RecruitingOBSERVATIONAL

Study for Epidemiology and Characterization of Myelodysplastic Syndromes (MDS) and Juvenile Myelomonocytic Leucemia (JMML) in Childhood

This study is focused on children and teenagers who have a type of blood cancer called Myelodysplastic Syndromes (MDS) or Juvenile Myelomonocytic Leukaemia (JMML). The main goal is to make diagnosing these conditions more accurate by looking closely at blood and bone marrow cells. Researchers will also study the genetic changes found in these conditions to understand them better. This improved understanding will help doctors give the correct diagnosis more quickly. The study also aims to learn more about how many children survive these conditions, how often they get better, and how often they might get sick again. They will look at children who have had a special type of treatment called a stem cell transplant. By gathering this information, the study hopes to improve how these serious conditions are managed and treated in the future for children and young people.

At a glance

Status

Recruiting

Sponsor

University Hospital Freiburg

Enrolment target

260

Start

01 Apr 2010

Estimated completion

01 Dec 2027

Myelodysplastic Syndromes Juvenile Myelomonocytic Leukemia

What is this study about?

This research is looking into two serious blood conditions that affect children and teenagers: Myelodysplastic Syndromes (MDS) and Juvenile Myelomonocytic Leukaemia (JMML). These conditions mean that the body doesn't make enough healthy blood cells, which can cause many health problems.

The main purpose of this study is to get a clearer picture of these conditions in young people. Researchers want to improve how doctors diagnose MDS by thoroughly checking blood and bone marrow samples. They will also use special tests to look at the genes and chromosomes within the cells. This helps them find tiny changes that might make diagnosing and understanding the condition much better than before. By having a more accurate diagnosis, doctors can make sure children get the right care from the start.

Beyond diagnosis, the study also aims to understand how children with MDS and JMML respond to treatment over time. They will follow children to see how long they live, how often they recover, and if the condition comes back. For children who have a treatment called a stem cell transplant, the study will also look at how well they do afterwards. All this information will help doctors and scientists worldwide learn more about these conditions and hopefully lead to better treatments and support for children and their families.

Key takeaways

This study aims to improve understanding and diagnosis of childhood blood conditions like MDS and JMML.
It involves examining existing medical information and samples, not new treatments.
The goal is to help future children with these conditions by learning more about them.
Participation is voluntary, and your child's medical care won't be affected if you choose not to take part.
The study includes children and teenagers up to 17 years old with a confirmed diagnosis.

Who may be eligible?

To be part of this study, a child or teenager must have been diagnosed with either Myelodysplastic Syndromes (MDS) or Juvenile Myelomonocytic Leukaemia (JMML). This diagnosis needs to be confirmed by doctors looking at their cells and genes. Children with a specific type of blood cancer linked to Down syndrome can also join if they are aged 6 or older.

Patients must be under 18 years old. Also, a parent or legal guardian will need to give written permission for their child to take part. Wherever possible, the child or teenager themselves will also be asked if they want to participate and understand what it means.

Children cannot join if their parents or they themselves decide not to give permission. Also, if a child with blood cancer linked to Down syndrome is under 6 years old, they cannot participate. If a child has recently taken part in another research study (within the last four weeks), they usually wouldn't be able to join this one, unless the other study was about improving cancer treatment or diagnosis.

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.

Has my child been diagnosed with Myelodysplastic Syndromes (MDS) or Juvenile Myelomonocytic Leukaemia (JMML)?
Is my child under 18 years old?
If my child has blood cancer linked to Down syndrome, are they 6 years old or older?
Are we happy to give consent for my child's medical information and samples to be reviewed?
Has my child not taken part in another major research study in the last month (unless it was about cancer treatment or diagnosis)?

Answer every question to see your result.

What does participation involve?

This study primarily involves collecting and carefully examining information and samples from children who have already been diagnosed with MDS or JMML. It doesn't involve any new treatments or procedures beyond what your child would receive as part of their usual medical care. The main participation will involve allowing the research team to review your child's medical records, including their diagnosis, treatment plans, and how they are doing over time. They will also need to analyse small samples, such as blood or bone marrow, that have already been taken for your child's diagnosis and treatment. There are no extra hospital visits or medication specifically for this study.

Potential risks and benefits

Participating in this study doesn't involve new medical procedures, so there are no additional physical risks beyond standard care. Your personal information will be kept private, but there's always a small chance of a data breach, though strict measures are in place to prevent this. The potential benefit is that the information gathered from your child, combined with that from many other children, will help doctors better understand MDS and JMML. This could lead to more accurate diagnoses and improved treatments for future children. You are free to withdraw your child from the study at any time without affecting their medical care.

Locations (1)

University Hospital of Freiburg
Verified postcode
Freiburg im Breisgau, Germany· Recruiting

Common questions

What are Myelodysplastic Syndromes (MDS) and Juvenile Myelomonocytic Leukaemia (JMML)?

These are types of blood cancers that affect children and teenagers. They mean the body doesn't make enough healthy blood cells.

Will my child receive new medicine in this study?

No, this study doesn't involve giving any new medicines. It focuses on understanding the conditions better through existing medical information and samples.

What information will the doctors collect?

They will look at your child's diagnosis, treatment journey, and how they are doing. They will also analyse blood and bone marrow samples that were taken for their care.

Do I have to let my child participate?

No, participation is completely voluntary. Your decision will not affect your child's medical care in any way.

Will this study help my child directly?

This study is not designed to directly help your child right now. However, the information gained will help doctors improve care for other children with these conditions in the future.

How to find out more

Charlotte M. Niemeyer, M.D.

charlotte.niemeyer@uniklinik-freiburg.de 49-761-270 Official trial record

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