YOLT-204 in Patients With Hemoglobinopathies
Trial statusNot yet recruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age3-17
SponsorGuangzhou Women and Children's Medical Center
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Aged 3-17 years (inclusive); any sex.
The subject and/or his/her legally authorized guardian/representative must fully understand the study and voluntarily sign a written informed-consent form.
Karnofsky Performance Status (KPS) ≥ 70 (if ≥ 16 years old) or Lansky Performance Scale (LPS) ≥ 70 (if < 16 years old).
Detailed medical records of red-cell transfusions during the 2 years before informed-consent signature must be available, including volume or units transfused and pre-/post-transfusion red-cell and hemoglobin levels.
Disqualifiers
1.History of multiple drug allergies or hypersensitivity to oligonucleotides or lipid nanoparticles (LNP).
Trial design
Design model
Single group
Treatments tested in this trial
YOLT-204
DrugThe intervention group will receive YOLT-204 on day0
Treatment groups
18 Participants
are divided into 1 treatment groupGroup A: ArmsExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Adverse event rate
Calculate the rate of various adverse events
Time frame
From baseline to 52 weeks after dose2
3 months of sustained transfusion reduction
Analysis begins one month after treatment with YOLT-204, and the proportion of patients who achieve at least 3 months of sustained transfusion reduction (sustained TR3) is obtained.
Time frame
From baseline to 52 weeks after dose3
3 months of sustained HbF level ≥20%
Proportion of patients who, starting one month after YOLT-204 treatment and without concomitant hydroxyurea, maintain HbF ≥ 20 % for at least three consecutive months.
Time frame
From baseline to 52 weeks after doseSecondary outcomes
1
The proportion of alleles with intended modifications
The proportion of alleles with intended modifications in peripheral blood leukocytes and bone marrow cells
Time frame
From baseline to 52 weeks after dose2
Concentration of Hemoglobin
The concentration of Hemoglobin was measured by laboratory
Time frame
From baseline to 52 weeks after dose3
Concentration of Fetal hemoglobin
The concentration of Fetal hemoglobin was measured by laboratory
Time frame
From baseline to 52 weeks after dose4
Concentration of proportion of F cell
The proportion of F cell was measured by laboratory
Time frame
From baseline to 52 weeks after doseSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: