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

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X-linked adrenoleukodystrophy
Related Access Program
University of Minnesota – Clinical and Translational Science Institute – X-linked Adrenoleukodystrophy
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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