Beta-ThalassemiaPhase 1/2NCT05577312
Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
A Phase 1/2 study testing BRL-101 in people with Beta-Thalassemia, run by Bioray Laboratories at 7 sites in China.
Trial 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) Read more about this study
- Who is it for?
- Ages 3 to 35 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- BRL-101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 7 sitesChina
- Study dates
- 2 Nov 2022to 10 Jul 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
China7 sites
Xiangya Hospital of Central South University
Changsha, Hunan, 510510
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510510
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen Universit
Guanzhou, Guangdong, 510006
No. 923 Hospital of Joint Support Force of Chinese People 's Liberation Army Hospital
Nanning, Guangxi, 530021
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 530021
Shenzhen Children 's Hospital
Shenzhen, Guangdong, 510006
Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300000
About this study
Official title: 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
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)
Read the detailed description
This clinical trial is a multi-center, single-arm, single-dose, open-label study without dose escalation. The proposed dose is ≥ 3 × 106 CD34 + cells/kg administered as a single intravenous infusion. The primary objective of Phase 1 is to explore the safety of the study drug in different age groups. For subjects of each age group, myeloablative conditioning and dosing of the remaining subjects was initiated only after completion of dosing and safety observations and assessments in sentinel subjects. The Phase 2 primary objective was to determine the effectiveness of BRL-101 administered intravenously to patients with TDT.
- Conditions
- Beta-Thalassemia
- Being tested
- BRL-101
- Keywords
- Transfusion-Dependent β-Thalassemia
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BRL-101Drug
Also called BRL-101 autologous hematopoietic stem and progenitor cells injection
CD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A enhancer site
| Study group | Role | What they receive |
|---|---|---|
| BRL-101 | Experimental | BRL-101 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Subjects will receive a single infusion of BRL-101. Receives: Drug: BRL-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of stem cell engrafted subjects
Stem cell engraftment was defined as an absolute peripheral blood neutrophil count of ≥ 0.5 × 109/L for 3 consecutive days within 42 days following BRL-101 intravenous infusion.
Measured: Within 42 Days After BRL-101 Infusion
Time to neutrophil engraftment
Defined as Day 1 of absolute peripheral blood neutrophil count ≥ 0.5 × 109/L for 3 consecutive days
Measured: 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
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Measured: Up to 12 Months After BRL-101 Infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2022
- Study start2 Nov 2022
- Primary completion20 Oct 2026 (estimated)
- Study completion10 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-ThalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioray Laboratories
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- First Affiliated Hospital of Guangxi Medical University, Xiangya Hospital of Central South University, Chinese Academy of Medical Sciences, Nanfang Hospital, Southern Medical University, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army, Shenzhen Children's Hospital
- Responsible party
- Sponsor
- Investigators
- Rongmei Liang, PhD, Study chair, Bioray Laboratories
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05577312
- Sponsor's protocol ID
- 2022-BRL-101
- Status last verified
- Jul 2026
- First submitted
- 9 Oct 2022
- Last updated
- 21 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05577312
- Acronym / sponsor ID
- 2022-BRL-101
- Conditions
- Beta-Thalassemia
- Keywords
- Transfusion-Dependent β-Thalassemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Bioray LaboratoriesIndustry (pharma, biotech, devices)
- Collaborators
- First Affiliated Hospital of Guangxi Medical University, Xiangya Hospital of Central South University, Chinese Academy of Medical Sciences, Nanfang Hospital, Southern Medical University, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army, Shenzhen Children's Hospital
- Study start
- 2 Nov 2022 (actual)
- Primary completion
- 20 Oct 2026 (estimated)
- Study completion
- 10 Jul 2027 (estimated)
- First posted
- 13 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Detailed description
This clinical trial is a multi-center, single-arm, single-dose, open-label study without dose escalation. The proposed dose is ≥ 3 × 106 CD34 + cells/kg administered as a single intravenous infusion. The primary objective of Phase 1 is to explore the safety of the study drug in different age groups. For subjects of each age group, myeloablative conditioning and dosing of the remaining subjects was initiated only after completion of dosing and safety observations and assessments in sentinel subjects. The Phase 2 primary objective was to determine the effectiveness of BRL-101 administered intravenously to patients with TDT.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BRL-101 | Experimental | Drug: BRL-101BRL-101 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Subjects will receive a single infusion of BRL-101. |
Who may qualify
- Ages
- 3 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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 (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BRL-101Also: BRL-101 autologous hematopoietic stem and progenitor cells injectionCD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A enhancer site | Drug | BRL-101 |
Primary outcome
Proportion of stem cell engrafted subjects
Time frame: Within 42 Days After BRL-101 Infusion
Stem cell engraftment was defined as an absolute peripheral blood neutrophil count of ≥ 0.5 × 109/L for 3 consecutive days within 42 days following BRL-101 intravenous infusion.
Time to neutrophil engraftment
Time frame: Up to 12 Months After BRL-101 Infusion
Defined as Day 1 of absolute peripheral blood neutrophil count ≥ 0.5 × 109/L for 3 consecutive days
Frequency, severity, and relationship to BRL-101 of adverse events over 12 months following BRL-101 infusion
Time frame: Up to 12 Months After BRL-101 Infusion
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rongmei Liang, PhD study chair
Bioray Laboratories
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing BRL-101 in people with Beta-Thalassemia, run by Bioray Laboratories at 7 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 21 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of stem cell engrafted subjects (time frame: Within 42 Days After BRL-101 Infusion). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jul 2026.