Clin2
NCT07190001Worth exploringNot yet recruiting

YOLT-204 for children with sickle cell disease

Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease)

Part of Blood & lymphatic, Genetic & congenital clinical trials.

This trial tests an experimental treatment called YOLT-204 for children and teens with sickle cell disease. The goal is to see if it can reduce or prevent severe complications like pain crises and acute chest syndrome.

Summary written for real people, not researchers, by Clin2.

Phase
Early Phase 1
Enrollment
18 people
Ages
3 years to 17 years
Study type
Interventional

Who can take part

  • Be between 3 and 17 years old.
  • Have a confirmed diagnosis of sickle cell disease (type HbSS or HbSβ0).
  • Have had at least 2 severe sickle cell events in the past year, such as painful crises, acute chest syndrome, or spleen problems.
  • Not have had a stem cell transplant or gene therapy before.
  • Be willing to use contraception (if applicable) and participate in long-term follow-up.

View the official record on ClinicalTrials.gov

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.

NCT06465550Recruiting· Phase 1
Gene therapy for beta-thalassemia in children and teens

This trial tests a new gene therapy (BD211) for children and teens with severe beta-thalassemia who need regular blood transfusions. The treatment uses your own blood stem cells, modified with a working gene, to help your body make healthy red blood cells and reduce the need for transfusions.

Guangzhou, Guandong
NCT07774351Not yet recruiting
Gene therapy study for sickle cell disease and thalassemia

This study tests an experimental gene therapy called YOLT-204 for people with sickle cell disease or thalassemia. It aims to see if the treatment is safe and can help reduce symptoms, and participants will be followed long-term.

NCT06678165Not yet recruiting· Early Phase 1
Study of YOLT-204 for transfusion-dependent beta-thalassemia

This study tests an experimental medicine called YOLT-204 for people with beta-thalassemia who need regular blood transfusions. The goal is to see if this treatment is safe and if it might reduce the need for transfusions.

Nanning, Guangxi
NCT07000318Not yet recruiting· Early Phase 1
Gene therapy for teenagers with sickle cell disease

This study tests an experimental gene therapy called CS-206 for teenagers with sickle cell disease. It aims to help reduce severe complications by modifying the patient's own blood stem cells.

Shanghai, Shanghai Municipality
NCT06399107Recruiting· Phase 1/Phase 2
Gene therapy trial for sickle cell disease using BAH243

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.

Beijing, Beijing Municipality
NCT06565026Recruiting· Early Phase 1
CS-206 treatment for sickle cell disease patients

This trial tests CS-206, an experimental therapy for people with sickle cell disease. It aims to reduce severe complications like pain crises, acute chest syndrome, strokes, and other serious events. You may qualify if you have a specific genetic type of sickle cell disease and have experienced complications despite standard treatments.

Nanning, Guangxi

Hear when a new Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease) trial opens

We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.