NCT07190001

YOLT-204 in Patients With Hemoglobinopathies

An Exploratory Clinical Study to Evaluate the Safety and Efficacy of YOLT-204 in Patients With Hemoglobinopathies (β-thalassemia and Sickle-cell Disease)

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: Guangzhou Women and Children's Medical Center · Academic / Other
• Sex: All
• Age: 3 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Conditions

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

Interventions

Type	Name	Description
DRUG	YOLT-204	The intervention group will receive YOLT-204 on day0

Timeline

• Start date: 2025-09-30
• Primary completion: 2027-04-30
• Completion: 2027-04-30
• First posted: 2025-09-24
• Last updated: 2025-09-24

Locations

1 site across 1 country: China