New drug YOLT-204 aims to reduce transfusions in blood disorders
NCT ID NCT07190001
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This early-phase trial will test a single dose of a new drug called YOLT-204 in 18 children and teens with transfusion-dependent β-thalassemia or sickle cell disease. The goal is to see if it is safe and can raise fetal hemoglobin levels enough to reduce the need for blood transfusions. Participants will be followed for at least a year, with long-term monitoring up to 15 years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- YOLT-204 (a drug given as a single dose to boost fetal hemoglobin)
- What this could lead to
- If it works, this could point toward a treatment that reduces or eliminates the need for regular blood transfusions in people with β-thalassemia or sickle cell disease.
- What could go wrong
- This is a very early, small trial (only 18 people) focused on safety. It may not work, and long-term effects are unknown. Participants will be followed for 15 years.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Guangzhou women and children's medical center
Guangzhou, Guangdong, 510405, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Gene therapy aims to free patients from lifelong blood transfusions
- Talking therapy could help thalassemia patients take their meds
- Drug cocktail may cut transfusions for kids with thalassemia
- Scientists track lifespan of transfused blood in sickle cell kids
- One-Shot gene fix for blood disorder enters human testing
- CRISPR stem cell therapy could end transfusions for sickle cell and thalassemia patients