Better diagnosis for inherited red blood cell diseases
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial aims to improve how doctors diagnose rare inherited anemias, including sickle cell disease and other conditions that destroy red blood cells. It may help if you have a confirmed or suspected diagnosis but need more clarity through advanced testing.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed or suspected inherited rare anemia, such as sickle cell disease or thalassemia.
- You have a type of anemia like hereditary spherocytosis with severe anemia (hemoglobin below 8 g/dL) or an unclear diagnosis.
- You have not had a stem cell transplant or gene therapy. If a transplant failed and you did not get a new one, you may still join.
- You are not just a carrier of a recessive anemia gene (you must have the disease, not just the trait).
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 is creating a European registry (database) to collect information about people with rare anemia disorders. By joining, you help researchers better understand these conditions and improve care for yourself and others.
This study looks at how your genes can affect inherited blood conditions like sickle cell disease and thalassemia. It may help researchers understand why symptoms differ from person to person, which could guide better future treatments.
This study looks at genetic (DNA) factors that may contribute to certain blood disorders. You may be invited if you or a close family member is receiving care for a blood condition like MDS or MPN, and the study also includes related relatives.
This trial tests a gene editing treatment for sickle cell disease. It aims to modify your own blood stem cells to produce more fetal hemoglobin, which can reduce pain episodes and other complications.
This trial tests a new gene therapy that edits your own blood stem cells to help them make more fetal hemoglobin, which can prevent sickling and reduce the need for transfusions. It is for people with severe sickle cell disease or beta thalassemia who do not have a matched sibling donor.
This trial tests a “biochip” device using samples from people with sickle cell disease. If it works, it may help doctors study sickle cell biology more accurately using patient samples.
Hear when a new Sickle Cell Disease trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.