Loading clinical trials...

A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia | Clinical Trials | Clareo Health

NOT_YET_RECRUITINGEARLY_PHASE1

A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia

NCT06678165•First Affiliated Hospital of Guangxi Medical University

View on ClinicalTrials.gov

Summary

This study is a single-arm, open-label, dose-escalation trial, planning to enroll 3-9 patients with transfusion-dependent β-thalassemia, aimed at assessing the safety and tolerability of a single-dose of YOLT-204 in patients with transfusion-dependent β-thalassemia; to preliminarily evaluate the impact of a single -dose of YOLT-204 on the levels of fetal hemoglobin in the plasma

Eligibility

Age

18 - 35 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Age between 18 to 35 years, gender not limited.
•2. The patient has fully understood this study and voluntarily signed an informed consent form.
•3. Clinically diagnosed as a patient with transfusion-dependent β-thalassemia, excluding the genotype: β0β0.
•4. Karnofsky Performance Status (KPS) score of at least 70.
•5. Obtain detailed medical records of red blood cell transfusions within 2 years prior to the patient signing the informed consent form, including the volume or units of transfusion and the levels of red blood cells and hemoglobin before and after transfusion.
•6. No severe hematopoietic dysfunction, with heart, lung, liver, and kidney functions essentially normal.
•7. Coagulation function: International Normalized Ratio (INR) and Activated Partial Thromboplastin Time (APTT) both ≤1.5×ULN (Upper Limit of Normal).
•8. Renal function: Creatinine ≤1.5×ULN, or when creatinine \>1.5×ULN, the creatinine clearance is \>50ml/min (calculated according to the Cockcroft-Gault formula).
•9. Liver function: Alanine Aminotransferase (ALT) ≤3×ULN and Aspartate Aminotransferase (AST) ≤3×ULN; Direct Bilirubin ≤2.5×ULN.
•10. Cardiac function: Left Ventricular Ejection Fraction (LVEF) ≥50%.
+2 more criteria

Exclusion Criteria

•1. History of multiple drug allergies or a history of allergic reactions to oligonucleotides or lipid nanoparticles (LNPs).
•2. Diagnosed with compound alpha-thalassemia.
•3. Clinically significant active bacterial, viral, fungal, or parasitic infections at the time of screening, as judged by the investigator.
•4. White blood cell count (WBC) \<3×10\^9/L and/or platelet count \<100×10\^9/L not due to hypersplenism, as judged by the investigator.
•5. Uncorrected bleeding disorders.
•6. Received treatment with erythropoietin (EPO) within the three months prior to enrollment.
•7. Severe iron overload, with serum ferritin levels ≥5000 ng/ml.
•8. Positive for hepatitis B surface antigen (HBsAg), antibodies to hepatitis C virus, antibodies to human immunodeficiency virus (HIV), or specific antibodies to Treponema pallidum (syphilis).
•9. History of hematopoietic stem cell transplantation, gene therapy, or gene editing therapy.
•10. Participation in another clinical study and use of investigational drugs within 3 months prior to starting the study drug.
+3 more criteria

Locations (1)

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

Key Dates

Start Date

December 31, 2024

Primary Completion Date

June 1, 2026

Completion Date

June 1, 2026

Last Updated

November 7, 2024

Enrollment

ESTIMATED participants

Conditions

Transfusion Dependent Beta Thalassemia

Interventions

YOLT-204

DRUG

Contacts

Rongrong Liu

CONTACT

+86 0771 5356510 liurongrong@stu.gxmu.edu.cn

Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia

NCT07292259

Transfusion Dependent Beta Thalassemia

View study

ACTIVE_NOT_RECRUITINGPHASE1/PHASE2

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

NCT05444894

Transfusion Dependent Beta ThalassemiaHemoglobinopathies+2 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: November 7, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia

Inclusion Criteria

Exclusion Criteria

Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

Glucagon-Like Peptide 1 Receptor Agonist in Diabetes Mellitus Management in Children and Adolescents With Transfusion-Dependent Thalassemia