Clinical Trial

YOLT-204 in Patients With Hemoglobinopathies

Not Yet Recruiting Early Phase 1
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Summary
This is a single-arm, open-label, single-dose, dose-escalation trial that plans to enrol 3-18 patients with transfusion-dependent β-thalassaemia (TDT) or sickle-cell disease (SCD). Its primary aims are to evaluate the safety and tolerability of a single administration of YOLT-204 and to obtain preliminary data on its effect on plasma fetal-haemoglobin levels. The main-study screening period may last up to 60 days; the treatment day is Day 0 (D0). Safety follow-up continues through Week 52 post-dose. After completion of the main study, participants will enter long-term follow-up extending to 15 years post-dose.
Trial Details
NCT Number NCT07190001
Lead Sponsor Guangzhou Women and Children's Medical Center
Conditions Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease)
Enrollment 18 participants
Start Date 2025-09-30
Primary Completion 2027-04-30 (estimated)
Study Completion 2027-04-30 (estimated)
Updated on ClinicalTrials.gov 2025-09-24