SNPMiner Trials (Home Page)
Report for Clinical Trial NCT02633943
Developed by Shray Alag, 2020.
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 transplantType: Other
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 ---
HPO Nodes