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Tests treatment safety and results for β-thalassemia Major
Official title Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia
ClinicalTrials.gov ID: NCT05776173
What this study is testing
What is BD211?
BD211 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for β-thalassemia major.
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 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.
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
- Ages 6 to 35 years old, including: people should be able to provide an ICF. Diagnosed as Transfusion Dependent β-thalassemia with any genotype (β0...
- 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.
You likely can't join 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.
See the full eligibility criteria
- Ages 6 to 35 years old, including: people 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...
- 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.
- 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).
- people 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...
- 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.
- people 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 people 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.
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, 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 Shanghai, 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.