Thalassemia MajorNCT05442346
Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
A study testing γ-globin reactivated autologous hematopoietic stem cells in people with Thalassemia Major, run by Bioray Laboratories at 1 site in China.
Trial summary
This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major. Read more about this study
- Who is it for?
- Ages 3 to 35 years
- Conditions the study is for
- Thalassemia Major
- What is being tested?
- γ-globin reactivated autologous hematopoietic stem cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteChina
- Study dates
- 25 Dec 2023to 30 Nov 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 EBV, HIV, CMV, TP, HAV, HBV and HCV.
- 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.
- Recently diagnosed as malaria.
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- History of complex autoimmune disease.
- Persistent aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin value >3 X the upper limit of normal (ULN).
- Subjects with severe heart, lung and kidney diseases.
- With serious iron overload, serum ferritin>5000mg/ml.
- Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician or Investigator.
- Subjects who are receiving treatment from another clinical study, or have received another gene therapy.
- Subjects or guardians had resisted the guidance of the attending doctor.
- Subjects whom the investigators do not consider appropriate for participating in this clinical study
Trial locations and contacts
1 site in 1 country.
China1 site
Shanghai Bioray Laboratories Inc
Shanghai, Shanghai Municipality, 200241
About this study
Official title: an Open Label Trial of Evaluation of the Safety and Efficacy of Treatment With γ-globin Reactivated Autologous Hematopoietic Stem Cells in Subjects With β-thalassemia Major
This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.
Read the detailed description
γ-globin reactivated autologous hematopoietic stem cells will be manufactured using Glycosylase Base Editors. Subject participation for this study will be 2 year. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 15 years post-transplant.
- Conditions
- Thalassemia Major
- Being tested
- γ-globin reactivated autologous hematopoietic stem cells
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
γ-globin reactivated autologous hematopoietic stem cellsBiological
gene edited autologous hematopoietic stem cells with γ-globin expression; BRL-103
| Study group | Role | What they receive |
|---|---|---|
| γ-globin reactivated autologous hematopoietic stem cells | Experimental | each subject will accept one dose of γ-globin reactivated autologous hematopoietic stem cells Receives: Biological: γ-globin reactivated autologous hematopoietic stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects achieving successful neutrophil engraftment within 42 days after BRL-103 infusion
Measured: From 12 months to 24 months post transplant
Time to neutrophil engraftment
Measured: From 12 months to 24 months post transplant
Time to platelet engraftment
Measured: From 12 months to 24 months post transplant
Frequency and severity of adverse events through 100 days after BRL-103 Infusion
Measured: From 12 months to 24 months post transplant
Proportion of subjects achieving sustained transfusion reduction for at least 3 months (TR3)
TR3 was defined as at least a 50% reduction in monthly red blood cell transfusion volume and transfusion frequency compared to baseline for at least 3 months
Measured: From 12 months to 24 months post transplant
Secondary outcomes (12)
Proportion of subjects achieving sustained transfusion independence for at least 3 months (TI3)
Routine transfusion without disease related and with Hb ≥ 90 g/L for at least 3 months
Measured: From 12 months to 24 months post transplant
Proportion of subjects achieving TR6
Measured: From 12 months to 24 months post transplant
Proportion of subjects achieving TR12
Measured: From 12 months to 24 months post transplant
Proportion of subjects achieving sustained transfusion independence for at least 6 months (TI6)
Measured: From 12 months to 24 months post transplant
Proportion of subjects achieving sustained transfusion independence for at least 12 months (TI12)
Measured: From 12 months to 24 months post transplant
Incidence of transplant related mortality (TRM) within 100 days and within 1 year
Measured: From 12 months to 24 months post transplant
Frequency, severity, and relationship to BRL-103 of adverse events over two years following BRL-103 infusion.
Measured: From 12 months to 24 months post transplant
All-cause mortality
Measured: From 12 months to 24 months post transplant
Proportion of alleles with intended genetic modification present in peripheral blood leukocytes over time
Measured: From 12 months to 24 months post transplant
Fetal hemoglobin concentration (pre-transfusion) over time
Measured: From 12 months to 24 months post transplant
Total hemoglobin concentration (pre-transfusion) over time
Measured: From 12 months to 24 months post transplant
Change in serum ferritin level from baseline over time
Measured: From 12 months to 24 months post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2022
- Study start25 Dec 2023
- Primary completion8 Sep 2024 (estimated)
- Study completion30 Nov 2024 (estimated)
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia MajorStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bioray Laboratories
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- First Affiliated Hospital of Guangxi Medical University
- Responsible party
- Sponsor
- Investigators
- lai yongrong, PhD, Principal investigator, First Affiliated Hospital of Guangxi Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05442346
- Sponsor's protocol ID
- 2021-BRL-103
- Status last verified
- Jul 2023
- First submitted
- 23 Sep 2021
- Last updated
- 28 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: clinical study protocol will be shared after Estimated Primary Completion Date
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- an Open Label Trial of Evaluation of the Safety and Efficacy of Treatment With γ-globin Reactivated Autologous Hematopoietic Stem Cells in Subjects With β-thalassemia Major
- ClinicalTrials.gov ID
- NCT05442346
- Acronym / sponsor ID
- 2021-BRL-103
- Conditions
- Thalassemia Major
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Sponsor decision
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Bioray LaboratoriesIndustry (pharma, biotech, devices)
- Collaborators
- First Affiliated Hospital of Guangxi Medical University
- Study start
- 25 Dec 2023 (actual)
- Primary completion
- 8 Sep 2024 (estimated)
- Study completion
- 30 Nov 2024 (estimated)
- First posted
- 5 Jul 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 single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.
Detailed description
γ-globin reactivated autologous hematopoietic stem cells will be manufactured using Glycosylase Base Editors. Subject participation for this study will be 2 year. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 15 years post-transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| γ-globin reactivated autologous hematopoietic stem cells | Experimental | Biological: γ-globin reactivated autologous hematopoietic stem cellseach subject will accept one dose of γ-globin reactivated autologous hematopoietic stem cells |
Who may qualify
- Ages
- 3 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 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 EBV, HIV, CMV, TP, HAV, HBV and HCV.
- Subjects body condition eligible for autologous stem cell transplant.
Key exclusion criteria (14 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 |
|---|---|---|
| γ-globin reactivated autologous hematopoietic stem cellsgene edited autologous hematopoietic stem cells with γ-globin expression; BRL-103 | Biological | γ-globin reactivated autologous hematopoietic stem cells |
Primary outcome
Proportion of subjects achieving successful neutrophil engraftment within 42 days after BRL-103 infusion
Time frame: From 12 months to 24 months post transplant
Time to neutrophil engraftment
Time frame: From 12 months to 24 months post transplant
Time to platelet engraftment
Time frame: From 12 months to 24 months post transplant
Frequency and severity of adverse events through 100 days after BRL-103 Infusion
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving sustained transfusion reduction for at least 3 months (TR3)
Time frame: From 12 months to 24 months post transplant
TR3 was defined as at least a 50% reduction in monthly red blood cell transfusion volume and transfusion frequency compared to baseline for at least 3 months
12 secondary outcomes
Proportion of subjects achieving sustained transfusion independence for at least 3 months (TI3)
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving TR6
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving TR12
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving sustained transfusion independence for at least 6 months (TI6)
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving sustained transfusion independence for at least 12 months (TI12)
Time frame: From 12 months to 24 months post transplant
Incidence of transplant related mortality (TRM) within 100 days and within 1 year
Time frame: From 12 months to 24 months post transplant
Frequency, severity, and relationship to BRL-103 of adverse events over two years following BRL-103 infusion.
Time frame: From 12 months to 24 months post transplant
All-cause mortality
Time frame: From 12 months to 24 months post transplant
Proportion of alleles with intended genetic modification present in peripheral blood leukocytes over time
Time frame: From 12 months to 24 months post transplant
Fetal hemoglobin concentration (pre-transfusion) over time
Time frame: From 12 months to 24 months post transplant
Total hemoglobin concentration (pre-transfusion) over time
Time frame: From 12 months to 24 months post transplant
Change in serum ferritin level from baseline over time
Time frame: From 12 months to 24 months post transplant
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Feb 2024.
- Individual participant dataSharing planned: Yes. clinical study protocol will be shared after Estimated Primary Completion Date
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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.
lai yongrong, PhD principal investigator
First Affiliated Hospital of Guangxi Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing γ-globin reactivated autologous hematopoietic stem cells in people with Thalassemia Major, run by Bioray Laboratories at 1 site 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 5 inclusion and 14 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: paused, as of the sponsor's last update on 28 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
Are results available for this study?
Results are not available in the source record.
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 subjects achieving successful neutrophil engraftment within 42 days after BRL-103 infusion (time frame: From 12 months to 24 months post transplant). The study lists 5 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 28 Feb 2024.