NCT05577312Active, not recruitingPhase 1/2
Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
A Phase 1/2 Clinical Study to Evaluate the Safety and Efficacy of Single Dose Intravenous Infusion of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
Source: ClinicalTrials.gov · Last updated 21 Jul 2026
Brief summary (registry, in English)
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)
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 39 (actual)
- First submitted
- 9 Oct 2022
- Start date
- 2 Nov 2022
- Primary completion date
- 20 Oct 2026
- Lead sponsor
- Bioray Laboratories
- Diseases
- Thalassaemia
- NCT number
- NCT05577312
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 39
- From 2 Nov 2022 to the primary completion date, 20 Oct 2026
- 7 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Subjects acceptable for allogeneic hematopoietic stem cell transplantation and have an available fully matched related donor.
- Treated with erythropoietin prior 3 months.
- Prior hematopoietic stem cell transplant (HSCT).
- Other protocol defined Inclusion/Exclusion criteria may apply.
Other illness
- Active bacterial, viral, or fungal infection.
- Subjects with severe psychiatric disorders to be unable to cooperate.
Other
- Immediate family member with any known hematological tumor.
Who the study is looking for
- 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.
Age in the registry: 3 years to 35 years · Sex: All · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Xiangya Hospital of Central South University Coordinator of this site? Confirm its status | Changsha | China | Unknown status |
| Nanfang Hospital, Southern Medical University Coordinator of this site? Confirm its status | Guangzhou | China | Unknown status |
| Sun Yat-Sen Memorial Hospital of Sun Yat-Sen Universit Coordinator of this site? Confirm its status | Guanzhou | China | Unknown status |
| No. 923 Hospital of Joint Support Force of Chinese People 's Liberation Army Hospital Coordinator of this site? Confirm its status | Nanning | China | Unknown status |
| The First Affiliated Hospital of Guangxi Medical University Coordinator of this site? Confirm its status | Nanning | China | Unknown status |
| Shenzhen Children's Hospital Coordinator of this site? Confirm its status | Shenzhen | China | Unknown status |
| Chinese Academy of Medical Sciences Coordinator of this site? Confirm its status | Tianjin | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.