Thalassemia, BetaNCT06314529
Long-term Follow-up Study of BHC001 for TDT
An observational study of Thalassemia, Beta, run by Bioray Laboratories at 2 sites in China.
Trial summary
Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects. Read more about this study
- Who is it for?
- Ages 5 to 35 years
- Conditions the study is for
- Thalassemia, Beta
- What is being tested?
- Assessments
- How many people
- 6 participants
- Where
- 2 sitesChina
- Study dates
- 14 Dec 2022to 10 May 2037
- Phase
- Observational
- 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 5 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Subjects with transfusion-dependent β-thalassemia who have been treated with BHC001.
Who may be able to join
- Provision of written informed consent for this study by subjects, or as applicable, subject's parent(s)/legal guardian(s)
- Treated with BRL-101 for therapy of transfusion-dependent β-thalassemia.
Who cannot join
- There are no exclusion criteria for this study
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
Xiangya Hospital of Central South University
Changsha, Hunan, 510510
Bin Fu, PhD
- Recruiting
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Nanning, Guangxi, 530021
Xinhua Zhang, PhD
About this study
Official title: Long-term Follow-up Study of BHC001 in the Treatment of Transfusion-dependent β-thalassemia
Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.
Read the detailed description
Observe long-term delayed safety risks such as emerging neoplasms, emerging haematological disorders, and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.
- Conditions
- Thalassemia, Beta
- Being tested
- Assessments
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AssessmentsOther
Safety and efficacy assessments
| Study group | Role | What they receive |
|---|---|---|
| BRL-101 | All patients who have received BRL-101 Receives: Other: Assessments |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of hematologic malignancies
Such as new lymphoma, leukemia, MDS, etc.
Measured: Up to 15 years post-drug product infusion
Frequency, grade, and relationship of SAEs
Frequency, grade, and relationship to BRL-101 of SAEs following BRL-101 reinfusion
Measured: Within 2 years to 5years 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 posted18 Mar 2024
- Study start14 Dec 2022
- Primary completion10 Dec 2036 (estimated)
- Study completion10 May 2037 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia, BetaStudies 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 6 participants (estimated)
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, Xiangya Hospital of Central South University
- Responsible party
- Sponsor
- Investigators
- Xiaochen Wang, PhD, Study chair, Bioray Laboratories
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06314529
- Sponsor's protocol ID
- 2022-LTF-BHC001
- Status last verified
- Mar 2024
- First submitted
- 6 Mar 2024
- Last updated
- 18 Mar 2024
- 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 18 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up Study of BHC001 in the Treatment of Transfusion-dependent β-thalassemia
- ClinicalTrials.gov ID
- NCT06314529
- Acronym / sponsor ID
- 2022-LTF-BHC001
- Conditions
- Thalassemia, Beta
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Bioray LaboratoriesIndustry (pharma, biotech, devices)
- Collaborators
- The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army, Xiangya Hospital of Central South University
- Study start
- 14 Dec 2022 (actual)
- Primary completion
- 10 Dec 2036 (estimated)
- Study completion
- 10 May 2037 (estimated)
- First posted
- 18 Mar 2024 (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.
Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.
Detailed description
Observe long-term delayed safety risks such as emerging neoplasms, emerging haematological disorders, and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BRL-101 | – | Other: AssessmentsAll patients who have received BRL-101 |
Who may qualify
- Ages
- 5 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Subjects with transfusion-dependent β-thalassemia who have been treated with BHC001.
Key inclusion criteria (2 listed)
- Provision of written informed consent for this study by subjects, or as applicable, subject's parent(s)/legal guardian(s)
- Treated with BRL-101 for therapy of transfusion-dependent β-thalassemia.
Key exclusion criteria (1 listed)
- There are no exclusion criteria for this 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 |
|---|---|---|
| AssessmentsSafety and efficacy assessments | Other | BRL-101 |
Primary outcome
Incidence of hematologic malignancies
Time frame: Up to 15 years post-drug product infusion
Such as new lymphoma, leukemia, MDS, etc.
Frequency, grade, and relationship of SAEs
Time frame: Within 2 years to 5years after BRL-101 Infusion
Frequency, grade, and relationship to BRL-101 of SAEs following BRL-101 reinfusion
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 18 Mar 2024.
- Individual participant dataSharing planned: No
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A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
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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.
Xiaochen Wang, PhD study chair
Bioray Laboratories
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thalassemia, Beta, run by Bioray Laboratories at 2 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 2 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 18 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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.
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?
Contact Xiaochen Wang, PhD (021-64340008, xcwang@brlmed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of hematologic malignancies (time frame: Up to 15 years post-drug product infusion). 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?
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 18 Mar 2024.