Gene therapy study for severe sickle cell disease in teens and adults
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new gene therapy called KL003 for people with severe sickle cell disease. It aims to fix your own stem cells so they make healthy red blood cells, reducing pain crises. You may qualify if you've had at least four severe pain episodes in the last two years and have not responded well to hydroxyurea.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 12 and 50 years old.
- You must have a severe type of sickle cell disease (like HbSS, HbSβ0, or HbSβ+).
- You must have had at least 4 severe pain crises in the last 2 years despite treatment.
- You must have tried hydroxyurea and it either didn't work or you couldn't take it.
- You must not have had gene therapy or a stem cell transplant before.
- Your major organs (heart, lungs, kidneys, liver) must work well enough for a stem cell transplant.
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 trial tests a new gene therapy for sickle cell disease. It uses your own blood stem cells (collected from bone marrow) that are modified in a lab to help produce healthy red blood cells, then given back to you after a short chemotherapy. The goal is to reduce or stop severe complications like pain crises and lung problems.
This trial tests a gene therapy that uses a modified virus to deliver a healthy gene to your blood stem cells. It aims to help people with sickle cell disease produce normal red blood cells and reduce painful crises.
This trial uses gene editing to fix your own blood stem cells, so you can stop having severe sickle cell crises. It is for young adults with frequent pain episodes or who need regular blood transfusions.
This trial tests a new treatment called CS-206 for adolescents with severe sickle cell disease. It aims to see if this treatment can help by making stem cell transplants safer and more effective for people who have not done well with standard care.
This trial tests a single dose of gene therapy (CTX001) to improve outcomes for people with severe sickle cell disease or transfusion-dependent beta-thalassemia. It aims to see how well it works and whether it is safe, especially after a planned stem cell transplant process.
This trial tests a gene-correction treatment that changes sickle hemoglobin (HbS) into normal hemoglobin (HbA) using your own stem cells. It may help reduce severe sickle cell complications for people with severe disease who meet specific safety and medical criteria.
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.