Β-Thalassemia Treatment with KL003 Cell Injection

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

41 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-05

Study Completion Date

2027-05-31

Brief Summary

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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.

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Detailed Description

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This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess KL003 Cell Injection in up to 41 participants with transfusion-dependent β-thalassemia (TDT) who are ≥3 and ≤35 years of age. KL003 Cell Injection is autologous CD34+ stem cells transduced Ex Vivo with a lentiviral Vector encoding βA-T87Q-Globin.

Conditions

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Transfusion-dependent Beta-Thalassemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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KL003 Cell Injection Drug Product

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

Group Type EXPERIMENTAL

KL003 Cell Injection Drug Product

Intervention Type DRUG

Administered by intravenous infusion after myeloablative conditioning with busulfan.

Interventions

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KL003 Cell Injection Drug Product

Administered by intravenous infusion after myeloablative conditioning with busulfan.

Intervention Type DRUG

Eligibility Criteria

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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;

Minimum Eligible Age

3 Years

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No