NCT02698579

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

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

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 64 (actual)
• Sponsor: Genetix Biotherapeutics Inc. · Industry
• Sex: Male
• Age: 19 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Cerebral Adrenoleukodystrophy (CALD)
• Adrenoleukodystrophy (ALD)
• X-Linked Adrenoleukodystrophy (X-ALD)

Interventions

Type	Name	Description
GENETIC	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.

Timeline

• Start date: 2016-01-22
• Primary completion: 2038-08-01
• Completion: 2038-08-01
• First posted: 2016-03-03
• Last updated: 2025-02-20

Locations

13 sites across 9 countries: United States, Argentina, Australia, Brazil, France, Germany, Italy, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study