NCT06298630

Brief Summary

Observe long-term safety risk and long-term efficacy after intravenous infusion of BRL-101 in TDT subjects.

Trial Health

63
Monitor

Trial Health Score

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

Enrollment
45

participants targeted

Target at P25-P50 for all trials

Timeline
152mo left

Started Dec 2024

Longer than P75 for all trials

Geographic Reach
1 country

7 active sites

Status
not yet 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 Progress10%
Dec 2024Oct 2038

First Submitted

Initial submission to the registry

March 1, 2024

Completed
6 days until next milestone

First Posted

Study publicly available on registry

March 7, 2024

Completed
9 months until next milestone

Study Start

First participant enrolled

December 8, 2024

Completed
13.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 20, 2038

Expected
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 15, 2038

Last Updated

March 22, 2024

Status Verified

February 1, 2024

Enrollment Period

13.7 years

First QC Date

March 1, 2024

Last Update Submit

March 21, 2024

Conditions

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

Safety and efficacy assessments

BRL-101

Eligibility Criteria

Age3 Years - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Subjects with transfusion-dependent β-thalassemia who have been treated with BRL-101.

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 (7)

Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, 510006, China

Location

Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

Guangzhou, Guangdong, 510006, China

Location

Shenzhen Children 's Hospital

Shenzhen, Guangdong, 510006, China

Location

No. 923 Hospital of Joint Support Force of Chinese People 's Liberation Army Hospital

Nanning, Guangxi, 530021, China

Location

Xiangya Hospital of Central South University

Changsha, Hunan, 510510, China

Location

Tianjin Institute of Hematology

Tianjin, Tianjin Municipality, 300000, China

Location

The First Affiliated Hospital of GUANGXI MEDICAL UNIVERSITY

Guangxi, 530021, China

Location

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

    Bioray Laboratories

    STUDY CHAIR

Central Study Contacts

Xiaochen Wang, phD

CONTACT

Study Design

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

Study Record Dates

First Submitted

March 1, 2024

First Posted

March 7, 2024

Study Start

December 8, 2024

Primary Completion (Estimated)

August 20, 2038

Study Completion (Estimated)

October 15, 2038

Last Updated

March 22, 2024

Record last verified: 2024-02

Locations