Brief Title
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
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 participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study. After completing a parent clinical study (approximately 2 years), eligible participants 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.
Study Type
Observational
Primary Outcome
Major Functional Disability (MFD)-Free Survival
Secondary Outcome
Overall Survival
Condition
Cerebral Adrenoleukodystrophy (CALD)
Intervention
Lenti-D
Study Arms / Comparison Groups
Participants treated with Lenti-D
Description: Partcipants who have received Lenti-D Drug Product in a parent clinical study (bluebird bio-sponsored clinical trial) and who meet the eligibility criteria for the study LTF-304.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Genetic
Estimated Enrollment
60
Start Date
January 22, 2016
Completion Date
May 2037
Primary Completion Date
May 2037
Eligibility Criteria
Inclusion Criteria: - Provision of written informed consent for this study by the participant or participant's parent(s)/ legal guardian(s) and written informed assent by participant, if applicable - Have received Lenti-D Drug Product in a parent clinical study - Able to comply with study requirements Exclusion Criteria: - There are no exclusion criteria for this Study
Gender
Male
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Jakob Sieker, MD, ,
Location Countries
Argentina
Location Countries
Argentina
Administrative Informations
NCT ID
NCT02698579
Organization ID
LTF-304
Secondary IDs
2015-002805-13
Responsible Party
Sponsor
Study Sponsor
bluebird bio
Study Sponsor
Jakob Sieker, MD, Study Director, bluebird bio, Inc.
Verification Date
March 2022