Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.

NCT05860595 | Active Not Recruiting

• 1 other identifier: IIT2023021
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells in subjects with transfusion-dependent β-thalassemia.

Conditions

Transfusion-dependent Beta-Thalassemia

Keywords

Beta-Thalassaemia; Hematopoietic Stem-Cell Transplantation

Outcome Measures

Primary Outcomes (5)

• Percentage of participants with successful lentiviral vector transduced CD34+ stem cell engraftment

  Successful engraftment was defined as neutrophil count \[ANC\] ≥0.5×10\^9/L for 3 consecutive days

  Up to 42 days post transplant

• Engraftment time of neutrophil

  The first day when neutrophils ≥ 0.5×10\^9/L for 3 consecutive days

  Up to 42 days post transplant

• Engraftment time of platelet

  The first day of platelet count ≥ 20.0×10\^9/L for 7 consecutive days after platelet transfusion independence

  Up to 42 days post transplant

• Transplant-related mortality within 100 days and within 1 year after reinfusion of KL003 drug product

  Up to 1 year post transplant

• The number, frequency and severity of adverse events (AE) within 1 year after reinfusion of KL003 drug products

  Frequency and severity of AEs \& SAEs identified according to NCI CTCAE 5.0

  Up to 24 months post transplant

Secondary Outcomes (3)

• The proportion of participants who meet the definition of transfusion independence (TI) for at least 6 months

  Up to 24 months post transplant

• The duration of transfusion independence

  Up to 24 months post transplant

• Changes in the frequency and volume of blood transfusion

  Up to 24 months post transplant

Study Arms (1)

KL003 cell injection Drug Product

EXPERIMENTAL

Each recruited subject will accept KL003 Transplantation.

Genetic: KL003 cell injection Drug Product

Interventions

KL003 cell injection Drug Product GENETIC

Transplant of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene.

KL003 cell injection Drug Product

Eligibility Criteria

• Age: 3 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify 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 2 years
• Documented baseline, or pretransfusion, Hb level≤7 g/dL
• Karnofsky performance status ≥70 for subjects≥16 years of age; Lansky performance status of ≥70 for subjects\<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 and maintain detailed medical records, including transfusion history
• Subject and/or legal guardians voluntarily participated in this clinical trial and signed the informed consent form, and can complete all follow-up in accordance with the protocol requirements

You may not qualify if:

• 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), Treponema pallidum antibody (TP-Ab)
• Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the clinical investigator
• Contraindication to bone marrow collection
• Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder
• A white blood cell (WBC) count \<3×10\^9/L, and/or platelet count \<100×10\^9/L not related to hypersplenism
• Diagnosis of composite α thalassemia
• Participants with severe iron overload at the time of screening: severe iron overload of the liver showed by MRI, serum ferritin ≥ 5000 ng/mL, or moderate to severe iron overload of the heart
• Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody
• Meet the criteria for allo-HSCT and with an identified willing donor with a full HLA match
• Prior receipt of gene therapy or allo-HSCT
• Immediate family member (i.e. parent or siblings) with a known Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome and familial adenomatous polyposis)
• Diagnosis of a significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study
• 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;

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• R&D Kanglin Biotech: collaborator

Study Sites (1)

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China

Location

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2023
• First Posted: May 16, 2023
• Study Start: May 23, 2023
• Primary Completion: August 20, 2025
• Study Completion: October 24, 2025
• Last Updated: August 5, 2025

Record last verified: 2025-03

Locations

CN (1)