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

Running, not enrolling

Conditions studied: Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)

In brief

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.

Key facts

Study ID

NCT02698579

Run by

Genetix Biotherapeutics Inc.

People needed

64

Starts

2016-01-22

Expected to finish

2038-08-01

Last updated by the study team

2025-02-20

Who can join

Age: any, up to 19. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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 may not qualify if…

• There are no exclusion criteria for this study

Where it is running

• Mattel Children's Hospital-UCLA — Los Angeles, California, United States
• Lucile Packard Children's Hospital - Stanford — Palo Alto, California, United States
• Boston Children's Hospital/Massachusetts General Hospital — Boston, Massachusetts, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Instituto Neurogenia — Caba, Argentina
• Women's and Children's Hospital — North Adelaide, Australia
• Hospital das Clínicas da Universidade de São Paulo — São Paulo, Brazil
• Hôpital Bicêtre — Le Kremlin-Bicêtre, Cedex, France
• Universitätsklinikum Leipzig AöR — Leipzig, Germany
• Ospedale Pediatrico Bambino Gesù — Rome, Italy
• Prinses Maxima Center — Utrecht, Netherlands
• Royal Free London Hospital — London, England, United Kingdom
• Great Ormond Street Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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