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.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Guangzhou women and children's medical center
Guangzhou, Guangdong, 510405, China
Contact Phone: •••-•••-•••• Email: •••••@•••••