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
Qualifiers
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
Treatments tested in this trial
- YOLT-204
Treatment groups
18 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov