankfurt, Germany· Not yet recruiting
- University Hospital Heidelberg, Department of Pediatric Hematology, Oncology and ImmunologyVerified postcodeHeidelberg, Germany· Recruiting
- University Hospital Regensburg, Dept. of Ped. Hematology, Oncology and Stem Cell TransplantationVerified postcodeRegensburg, Germany· Recruiting
- University Children's Hospital TübingenVerified postcodeTübingen, Germany· Recruiting
- University Children's Hospital WürzburgVerified postcodeWürzburg, Germany· Recruiting
Common questions
What is a 'haploidentical' transplant?
It means the stem cell donor is a family member who is only a partial genetic match, not a perfect one. This could allow more people to find a donor.
What is 'GvHD' and why is the study trying to prevent it?
GvHD stands for Graft-versus-Host Disease. It's when the new transplanted cells attack the patient's body. The study aims to reduce this serious complication.
Will I still need medicine after the transplant?
Yes, you will likely need medications after the transplant to help prevent infection and manage any potential side effects or complications.
How long will I be in the hospital for the transplant?
The hospital stay for a stem cell transplant is usually several weeks, followed by careful monitoring as an outpatient.
What happens if the transplant doesn't work?
The study team will discuss all possible outcomes and your treatment options in detail before you decide to take part.
How to find out more
Selim Corbacioglu, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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