Intrathecal and Intravenous Lentiviral Gene Therapy for X-linked Adrenoleukodystrophy (X-ALD)

What this Trial Is About

Researchers are evaluating a gene therapy approach to treat X-linked adrenoleukodystrophy (X-ALD), a serious neurological disorder caused by mutations in the ABCD1 gene. This trial focuses on using a self-inactivating lentiviral vector called TYF-ABCD1 to deliver a functional ABCD1 gene. The aim is to assess the safety and effectiveness of this therapy when given through both intrathecal (into the spine) and intravenous (into the vein) injections, potentially offering treatment options beyond the early stages of the disease. The treatment involves direct delivery of the TYF-ABCD1 lentiviral vector carrying the working gene into the central nervous system and bloodstream via intrathecal and intravenous routes. This method targets patients with symptoms at various stages and seeks to simplify the treatment process compared to traditional stem cell transplantation, reducing associated risks. Participants will receive the gene therapy through these injections as part of the protocol. During the study, participants will be monitored closely through scheduled assessments including vital signs, physical exams, and imaging to evaluate safety and measure disease progression. The main outcomes include tracking any treatment-related adverse events and assessing changes in disease status over time, with follow-up lasting from one day up to three years. The study provides ongoing evaluation of the therapy's impact on the disease course and participants' neurological function.

IT and IV Lentiviral Gene Therapy for X-ALD