This study is in progress, not accepting new patients

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

a study on Adrenoleukodystrophy

Summary

Eligibility
for males ages up to 19 years (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
completion around

Description

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 (eli-cel) in a parent clinical study (Study ALD-102 or Study ALD-104).

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.

Official Title

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

Keywords

Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD), Adrenoleukodystrophy, X-linked Adrenoleukodystrophy, Hematopoietic Stem Cells, No interventional drug product utilized in this follow-up study

Eligibility

You can join if…

Open to males ages up to 19 years

  • 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 eli-cel in a parent clinical study

You CAN'T join if...

  • There are no exclusion criteria for this study

Locations

  • Mattel Children's Hospital-UCLA
    Los Angeles California 90095 United States
  • Lucile Packard Children's Hospital - Stanford
    Palo Alto California 94304 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
bluebird bio
ID
NCT02698579
Study Type
Observational
Participants
About 64 people participating
Last Updated