Clinical Trial: Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Observational
Official Title: Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Brief Summary:
This is a multi-center, long-term safety and efficacy follow-up study for subjects with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in Study ALD-102.
After completing Study ALD-102 (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.
Detailed Summary:
Sponsor: bluebird bio
Current Primary Outcome:
- Overall survival of subjects with cerebral adrenoleukodystrophy (CALD) treated with Lenti-D Drug Product [ Time Frame: 15 years post-drug-product infusion ]
- All drug product-related AEs [ Time Frame: 15 years post-drug-product infusion ]
- Monitoring neurological status using the Neurological Function Score (NFS) [ Time Frame: 15 years post-drug-product infusion ]
- Monitoring for clinical signs of oncogenesis [ Time Frame: 15 years post-drug-product infusion ]
- Brain magnetic resonance imaging (MRI) (with and without gadolinium enhancement) [ Time Frame: 15 years post-drug-product infusion ]
- Archiving samples and potentially monitoring for the presence of vector-derived replication competent lentivirus (RCL) in archived samples using RCL detection assay, if clinical outcomes are suggestive of retroviral disease [ Time Frame: 5 years post-drug-product infusion ]
- Monitoring for clonal dominance using integration site analysis (ISA) [ Time Frame: 15 years post-drug-product infusion ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Assessment of adrenoleukodystrophy protein (ALDP) expression in peripheral blood [ Time Frame: 5 years post-drug-product infusion ]
- Assessment of vector copy number (VCN) in peripheral blood [ Time Frame: 15 years post-drug-product infusion ]
- Assessment of changes from baseline in very long chain fatty acids (VLCFA) levels in fasting serum [ Time Frame: 5 years post-drug-product infusion ]
- Assessment of changes from baseline in IQ using the age-appropriate test from the Wechsler panel [ Time Frame: 15 years post-drug-product infusion ]
- Health related quality of life assessment (PedsQL) [ Time Frame: 15 years post-drug-product infusion ]
Original Secondary Outcome:
- Assessment of adrenoleukodystrophy protein (ALDP) expression in peripheral blood leukocytes and CD14+ cells [ Time Frame: 5 years post-drug-product infusion ]
- Assessment of vector copy number (VCN) in peripheral blood leukocytes and CD14+ cells [ Time Frame: 15 years post-drug-product infusion ]
- Assessment of changes from baseline in very long chain fatty acids (VLCFA) levels in fasting serum [ Time Frame: 5 years post-drug-product infusion ]
- Assessment of changes from baseline in IQ using the age-appropriate test from the Wechsler panel [ Time Frame: 15 years post-drug-product infusion ]
- Health related quality of life assessment (PedsQL) [ Time Frame: 15 years post-drug-product infusion ]
Information By: bluebird bio
Dates:
Date Received: February 17, 2016
Date Started: January 2016
Date Completion: November 2033
Last Updated: January 13, 2017
Last Verified: January 2017