NCT06314529

Brief Summary

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

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for all trials

Timeline
134mo left

Started Dec 2022

Longer than P75 for all trials

Geographic Reach
1 country

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress24%
Dec 2022May 2037

Study Start

First participant enrolled

December 14, 2022

Completed
1.2 years until next milestone

First Submitted

Initial submission to the registry

March 6, 2024

Completed
12 days until next milestone

First Posted

Study publicly available on registry

March 18, 2024

Completed
12.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 10, 2036

Expected
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 10, 2037

Last Updated

March 18, 2024

Status Verified

March 1, 2024

Enrollment Period

14 years

First QC Date

March 6, 2024

Last Update Submit

March 11, 2024

Conditions

Thalassemia, Beta

Outcome Measures

Primary Outcomes (2)

  • Incidence of hematologic malignancies

    Such as new lymphoma, leukemia, MDS, etc.

    Up to 15 years post-drug product infusion

  • Frequency, grade, and relationship of SAEs

    Frequency, grade, and relationship to BRL-101 of SAEs following BRL-101 reinfusion

    Within 2 years to 5years after BRL-101 Infusion

Study Arms (1)

BRL-101

All patients who have received BRL-101

Other: Assessments

Interventions

AssessmentsOTHER

Safety and efficacy assessments

BRL-101

Eligibility Criteria

Age5 Years - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodProbability Sample
Study Population

Subjects with transfusion-dependent β-thalassemia who have been treated with BHC001.

You may qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

Nanning, Guangxi, 530021, China

RECRUITING

Xiangya Hospital of Central South University

Changsha, Hunan, 510510, China

RECRUITING

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

ThalassemiaAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Xiaochen Wang, PhD

    Bioray Laboratories

    STUDY CHAIR

Central Study Contacts

Xiaochen Wang, PhD

CONTACT

021-64340008xcwang@brlmed.com

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 6, 2024

First Posted

March 18, 2024

Study Start

December 14, 2022

Primary Completion (Estimated)

December 10, 2036

Study Completion (Estimated)

May 10, 2037

Last Updated

March 18, 2024

Record last verified: 2024-03

Data Sharing

IPD Sharing
Will not share

Locations

CN(2)