β-thalassemia MajorPhase 1/2NCT04205435
β-globin Restored Autologous HSC in β-thalassemia Major Patients
A Phase 1/2 study testing β-globin restored autologous HSC in people with β-thalassemia Major, run by Bioray Laboratories at 1 site in China.
Trial summary
This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation. 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?
- β-globin restored autologous HSC
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteChina
- Study dates
- 1 Nov 2021to 25 Jul 2022
- Phase
- Phase 1/2
- 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
- 5-15 years old. Clinically diagnosed as β-thalassemia major with IVS-654 gene mutation phenotype;
- Subjects or at least one legal guardian/agent understand and voluntarily sign informed consent.
- 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. 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. 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 Bioraylaboratory Inc
Shanghai, Shanghai Municipality, 200241
About this study
Official title: a Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation
This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.
Read the detailed description
β-globin restored 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
- β-globin restored autologous HSC
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
β-globin restored autologous HSCBiological
gene edited autologous hematopoietic stem cells with β-globin restoration
| Study group | Role | What they receive |
|---|---|---|
| β-globin restored autologous HSC | Experimental | each subject will accept one dose of β-globin restored autologous hematopoietic stem cells Receives: Biological: β-globin restored autologous HSC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects with engraftment;
Measured: up to 42 days 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
Measured: up to 60 days post transplant
Secondary outcomes (2)
Proportion of subjects achieving transfusion independence;
Measured: up to 24 months post transplant
Proportion of subjects with a > = 50% reduced annualized 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 posted19 Dec 2019
- Study start1 Nov 2021
- Primary completion1 Jun 2022
- Study completion25 Jul 2022
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioray Laboratories
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
- Responsible party
- Sponsor
- Investigators
- Xinhua Zhang, Prof, Principal investigator, The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04205435
- Sponsor's protocol ID
- 2019-BRL-00CH2
- Status last verified
- Sep 2021
- First submitted
- 18 Dec 2019
- Last updated
- 13 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Brusson 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 13 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- a Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation
- ClinicalTrials.gov ID
- NCT04205435
- Acronym / sponsor ID
- 2019-BRL-00CH2
- Conditions
- β-thalassemia Major
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Bioray LaboratoriesIndustry (pharma, biotech, devices)
- Collaborators
- The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
- Study start
- 1 Nov 2021 (actual)
- Primary completion
- 1 Jun 2022 (actual)
- Study completion
- 25 Jul 2022 (actual)
- First posted
- 19 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 single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.
Detailed description
β-globin restored 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 |
|---|---|---|
| β-globin restored autologous HSC | Experimental | Biological: β-globin restored autologous HSCeach subject will accept one dose of β-globin restored 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)
- 5-15 years old. Clinically diagnosed as β-thalassemia major with IVS-654 gene mutation phenotype;
- Subjects or at least one legal guardian/agent understand and voluntarily sign informed consent.
- 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 (5 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. Recently diagnosed as malaria. 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. 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.
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 |
|---|---|---|
| β-globin restored autologous HSCgene edited autologous hematopoietic stem cells with β-globin restoration | Biological | β-globin restored autologous HSC |
Primary outcome
Proportion of subjects with engraftment;
Time frame: up to 42 days 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
Time frame: up to 60 days post transplant
2 secondary outcomes
Proportion of subjects achieving transfusion independence;
Time frame: up to 24 months post transplant
Proportion of subjects with a > = 50% reduced annualized volume of packed RBC transfusions.
Time frame: up to 24 months post transplant
Results and safety
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2022.
- 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: 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.
Xinhua Zhang, Prof principal investigator
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
1 site 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 β-globin restored autologous HSC 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 5 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: stopped early, as of the sponsor's last update on 13 Oct 2022. 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.
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?
The study was stopped early (reason given: Sponsor decision). 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 with engraftment; (time frame: up to 42 days 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 NCT04205435, which also lists 1 linked publication.
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 13 Oct 2022.