Transfusion Dependent Beta ThalassemiaEarly Phase 1NCT06678165
A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia
A Early Phase 1 study testing YOLT-204 in people with Transfusion Dependent Beta Thalassemia, run by First Affiliated Hospital of Guangxi Medical University at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 35 years
- Conditions the study is for
- Transfusion Dependent Beta Thalassemia
- What is being tested?
- YOLT-204
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteChina
- Study dates
- 31 Dec 2024to 1 Jun 2026
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 35 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age between 18 to 35 years, gender not limited.
- The patient has fully understood this study and voluntarily signed an informed consent form.
- Clinically diagnosed as a patient with transfusion-dependent β-thalassemia, excluding the genotype: β0β0.
- Karnofsky Performance Status (KPS) score of at least 70.
- 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.
- No severe hematopoietic dysfunction, with heart, lung, liver, and kidney functions essentially normal.
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- Coagulation function: International Normalized Ratio (INR) and Activated Partial Thromboplastin Time (APTT) both ≤1.5×ULN (Upper Limit of Normal).
- 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).
- Liver function: Alanine Aminotransferase (ALT) ≤3×ULN and Aspartate Aminotransferase (AST) ≤3×ULN; Direct Bilirubin ≤2.5×ULN.
- Cardiac function: Left Ventricular Ejection Fraction (LVEF) ≥50%.
- Good compliance, willing to adhere to visit schedules, trial plans, laboratory tests, and other trial steps.
- Willing to participate in long-term follow-up studies.
Who cannot join
- History of multiple drug allergies or a history of allergic reactions to oligonucleotides or lipid nanoparticles (LNPs).
- Diagnosed with compound alpha-thalassemia.
- Clinically significant active bacterial, viral, fungal, or parasitic infections at the time of screening, as judged by the investigator.
- 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.
- Uncorrected bleeding disorders.
- Received treatment with erythropoietin (EPO) within the three months prior to enrollment.
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- Severe iron overload, with serum ferritin levels ≥5000 ng/ml.
- 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).
- History of hematopoietic stem cell transplantation, gene therapy, or gene editing therapy.
- Participation in another clinical study and use of investigational drugs within 3 months prior to starting the study drug.
- History or current presence of malignant tumors or myeloproliferative diseases or immunodeficiency diseases.
- Presence of severe mental illness that prevents cooperation with treatment; significant pulmonary arterial hypertension requiring medical intervention; recent malaria; a history of hematological tumors in immediate family members.
- Any past or current disease, treatment, or laboratory abnormality that may interfere with the study results, affect the patient's full participation in the study, or that the investigator deems unsuitable for participation in this clinical study.
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 530000
About this study
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
- Conditions
- Transfusion Dependent Beta Thalassemia
- Being tested
- YOLT-204
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
YOLT-204Drug
The intervention group will receive YOLT-204 on day0
| Study group | Role | What they receive |
|---|---|---|
| The intervention group will receive YOLT-204 on day0 | Experimental | Not described Receives: Drug: YOLT-204 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-Emergent Adverse Events
Number of Participants with Treatment-Emergent Adverse Events
Measured: 52 weeks after dose
Laboratory Test Findings
Number of Participants with Clinically Significant Clinical Laboratory Test Findings
Measured: 52 weeks after dose
Safety Measurements
Number of Participants with Clinically Significant Safety Measurements
Measured: 52 weeks after dose
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.
Measured: 4 months after dose
3 months of transfusion independence
Analysis begins one month after treatment with YOLT-204, and the proportion of patients who achieve at least 3 months of transfusion independence (sustained TI3) is obtained.
Measured: 4 months after dose
Secondary outcomes (5)
6 months of sustained transfusion reduction
Analysis begins one month after treatment with YOLT-204, and the proportion of patients who achieve at least 6 months of sustained transfusion reduction (sustained TR6) is obtained.
Measured: 7 months after dose
6 months of transfusion independence
Analysis begins one month after treatment with YOLT-204, and the proportion of patients who achieve at least 6 months of transfusion independence (sustained TI6) is obtained.
Measured: 7 months after dose
The proportion of alleles with intended modifications
The proportion of alleles with intended modifications in peripheral blood leukocytes and bone marrow cells over time.
Measured: 52 weeks after dose
Fetal hemoglobin concentration
The change in fetal hemoglobin concentration over time after YOLT-204 infusion
Measured: 52 weeks after dose
Total hemoglobin concentration
The change in total hemoglobin concentration over time after YOLT-204 infusion.
Measured: 52 weeks after dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2024
- Study start31 Dec 2024 (estimated)
- Primary completion1 Jun 2026 (estimated)
- Study completion1 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transfusion Dependent Beta ThalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- First Affiliated Hospital of Guangxi Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06678165
- Sponsor's protocol ID
- YOLT-204-IIT
- Status last verified
- Oct 2024
- First submitted
- 31 Oct 2024
- Last updated
- 7 Nov 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia
- ClinicalTrials.gov ID
- NCT06678165
- Acronym / sponsor ID
- YOLT-204-IIT
- Conditions
- Transfusion Dependent Beta Thalassemia
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- First Affiliated Hospital of Guangxi Medical UniversityUniversity, hospital or other
- Study start
- 31 Dec 2024 (estimated)
- Primary completion
- 1 Jun 2026 (estimated)
- Study completion
- 1 Jun 2026 (estimated)
- First posted
- 7 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| The intervention group will receive YOLT-204 on day0 | Experimental | Drug: YOLT-204 |
Who may qualify
- Ages
- 18 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (12 listed)
- Age between 18 to 35 years, gender not limited.
- The patient has fully understood this study and voluntarily signed an informed consent form.
- Clinically diagnosed as a patient with transfusion-dependent β-thalassemia, excluding the genotype: β0β0.
- Karnofsky Performance Status (KPS) score of at least 70.
- 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.
Key exclusion criteria (13 listed)
- History of multiple drug allergies or a history of allergic reactions to oligonucleotides or lipid nanoparticles (LNPs).
- Diagnosed with compound alpha-thalassemia.
- Clinically significant active bacterial, viral, fungal, or parasitic infections at the time of screening, as judged by the investigator.
- 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.
- Uncorrected bleeding disorders.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| YOLT-204The intervention group will receive YOLT-204 on day0 | Drug | The intervention group will receive YOLT-204 on day0 |
Primary outcome
Treatment-Emergent Adverse Events
Time frame: 52 weeks after dose
Number of Participants with Treatment-Emergent Adverse Events
Laboratory Test Findings
Time frame: 52 weeks after dose
Number of Participants with Clinically Significant Clinical Laboratory Test Findings
Safety Measurements
Time frame: 52 weeks after dose
Number of Participants with Clinically Significant Safety Measurements
3 months of sustained transfusion reduction
Time frame: 4 months after dose
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.
3 months of transfusion independence
Time frame: 4 months after dose
Analysis begins one month after treatment with YOLT-204, and the proportion of patients who achieve at least 3 months of transfusion independence (sustained TI3) is obtained.
5 secondary outcomes
6 months of sustained transfusion reduction
Time frame: 7 months after dose
6 months of transfusion independence
Time frame: 7 months after dose
The proportion of alleles with intended modifications
Time frame: 52 weeks after dose
Fetal hemoglobin concentration
Time frame: 52 weeks after dose
Total hemoglobin concentration
Time frame: 52 weeks after dose
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing YOLT-204 in people with Transfusion Dependent Beta Thalassemia, run by First Affiliated Hospital of Guangxi Medical University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 7 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Rongrong Liu (+86 0771 5356510, liurongrong@stu.gxmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment-Emergent Adverse Events (time frame: 52 weeks after dose). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Nov 2024.