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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02633943
Registration number
NCT02633943
Ethics application status
Date submitted
30/11/2015
Date registered
17/12/2015
Titles & IDs
Public title
Long-term Follow-up of Subjects With Transfusion-Dependent ß-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
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Scientific title
Long-term Follow-up of Subjects With Transfusion-Dependent ß-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
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Secondary ID [1]
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LTF-303
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Transfusion-dependent Beta-Thalassemia
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Condition category
Condition code
Blood
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Haematological diseases
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Blood
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0
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Other blood disorders
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Human Genetics and Inherited Disorders
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0
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Other interventions - Safety and efficacy assessments
Subjects with Transfusion-Dependent ß-Thalassemia - Subjects treated with ex vivo gene therapy product in an applicable bluebird bio-sponsored clinical trial who agree to participate in this long-term follow-up study
Other interventions: Safety and efficacy assessments
Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term effects of autologous transplant
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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The number of subjects with malignancies
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Assessment method [1]
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Timepoint [1]
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Up to 15 years post-drug product infusion
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Primary outcome [2]
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The number of subjects with immune-related AEs
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Assessment method [2]
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Timepoint [2]
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Up to 15 years post-drug product infusion
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Primary outcome [3]
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The number of subjects with new or worsening hematologic disorders
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Assessment method [3]
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0
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Timepoint [3]
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Up to 15 years post-drug product infusion
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Primary outcome [4]
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The number of subjects with new or worsening neurologic disorders
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Assessment method [4]
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0
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Timepoint [4]
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Up to 15 years post-drug product infusion
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Secondary outcome [1]
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ßA-T87Q-globin expression
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Assessment method [1]
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Median (min, max) ßA-T87Q-globin expression
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Timepoint [1]
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Up to 15 years post-drug product infusion
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Secondary outcome [2]
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Proportion of subjects treated with beti-cel who achieved Transfusion Independence (TI)
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Assessment method [2]
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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
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Timepoint [2]
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Up to 15 years post-drug product infusion
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Secondary outcome [3]
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Proportion of subjects treated with beti-cel who achieved Transfusion Independence at yearly timepoints
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Assessment method [3]
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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
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Timepoint [3]
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Up to 15 years post-drug product infusion
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Secondary outcome [4]
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Time from drug product infusion to achievement of Transfusion Independence (in parent study or Study LTF-303)
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Assessment method [4]
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Timepoint [4]
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Up to 15 years post-drug product infusion
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Secondary outcome [5]
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Duration of Transfusion Independence
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Assessment method [5]
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Timepoint [5]
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Up to 15 years post-drug product infusion
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Secondary outcome [6]
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Weighted average Hb during Transfusion Independence
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Assessment method [6]
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Timepoint [6]
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Up to 15 years post-drug product infusion
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Secondary outcome [7]
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Change in annualized pRBC transfusion volume (among subjects who achieved TI), from 6 months post-drug product infusion (parent study) through last follow-up
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Assessment method [7]
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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
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Timepoint [7]
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Up to 15 years post-drug product infusion
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Secondary outcome [8]
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Annualized pRBC transfusion volume, from 6 months post-drug product infusion (parent study) through last follow-up
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Assessment method [8]
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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
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Timepoint [8]
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Up to 15 years post-drug product infusion
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Secondary outcome [9]
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pRBC transfusion frequency, from 6 months post-drug product infusion (parent study) through last follow-up
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Assessment method [9]
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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
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Timepoint [9]
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0
Up to 15 years post-drug product infusion
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Secondary outcome [10]
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Time from drug product infusion to last pRBC transfusion (in parent study or Study LTF-303)
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Assessment method [10]
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0
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Timepoint [10]
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Up to 15 years post-drug product infusion
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Secondary outcome [11]
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Time from last pRBC transfusion (in parent study or Study LTF-303) to last follow-up
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Assessment method [11]
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0
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Timepoint [11]
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Up to 15 years post-drug product infusion
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Secondary outcome [12]
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Weighted average nadir Hb from 6 months post-drug product infusion (parent study) through last follow-up
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Assessment method [12]
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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
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Timepoint [12]
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Up to 15 years post-drug product infusion
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Secondary outcome [13]
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Unsupported total Hb levels over time through last follow-up
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Assessment method [13]
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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.
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Timepoint [13]
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Up to 15 years post-drug product infusion
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Secondary outcome [14]
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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
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Assessment method [14]
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Timepoint [14]
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Up to 15 years post-drug product infusion
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Secondary outcome [15]
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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
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Assessment method [15]
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0
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Timepoint [15]
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Up to 15 years post-drug product infusion
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Secondary outcome [16]
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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
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Assessment method [16]
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Timepoint [16]
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Up to 15 years post-drug product infusion
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Secondary outcome [17]
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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
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Assessment method [17]
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Timepoint [17]
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Up to 15 years post-drug product infusion
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Secondary outcome [18]
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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
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Assessment method [18]
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0
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Timepoint [18]
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Up to 15 years post-drug product infusion
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Secondary outcome [19]
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Liver iron content (LIC) by magnetic resonance imaging (MRI)/Superconducting Quantum Interference Device (SQUID) over time at yearly timepoints through last follow-up
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Assessment method [19]
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Timepoint [19]
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Up to 15 years post-drug product infusion
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Secondary outcome [20]
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Change from parent study baseline in LIC by MRI/SQUID over time at yearly timepoints through last follow-up
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Assessment method [20]
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Timepoint [20]
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Up to 15 years post-drug product infusion
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Secondary outcome [21]
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Cardiac T2* by MRI over time at yearly timepoints through last follow-up
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Assessment method [21]
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0
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Timepoint [21]
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Up to 15 years post-drug product infusion
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Secondary outcome [22]
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Change from parent study baseline in cardiac T2* by MRI over time at yearly timepoints through last follow-up
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Assessment method [22]
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0
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Timepoint [22]
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Up to 15 years post-drug product infusion
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Secondary outcome [23]
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Serum ferritin over time at yearly timepoints through last follow-up
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Assessment method [23]
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0
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Timepoint [23]
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Up to 15 years post-drug product infusion
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Secondary outcome [24]
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Change from parent study baseline in serum ferritin over time at yearly timepoints through last follow-up
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Assessment method [24]
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0
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Timepoint [24]
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Up to 15 years post-drug product infusion
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Secondary outcome [25]
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Number of subjects who stopped iron chelation post-DP infusion
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Assessment method [25]
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Defined as subjects who stopped iron chelation or never restarted chelation after DP infusion.
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Timepoint [25]
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Up to 15 years post-drug product infusion
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Secondary outcome [26]
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Number of subjects who stopped iron chelation for at least 6 months post-drug product infusion
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Assessment method [26]
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Timepoint [26]
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Up to 15 years post-drug product infusion
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Secondary outcome [27]
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Time from stopping chelation to last follow-up
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Assessment method [27]
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Among subjects that never restart chelation after DP infusion.
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Timepoint [27]
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Up to 15 years post-drug product infusion
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Secondary outcome [28]
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Proportion of subjects using phlebotomy therapy post-drug product infusion
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Assessment method [28]
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Timepoint [28]
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Up to 15 years post-drug product infusion
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Secondary outcome [29]
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Annualized frequency of phlebotomy therapy usage
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Assessment method [29]
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Annualized frequency of phlebotomy therapy usage is defined as the number of procedures per year, calculated from DP infusion through last follow-up.
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Timepoint [29]
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Up to 15 years post-drug product infusion
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Secondary outcome [30]
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Reticulocyte counts over time at yearly timepoints through last follow-up
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Assessment method [30]
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Timepoint [30]
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Up to 15 years post-drug product infusion
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Secondary outcome [31]
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Change from Baseline in reticulocyte counts at yearly timepoints through last follow-up
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Assessment method [31]
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Baseline defined as value closest, but prior to, conditioning in parent study.
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Timepoint [31]
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15 years post-drug product infusion
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Secondary outcome [32]
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Proportion of subject with nucleated RBC over time at yearly timepoints through last follow-up
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Assessment method [32]
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Timepoint [32]
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Up to 15 years post-drug product infusion
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Secondary outcome [33]
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Change from Baseline in patient reported outcome (PRO) as assessed by Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (GCS) Score
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Assessment method [33]
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Timepoint [33]
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5 years post-drug product infusion
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Secondary outcome [34]
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Change from Baseline in PRO as assessed by EuroQol-5D Youth version (EQ-5D-Y)
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Assessment method [34]
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Timepoint [34]
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5 years post-drug product infusion
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Secondary outcome [35]
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Change from Baseline in PRO as assessed by EuroQol-5D (EQ-5D-3L)
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Assessment method [35]
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Timepoint [35]
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5 years post-drug product infusion
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Secondary outcome [36]
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Change From Baseline in PRO as assessed by Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Score
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Assessment method [36]
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Timepoint [36]
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5 years post-drug product infusion
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Secondary outcome [37]
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Change from Baseline in PRO as assessed by Short Form-36 Health Survey (SF-36)
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Assessment method [37]
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Timepoint [37]
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5 years post-drug product infusion
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Eligibility
Key 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 ß-thalassemia in a bluebird bio-sponsored clinical study
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Minimum age
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Years
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Maximum age
50
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* There are no exclusion criteria for this study
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/01/2014
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/11/2035
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Actual
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Sample size
Target
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Accrual to date
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Final
66
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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- Sydney
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Recruitment postcode(s) [1]
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- Sydney
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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0
United States of America
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State/province [2]
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Illinois
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Country [3]
0
0
United States of America
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State/province [3]
0
0
Maryland
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Country [4]
0
0
United States of America
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State/province [4]
0
0
New York
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Country [5]
0
0
United States of America
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State/province [5]
0
0
Pennsylvania
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Country [6]
0
0
United States of America
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State/province [6]
0
0
South Carolina
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Country [7]
0
0
France
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State/province [7]
0
0
Marseille
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Country [8]
0
0
France
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State/province [8]
0
0
Paris
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Country [9]
0
0
Germany
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State/province [9]
0
0
Hannover
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Country [10]
0
0
Germany
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State/province [10]
0
0
Heidelberg
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Country [11]
0
0
Greece
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State/province [11]
0
0
Thessaloníki
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Country [12]
0
0
Italy
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State/province [12]
0
0
Rome
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Country [13]
0
0
Thailand
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State/province [13]
0
0
Bangkok
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Country [14]
0
0
United Kingdom
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State/province [14]
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0
London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
bluebird bio
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent ß-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.
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Trial website
https://clinicaltrials.gov/study/NCT02633943
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Trial related presentations / publications
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.
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Public notes
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Contacts
Principal investigator
Name
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Himal L Thakar, MD
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Address
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bluebird bio, Inc.
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Country
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0
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Phone
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Fax
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0
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Email
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0
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Contact person for public queries
Name
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0
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Address
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0
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Country
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0
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Phone
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Fax
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0
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Email
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0
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
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
[email protected]
.
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT02633943