By InvitationPhase 1/2

Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia

China39 participantsStarted 2022-11-01

Plain-language summary

This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in subjects with Transfusion-Dependent β-Thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101)

Who can participate

Age range

3 Years – 35 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Key Inclusion Criteria: * Fully understand and voluntarily sign informed consent. 3-35years old. At least one legal guardian and/or Subjects to sign informed consent. * Clinically diagnosed as β-thalassemia major, phenotypes including β0β0, β+β+, β+β0, βEβ0 genotype. * Subjects with no affection with HIV, TP, HBV, HCV, CMV and EBV. * Subjects body condition eligible for autologous stem cell transplant. Key Exclusion Criteria: * Subjects acceptable for allogeneic hematopoietic stem cell transplantation and have an available fully matched related donor. * Active bacterial, viral, or fungal infection. * Treated with erythropoietin prior 3 months. * Immediate family member with any known hematological tumor. * Subjects with severe psychiatric disorders to be unable to cooperate. * Prior hematopoietic stem cell transplant (HSCT). Other protocol defined Inclusion/Exclusion criteria may apply.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Proportion of stem cell engrafted subjects

Timeframe: Within 42 Days After BRL-101 Infusion

Time to neutrophil engraftment

Timeframe: Up to 12 Months After BRL-101 Infusion

Frequency, severity, and relationship to BRL-101 of adverse events over 12 months following BRL-101 infusion

Timeframe: Up to 12 Months After BRL-101 Infusion

Trial details

NCT IDNCT05577312

SponsorBioray Laboratories

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-08-20

View on ClinicalTrials.gov More Beta-Thalassemia trials

Who can participate

Age range

3 Years – 35 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Proportion of stem cell engrafted subjects

Timeframe: Within 42 Days After BRL-101 Infusion

Time to neutrophil engraftment

Timeframe: Up to 12 Months After BRL-101 Infusion

Frequency, severity, and relationship to BRL-101 of adverse events over 12 months following BRL-101 infusion

Timeframe: Up to 12 Months After BRL-101 Infusion