Transfusion-dependent Beta-ThalassemiaNCT02633943
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
An observational study of Transfusion-dependent Beta-Thalassemia, run by Genetix Biotherapeutics Inc. at 15 sites in 8 countries.
Trial summary
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent clinical studies. Read more about this study
- Who is it for?
- Ages 0 to 50 years
- Conditions the study is for
- Transfusion-dependent Beta-Thalassemia
- What is being tested?
- Safety and efficacy assessments
- Type of treatment
- Injection or infusion
- How many people
- 66 participants
- Where
- 15 sitesin 8 countries
- Study dates
- Jan 2014to Nov 2035
- 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 0 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Subjects with transfusion-dependent β-thalassemia who have been treated with ex vivo gene therapy product in bluebird bio-sponsored clinical studies
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 drug product for therapy of transfusion-dependent β-thalassemia in a bluebird bio-sponsored clinical study
Who cannot join
- There are no exclusion criteria for this study
Trial locations and contacts
15 sites in 8 countries.
15 sites in 8 countries
United States6 sites
Study site
Bethesda, Maryland
Study site
Charleston, South Carolina
Study site
Chicago, Illinois
Study site
New York, New York
Study site
Oakland, California
Study site
Philadelphia, Pennsylvania
About this study
Official title: Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent clinical studies. After completing the parent clinical studies (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Being tested
- Safety and efficacy assessments
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Safety and efficacy assessmentsOther
Genetic: No interventional drug product utilized in this follow-up study
Participants received a single IV infusion of LentiGlobin BB305 Drug Product in the parent studies. Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term effects of autologous transplant are conducted in this study.
| Study group | Role | What they receive |
|---|---|---|
| Subjects with Transfusion-Dependent β-Thalassemia | Long-term follow-up for participants treated with ex vivo gene therapy product in applicable bluebird bio-sponsored parent clinical trials and who agreed to participate in this study. Participants will be followed in this study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent studies) Receives: Other: Safety and efficacy assessments |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number of subjects with malignancies
Measured: Up to 15 years post-drug product infusion
The number of subjects with immune-related AEs
Measured: Up to 15 years post-drug product infusion
The number of subjects with new or worsening hematologic disorders
Measured: Up to 15 years post-drug product infusion
The number of subjects with new or worsening neurologic disorders
Measured: Up to 15 years post-drug product infusion
Secondary outcomes (37)
βA-T87Q-globin expression
Median (min, max) βA-T87Q-globin expression
Measured: Up to 15 years post-drug product infusion
Proportion of subjects treated with beti-cel who achieved Transfusion Independence (TI)
Proportion of subjects who achieved TI, defined as a weighted average Hb ≥ 9 g/dL without any packed red blood cell (pRBC) transfusions for a continuous period of ≥ 12 months at any time after drug product infusion in parent study and/or Study LTF-303
Measured: Up to 15 years post-drug product infusion
Proportion of subjects treated with beti-cel who achieved Transfusion Independence at yearly timepoints
Proportion of subjects treated with beti-cel who achieved TI at yearly timepoints including Year 5, Year 10, and Year 15 post-drug product infusion, and at last follow-up
Measured: Up to 15 years post-drug product infusion
Time from drug product infusion to achievement of Transfusion Independence (in parent study or Study LTF-303)
Measured: Up to 15 years post-drug product infusion
Duration of Transfusion Independence
Measured: Up to 15 years post-drug product infusion
Weighted average Hb during Transfusion Independence
Measured: Up to 15 years post-drug product infusion
Change in annualized pRBC transfusion volume (among subjects who achieved TI), from 6 months post-drug product infusion (parent study) through last follow-up
Reduction in annualized pRBC transfusion volume (mL/kg/year) from 6 months post-drug product infusion (parent study) through last follow-up of at least 50%, 60%, 75%, 90%, or 100% as compared to the annualized pRBC transfusion volume during the 2 years prior to parent study enrollment
Measured: Up to 15 years post-drug product infusion
Annualized pRBC transfusion volume, from 6 months post-drug product infusion (parent study) through last follow-up
Annualized pRBC transfusion volume (mL/kg/year from 6 months post-drug product infusion (parent study) through last follow-up as compared to the annualized pRBC transfusion requirements during the 2 years prior to parent study enrollment
Measured: Up to 15 years post-drug product infusion
pRBC transfusion frequency, from 6 months post-drug product infusion (parent study) through last follow-up
Annualized pRBC frequency (number/year) from 6 months post-drug product infusion (parent study) through last follow-up as compared to the annualized pRBC transfusion requirements during the 2 years prior to parent study enrollment
Measured: Up to 15 years post-drug product infusion
Time from drug product infusion to last pRBC transfusion (in parent study or Study LTF-303)
Measured: Up to 15 years post-drug product infusion
Time from last pRBC transfusion (in parent study or Study LTF-303) to last follow-up
Measured: Up to 15 years post-drug product infusion
Weighted average nadir Hb from 6 months post-drug product infusion (parent study) through last follow-up
Weighted average nadir Hb from 6 months post-drug product infusion (parent study) through last follow-up as compared to the weighted average nadir Hb during the 2 years prior to parent study enrollment
Measured: Up to 15 years post-drug product infusion
Unsupported total Hb levels over time through last follow-up
Unsupported total Hb level is defined as the total Hb measurement level without any acute or chronic pRBC transfusions within 60 days prior to the measurement date.
Measured: Up to 15 years post-drug product infusion
Proportion of subjects with unsupported total Hb levels ≥ 10 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Measured: Up to 15 years post-drug product infusion
Proportion of subjects with unsupported total Hb levels ≥ 11 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Measured: Up to 15 years post-drug product infusion
Proportion of subjects with unsupported total Hb levels ≥ 12 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Measured: Up to 15 years post-drug product infusion
Proportion of subjects with unsupported total Hb levels ≥ 13 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Measured: Up to 15 years post-drug product infusion
Proportion of subjects with unsupported total Hb levels ≥ 14 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Measured: Up to 15 years post-drug product infusion
Liver iron content (LIC) by magnetic resonance imaging (MRI)/Superconducting Quantum Interference Device (SQUID) over time at yearly timepoints through last follow-up
Measured: Up to 15 years post-drug product infusion
Change from parent study baseline in LIC by MRI/SQUID over time at yearly timepoints through last follow-up
Measured: Up to 15 years post-drug product infusion
Cardiac T2* by MRI over time at yearly timepoints through last follow-up
Measured: Up to 15 years post-drug product infusion
Change from parent study baseline in cardiac T2* by MRI over time at yearly timepoints through last follow-up
Measured: Up to 15 years post-drug product infusion
Serum ferritin over time at yearly timepoints through last follow-up
Measured: Up to 15 years post-drug product infusion
Change from parent study baseline in serum ferritin over time at yearly timepoints through last follow-up
Measured: Up to 15 years post-drug product infusion
Number of subjects who stopped iron chelation post-DP infusion
Defined as subjects who stopped iron chelation or never restarted chelation after DP infusion.
Measured: Up to 15 years post-drug product infusion
Number of subjects who stopped iron chelation for at least 6 months post-drug product infusion
Measured: Up to 15 years post-drug product infusion
Time from stopping chelation to last follow-up
Among subjects that never restart chelation after DP infusion.
Measured: Up to 15 years post-drug product infusion
Proportion of subjects using phlebotomy therapy post-drug product infusion
Measured: Up to 15 years post-drug product infusion
Annualized frequency of phlebotomy therapy usage
Annualized frequency of phlebotomy therapy usage is defined as the number of procedures per year, calculated from DP infusion through last follow-up.
Measured: Up to 15 years post-drug product infusion
Reticulocyte counts over time at yearly timepoints through last follow-up
Measured: Up to 15 years post-drug product infusion
Change from Baseline in reticulocyte counts at yearly timepoints through last follow-up
Baseline defined as value closest, but prior to, conditioning in parent study.
Measured: 15 years post-drug product infusion
Proportion of subject with nucleated RBC over time at yearly timepoints through last follow-up
Measured: Up to 15 years post-drug product infusion
Change from Baseline in patient reported outcome (PRO) as assessed by Pediatric Quality of Life Inventory (PedsQL
Measured: 5 years post-drug product infusion
Change from Baseline in PRO as assessed by EuroQol-5D Youth version (EQ-5D-Y)
Measured: 5 years post-drug product infusion
Change from Baseline in PRO as assessed by EuroQol-5D (EQ-5D-3L)
Measured: 5 years post-drug product infusion
Change From Baseline in PRO as assessed by Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score
Measured: 5 years post-drug product infusion
Change from Baseline in PRO as assessed by Short Form-36 Health Survey (SF-36)
Measured: 5 years post-drug product infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2015 (estimated)
- Study startJan 2014
- Primary completionNov 2035 (estimated)
- Study completionNov 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Transfusion-dependent 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 66 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Himal L Thakar, MD, Study director, bluebird bio, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02633943
- Sponsor's protocol ID
- LTF-303
- Status last verified
- Apr 2025
- First submitted
- 30 Nov 2015
- Last updated
- 9 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Bluebird bio is committed to transparency and appropriately de-identified subject-level datasets and supporting documents may be shared after all participants have completed study participation and following attainment of applicable marketing approvals associated with a given study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
Publications
Kwiatkowski JL, Thompson AA, Schneiderman J, Thuret I, Kulozik AE, Yannaki E, Cavazzana M, Hongeng S, Olson TS, Sauer MG, Thrasher AJ, Lal A, Rasko JEJ, Kunz JB, Kinney MA, Chawla A, Ali S, Tao G, Thakar HL, Paramore C, Witthuhn N, Walters MC, Locatelli F. Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent beta-thalassemia. Blood. 2026 May 7;147(19):2203-2214. doi: 10.1182/blood.2025029196.
PubMed 41525466Kwiatkowski JL, Walters MC, Hongeng S, Yannaki E, Kulozik AE, Kunz JB, Sauer MG, Thrasher AJ, Thuret I, Lal A, Tao G, Ali S, Thakar HL, Elliot H, Lodaya A, Lee J, Colvin RA, Locatelli F, Thompson AA. Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype beta-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial. Lancet. 2024 Nov 30;404(10468):2175-2186. doi: 10.1016/S0140-6736(24)01884-1. Epub 2024 Nov 8.
PubMed 39527960Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Treluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poiree M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P, Cavazzana M. Long-term outcomes of lentiviral gene therapy for the beta-hemoglobinopathies: the HGB-205 trial. Nat Med. 2022 Jan;28(1):81-88. doi: 10.1038/s41591-021-01650-w. Epub 2022 Jan 24.
PubMed 35075288
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
- ClinicalTrials.gov ID
- NCT02633943
- Acronym / sponsor ID
- LTF-303
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 66 participants (actual)
- Lead sponsor
- Genetix Biotherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- Jan 2014 (actual)
- Primary completion
- Nov 2035 (estimated)
- Study completion
- Nov 2035 (estimated)
- First posted
- 17 Dec 2015 (estimated)
- 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 multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent clinical studies. After completing the parent clinical studies (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Subjects with Transfusion-Dependent β-Thalassemia | – | Other: Safety and efficacy assessmentsLong-term follow-up for participants treated with ex vivo gene therapy product in applicable bluebird bio-sponsored parent clinical trials and who agreed to participate in this study. Participants will be followed in this study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent studies) |
Who may qualify
- Ages
- 0 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Subjects with transfusion-dependent β-thalassemia who have been treated with ex vivo gene therapy product in bluebird bio-sponsored clinical studies
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 drug product for therapy of transfusion-dependent β-thalassemia in a bluebird bio-sponsored clinical study
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 |
|---|---|---|
| Safety and efficacy assessmentsGenetic: No interventional drug product utilized in this follow-up study Participants received a single IV infusion of LentiGlobin BB305 Drug Product in the parent studies. Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term effects of autologous transplant are conducted in this study. | Other | Subjects with Transfusion-Dependent β-Thalassemia |
Primary outcome
The number of subjects with malignancies
Time frame: Up to 15 years post-drug product infusion
The number of subjects with immune-related AEs
Time frame: Up to 15 years post-drug product infusion
The number of subjects with new or worsening hematologic disorders
Time frame: Up to 15 years post-drug product infusion
The number of subjects with new or worsening neurologic disorders
Time frame: Up to 15 years post-drug product infusion
37 secondary outcomes
βA-T87Q-globin expression
Time frame: Up to 15 years post-drug product infusion
Proportion of subjects treated with beti-cel who achieved Transfusion Independence (TI)
Time frame: Up to 15 years post-drug product infusion
Proportion of subjects treated with beti-cel who achieved Transfusion Independence at yearly timepoints
Time frame: Up to 15 years post-drug product infusion
Time from drug product infusion to achievement of Transfusion Independence (in parent study or Study LTF-303)
Time frame: Up to 15 years post-drug product infusion
Duration of Transfusion Independence
Time frame: Up to 15 years post-drug product infusion
Weighted average Hb during Transfusion Independence
Time frame: Up to 15 years post-drug product infusion
Change in annualized pRBC transfusion volume (among subjects who achieved TI), from 6 months post-drug product infusion (parent study) through last follow-up
Time frame: Up to 15 years post-drug product infusion
Annualized pRBC transfusion volume, from 6 months post-drug product infusion (parent study) through last follow-up
Time frame: Up to 15 years post-drug product infusion
pRBC transfusion frequency, from 6 months post-drug product infusion (parent study) through last follow-up
Time frame: Up to 15 years post-drug product infusion
Time from drug product infusion to last pRBC transfusion (in parent study or Study LTF-303)
Time frame: Up to 15 years post-drug product infusion
Time from last pRBC transfusion (in parent study or Study LTF-303) to last follow-up
Time frame: Up to 15 years post-drug product infusion
Weighted average nadir Hb from 6 months post-drug product infusion (parent study) through last follow-up
Time frame: Up to 15 years post-drug product infusion
Unsupported total Hb levels over time through last follow-up
Time frame: Up to 15 years post-drug product infusion
Proportion of subjects with unsupported total Hb levels ≥ 10 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Time frame: Up to 15 years post-drug product infusion
Proportion of subjects with unsupported total Hb levels ≥ 11 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Time frame: Up to 15 years post-drug product infusion
Proportion of subjects with unsupported total Hb levels ≥ 12 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Time frame: Up to 15 years post-drug product infusion
Proportion of subjects with unsupported total Hb levels ≥ 13 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Time frame: Up to 15 years post-drug product infusion
Proportion of subjects with unsupported total Hb levels ≥ 14 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15
Time frame: Up to 15 years post-drug product infusion
Liver iron content (LIC) by magnetic resonance imaging (MRI)/Superconducting Quantum Interference Device (SQUID) over time at yearly timepoints through last follow-up
Time frame: Up to 15 years post-drug product infusion
Change from parent study baseline in LIC by MRI/SQUID over time at yearly timepoints through last follow-up
Time frame: Up to 15 years post-drug product infusion
Cardiac T2* by MRI over time at yearly timepoints through last follow-up
Time frame: Up to 15 years post-drug product infusion
Change from parent study baseline in cardiac T2* by MRI over time at yearly timepoints through last follow-up
Time frame: Up to 15 years post-drug product infusion
Serum ferritin over time at yearly timepoints through last follow-up
Time frame: Up to 15 years post-drug product infusion
Change from parent study baseline in serum ferritin over time at yearly timepoints through last follow-up
Time frame: Up to 15 years post-drug product infusion
Number of subjects who stopped iron chelation post-DP infusion
Time frame: Up to 15 years post-drug product infusion
Number of subjects who stopped iron chelation for at least 6 months post-drug product infusion
Time frame: Up to 15 years post-drug product infusion
Time from stopping chelation to last follow-up
Time frame: Up to 15 years post-drug product infusion
Proportion of subjects using phlebotomy therapy post-drug product infusion
Time frame: Up to 15 years post-drug product infusion
Annualized frequency of phlebotomy therapy usage
Time frame: Up to 15 years post-drug product infusion
Reticulocyte counts over time at yearly timepoints through last follow-up
Time frame: Up to 15 years post-drug product infusion
Change from Baseline in reticulocyte counts at yearly timepoints through last follow-up
Time frame: 15 years post-drug product infusion
Proportion of subject with nucleated RBC over time at yearly timepoints through last follow-up
Time frame: Up to 15 years post-drug product infusion
Change from Baseline in patient reported outcome (PRO) as assessed by Pediatric Quality of Life Inventory (PedsQL
Time frame: 5 years post-drug product infusion
Change from Baseline in PRO as assessed by EuroQol-5D Youth version (EQ-5D-Y)
Time frame: 5 years post-drug product infusion
Change from Baseline in PRO as assessed by EuroQol-5D (EQ-5D-3L)
Time frame: 5 years post-drug product infusion
Change From Baseline in PRO as assessed by Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score
Time frame: 5 years post-drug product infusion
Change from Baseline in PRO as assessed by Short Form-36 Health Survey (SF-36)
Time frame: 5 years post-drug product 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 9 Apr 2025.
- Derived from this study: PubMed 41525466Kwiatkowski JL, Thompson AA, Schneiderman J, Thuret I, Kulozik AE, Yannaki E, Cavazzana M, Hongeng S, Olson TS, Sauer MG, Thrasher AJ, Lal A, Rasko JEJ, Kunz JB, Kinney MA, Chawla A, Ali S, Tao G, Thakar HL, Paramore C, Witthuhn N, Walters MC, Locatelli F. Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent beta-thalassemia. Blood. 2026 May 7;147(19):2203-2214. doi: 10.1182/blood.2025029196.
- Derived from this study: PubMed 39527960Kwiatkowski JL, Walters MC, Hongeng S, Yannaki E, Kulozik AE, Kunz JB, Sauer MG, Thrasher AJ, Thuret I, Lal A, Tao G, Ali S, Thakar HL, Elliot H, Lodaya A, Lee J, Colvin RA, Locatelli F, Thompson AA. Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype beta-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial. Lancet. 2024 Nov 30;404(10468):2175-2186. doi: 10.1016/S0140-6736(24)01884-1. Epub 2024 Nov 8.
- Derived from this study: PubMed 35075288Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Treluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poiree M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P, Cavazzana M. Long-term outcomes of lentiviral gene therapy for the beta-hemoglobinopathies: the HGB-205 trial. Nat Med. 2022 Jan;28(1):81-88. doi: 10.1038/s41591-021-01650-w. Epub 2022 Jan 24.
- HGB-204 Clinicaltrials.govLink provided by the sponsor.
- HGB-205 Clinicaltrials.govLink provided by the sponsor.
- HGB-207 Clinicaltrials.govLink provided by the sponsor.
- HGB-212 Clinicaltrials.govLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Bluebird bio is committed to transparency and appropriately de-identified subject-level datasets and supporting documents may be shared after all participants have completed study participation and following attainment of applicable marketing approvals associated with a given study and consistent with criteria established by bluebird bio and/or industry best practices to 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. This study is not recruiting at the moment.
Himal L Thakar, MD study director
bluebird bio, Inc.
15 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Transfusion-dependent Beta-Thalassemia, run by Genetix Biotherapeutics Inc. at 15 sites in 8 countries.
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: under way, not looking for participants, as of the sponsor's last update on 9 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, France, Germany, Australia, Greece and 3 more countries. 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?
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?
The number of subjects with malignancies (time frame: Up to 15 years post-drug product infusion). The study lists 4 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 NCT02633943, which also lists 3 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 9 Apr 2025.