Beta-ThalassemiaNCT06271512
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
An observational study of Beta-Thalassemia, run by Genetix Biotherapeutics Inc. at 6 sites in United States.
Trial summary
The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Beta-Thalassemia
- Type of treatment
- Injection or infusion
- How many people
- 150 participants
- Where
- 6 sitesUnited States
- Study dates
- 23 Jan 2024to Dec 2043
- How long will I take part?
- 15 Years
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants with β-thalassemia treated with beti-cel in the post marketing setting at a center in the United States (US) that participates in the Registry.
Who may be able to join
- Participant must be treated with beti-cel in the post marketing setting at a center in the US that participates in the Registry.
- Participant must sign an informed consent and/or assent prior to enrollment as required under applicable laws and regulations.
- Participant must have signed an informed consent and/or assent permitting data to be shared with Center for International Blood and Marrow Transplant Research (CIBMTR).
- Participant must be followed by a hematologist based in the US.
Who cannot join
- There are no exclusion criteria for Registry participation.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
Texas Children's Hospital
Houston, Texas, 77030
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55455
- Recruiting
Cohen Children's Medical Center
New Hyde Park, New York, 11040
- Recruiting
UCSF Benioff Children's Hospitals
Oakland, California, 94609
- Recruiting
Stanford University
Palo Alto, California, 94043
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)
The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.
- Conditions
- Beta-Thalassemia
- Being tested
- No Intervention
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
This is non-interventional study.
| Study group | Role | What they receive |
|---|---|---|
| All Participants | Participants with β-thalassemia treated with beti-cel in the post-marketing setting will be followed in this registry study for up to 15 years after infusion with beti-cel to collect real-world longitudinal data. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Event [SAE]):
- Any newly diagnosed malignancy
- Neutrophil engraftment failure: defined as healthcare provider (HCP) decision to administer back-up cells or subsequent hematopoietic stem cell transplantation (HSCT) due to neutrophil recovery failure
- Newly acquired human immunodeficiency virus-1 (HIV-1), HIV-2 infection and human t-lymphotropic virus (HTLV) infection
- Any newly diagnosed autoimmune disorder
- Any hepatic veno-occlusive disease (VOD)
- Any clinically significant bleeding events.
Measured: Through 15 years post-beti-cel infusion
Secondary outcomes (4)
Number of Participants with Serious Adverse Events (SAEs)
Measured: Through 15 years post-beti-cel infusion
Number of Participants with beti-cel related AEs
Measured: Through 15 years post-beti-cel infusion
Event-Free Survival
Measured: Through 15 years post-beti-cel infusion
Percentage of Participants Achieving Transfusion Independence
Measured: Through 15 years post-beti-cel infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2024
- Study start23 Jan 2024
- Primary completionDec 2043 (estimated)
- Study completionDec 2043 (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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 150 participants (estimated)
- Target duration
- 15 Years
Sponsor and investigators
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Center for International Blood and Marrow Transplant Research
- Responsible party
- Sponsor
- Investigators
- Himal Lal Thakar, MD, Study director, bluebird bio, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06271512
- Sponsor's protocol ID
- REG-501
- Status last verified
- Feb 2025
- First submitted
- 14 Feb 2024
- Last updated
- 11 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: bluebird bio is committed to transparency. Appropriately de-identified participant-level datasets and supporting documents may be shared (if bluebird bio is contractually permitted to do so) following completion of this study, completion of all applicable regulatory submissions and consistent with criteria established by bluebird bio, our collaborators and/or industry best practices to protect confidential information and maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)
- ClinicalTrials.gov ID
- NCT06271512
- Acronym / sponsor ID
- REG-501
- Conditions
- Beta-Thalassemia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Genetix Biotherapeutics Inc.Industry (pharma, biotech, devices)
- Collaborators
- Center for International Blood and Marrow Transplant Research
- Study start
- 23 Jan 2024 (actual)
- Primary completion
- Dec 2043 (estimated)
- Study completion
- Dec 2043 (estimated)
- First posted
- 21 Feb 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.
The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 15 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Participants | – | Other: No InterventionParticipants with β-thalassemia treated with beti-cel in the post-marketing setting will be followed in this registry study for up to 15 years after infusion with beti-cel to collect real-world longitudinal data. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with β-thalassemia treated with beti-cel in the post marketing setting at a center in the United States (US) that participates in the Registry.
Key inclusion criteria (4 listed)
- Participant must be treated with beti-cel in the post marketing setting at a center in the US that participates in the Registry.
- Participant must sign an informed consent and/or assent prior to enrollment as required under applicable laws and regulations.
- Participant must have signed an informed consent and/or assent permitting data to be shared with Center for International Blood and Marrow Transplant Research (CIBMTR).
- Participant must be followed by a hematologist based in the US.
Key exclusion criteria (1 listed)
- There are no exclusion criteria for Registry participation.
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 |
|---|---|---|
| No InterventionThis is non-interventional study. | Other | All Participants |
Primary outcome
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
Time frame: Through 15 years post-beti-cel infusion
The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Event [SAE]): * Any newly diagnosed malignancy * Neutrophil engraftment failure: defined as healthcare provider (HCP) decision to administer back-up cells or subsequent hematopoietic stem cell transplantation (HSCT) due to neutrophil recovery failure * Newly acquired human immunodeficiency virus-1 (HIV-1), HIV-2 infection and human t-lymphotropic virus (HTLV) infection * Any newly diagnosed autoimmune disorder * Any hepatic veno-occlusive disease (VOD) * Any clinically significant bleeding events.
4 secondary outcomes
Number of Participants with Serious Adverse Events (SAEs)
Time frame: Through 15 years post-beti-cel infusion
Number of Participants with beti-cel related AEs
Time frame: Through 15 years post-beti-cel infusion
Event-Free Survival
Time frame: Through 15 years post-beti-cel infusion
Percentage of Participants Achieving Transfusion Independence
Time frame: Through 15 years post-beti-cel infusion
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 11 Feb 2025.
- Individual participant dataSharing planned: Yes. bluebird bio is committed to transparency. Appropriately de-identified participant-level datasets and supporting documents may be shared (if bluebird bio is contractually permitted to do so) following completion of this study, completion of all applicable regulatory submissions and consistent with criteria established by bluebird bio, our collaborators and/or industry best practices to protect confidential information and maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
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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.
Himal Lal Thakar, MD study director
bluebird bio, Inc.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Beta-Thalassemia, run by Genetix Biotherapeutics Inc. at 6 sites in United States.
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 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 11 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. 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 bluebird bio (+1-833-999-6378, clinicaltrials@bluebirdbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) (time frame: Through 15 years post-beti-cel infusion). 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 11 Feb 2025.