β Thalassemia MajorNCT04211480
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 β Thalassemia Major, run by Bioray Laboratories at 1 site in China.
Trial summary
This is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to 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 5 to 15 years
- Conditions the study is for
- β 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
- 6 participants
- Where
- 1 siteChina
- Study dates
- 1 Oct 2020to 27 Nov 2023
- 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 5 to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Fully understand and voluntarily sign informed consent. 5-15years old. At least one legal guardian and/or Subjects to sign informed consent.
- Clinically diagnosed as β-thalassemia major, phenotypes including β0β0, 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.
Show all 14Show fewer
- 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 non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to 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 Crispr/Cas9 gene editing system. Subject participation for this study will be 1 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
- β Thalassemia Major
- Being tested
- γ-globin reactivated autologous hematopoietic stem cells
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
| 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.
Safety evaluation of γ-globin reactivated autologous hematopoietic stem cells
Proportion of subjects with engraftment; Overall survival.
Measured: up to 24 months post transplant
Incidence and severity of adverse events as a measure of safety and tolerability. Adverse events assessed according to NCI-CTCAE v5.0 criteria
Incidence of AEs and SAEs post transplant
Measured: up to 24 months post transplant
Secondary outcomes (1)
Efficacy evaluation of γ-globin reactivated autologous hematopoietic stem cells
Proportion of subjects achieving transfusion independence for at least 6 months (TI6); Proportion of subjects achieving TI12; Proportion of alleles with intended genetic modification in bone marrow cells; Change in total hemoglobin concentration; Change from baseline in annualized frequency and volume of packed RBC transfusions.
Measured: up 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 posted26 Dec 2019
- Study start1 Oct 2020
- Primary completion15 Oct 2023
- Study completion27 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for β 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
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioray Laboratories
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Xiangya Hospital of Central South University, The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
- Responsible party
- Sponsor
- Investigators
- Bin Fu, Prof., Principal investigator, Xiangya Hospital Central University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04211480
- Sponsor's protocol ID
- 2019-BRL-00CH1
- Status last verified
- Jan 2024
- First submitted
- 23 Dec 2019
- Last updated
- 5 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant data (IPD) that underlie the results reported in published article will be shared, after deidentification (text, tables,figures and appendices). Other available documents include study protocol.
Publications
Fu B, Liao J, Chen S, Li W, Wang Q, Hu J, Yang F, Hsiao S, Jiang Y, Wang L, Chen F, Zhang Y, Wang X, Li D, Liu M, Wu Y. CRISPR-Cas9-mediated gene editing of the BCL11A enhancer for pediatric beta0/beta0 transfusion-dependent beta-thalassemia. Nat Med. 2022 Aug;28(8):1573-1580. doi: 10.1038/s41591-022-01906-z. Epub 2022 Aug 4.
PubMed 35922667Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.
PubMed 34175041
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 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
- NCT04211480
- Acronym / sponsor ID
- 2019-BRL-00CH1
- Conditions
- β Thalassemia Major
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Bioray LaboratoriesIndustry (pharma, biotech, devices)
- Collaborators
- Xiangya Hospital of Central South University, The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
- Study start
- 1 Oct 2020 (actual)
- Primary completion
- 15 Oct 2023 (actual)
- Study completion
- 27 Nov 2023 (actual)
- First posted
- 26 Dec 2019 (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, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to 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 Crispr/Cas9 gene editing system. Subject participation for this study will be 1 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
- 5 to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Fully understand and voluntarily sign informed consent. 5-15years old. At least one legal guardian and/or Subjects to sign informed consent.
- Clinically diagnosed as β-thalassemia major, phenotypes including β0β0, 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 | Biological | γ-globin reactivated autologous hematopoietic stem cells |
Primary outcome
Safety evaluation of γ-globin reactivated autologous hematopoietic stem cells
Time frame: up to 24 months post transplant
Proportion of subjects with engraftment; Overall survival.
Incidence and severity of adverse events as a measure of safety and tolerability. Adverse events assessed according to NCI-CTCAE v5.0 criteria
Time frame: up to 24 months post transplant
Incidence of AEs and SAEs post transplant
1 secondary outcome
Efficacy evaluation of γ-globin reactivated autologous hematopoietic stem cells
Time frame: up to 24 months post transplant
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2024.
- Derived from this study: PubMed 35922667Fu B, Liao J, Chen S, Li W, Wang Q, Hu J, Yang F, Hsiao S, Jiang Y, Wang L, Chen F, Zhang Y, Wang X, Li D, Liu M, Wu Y. CRISPR-Cas9-mediated gene editing of the BCL11A enhancer for pediatric beta0/beta0 transfusion-dependent beta-thalassemia. Nat Med. 2022 Aug;28(8):1573-1580. doi: 10.1038/s41591-022-01906-z. Epub 2022 Aug 4.
- Derived from this study: PubMed 34175041Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.
- Individual participant dataSharing planned: Yes. Individual participant data (IPD) that underlie the results reported in published article will be shared, after deidentification (text, tables,figures and appendices). Other available documents include study protocol.
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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.
Bin Fu, Prof. principal investigator
Xiangya Hospital Central 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 β 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 4 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: finished, as of the sponsor's last update on 5 Jan 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety evaluation of γ-globin reactivated autologous hematopoietic stem cells (time frame: up to 24 months post transplant). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04211480, which also lists 2 linked publications.
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 5 Jan 2024.