Hematologic DiseasesNCT05015920
A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants
A study testing BD211 Drug Product in people with Hematologic Diseases, run by Shanghai BDgene Co., Ltd. at 1 site in China.
Trial summary
This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration. Read more about this study
- Who is it for?
- Ages 5 to 35 years
- Conditions the study is for
- Hematologic Diseases
- What is being tested?
- BD211 Drug Product
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteChina
- Study dates
- 10 Jul 2021to 31 Dec 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 5 to 35 years of age.
- Be eligible for allogeneic HSCT based on institutional medical guideline, but without a matched related donor.
- Transfusion-dependent β-Thalassemia Major, regardless of the genotype, with the diagnosis confirmed by Hb studies. Subjects must be stable and maintained on an appropriate iron chelation regimen. Transfusion dependence is defined as requiring at least 100 mL/kg/year of packed red blood cells(pRBCs).
- Have been treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.
- Be willing and able, in the Investigator's opinion, to comply with the study procedures outlined in the study protocol. If a pediatric subject, the subject's parent/legal guardian also must be willing and able to comply with the study procedures outlined in the study protocol.
Who cannot join
- Availability of a willing matched HLA-identical sibling hematopoietic cell donor.
- Positive for presence of human immunodeficiency virus, human T-lymphotropic virus, vesicular stomatitis virus G antibody.
- Clinically significant, active bacterial, viral, fungal, or parasitic infection.
- A white blood cell (WBC) count<3x109/L and/or platelet count<120x109/L
- Receipt of an allogeneic transplant.
- Receipt of erythropoietin within 3 months before HSCT harvest.
Show all 19Show fewer
- Contraindication to anesthesia for bone marrow harvesting.
- Any of prior or current malignancy, myeloproliferative or immunodeficiency disorder.
- Active relapsing malaria
- Immediate family member with a known or suspected Familial Cancer Syndrome.
- Diagnosis of significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study.
- Pregnancy or breastfeeding in a postpartum female or absence of adequate contraception for fertile subjects.
- Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician.
- History of major organ damage.including Liver, Heart, Kidney disease, pulmonary hypertension ,severe iron overload, which in the opinion of the physician is grounds for exclusion.
- Participation in another clinical study with an investigational drug within 30 days of screening.
- Hydroxyurea therapy within 3 months before hematopoietic stem cell collection.
- An assessment by the Investigator that the subject or parents of the subject will not comply with the study procedures outlined in the study protocol.
- Subjects who have the desire to become a parent within the 27-month study period.
- Prior receipt of gene therapy.
Trial locations and contacts
1 site in 1 country.
China1 site
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Kunming, Yunnan, 650000
About this study
Official title: A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+Stem Cells Transduced With a Lentiviral Vector Encoding βA-T87Q-Globin
This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.
Read the detailed description
After collection of mobilised peripheral blood samples, the patient's autologous cells,enriched for CD34+ HSCs, undergo ex vivo transduction with lentiviral vector encoding βA-T87Q-globin to BD211 finished product,which is then infused intravenously into the patient after myeloablative busulfan conditioning to prepare bone marrow "niches" for engraftment of the HSCs.
After discharge, subjects will be followed monthly, at a minimum, for 6 months and thereafter every 3 months for the remainder of the 24 months post-transplant.
Evaluation will include Routine and special biological testing at regular intervals, collection of AEs and concomitant medications, and evaluation of disease specific biological and clinical parameters.
Subjects will then be enrolled in a long-term follow-up protocol with annual evaluations for an additional 13 years post-transplant.
The long-term follow-up study will focus on long-term safety, with an emphasis on integration site analysis, and long-term efficacy.
This study will end when the last subject completes the Month 24 visit or discontinues from the study.
- Conditions
- Hematologic Diseases
- Being tested
- BD211 Drug Product
- Keywords
- TDT, β-thalassemia major
- Medical subject headings
- Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BD211 Drug ProductGenetic
Transplantation of Autologous CD34+Stem Cells Transduced to BD211 finished Product with a Lentiviral Vector coding βA-T87Q-Globin.
| Study group | Role | What they receive |
|---|---|---|
| Mobilization,harvest,transduction,conditioning,treatment,engraftment | Experimental | Subjects will participate in this study for a total of approximately 27 months, consisting of an up to 3 months pre-transplant period(consisting of a screening period followed by autologous cell harvest, followed by a waiting period during which the harvested cells are transduced and undergo release testing, followed by treatment with busulfan IV, and a single infusion of BD211 Drug Product) and a 24-month post-transplant evaluation period. Following completion of this study, all subjects will be asked to provided consent to participate in a follow-up study for another 13 years, which will focus on long-term safety, with an emphasis on integration site analysis, and long-term efficacy. Receives: Genetic: BD211 Drug Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the success and kinetics of HSC engraftment.
Three consecutive absolute neutrophil counts≥500 cells/uL, three consecutive platelet values ≥20 e9/L, measure blood samples monthly after BD211 drug product infusion.
Measured: At multiple timepoints after infusion for 24 months.
Incidence of transplant-related mortality through 100 days post-transplant.
Incidence of transplant-related mortality through 100 days post-transplant.
Measured: Up to 100 days post-HSCT.
Overall survival of maintenance phase.
Overall survival up to 24 months post-HSCT.
Measured: Up to 24 months post-HSCT.
Post-transplant blood samples for replication competent lentivirus (RCL) testing.
The testing of any subject positivity will be considered an SAE and suspend the inclusion of new subjects.
Measured: At multiple timepoints after infusion for 24 months.
Assessment of Clonal dominance or leukemia/lymphoma and other malignancies.
Using peripheral blood of subjects for integration site analysis via LAM-PCR \& deep sequencing.
Measured: At multiple timepoints after infusion for 24 months.
Incidence of treatment- related adverse events.
According to the requirements of the National Cancer Institute Common Terminology Standards for Adverse Events (NCI CTCAE) version 5.0, monitor laboratory parameters and the frequency and severity of clinical AEs.
Measured: Up to 24 months after BD211 drug product infusion.
Secondary outcomes (8)
Quantify gene transfer efficiency and expression of BD211 drug product.
Expression of βA-T87Q-globin chain in whole blood by assessing the ratio of βA-T87Q-globin to α-globin, as well as βA-T87Q-globin fractions in all β-chains over time by HPLC.
Measured: Up to 24 months after engraftment.
Quantify the hematopoietic chimerism resulting from treatment with BD211 drug product.
Measuring lentiviral vector copy number per diploid genome(c/dg) for evaluation.
Measured: Up to 24 months after engraftment.
HbAT87Q in peripheral blood
Measure the effects of transplantation with BD211 on the expression of disease-specific biological parameters and clinical events, including amounts of HbAT87Q in peripheral blood in grams per deciliter (g/dL).
Measured: Up to 24 months after engraftment.
Reduction of RBC transfusion requirements from baseline
Reduction from baseline of RBC transfusion requirements (mL/kg) per month and year post-transplant, Up to 24 months after engraftment.
Measured: Up to 24 months after engraftment.
Duration of transfusion independence (months).
Duration of transfusion independence up to 24 months after engraftment.
Measured: Up to 24 months after engraftment.
Weighted average Hemoglobin
Weighted average Hemoglobin during transfusion independence in grams per deciliter (g/dL), up to 24 months after engraftment.
Measured: Up to 24 months after engraftment.
Changes of liver iron burden from baseline
Changes from baseline in iron burden by assessing liver iron content (mg Fe/g dry weight).
Measured: Up to 24 months after engraftment.
Changes of cardiac iron burden from baseline
Changes from baseline in iron burden by assessing cardiac iron content using magnetic resonance imaging (MRI) T2* value (milliseconds, ms).
Measured: Up to 24 months after engraftment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2021
- Study start10 Jul 2021
- Primary completion23 Aug 2024
- Study completion31 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematologic DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shanghai BDgene Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
- Responsible party
- Sponsor
- Investigators
- Sanbin Wang, Dr., Study chair, 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05015920
- Sponsor's protocol ID
- 920IEC/AF/61/2019-01.0
- Status last verified
- May 2025
- First submitted
- 7 Jun 2021
- Last updated
- 18 May 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Li S, Ling S, Wang D, Wang X, Hao F, Yin L, Yuan Z, Liu L, Zhang L, Li Y, Chen Y, Luo L, Dai Y, Zhang L, Chen L, Deng D, Tang W, Zhang S, Wang S, Cai Y. Modified lentiviral globin gene therapy for pediatric beta0/beta0 transfusion-dependent beta-thalassemia: A single-center, single-arm pilot trial. Cell Stem Cell. 2024 Jul 5;31(7):961-973.e8. doi: 10.1016/j.stem.2024.04.021. Epub 2024 May 16.
PubMed 38759653
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+Stem Cells Transduced With a Lentiviral Vector Encoding βA-T87Q-Globin
- ClinicalTrials.gov ID
- NCT05015920
- Acronym / sponsor ID
- 920IEC/AF/61/2019-01.0
- Conditions
- Hematologic Diseases
- Keywords
- TDT, β-thalassemia major
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Shanghai BDgene Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
- Study start
- 10 Jul 2021 (actual)
- Primary completion
- 23 Aug 2024 (actual)
- Study completion
- 31 Dec 2024 (actual)
- First posted
- 23 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.
Detailed description
After collection of mobilised peripheral blood samples, the patient's autologous cells,enriched for CD34+ HSCs, undergo ex vivo transduction with lentiviral vector encoding βA-T87Q-globin to BD211 finished product,which is then infused intravenously into the patient after myeloablative busulfan conditioning to prepare bone marrow "niches" for engraftment of the HSCs.
After discharge, subjects will be followed monthly, at a minimum, for 6 months and thereafter every 3 months for the remainder of the 24 months post-transplant.
Evaluation will include Routine and special biological testing at regular intervals, collection of AEs and concomitant medications, and evaluation of disease specific biological and clinical parameters.
Subjects will then be enrolled in a long-term follow-up protocol with annual evaluations for an additional 13 years post-transplant.
The long-term follow-up study will focus on long-term safety, with an emphasis on integration site analysis, and long-term efficacy.
This study will end when the last subject completes the Month 24 visit or discontinues from the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Mobilization,harvest,transduction,conditioning,treatment,engraftment | Experimental | Genetic: BD211 Drug ProductSubjects will participate in this study for a total of approximately 27 months, consisting of an up to 3 months pre-transplant period(consisting of a screening period followed by autologous cell harvest, followed by a waiting period during which the harvested cells are transduced and undergo release testing, followed by treatment with busulfan IV, and a single infusion of BD211 Drug Product) and a 24-month post-transplant evaluation period. Following completion of this study, all subjects will be asked to provided consent to participate in a follow-up study for another 13 years, which will focus on long-term safety, with an emphasis on integration site analysis, and long-term efficacy. |
Who may qualify
- Ages
- 5 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 5 to 35 years of age.
- Be eligible for allogeneic HSCT based on institutional medical guideline, but without a matched related donor.
- Transfusion-dependent β-Thalassemia Major, regardless of the genotype, with the diagnosis confirmed by Hb studies. Subjects must be stable and maintained on an appropriate iron chelation regimen. Transfusion dependence is defined as requiring at least 100 mL/kg/year of packed red blood cells(pRBCs).
- Have been treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.
- Be willing and able, in the Investigator's opinion, to comply with the study procedures outlined in the study protocol. If a pediatric subject, the subject's parent/legal guardian also must be willing and able to comply with the study procedures outlined in the study protocol.
Key exclusion criteria (19 listed)
- Availability of a willing matched HLA-identical sibling hematopoietic cell donor.
- Positive for presence of human immunodeficiency virus, human T-lymphotropic virus, vesicular stomatitis virus G antibody.
- Clinically significant, active bacterial, viral, fungal, or parasitic infection.
- A white blood cell (WBC) count<3x109/L and/or platelet count<120x109/L
- Receipt of an allogeneic transplant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BD211 Drug ProductTransplantation of Autologous CD34+Stem Cells Transduced to BD211 finished Product with a Lentiviral Vector coding βA-T87Q-Globin. | Genetic | Mobilization,harvest,transduction,conditioning,treatment,engraftment |
Primary outcome
Evaluate the success and kinetics of HSC engraftment.
Time frame: At multiple timepoints after infusion for 24 months.
Three consecutive absolute neutrophil counts≥500 cells/uL, three consecutive platelet values ≥20 e9/L, measure blood samples monthly after BD211 drug product infusion.
Incidence of transplant-related mortality through 100 days post-transplant.
Time frame: Up to 100 days post-HSCT.
Incidence of transplant-related mortality through 100 days post-transplant.
Overall survival of maintenance phase.
Time frame: Up to 24 months post-HSCT.
Overall survival up to 24 months post-HSCT.
Post-transplant blood samples for replication competent lentivirus (RCL) testing.
Time frame: At multiple timepoints after infusion for 24 months.
The testing of any subject positivity will be considered an SAE and suspend the inclusion of new subjects.
Assessment of Clonal dominance or leukemia/lymphoma and other malignancies.
Time frame: At multiple timepoints after infusion for 24 months.
Using peripheral blood of subjects for integration site analysis via LAM-PCR \& deep sequencing.
Incidence of treatment- related adverse events.
Time frame: Up to 24 months after BD211 drug product infusion.
According to the requirements of the National Cancer Institute Common Terminology Standards for Adverse Events (NCI CTCAE) version 5.0, monitor laboratory parameters and the frequency and severity of clinical AEs.
8 secondary outcomes
Quantify gene transfer efficiency and expression of BD211 drug product.
Time frame: Up to 24 months after engraftment.
Quantify the hematopoietic chimerism resulting from treatment with BD211 drug product.
Time frame: Up to 24 months after engraftment.
HbAT87Q in peripheral blood
Time frame: Up to 24 months after engraftment.
Reduction of RBC transfusion requirements from baseline
Time frame: Up to 24 months after engraftment.
Duration of transfusion independence (months).
Time frame: Up to 24 months after engraftment.
Weighted average Hemoglobin
Time frame: Up to 24 months after engraftment.
Changes of liver iron burden from baseline
Time frame: Up to 24 months after engraftment.
Changes of cardiac iron burden from baseline
Time frame: Up to 24 months after engraftment.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2025.
- Derived from this study: PubMed 38759653Li S, Ling S, Wang D, Wang X, Hao F, Yin L, Yuan Z, Liu L, Zhang L, Li Y, Chen Y, Luo L, Dai Y, Zhang L, Chen L, Deng D, Tang W, Zhang S, Wang S, Cai Y. Modified lentiviral globin gene therapy for pediatric beta0/beta0 transfusion-dependent beta-thalassemia: A single-center, single-arm pilot trial. Cell Stem Cell. 2024 Jul 5;31(7):961-973.e8. doi: 10.1016/j.stem.2024.04.021. Epub 2024 May 16.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sanbin Wang, Dr. study chair
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing BD211 Drug Product in people with Hematologic Diseases, run by Shanghai BDgene Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the success and kinetics of HSC engraftment. (time frame: At multiple timepoints after infusion for 24 months.). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05015920, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2025.