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New treatment option for Transfusion-dependent Beta-Thalassemia
Official title Β-Thalassemia Treatment with KL003 Cell Injection
ClinicalTrials.gov ID: NCT06280378
What this study is testing
What is KL003 Cell Injection Drug Product?
KL003 Cell Injection Drug Product is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for transfusion-dependent beta-thalassemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 35
You may be able to join if
- Male or female age between 3-35 years;
- Diagnosis of transfusion-dependent β-thalassemia and a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior...
- Karnofsky performance status ≥70 for participants≥16 years of age; Lansky performance status of ≥70 for participants\<16 years of age;
- Eligible to undergo auto-HSCT;
- Willing and able to follow the research procedures and conditions, with good compliance;
You likely can't join if
- Diagnosis of composite α thalassemia;
- Prior receipt of gene therapy or allo-HSCT;
- Meet the criteria for allo-HSCT and with an identified willing donor with full HLA match;
- Participants with severe iron overload at the time of screening;
- Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody;
- Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.);
See the full eligibility criteria
- Male or female age between 3-35 years;
- Diagnosis of transfusion-dependent β-thalassemia and a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years;
- Karnofsky performance status ≥70 for participants≥16 years of age; Lansky performance status of ≥70 for participants\<16 years of age;
- Eligible to undergo auto-HSCT;
- Willing and able to follow the research procedures and conditions, with good compliance;
- Willing to receive at least the 2 years follow-up;
- Participant and/or legal guardians voluntarily participated in this clinical trial and signed the informed consent form.
- Diagnosis of composite α thalassemia;
- Prior receipt of gene therapy or allo-HSCT;
- Meet the criteria for allo-HSCT and with an identified willing donor with full HLA match;
- Participants with severe iron overload at the time of screening;
- Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody;
- Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.);
- Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the clinical investigator;
- people positive with the following etiological tests: human immunodeficiency virus(HIV-1-2),human cytomegalovirus (HCMV-DNA),EB virus（EBV-DNA）,HBV (HBsAg/HBV-DNA positive),HCV antibody (HCV-Ab), Human T-lymphotropic...
- Uncorrectable coagulation dysfunction or history of severe bleeding disorder;
- History of major organ damage including:
- Liver function test suggest AST or ALT levels \>3× upper limit of normal（ULN）;
- Total serum bilirubin value\>2.5×ULN;if combined with Gilbert syndrome, total bilirubin\>3×ULN and direct bilirubin value\>2.5×ULN;
- Left ventricular ejection fraction \<45%;
- Baseline calculated eGFR\<60mL/min/1.73m2;
- Pulmonary function:FEV1/FVC\<60% and/or diffusion capacity of carbon monoxide (DLco) \<60% of prediction;
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, Shanghai Municipality, China
- Tianjin, Tianjin Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, Shanghai Municipality, China; Tianjin, Tianjin Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.