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Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

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Disease Information

Descriptive Information
Brief Title † Long-term Follow-up of Subjects 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 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 Description
Study Phase N/A
Study Type † Observational
Study Design †
Primary Outcome Measure † Overall survival of subjects with cerebral adrenoleukodystrophy (CALD) treated with Lenti-D Drug Product
Secondary Outcome Measure † Assessment of adrenoleukodystrophy protein (ALDP) expression in peripheral blood
Condition † Cerebral Adrenoleukodystrophy (CALD) Adrenoleukodystrophy (ALD) X-Linked Adrenoleukodystrophy (X-ALD)
Intervention † OtherSafety and efficacy assessments
Study Arms / Comparison Groups Subjects treated with Lenti-D Subjects treated with Lenti-D Drug Product in Study ALD-102 (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 † Other
Estimated Enrollment † 17
Start Date † January 2016
Completion Date November 2033
Primary Completion Date November 2033
Eligibility Criteria † Inclusion Criteria: - Provision of written informed consent for this study by the subject or subject's parent(s)/ legal guardian(s) and written informed assent by subject, if applicable - Have received Lenti-D Drug Product in Study ALD-102 - Able to comply with study requirements. Exclusion Criteria: - Met the VCN discontinuation criterion on Study ALD-102, as follows: the subject has undetectable VCN (
Gender Male
Ages N/A - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † Argentina
Administrative Information
NCT ID † NCT02698579
Organization ID LTF-304
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † bluebird bio
Collaborators ††
Investigators † Study Director: Mohammed Asmal, MD, PhD, bluebird bio, Inc.
Information Provided By
Verification Date January 2017
First Received Date † February 17, 2016
Last Updated Date January 13, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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