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Clinical Trials/NCT02633943
NCT02633943Active, not recruitingNot Applicable

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

bluebird bio0 sites66 target enrollmentStarted: January 2014Last updated:
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Conditions

Trial Snapshot

Phase
Not Applicable
Status
Active, not recruiting
Enrollment
66
Primary Endpoint
The number of subjects with new or worsening hematologic disorders
Study OverviewStudy DesignEligibility CriteriaOutcomesInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief 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. After completing the parent clinical study (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

Study Type
Observational
Observational Model
Case Only
Time Perspective
Prospective

Eligibility Criteria

Ages
0 Years to 50 Years (Child, Adult)
Sex
All
Accepts Healthy Volunteers
No

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

Outcomes

Primary Outcomes

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 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 neurologic disorders

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

Secondary Outcomes

  • βA-T87Q-globin expression(Up to 15 years post-drug product infusion)
  • Proportion of subjects treated with beti-cel who achieved Transfusion Independence (TI)(Up to 15 years post-drug product infusion)
  • Proportion of subjects treated with beti-cel who achieved Transfusion Independence at yearly timepoints(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)
  • Time from drug product infusion to last pRBC transfusion (in parent study or Study LTF-303)(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(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(Up to 15 years post-drug product infusion)
  • pRBC transfusion frequency, from 6 months post-drug product infusion (parent study) through last follow-up(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(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)
  • Unsupported total Hb levels over time through last follow-up(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)
  • Cardiac T2* by MRI over time at yearly timepoints through last follow-up(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)
  • 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)
  • 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)
  • 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(Up to 15 years post-drug product infusion)
  • Time from stopping chelation to last follow-up(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(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(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 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)
  • 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)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

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