The Starbeam Study

The Starbeam Study, a clinical research study, has enrolled 18 boys, aged 17 and younger, who have been diagnosed with CALD.

The purpose of the Starbeam Study is to assess the effectiveness and safety of an investigational gene therapy approach, known as gene transfer. The study involves transplantation with a patient’s own stem cells, which will be modified by gene transfer to contain a functioning copy of the ABCD-1 gene. The functioning copy of the gene is intended to allow the body to produce ALDP, a protein critical for the breakdown of very long chain fatty acids.

In this investigational gene therapy study, a patient receives his own stem cells through autologous HSCT. GVHD and graft rejection do not occur with autologous HSCTs, and long-term immunosuppressive therapy is not needed.

The goal of the Starbeam Study is to determine if the one-time investigational gene therapy treatment can stop the progression of CALD, and if it is safe and well tolerated.