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NCT Number: NCT02633943

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

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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This study is active but is not currently recruiting participants.

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Key information

Conditions

Transfusion-dependent Beta-Thalassemia

Age range

0 year–50 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Sydney, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • There are no exclusion criteria for this study

Treatment and study plan

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.

Primary outcomes

  1. The number of subjects with malignancies

    Time frame: Up to 15 years post-drug product infusion

  2. The number of subjects with immune-related AEs

    Time frame: Up to 15 years post-drug product infusion

  3. The number of subjects with new or worsening hematologic disorders

    Time frame: Up to 15 years post-drug product infusion

  4. The number of subjects with new or worsening neurologic disorders

    Time frame: Up to 15 years post-drug product infusion

Secondary outcomes

  1. βA-T87Q-globin expression

    Time frame: Up to 15 years post-drug product infusion

    Median (min, max) βA-T87Q-globin expression

  2. 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 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

  3. Proportion of subjects treated with beti-cel who achieved Transfusion Independence at yearly timepoints

    Time frame: Up to 15 years post-drug product infusion

    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

  4. 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

  5. Duration of Transfusion Independence

    Time frame: Up to 15 years post-drug product infusion

  6. Weighted average Hb during Transfusion Independence

    Time frame: Up to 15 years post-drug product infusion

  7. 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

    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

  8. 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

    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

  9. 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

    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

  10. 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

  11. 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

  12. 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

    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

  13. Unsupported total Hb levels over time through last follow-up

    Time frame: Up to 15 years post-drug product infusion

    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.

  14. 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

  15. 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

  16. 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

  17. 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

  18. 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

  19. 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

  20. 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

  21. Cardiac T2* by MRI over time at yearly timepoints through last follow-up

    Time frame: Up to 15 years post-drug product infusion

  22. 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

  23. Serum ferritin over time at yearly timepoints through last follow-up

    Time frame: Up to 15 years post-drug product infusion

  24. 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

  25. Number of subjects who stopped iron chelation post-DP infusion

    Time frame: Up to 15 years post-drug product infusion

    Defined as subjects who stopped iron chelation or never restarted chelation after DP infusion.

  26. 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

  27. Time from stopping chelation to last follow-up

    Time frame: Up to 15 years post-drug product infusion

    Among subjects that never restart chelation after DP infusion.

  28. Proportion of subjects using phlebotomy therapy post-drug product infusion

    Time frame: Up to 15 years post-drug product infusion

  29. Annualized frequency of phlebotomy therapy usage

    Time frame: Up to 15 years post-drug product infusion

    Annualized frequency of phlebotomy therapy usage is defined as the number of procedures per year, calculated from DP infusion through last follow-up.

  30. Reticulocyte counts over time at yearly timepoints through last follow-up

    Time frame: Up to 15 years post-drug product infusion

  31. Change from Baseline in reticulocyte counts at yearly timepoints through last follow-up

    Time frame: 15 years post-drug product infusion

    Baseline defined as value closest, but prior to, conditioning in parent study.

  32. 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

  33. 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

  34. Change from Baseline in PRO as assessed by EuroQol-5D Youth version (EQ-5D-Y)

    Time frame: 5 years post-drug product infusion

  35. Change from Baseline in PRO as assessed by EuroQol-5D (EQ-5D-3L)

    Time frame: 5 years post-drug product infusion

  36. 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

  37. Change from Baseline in PRO as assessed by Short Form-36 Health Survey (SF-36)

    Time frame: 5 years post-drug product infusion

Sponsors and collaborators

Lead sponsor

Genetix Biotherapeutics Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2014
Primary completion
2035
Study completion
2035
First posted
Dec 17, 2015
Registry last updated
Apr 9, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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