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Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Sponsor: Genetix Biotherapeutics Inc.
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
Key Details
Gender
MALE
Age Range
Any - 19 Years
Study Type
OBSERVATIONAL
Enrollment
64
Start Date
2016-01-22
Completion Date
2038-08
Last Updated
2025-02-20
Healthy Volunteers
No
Conditions
Interventions
No interventional drug product utilized in this follow-up study
Participants received a single IV infusion of Lenti-D Drug Product (also known as elivaldogene autotemcel or eli-cel) in either parent Study ALD-102 or ALD-104. The objectives of this long-term follow-up study are to assess long-term safety and efficacy following completion of participation in parent studies. Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant are conducted in this study.
Locations (13)
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