BridgeMD
Tests treatment safety and results for Transfusion-dependent Beta-Thalassemia
Official title Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
ClinicalTrials.gov ID: NCT06219239
What this study is testing
What is KL003 cell injection Drug Product?
KL003 cell injection Drug Product is an investigational medicine, 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. 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 transfusion in subjects with transfusion-dependent β-thalassemia.
- 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...
- Documented baseline, or pretransfusion, Hb level≤7 g/dL
- Karnofsky performance status ≥70 for people≥16 years of age; Lansky performance status of ≥70 for people\<16 years of age
- Eligible to undergo auto-HSCT
You likely can't join if
- Presence of clear contraindications for hematopoietic stem cell collection
- Diagnosis of composite α thalassemia
- A white blood cell (WBC) count \<3×10\^9/L, and/or platelet count \<100×10\^9/L not related to hypersplenism
- people 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...
- Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder
- Meet the criteria for allo-HSCT and with an identified willing donor with a full HLA match
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
- Documented baseline, or pretransfusion, Hb level≤7 g/dL
- Karnofsky performance status ≥70 for people≥16 years of age; Lansky performance status of ≥70 for people\<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-ups in accordance with the protocol requirements
- Presence of clear contraindications for hematopoietic stem cell collection
- Diagnosis of composite α thalassemia
- A white blood cell (WBC) count \<3×10\^9/L, and/or platelet count \<100×10\^9/L not related to hypersplenism
- people 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
- Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder
- 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
- people with any severe active fungal, bacterial, viral, tuberculosis or other infection, including active hepatitis B (defined as serum HBV-DNA ≥2000 IU/ml), active hepatitis C virus, HCV) infection, human...
- 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...
- 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;
- History of bridging fibrosis, cirrhosis;
- Left ventricular ejection fraction \<45%;
- New York Heart Association (NYHA) class III or IV congestive heart failure;
- Severe arrhythmia requiring medical treatment;
- Uncontrolled hypertension or unstable angina pectoris;
- Myocardial infarction or bypass or stent surgery within 12 months before drug administration;
- Valvular disease with clinical significance;
- Baseline calculated eGFR\<60mL/min/1.73m2;
- Pulmonary function: FEV1/FVC\<60% and/or diffusion capacity of carbon monoxide (DLco) \<60% of prediction;
- Evidence of clinically significant pulmonary hypertension requiring medical intervention.
- Uncorrectable coagulation dysfunction or history of severe bleeding disorder
- Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician
- Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.)
- Participated in other clinical studies within 3 months prior to screening
- Inoculated live vaccine within 6 weeks prior to screening
- Pregnancy or breastfeeding women; people or their sexual partners were unable to take medically recognized effective contraceptive measures during the 27-month study period
- The people or their parents would not comply with the study procedures outlined in the protocol
- The people received hydroxyurea or thalidomide or hypomethylating drugs within 3 months before hematopoietic stem cell collection
- Patients considered to be ineligible for the study by the investigator for reasons other than the above
The study team makes the final eligibility decision.
Where it's taking place
- 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 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.