Faster genetic testing for children with familial hemophagocytic lymphohistiocytosis
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This study tests whether getting genetic test results faster for children with a rare immune disorder called familial hemophagocytic lymphohistiocytosis (FHL) can speed up the time to a bone marrow transplant and improve survival. It aims to see if quicker diagnosis leads to better outcomes.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be under 18 years old.
- You must have a confirmed or suspected diagnosis of FHL or a related genetic condition that can cause HLH (such as Griscelli syndrome, Chédiak-Higashi syndrome, or X-linked lymphoproliferative disease), or have a family history of HLH.
- You must have at least 5 of 8 specific symptoms or lab findings, including fever, enlarged spleen, high triglycerides or low fibrinogen, evidence of hemophagocytosis (a process where certain cells eat other blood cells), low NK cell function, high ferritin, high soluble CD25 or activated T cells, or low blood counts in at least two cell lines.
- You must be covered by social security (in France).
- Your legal guardian must sign an informed consent form.
- You cannot have a solid tumor, leukemia, or lymphoma.
- You must not be in the exclusion period of another research study at the time of signing consent.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study aims to create an early warning system to identify patients with hemophagocytic lymphohistiocytosis (HLH), a rare but serious immune overreaction. If you have certain infections, autoimmune diseases, or lymphoma, this screening could help detect HLH early.
This trial tests a one-time gene therapy for FHL type 3, a rare immune disorder. Doctors take your own blood stem cells, add a working copy of the UNC13D gene, and then put them back into your body. The goal is to fix the immune system so you don't need a transplant from a donor.
This study is for people with hemophagocytic lymphohistiocytosis (HLH), a condition where the immune system becomes overactive. Researchers want to collect medical data and blood samples to better understand HLH and find better ways to diagnose and treat it.
This trial tests a combination of a targeted drug (ruxolitinib) with a less intense version of standard chemo for adults with Hemophagocytic Lymphohistiocytosis (HLH). It aims to control the overactive immune response while reducing side effects.
This trial looks for a DNA marker in people with HLH caused by lymphoma. It may help doctors better confirm the cause of HLH and guide treatment decisions.
This trial tests a new combination of medicines (the GO regimen) for people with hemophagocytic lymphohistiocytosis (HLH) that is either newly diagnosed, has come back, or hasn't responded to other treatments. It aims to see if this approach can help when a stem cell transplant isn't an option right now.
Hear when a new Familial Lymphohistiocytosis trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.