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YOLT-204 in Patients With Hemoglobinopathies

NCT07190001 · Status: NOT_YET_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2025-09-24

No results posted yet for this study

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

DRUG

YOLT-204

The intervention group will receive YOLT-204 on day0

Sponsors & Collaborators

  • Guangzhou Women and Children's Medical Center

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-09-30
Primary Completion
2027-04-30
Completion
2027-04-30

Countries

  • China

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07190001 on ClinicalTrials.gov