A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Parti… (NCT05015920) | Clinical Trial Compass
CompletedNot Applicable
A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants
China2 participantsStarted 2021-07-10
Plain-language 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.
Who can participate
Age range
5 Years – 35 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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.
Exclusion criteria
. 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Evaluate the success and kinetics of HSC engraftment.
Timeframe: At multiple timepoints after infusion for 24 months.
2
Incidence of transplant-related mortality through 100 days post-transplant.
Timeframe: Up to 100 days post-HSCT.
3
Overall survival of maintenance phase.
Timeframe: Up to 24 months post-HSCT.
4
Post-transplant blood samples for replication competent lentivirus (RCL) testing.
Timeframe: At multiple timepoints after infusion for 24 months.
5
Assessment of Clonal dominance or leukemia/lymphoma and other malignancies.
Timeframe: At multiple timepoints after infusion for 24 months.
6
Incidence of treatment- related adverse events.
Timeframe: Up to 24 months after BD211 drug product infusion.