NCT06280378 Β-Thalassemia Treatment with KL003 Cell Injection
| NCT ID | NCT06280378 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Kanglin Biotechnology (Hangzhou) Co., Ltd. |
| Condition | Transfusion-dependent Beta-Thalassemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 41 participants |
| Start Date | 2024-04-05 |
| Primary Completion | 2025-10 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 41 participants in total. It began in 2024-04-05 with a primary completion date of 2025-10.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion 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. Exclusion Criteria: * 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; * Subjects 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 virus antibody (HTLV-Ab), Treponema pallidum antibody (TP-Ab); * Uncorrectable coagulation dysfunction or history of severe bleeding disorder; * History of major organ damage including: 1. Liver function test suggest AST or ALT levels \>3× upper limit of normal（ULN）; 2. Total serum bilirubin value\>2.5×ULN;if combined with Gilbert syndrome, total bilirubin\>3×ULN and direct bilirubin value\>2.5×ULN; 3. Left ventricular ejection fraction \<45%; 4. Baseline calculated eGFR\<60mL/min/1.73m2; 5. Pulmonary function:FEV1/FVC\<60% and/or diffusion capacity of carbon monoxide (DLco) \<60% of prediction;
Contact & Investigator
Haoquan Wu, PhD
STUDY DIRECTOR
R&D Kanglin Biotech
Frequently Asked Questions
Who can join the NCT06280378 clinical trial?
This trial is open to participants of all sexes, aged 3 Years or older, up to 35 Years, studying Transfusion-dependent Beta-Thalassemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06280378 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06280378 currently recruiting?
Yes, NCT06280378 is actively recruiting participants. Contact the research team at yanjingfeng@kanglinbio.com for enrollment information.
Where is the NCT06280378 trial being conducted?
This trial is being conducted at Shanghai, China, Tianjin, China.
Who is sponsoring the NCT06280378 clinical trial?
NCT06280378 is sponsored by Kanglin Biotechnology (Hangzhou) Co., Ltd.. The principal investigator is Haoquan Wu, PhD at R&D Kanglin Biotech. The trial plans to enroll 41 participants.