Is anyone participating in the Starbeam Study for gene therapy for CCALD?



The Starbeam Study, a clinical research study, is now enrolling boys, aged 17 and younger, who have been diagnosed with CCALD.

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 CCALD, and if it is safe and well tolerated.

Participation in the Starbeam Study requires a commitment. You and your family member should expect to visit the study sites regularly and, at times, your family member will need to stay in a hospital or hospitals for various procedures, monitoring and other care. Your family member may withdraw from the study at any time, for any reason.

Your family member’s doctor also may be able to help determine if your family member is a candidate for the Starbeam Study and if it is the right choice for him. If he is eligible and you decide that he should participate in the Starbeam Study, you will discuss all potential benefits and risks in detail with your study doctor. It is not possible to know in advance if your family member will benefit from participating in the study. Some of the potential risks are serious and could lead to death. It will be important that you have a full understanding of the potential risks and benefits.

Specific details about the potential risks associated with the Starbeam Study appear in the informed consent form you will sign before your family member is enrolled in the study. You will be able to ask questions about the study, including the risks, when you provide written consent and throughout the study.

At the completion of the Starbeam Study, you will be asked if you are willing to have your family member participate in a separate, long-term follow-up study. It is important that he takes part in the long-term follow-up study, as the long-term benefits and risks of gene therapy are unknown.

The Starbeam Study will last about 26 months and include the following activities and procedures:

  • Informed consent — You and your family member will receive, and be asked to sign, a form that explains the study in detail, including the potential risks and benefits.
  • Screening — Your family member will undergo several tests to see if he qualifies for the study.
  • Mobilization — This procedure involves receiving medicine to make stem cells circulate in the bloodstream.
  • Apheresis — During this procedure, stem cells will be collected from your family member’s blood through an intravenous catheter.
  • Transduction — This procedure involves transferring the functioning gene to your family member’s own stem cells in a laboratory.
  • Myeloablation — In this stage of the study, your family member will receive several days of chemotherapy to remove existing bone marrow stem cells to make room for the new ones, which contain the functioning gene. This is intended to allow the modified stem cells to establish a home and multiply.
  • Transplantation — During this procedure, the modified stem cells are reintroduced back into your family member’s body through an intravenous infusion.
  • Maintenance — In this stage, your family member will recover in the hospital and be monitored closely by your study team for approximately one to two months. After he leaves the hospital, he will periodically return for check-ups, so the clinical team can evaluate his health and the outcome of treatment with the investigational gene therapy.