Long-term Follow-up Study of BHC001 for TDT

Recruiting

• Conditions: Thalassemia, Beta
• Interventions: Other: Assessments

• Registration Number: NCT06314529
• Lead Sponsor: Bioray Laboratories

Brief Summary

Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.

Detailed Description

Observe long-term delayed safety risks such as emerging neoplasms, emerging haematological disorders, and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.

Study Timeline

• Study Start: 2022-12-14
• Status Verified: 2024-03
• Study First Submitted: 2024-03-06
• Study First Submitted QC: 2024-03-11
• Last Update Submitted: 2024-03-11
• Study First Posted: 2024-03-18
• Last Update Posted: 2024-03-18
• Primary Completion: 2036-12-10
• Study Completion: 2037-05-10

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 6

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
BRL-101	Assessments	All patients who have received BRL-101

Primary Outcome Measures

Name	Time	Method
Frequency, grade, and relationship of SAEs	Within 2 years to 5years after BRL-101 Infusion	Frequency, grade, and relationship to BRL-101 of SAEs following BRL-101 reinfusion
Incidence of hematologic malignancies	Up to 15 years post-drug product infusion	Such as new lymphoma, leukemia, MDS, etc.

Trial Locations

Locations (2)

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army; 🇨🇳 Nanning, Guangxi, China

Xiangya Hospital of Central South University; 🇨🇳 Changsha, Hunan, China