Β-thalassemia study: BD211
Official title A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major
ClinicalTrials.gov ID: NCT06465550
What this study is testing
What is BD211?
BD211 is an investigational gene therapy being studied for transfusion‑dependent β‑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
- The study will look at how safe and well the new stem cells work after a strong pre‑treatment that wipes out the body’s blood cells.
- Who it's for
- It is for adults who have β‑thalassemia that requires regular blood transfusions.
- What's involved
- Participants will receive a high‑dose chemotherapy or radiation to clear their blood cells, then a transplant of their own stem cells that have been changed to produce a normal form of hemoglobin. They will be monitored with blood tests and other checks.
- Time commitment
- The study lasts about 12 months, with frequent visits in the first few months and then regular follow‑up visits over the year.
- 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 3 to 35
You may be able to join if
- Participants aged 3 years (inclusive) to 18 years (exclusive), with no gender restrictions.
- Parents/legal guardians have fully understood and voluntarily signed a written informed consent form
- Transfusion-dependent β-thalassemia patients. "Transfusion-dependent" is defined as: requiring at least 100 mL/kg of packed red blood cells annually
- Eligible for allogeneic hematopoietic stem cell transplantation, but without a donor or those refusing to undergo allogeneic hematopoietic stem cell...
- Have undergone symptomatic treatment for at least the past 2 years and have retained medical records including transfusion history.
You likely can't join if
- Has a fully HLA-matched hematopoietic stem cell donor and is willing to receive a fully HLA-matched hematopoietic stem cell transplant.
- Positive for antibodies against Human Immunodeficiency Virus 1/2 (HIV-1/HIV-2), Treponema pallidum (TP) specific antibodies, Human T-lymphotropic...
- Positive for Hepatitis B Virus (HBV) HbsAg or HBV-DNA; Hepatitis C Virus (HCV) HCAb positive
- Severe active bacterial, viral, fungal, malarial, or parasitic infections.
- Has had, or currently has, a malignant, myeloproliferative, or immunodeficiency disorder.
- Direct relatives with known or suspected hereditary cancer syndromes (including but not limited to breast cancer, colorectal cancer, ovarian cancer...
See the full eligibility criteria
- Participants aged 3 years (inclusive) to 18 years (exclusive), with no gender restrictions.
- Parents/legal guardians have fully understood and voluntarily signed a written informed consent form
- Transfusion-dependent β-thalassemia patients. "Transfusion-dependent" is defined as: requiring at least 100 mL/kg of packed red blood cells annually
- Eligible for allogeneic hematopoietic stem cell transplantation, but without a donor or those refusing to undergo allogeneic hematopoietic stem cell transplantation.
- Have undergone symptomatic treatment for at least the past 2 years and have retained medical records including transfusion history.
- Stable condition and maintained an appropriate iron chelation regimen.
- Good status of organ function.
- Good compliance from the individual and parents/legal guardians, willing to adhere to visit schedules, trial plans, laboratory tests, and other trial procedures as stipulated in this protocol.
- Willing to participate in long-term follow-up research.
- Has a fully HLA-matched hematopoietic stem cell donor and is willing to receive a fully HLA-matched hematopoietic stem cell transplant. Enrollment is otherwise only advised after review by the safety review committee.
- Positive for antibodies against Human Immunodeficiency Virus 1/2 (HIV-1/HIV-2), Treponema pallidum (TP) specific antibodies, Human T-lymphotropic Virus 1 or 2 (HTLV-1/HTLV-2) antibodies, and Vesicular Stomatitis Virus G...
- Positive for Hepatitis B Virus (HBV) HbsAg or HBV-DNA; Hepatitis C Virus (HCV) HCAb positive; positive nucleic acid test for Epstein-Barr Virus (EBV) or Cytomegalovirus (CMV).
- Severe active bacterial, viral, fungal, malarial, or parasitic infections.
- Has had, or currently has, a malignant, myeloproliferative, or immunodeficiency disorder.
- Direct relatives with known or suspected hereditary cancer syndromes (including but not limited to breast cancer, colorectal cancer, ovarian cancer, prostate cancer, and pancreatic cancer).
- Autoimmune diseases that could result in transfusion difficulties.
- Liver cirrhosis, fibrosis, or active hepatitis, and/or abnormal liver function tests (Serum total bilirubin (TBIL) ≥ 1.5x Upper Limit of Normal (ULN)
- Heart disease, or Left Ventricular Ejection Fraction (LVEF) \< 60%.
- Kidney diseases, or serum creatinine ≥ 1.5ULN, creatinine clearance rate \< 30% of the normal level (measured or calculated by the Cockcroft-Gault equation).
- Endocrine disorders, such as insulin-dependent diabetes, hyperthyroidism, or hypothyroidism.
- Severe iron overload, serum ferritin ≥ 5000 ng/mL.
- Cardiac T2\ \< 20 ms, and/or liver iron content (LIC) ≥ 15mg/g liver weight by MRI.
- Significant pulmonary hypertension diagnosed clinically according to guidelines, requiring clinical medical intervention.
- Uncorrected bleeding disorders.
- Severe psychiatric disorders.
- Peripheral blood white cell (WBC) count \< 3x10\^9/L or platelets count \< 120x10\^9/L.
- Received hydroxyurea treatment within the last 3 months before stem cell collection.
- Used erythropoiesis-stimulating agents within the 3 months prior to HSC collection.
- History of allogeneic transplantation.
- Previously received any type of gene and/or cell therapy.
- Participating in another clinical trial and is within a 30-day screening period.
- Has contraindications to anesthesia.
- Has contraindications to hematopoietic stem cell collection.
- Allergic to the investigational drug or its excipients.
- Any other conditions determined by the investigator as unsuitable for participation in this clinical trial.
The study team makes the final eligibility decision.
Where it is enrolling
- Guangzhou, Guandong, China
- Nanning, Guangxi, China
- Shanghai, Shanghai City, 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, but 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?
This study is enrolling now at Guangzhou, Guandong, China; Nanning, Guangxi, China; Shanghai, Shanghai City, China. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.