Clinical Trial: Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy
Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Observational
Official Title: Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
Brief Summary: 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.
Detailed Summary:
Sponsor: bluebird bio
Current Primary Outcome:
- • Overall survival of subjects with hemoglobinopathies treated with gene therapy drug product in a bluebird bio-sponsored clinical study [ Time Frame: 15 years post-drug product infusion ]
- All adverse events (AEs) related to drug product [ Time Frame: 15 years post-drug product infusion ]
- All serious adverse events (SAEs) [ Time Frame: 15 years post-drug product infusion ]
- Monitoring for persistence of vector sequences by polymerase chain reaction to determine vector copy number (VCN) [ Time Frame: 15 years post-drug product infusion ]
- Monitoring of βA-T87Q-globin [ Time Frame: 15 years post-drug product infusion ]
- Assessment of transfusions required (mL of packed red blood cells/kg/year) in subjects with β-thalassemia [ Time Frame: 15 years post-drug product infusion ]
- Assessment of iron content in the liver and heart by cardiac magnetic resonance imaging (MRI) and blood draws in subjects with β-thalassemia [ Time Frame: 15 years post-drug product infusion ]
- 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 Frame: 15 years post-drug product infusion ]ACS is defined as an acute event with pneumonia-like symptoms and the presence of a new pulmonary infiltrate. A severe VOC is defined as an episode of pain, lasting more than 2 hours and requiring care at a medical facility.
Original Primary Outcome:
- Overall survival of subjects with hemoglobinopathies treated with gene therapy drug product in a bluebird bio-sponsored clinical study [ Time Frame: 15 years post-drug product infusion ]
- All serious adverse events (SAEs) related to drug product [ Time Frame: 15 years post-drug product infusion ]
- Monitoring for persistence of vector sequences by polymerase chain reaction to determine vector copy number (VCN) [ Time Frame: 15 years post-drug product infusion ]
- Monitoring of βA-T87Q-globin analysis by high-performance liquid chromatography [ Time Frame: 15 years post-drug product infusion ]
- Assessment of transfusions required (mL of packed red blood cells/kg/year) in subjects with β-thalassemia [ Time Frame: 15 years post-drug product infusion ]
- Assessment of iron content in the liver and heart by cardiac magnetic resonance imaging (MRI) and blood draws in subjects with β-thalassemia [ Time Frame: 15 years post-drug product infusion ]
- 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 Frame: 15 years post-drug product infusion ]ACS is defined as an acute event with pneumonia-like symptoms and the presence of a new pulmonary infiltrate. A severe VOC is defined as an episode of pain, lasting more than 2 hours and requiring care at a medical facility.
Current Secondary Outcome:
Original Secondary Outcome:
Information By: bluebird bio
Dates:
Date Received: November 30, 2015
Date Started: September 2013
Date Completion: March 2031
Last Updated: April 3, 2017
Last Verified: April 2017