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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Safety and efficacy assessments | Other | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| The number of subjects with malignancies | Up to 15 years post-drug product infusion | |
| The number of subjects with immune-related AEs | Up to 15 years post-drug product infusion | |
| The number of subjects with new or worsening hematologic disorders | Up to 15 years post-drug product infusion | |
| The number of subjects with new or worsening neurologic disorders | Up to 15 years post-drug product infusion |
| Measure | Description | Time Frame |
|---|---|---|
| βA-T87Q-globin expression | Median (min, max) βA-T87Q-globin expression | Up to 15 years post-drug product infusion |
| Proportion of subjects treated with beti-cel who achieved Transfusion Independence (TI) |
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Inclusion Criteria:
Exclusion Criteria:
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Subjects with transfusion-dependent β-thalassemia who have been treated with ex vivo gene therapy product in bluebird bio-sponsored clinical studies
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| Name | Affiliation | Role |
|---|---|---|
| Himal L Thakar, MD | bluebird bio, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Oakland | California | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41525466 | Derived | 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. | |
| 39527960 |
| Label | URL |
|---|---|
| HGB-204 Clinicaltrials.gov | View source |
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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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|
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
| 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 | 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) | Up to 15 years post-drug product infusion |
| Duration of Transfusion Independence | Up to 15 years post-drug product infusion |
| Weighted average Hb during Transfusion Independence | 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 | 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 | 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 | Up to 15 years post-drug product infusion |
| Time from drug product infusion to last pRBC transfusion (in parent study or Study LTF-303) | Up to 15 years post-drug product infusion |
| Time from last pRBC transfusion (in parent study or Study LTF-303) to last follow-up | 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 | 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. | 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 | 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 | 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 | 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 | 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 | 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 | 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 | Up to 15 years post-drug product infusion |
| Cardiac T2* by MRI over time at yearly timepoints through last follow-up | 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 | Up to 15 years post-drug product infusion |
| Serum ferritin over time at yearly timepoints through last follow-up | 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 | 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. | Up to 15 years post-drug product infusion |
| Number of subjects who stopped iron chelation for at least 6 months post-drug product infusion | 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. | Up to 15 years post-drug product infusion |
| Proportion of subjects using phlebotomy therapy post-drug product infusion | 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. | Up to 15 years post-drug product infusion |
| Reticulocyte counts over time at yearly timepoints through last follow-up | 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. | 15 years post-drug product infusion |
| Proportion of subject with nucleated RBC over time at yearly timepoints through last follow-up | 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 | 5 years post-drug product infusion |
| Change from Baseline in PRO as assessed by EuroQol-5D Youth version (EQ-5D-Y) | 5 years post-drug product infusion |
| Change from Baseline in PRO as assessed by EuroQol-5D (EQ-5D-3L) | 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 | 5 years post-drug product infusion |
| Change from Baseline in PRO as assessed by Short Form-36 Health Survey (SF-36) | 5 years post-drug product infusion |
| Chicago |
| Illinois |
| United States |
| Bethesda | Maryland | United States |
| New York | New York | United States |
| Philadelphia | Pennsylvania | United States |
| Charleston | South Carolina | United States |
| Sydney | Australia |
| Marseille | France |
| Paris | France |
| Hanover | Germany |
| Heidelberg | Germany |
| Thessaloniki | Greece |
| Rome | Italy |
| Bangkok | Thailand |
| London | United Kingdom |
| Derived |
| Kwiatkowski 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. |
| 35075288 | Derived | Magrin 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-205 Clinicaltrials.gov | View source |
| HGB-207 Clinicaltrials.gov | View source |
| HGB-212 Clinicaltrials.gov | View source |
| ID | Term |
|---|---|
| D012449 | Safety |
| ID | Term |
|---|---|
| D000056 | Accident Prevention |
| D000059 | Accidents |
| D011634 | Public Health |
| D004778 | Environment and Public Health |
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