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Report for Clinical Trial NCT02633943
 Developed by Shray Alag, 2020-2021.
SNP Clinical Trial Gene 
      This is a multi-center, long-term safety and efficacy follow-up study for subjects with
      hemoglobinopathies (β-thalassemia or severe sickle cell disease) who have been treated with
      ex vivo gene therapy drug product in bluebird bio-sponsored 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 the study.
    
  1  Interventions 
 Name: Safety and efficacy assessments
Description: Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant
 Type: Other
Group Labels:  1  Subjects with hemoglobinopathies  
  
Primary Outcomes 
 Measure: Overall survival of subjects with hemoglobinopathies treated with gene therapy drug product in a bluebird bio-sponsored clinical study Time: 15 years post-drug product infusion
 Measure: All adverse events (AEs) related to drug product Time: 15 years post-drug product infusion
 Measure: All serious adverse events (SAEs) Time: 15 years post-drug product infusion
 Measure: Monitoring for persistence of vector sequences by polymerase chain reaction to determine vector copy number (VCN) Time: 15 years post-drug product infusion
 Measure: Monitoring of βA-T87Q-globin Time: 15 years post-drug product infusion
 Measure: Assessment of transfusions required (mL of packed red blood cells/kg/year) in subjects with β-thalassemia Time: 15 years post-drug product infusion
 Measure: Assessment of iron content in the liver and heart by cardiac magnetic resonance imaging (MRI) and blood draws in subjects with β-thalassemia Time: 15 years post-drug product infusion
 Measure: Frequency of AEs from subjects with severe sickle cell disease including acute chest syndrome (ACS), severe vaso-occlusive episodes (VOC), and stroke or ischemic attacks Time: 15 years post-drug product infusion
  
Time Perspective: Prospective
  Case-Only 
There is one SNP
SNPs
1 T87Q 
Monitoring of βA-T87Q-globin. --- T87Q ---