A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants

NCT05015920 | Completed

• 1 other identifier: 920IEC/AF/61/2019-01.0
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Hematologic Diseases

Keywords

TDT; β-thalassemia major

Outcome Measures

Primary Outcomes (6)

• 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.

  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.

  Up to 100 days post-HSCT.

• Overall survival of maintenance phase.

  Overall survival up to 24 months post-HSCT.

  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.

  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.

  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.

  Up to 24 months after BD211 drug product infusion.

Secondary Outcomes (8)

• Quantify gene transfer efficiency and expression of BD211 drug product.

  Up to 24 months after engraftment.

• Quantify the hematopoietic chimerism resulting from treatment with BD211 drug product.

  Up to 24 months after engraftment.

• HbAT87Q in peripheral blood

  Up to 24 months after engraftment.

• Reduction of RBC transfusion requirements from baseline

  Up to 24 months after engraftment.

• Duration of transfusion independence (months).

  Up to 24 months after engraftment.

Study Arms (1)

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.

Genetic: BD211 Drug Product

Interventions

BD211 Drug Product GENETIC

Transplantation of Autologous CD34+Stem Cells Transduced to BD211 finished Product with a Lentiviral Vector coding βA-T87Q-Globin.

Mobilization,harvest,transduction,conditioning,treatment,engraftment

Eligibility Criteria

• Age: 5 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.
• Participation in another clinical study with an investigational drug within 30 days of screening.
• Hydroxyurea therapy within 3 months before hematopoietic stem cell collection.

Sponsors & Collaborators

• Shanghai BDgene Co., Ltd.: lead
• 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China: collaborator

Study Sites (1)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

Kunming, Yunnan, 650000, China

Location

MeSH Terms

Conditions

Hematologic Diseases

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases

Study Officials

• Sanbin Wang, Dr.

  920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 7, 2021
• First Posted: August 23, 2021
• Study Start: July 10, 2021
• Primary Completion: August 23, 2024
• Study Completion: December 31, 2024
• Last Updated: May 18, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)