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

Condition(s) studied

Investigational drug(s) / intervention(s)

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.

Study 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.

Eligibility

Primary outcome measure(s)

• Major functional disability (MFD)-free survival — 15 years post-drug-product infusion
  The MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement.
• Number of participants with malignancies — 15 years post-drug-product infusion
• Number of participants who experience graft versus host disease (GVHD) — 15 years post-drug-product infusion
• Number of participants with immune-related adverse events (AEs) — 15 years post-drug-product infusion
• Number of participants with new or worsening hematologic disorders — 15 years post-drug-product infusion
• Number of participants with new or worsening neurologic disorders — 15 years post-drug-product infusion

Trial sites (13)