Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia

Recruiting now · Not applicable

Conditions studied: β-thalassemia Major

In brief

This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.

Key facts

Study ID

NCT05776173

Run by

Shanghai BDgene Co., Ltd.

People needed

10

Starts

2023-08-10

Expected to finish

2026-10-01

Last updated by the study team

2024-06-14

Who can join

Age: 6 and older, up to 35. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Ages 6 to 35 years old, including:
• Subjects should be able to provide an ICF. Diagnosed as Transfusion Dependent β-thalassemia with any genotype (β0, β+, βE/β0, βS/S, βS/β0, βS/β+), confirmed the Hb analysis. No alfa chain genetic abnormalities. Subjects must stabilize and maintain an appropriate iron chelation regimen. Transfusion-dependent types are defined as requiring at least 100 mL/kg/ year of red blood cells (pRBCs).
• The tumor genes chip detection results about acute leukemia and myeloid tumor gene mutations (panel) showed no abnormality.
• There were candidates for HLA gene semi-compatible hematopoietic stem cell transplantation.
• No eligiblity for allogeneic hematopoietic stem cell transplantation.
• The treatment of erythrocyte maturation agent luspatercept cannot be financially supported.
• The investigator confirmed that subject was willing to follow the research procedures.
• Having complete medical records including a history of blood transfusions testified subject received treatment and followed up for at least two years prior to screening.

You may not qualify if…

• Availability of voluntary, fully HLA-matched hematopoietic cell donors, unless recommended for inclusion by the Monitoring Committee.
• HIV-1 and HIV-2 were positive, and / or HTLV-1, HTLV-2 and VSV-G antibodies were positive.
• An active bacterial, viral, fungal or parasitic infection.
• Contraindicated for the extraction of bone marrow under anesthesia.
• Any malignancy, myeloproliferative, or immunodeficient disease and relevant medical history.
• Peripheral blood white blood cell (WBC) count < 3×10\^9/L or platelet count < 120×10\^9/L.
• A history of allo-transplantation.
• Erythropoietin was used within 3 months prior to HSC cell collection.
• Immediate family members with known or suspected familial cancer syndromes (including but not limited to breast, colorectal, ovarian, prostate, and pancreatic cancers).
• Subjects with a diagnosis of major mental illness may had a serious disability to participate in the study.
• Active recurrent malaria.
• Pregnant or postpartum nursing or unable to use contraception.
• History of major organ injury including:
• Liver disease, transaminase > 3 times the upper limit of normal. (If the liver biopsy does not reveal evidence of widespread bridging fibrosis, cirrhosis, or acute hepatitis, this indicator will not be used as a criterion for the exclusion); Widely bridging fibrosis, histopathological evidence of acute hepatitis or cirrhosis showed in liver biopsy Heart disease, left ventricular ejection fraction < 25%; Kidney disease, creatinine clearance < 30% normal level; Of severe iron overload, confirmed by the study doctor; An heart MRI detection of T2 * < 10 ms; Significant pulmonary hypertension needing clinical medical intervention.
• Any other conditions being ineligible for HSC transplantation determined by the investigator.
• The subject involved with another clinical study in a 30-day screening period.
• Subjects who expected to become parents during the 27-month study period.
• Prior treatment with any type of gene and/or cell therapy.
• As assessed by the investigator, the subjects or their parents are unable to comply well with the study procedures per protocol.
• Hydroxyurea treatment within 3 months prior to hematopoietic stem cell collection.

Where it is running

• Shanghai Ruijin Hospital — Shanghai, China (enrolling)

Full record on ClinicalTrials.gov

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