Treatment for Beta-Thalassemia using KL003 Cell Injection

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial is a non-randomized, open-label, single-dose study aimed at evaluating the safety and efficacy of KL003 cell injection in participants with transfusion-dependent β-thalassemia major. The study will involve up to 41 participants aged between 3 and 35 years, who will receive autologous CD34+ stem cells that have been modified using a lentiviral vector to produce βA-T87Q-Globin. The trial will assess the outcomes of this innovative gene therapy approach over a follow-up period of at least two years.

Why it matters

Who can join: the full rules

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;

Where it's running

• Ruijin Hospital, Shanghai Jiaotong University School of Medicine
  Shanghai, Shanghai Municipality, China Recruiting
• Institute of Hematology & Blood Diseases Hospital
  Tianjin, Tianjin Municipality, China Recruiting

Study contacts

• Study coordinator: jingfeng Yan
• Email: yanjingfeng@kanglinbio.com
• Phone: +86 18852138866

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Gene-modified stem cell therapy for transfusion-dependent beta-thalassemia

  A small, early-phase study will test a one-time infusion of a patient’s own genetically edited stem cells to see if it reduces transfusion needs in adults and children with transfusion-dependent beta-thalassemia.

  Recruiting at 2 sites Phase 2 Fondazione Telethon · Rome, Lazio and 1 other locations · NCT07680803

• Mitapivat for pediatric transfusion-dependent thalassemia (α- or β-TDT)

  This pediatric trial tests whether mitapivat can reduce or lessen the need for transfusions in children with transfusion-dependent alpha- or beta-thalassemia.

  Recruiting at 4 sites Phase 3 Agios Pharmaceuticals, Inc. · Atlanta, Georgia and 3 other locations · NCT07506863

• Comparing growth and development in children with beta-thalassemia after gene therapy

  This study looks at how gene therapy affects the growth and development of children with beta-thalassemia compared to those getting regular treatments and healthy kids.

  Recruiting Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT05991336

• Evaluating a new gene therapy for transfusion-dependent beta-thalassemia

  This study is testing a new gene therapy to see if it can help people with transfusion-dependent beta-thalassemia reduce or stop their need for regular blood transfusions.

  Recruiting Early Phase 1 First Affiliated Hospital of Guangxi Medical University · Nanning, Guangxi · NCT05762510

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.