Health TrialsbyHealthPanel

A Clinical Exploratory Study of YOLT-204 in the Treatment of Hemoglobinopathies

ConditionHemoglobinopathies
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age14-35
SponsorUnion Hospital, Tongji Medical College, Huazhong University of Science and Technology

About this trial

This is a single-arm, open-label, dose-escalation study designed to enroll approximately 5-30 patients with TDT or SCD. The objectives are to evaluate the safety and tolerability in patients following intravenous administration of YOLT-204 and to preliminarily assess its effect on fetal hemoglobin levels in plasma.

In this study, the maximum screening period of the main study is 60 days, the treatment day is Day 0 (D0), and the safety follow-up period is up to Week 52 after administration.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male or female subjects aged 14 to 35 years (inclusive).

The subject and/or their legal guardian has been fully informed about the study and has voluntarily signed a written ICF.

KPS score ≥ 70 for subjects aged 16 years or older; LPS score ≥ 70 for subjects under 16 years of age.

Detailed medical records regarding red blood cell transfusions within the 2-year period prior to signing the ICF are available, including the volume or number of units transfused, as well as pre- and post-transfusion red blood cell and hemoglobin levels.

Disqualifiers

History of multiple drug allergies or allergic reaction history to oligonucleotides or LNP;

Presence of clinically significant active bacterial, viral, fungal, or parasitic infection as judged by the investigator at screening;

White blood cell (WBC) count < 3 × 109/L and/or platelet count < 100 × 109/L at screening (using pre-lymphodepletion results if lymphodepletion is considered);

Uncorrected hemorrhagic disorders;

Trial design

Design model

Single group

Treatments tested in this trial

  • YOLT-204

    Genetic

    YOLT-204 treatment on Day0

Treatment groups

30 Participants
are divided into 4 treatment groups
Group A: YOLT-204 treatment with 0.6 mg/kgExperimental treatment 1 intervention
Group B: YOLT-204 treatment with 1.0 mg/kgExperimental treatment 1 intervention
Group C: YOLT-204 treatment with 1.25 mg/kgExperimental treatment 1 intervention
Group D: YOLT-204 treatment with TBDExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

adverse event

Time frame
From screening to 52 weeks after treatment
2

Proportion of patients achieving sustained transfusion reduction (TR) for at least 3 months (sustained TR3)

Time frame
From 1 month to the 52 weeks after treatment
3

Proportion of patients achieving HbF ≥ 20% for at least 3 months without using hydroxyurea

Time frame
From 1 month to the 52 weeks after treatment

Secondary outcomes

1

Proportion of intentionally modified alleles in peripheral blood leukocytes over time.

Time frame
From enrollment to the 52 weeks after treatment
2

Proportion of intentionally modified alleles in bone marrow cells over time.

Time frame
From enrollment to the 52 weeks after treatment
3

Changes in total hemoglobin concentration over time following YOLT-204 infusion.

Time frame
From enrollment to the 52 weeks after treatment
4

Changes in fetal hemoglobin concentration over time following YOLT-204 infusion.

Time frame
From enrollment to the 52 weeks after treatment

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: