SNPMiner Trials by Shray Alag


SNPMiner SNPMiner Trials (Home Page)


Report for Clinical Trial NCT02633943

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

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.

NCT02633943 Beta-Thalassemia Sickle Cell Disease
MeSH:Anemia, Sickle Cell Thalassemia beta-Thalassemia Hemoglobinopathies

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 ---



HPO Nodes