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Tests treatment safety and results for Β-thalassemia
Official title Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
ClinicalTrials.gov ID: NCT06655662
What this study is testing
- What it's testing
- This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia. The study aims to evaluate the safety and efficacy of autologous lentiviral-transduced CD34+ human hematopoietic stem cells (hHSCs) using the gene therapy product HGI-001.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 35
You may be able to join if
- Aged 6-35 years (inclusive), ICF can be provided by the patient and/or legal guardian;
- Definitively diagnosed with severe TDT without genotype restriction (excluding patients with coexisting α-thalassemia), and a valid test report can...
- Average transfusion volume \> 100 mL/kg/year or transfusion frequency \> 8 times/year within 2 years prior to enrollment；
- At least 3 months of full volume transfusion (verification of blood transfusion records can be provided) prior to screening, and Hb is maintained at...
- Serum ferritin level less than 5000μg/L, with moderate or lower iron overload in the heart and liver as indicated by magnetic resonance imaging (MRI...
You likely can't join if
- Patients with fully HLA-matched donors;
- Having previously received gene therapy, gene editing therapy, or allogeneic hematopoietic stem cell transplantation;
- Uncorrected bleeding disorder;
- Uncontrolled epilepsy and mental illness;
- Within the past 3 months prior to enrollment, the use of Luspatercept, Hydroxyurea, Ruxolitinib, Thalidomide, Decitabine, or Ara-c has been...
- Psychoactive substance abuse, drug or alcohol abuse within 6 months prior to enrollment;
See the full eligibility criteria
- Aged 6-35 years (inclusive), ICF can be provided by the patient and/or legal guardian;
- Definitively diagnosed with severe TDT without genotype restriction (excluding patients with coexisting α-thalassemia), and a valid test report can be provided;
- Average transfusion volume \> 100 mL/kg/year or transfusion frequency \> 8 times/year within 2 years prior to enrollment；
- At least 3 months of full volume transfusion (verification of blood transfusion records can be provided) prior to screening, and Hb is maintained at ≥ 9.0 g/dL;
- Serum ferritin level less than 5000μg/L, with moderate or lower iron overload in the heart and liver as indicated by magnetic resonance imaging (MRI T2\ ), specifically liver MRI T2\ greater than 1.4ms and cardiac MRI...
- Acceptable organ functions (including heart, liver, kidney, lung and coagulation functions), stable disease condition, and suitable for busulfan pre-treatment and hematopoietic stem cell (HSC) transplantation as judged...
- Meets follow-up requirements, adheres to treatment arrangements, and is able to return to the hospital regularly to undergo various examinations within 2 years after reinfusion of HGI-001 injection.
- Patients with fully HLA-matched donors;
- Having previously received gene therapy, gene editing therapy, or allogeneic hematopoietic stem cell transplantation;
- Uncorrected bleeding disorder;
- Uncontrolled epilepsy and mental illness;
- Within the past 3 months prior to enrollment, the use of Luspatercept, Hydroxyurea, Ruxolitinib, Thalidomide, Decitabine, or Ara-c has been administered；
- Psychoactive substance abuse, drug or alcohol abuse within 6 months prior to enrollment;
- Patients with pulmonary hypertension who have not been given effective intervention;
- Positive for anti-RBC antibodies in antibody screening;
- Hepatitis B surface antigen (HBsAg) is positive and the HBV DNA copy number is greater than the upper limit of the normal value of the detection unit (those who are negative do not need to test for HBV DNA copy number)...
- Have or have had malignant tumors or myeloproliferative diseases or immunodeficiency disorders or autoimmune diseases;
- Have a first-degree relative with a history of or suspected hereditary cancer (including but not limited to hereditary breast and ovarian cancer, nonpolyposis colorectal cancer, and adenomatous polyposis);
- Severe bacterial, viral, fungal or parasitic infection;
- Other illnesses which render the subject unsuitable for participation (e.g., severe liver, kidney or heart disease); Definition of severe liver and kidney disease: a. Aspartate aminotransferase (AST), alanine...
- WBC \< 3 × 10\^9/L and/or PLT \< 100 × 10\^9/L;
- Has diabetes, abnormal thyroid functions or other endocrine disorder;
- Participated in other treatment clinical studies within 4 weeks before the trial;
- Poor adherence or other conditions that renders the subject unsuitable for participation as judged by the investigator.
The study team makes the final eligibility decision.
Where it's taking place
- Guangxi, China
- Shenzhen, 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, 6 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Guangxi, China; Shenzhen, 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.