Long term follow-up of subjects with CALD treated with Lenti-D
Research type
Research Study
Full title
Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
IRAS ID
129575
Contact name
Suman Raj
Contact email
Eudract number
2015-002805-13
Duration of Study in the UK
16 years, 11 months, 1 days
Research summary
This is a multi-centre, 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. Therefore, after a subject has completed Study ALD-102, they will be asked to participate in a this long-term follow-up , in which they will be followed every 6 months through 5 years post-drug-product infusion, and then annually through 15 years post-drug-product infusion. Safety evaluations will include documentation of serious adverse events (SAEs) and archiving for testing for replication competent lentivirus (RCL) through 5 years post-drug-product infusion; additionally, documentation of drug product-related AEs and long-term surveillance for evidence of insertional mutagenesis leading to oncogenesis where appropriate, will continue throughout 15 years post-drug-product infusion.
Efficacy evaluations will include CALD disease-specific assessments, primarily assessment of major functional disabilities and brain MRI, with additional exploratory assessments for ALDP expression, very long chain fatty acids (VLCFA), intelligence quotients (IQ), quality of life (QoL) assessment.
To monitor persistence of vector sequences, vector copy number (VCN) will be measured at designated study visits; subjects meeting the VCN discontinuation criterion may discontinue from the study.REC name
London - West London & GTAC Research Ethics Committee
REC reference
15/LO/1677
Date of REC Opinion
27 Nov 2015
REC opinion
Further Information Favourable Opinion