Long-term observation of BHC001 treatment for beta thalassemia

Long-term Follow-up Study of BHC001 in the Treatment of Transfusion-dependent β-thalassemia

Observational Bioray Laboratories · NCT06314529

This study looks at the long-term safety and effects of a treatment called BHC001 in people with beta thalassemia who need regular blood transfusions.

Quick facts

Study typeObservational
Enrollment6 (estimated)
Ages5 Years to 35 Years
SexAll
SponsorBioray Laboratories Industry-sponsored
Locations2 sites (Nanning, Guangxi and 1 other locations)
Trial IDNCT06314529 on ClinicalTrials.gov

What this trial studies

This study observes the long-term safety and efficacy of BHC001 in patients with transfusion-dependent beta thalassemia (TDT). Participants who have been treated with BRL-101 will be monitored for delayed safety risks, including the emergence of neoplasms and hematological disorders. The study aims to gather comprehensive data on the long-term effects of the treatment following intravenous infusion. It is an observational study, meaning it will focus on collecting data without any experimental intervention.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals with transfusion-dependent beta thalassemia who have previously been treated with BRL-101.

Not a fit: Patients who have not been treated with BRL-101 or those with other forms of thalassemia may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term safety and effectiveness of BHC001 for patients with beta thalassemia.

How similar studies have performed: While this study focuses on long-term effects, similar observational studies have shown success in monitoring treatment outcomes in chronic conditions, suggesting a potential for valuable findings.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

1. Provision of written informed consent for this study by subjects, or as applicable, subject's parent(s)/legal guardian(s)
2. Treated with BRL-101 for therapy of transfusion-dependent β-thalassemia.

Exclusion Criteria:

\- There are no exclusion criteria for this study

Where this trial is running

Nanning, Guangxi and 1 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Thalassemia, Beta
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.