This clinical trial focuses on a rare genetic disease called Cerebral Adrenoleukodystrophy (CALD). CALD affects the brain and can lead to severe neurological problems. The study is examining a treatment known as elivaldogene autotemcel, also referred to as Lenti-D. This treatment involves using a patient’s own blood stem cells, which are modified to include a healthy version of a gene that is faulty in people with CALD. The modified cells are then infused back into the patient through a process called intravenous infusion.

The purpose of this study is to monitor the long-term safety and effectiveness of the Lenti-D treatment in individuals who have already received it in previous studies. Participants will be followed for up to 15 years after their initial treatment. During this time, researchers will observe how well the treatment works in preventing major disabilities and whether any side effects or new health issues arise. This includes checking for conditions like graft versus host disease (GVHD), which can occur when the infused cells attack the patient’s body, and other immune-related or neurological disorders.

Throughout the study, participants will undergo regular health assessments, including MRI scans to monitor changes in the brain. The study aims to provide valuable information on the long-term outcomes of using Lenti-D for treating CALD, helping to understand its potential benefits and risks over an extended period. This information will be crucial for improving treatment strategies for individuals affected by this challenging condition.