Not Yet RecruitingEarly Phase 1

YOLT-204 in Patients With Hemoglobinopathies

China18 participantsStarted 2025-09-30

Plain-language 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.

Who can participate

Age range3 Years – 17 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

✕.Clinically significant active bacterial, viral, fungal, or parasitic infection at screening, as judged by the investigator.
✕.White blood cell (WBC) count \< 3 × 10⁹/L and/or platelet count \< 100 × 10⁹/L at screening.
✕.Uncorrected bleeding diathesis. 5.Massive splenomegaly at screening (spleen edge below the umbilicus or \> 4 cm below the costal margin) deemed by the investigator to preclude enrollment.
✕.Serum ferritin ≥ 5 000 ng/mL, or MRI T2\* evidence of severe cardiac or hepatic iron overload.
✕.Positive for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus antibody, anti-HIV antibody, or specific anti-Treponema pallidum antibody.
✕.Prior hematopoietic stem-cell transplantation, gene therapy, or gene-editing therapy.
✕.Participation in another clinical trial and receipt of investigational product within 3 months before first dose of study drug.
✕0.Current or prior malignancy, myeloproliferative disorder, or immunodeficiency disease.

What they're measuring

Adverse event rate

Timeframe: From baseline to 52 weeks after dose

3 months of sustained transfusion reduction

Timeframe: From baseline to 52 weeks after dose

3 months of sustained HbF level ≥20%

Timeframe: From baseline to 52 weeks after dose

Trial details

NCT IDNCT07190001

SponsorGuangzhou Women and Children's Medical Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-04-30

Contact for this trial

Jiang Hua

+8613533330985 jiang_hua18@sina.cn

View on ClinicalTrials.gov More Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease) trials

Plain-language summary

Who can participate

Age range3 Years – 17 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

✕.Clinically significant active bacterial, viral, fungal, or parasitic infection at screening, as judged by the investigator.
✕.White blood cell (WBC) count \< 3 × 10⁹/L and/or platelet count \< 100 × 10⁹/L at screening.
✕.Uncorrected bleeding diathesis. 5.Massive splenomegaly at screening (spleen edge below the umbilicus or \> 4 cm below the costal margin) deemed by the investigator to preclude enrollment.
✕.Serum ferritin ≥ 5 000 ng/mL, or MRI T2\* evidence of severe cardiac or hepatic iron overload.
✕.Positive for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus antibody, anti-HIV antibody, or specific anti-Treponema pallidum antibody.
✕.Prior hematopoietic stem-cell transplantation, gene therapy, or gene-editing therapy.
✕.Participation in another clinical trial and receipt of investigational product within 3 months before first dose of study drug.
✕0.Current or prior malignancy, myeloproliferative disorder, or immunodeficiency disease.